Nervous system disorder

This study tests a new gene therapy called DB-OTO for children with hearing loss caused by changes in the otoferlin gene. The treatment is injected into the inner ear to help restore hearing. Up to 30 children and infants will take part to check safety and how well it works.

This early-phase trial tests a personalized RNA vaccine for people with recurrent medulloblastoma, a type of brain tumor. The vaccine is made from the patient's own tumor material and aims to train the immune system to fight the cancer. The study includes up to 24 participants ag…

This study tests a new treatment called AZD0120 for people with AL amyloidosis, a rare disease where abnormal proteins build up in organs. The therapy uses a patient's own immune cells, modified to target and destroy harmful cells. The study aims to see if it is safe and effectiv…

This study tests brain surgery as a treatment for people whose epilepsy doesn't improve with medication. About 300 children and adults aged 8 and older will have surgery to remove or treat the part of the brain causing seizures. The goal is to stop or greatly reduce seizures, tho…

This phase 3 trial is testing whether adding a targeted therapy called Sacituzumab tirumotecan (Sac-TMT) to standard chemotherapy and immunotherapy (pembrolizumab) can better eliminate cancer before surgery in people with high-risk, early-stage triple-negative or hormone-receptor…

This Phase 3 trial tests an oral drug called gildeuretinol acetate (ALK 001) in 230 people aged 8 to 45 with Stargardt disease, a genetic condition that causes progressive vision loss. Participants take the drug or a placebo daily for 24 months. The goal is to see if the drug can…

This phase 2 trial tests whether specially selected immune cells taken from a patient's own tumor, grown in large numbers in the lab, and given back along with the drug pembrolizumab (Keytruda) can shrink tumors in people with metastatic colorectal, pancreatic, ovarian, breast, o…

This Phase 2 trial tests a new way to give radiation for sinus cancer after endoscopic surgery. Instead of radiating the whole sinus area, doctors will use a 'dose painting' technique that targets only the areas where cancer was found. The goal is to see if this reduces side effe…

This study tests an oral medication called LUM-201 for children with growth hormone deficiency who have never been treated before. The goal is to see if taking a pill every day for 12 months can improve growth rate compared to a placebo. The study involves 150 prepubertal childre…

This study tests a new drug called LMP744 in people whose glioblastoma has come back after standard treatment. The drug is designed to kill cancer cells by blocking a key growth signal and interfering with DNA repair. Participants will receive the drug through a vein for 5 days e…

This early-stage trial is testing an experimental drug called EVOLVE104 in people with advanced bladder cancer or several types of squamous cell carcinoma (lung, esophagus, skin, and others). The study aims to find a safe dose and see if the drug can shrink tumors. It is for pati…

This early-phase trial at Mayo Clinic is testing combinations of four drugs (dasatinib, quercetin, fisetin, and temozolomide) in 30 adults with previously treated glioma that still has residual tumor cells. The goal is to see if it's feasible to use individual patient data from b…

This study tests whether clemizole HCl (EPX-100) can safely reduce seizures when added to current treatments for Lennox-Gastaut syndrome, a severe form of epilepsy. About 260 children and adults aged 2 to 55 will receive either the drug or a placebo. The main goal is to measure c…

This Phase 3 trial tests trontinemab, an antibody given by IV, in 800 people with early Alzheimer's (mild cognitive impairment to mild dementia). Half get the drug, half get a placebo, for 72 weeks. The goal is to see if it slows decline in memory, thinking, and daily activities.

This early-stage trial is testing whether a personalized natural killer (NK) cell therapy called SNK01 is safe and might help people with moderate Alzheimer's disease. The treatment is made from each patient's own immune cells and given as an IV infusion every three weeks for up …

This study tests whether adding a personalized mRNA vaccine (V940) to standard immunotherapy (pembrolizumab) can stop advanced melanoma from growing or spreading. About 160 people with stage III or IV melanoma that cannot be removed by surgery will receive either the vaccine plus…

This trial investigates a 'maximal medical treatment' bundle for people with intracerebral hemorrhage (bleeding in the brain). The bundle includes rapid blood pressure control, reversal of blood thinners, and a drug to reduce bleeding, all delivered within the first 72 hours in a…

This study follows about 300 people with Duchenne muscular dystrophy who are taking the drug givinostat as part of their normal care. Researchers will track safety issues like low platelet counts and bleeding, and see how patients' muscle function changes over up to 5 years. The …

This study tests a telemonitoring device called ANTALGEEK® that helps people manage acute pain at home after outpatient surgery. The device tracks pain levels and guides medication use, aiming to improve recovery and lower the risk of chronic pain. About 150 adults having surgery…

This study tests whether programming a cochlear implant based on each person's unique inner ear structure improves hearing compared to the standard one-size-fits-all approach. Adults with severe to profound hearing loss who already have a MED-EL cochlear implant will receive both…

This phase 3 trial tests whether adding the experimental drug sacituzumab tirumotecan (sac-TMT) to standard immunotherapy (pembrolizumab) and a targeted therapy (bevacizumab) helps people with metastatic cervical cancer live longer. About 1,000 participants whose cancer has sprea…

This study is testing a new type of immunotherapy called liso-cel (Breyanzi) as the first treatment for primary central nervous system lymphoma (PCNSL), a rare brain cancer. The trial is for 65 adults who cannot have a stem cell transplant. Liso-cel is made from the patient's own…

This study tests four drugs (vismodegib, GSK2256098, capivasertib, and abemaciclib) in people with progressive meningiomas that have specific gene changes. The goal is to see if these drugs can stop tumor growth or shrink tumors. About 124 adults with confirmed mutations will rec…

This study tests whether adding the drug dinutuximab to standard chemotherapy and other treatments helps children with high-risk neuroblastoma live longer without their cancer returning. About 478 children and young adults up to age 30 will be randomly assigned to receive either …

This early-phase trial tests a new drug called metarrestin in people with advanced solid tumors that have spread (metastasized). The goal is to find a safe dose and see if it can shrink tumors. The study includes adults with pancreatic, breast, or other solid tumors, and children…

This study looks at people with narrowed carotid arteries who are getting a stent. Some people do not respond well to the common blood thinner clopidogrel. The study compares giving these 'non-responders' a different drug, ticagrelor, versus continuing clopidogrel in those who re…

This study compares two versions of the drug ocrelizumab given as a shot under the skin in people with multiple sclerosis. The goal is to see if the new test version works the same as the current one. About 182 participants with relapsing or primary progressive MS will take part.

This study tests whether low intensity focused ultrasound (LIFU) — sound waves delivered through a wearable headband — can reduce seizures in adults with drug-resistant epilepsy. Participants wear the device during treatment sessions, and researchers track seizure frequency and b…

This study tests an experimental drug called THN391, given as an injection into the eye, for people with diabetic macular edema (DME) — a condition where fluid builds up in the retina due to diabetes, causing vision loss. The trial involves 21 adults aged 18 to 80 with DME and vi…

This early-phase study tests a new drug called NX-5948 in adults with advanced B-cell blood cancers (like certain leukemias and lymphomas) that have not responded to other treatments. The drug works by breaking down a protein (BTK) that helps these cancer cells grow. The main goa…

This study tests two experimental drugs, sapanisertib and serabelisib, combined with standard therapy for people with HR+/HER2- advanced or metastatic breast cancer. The goal is to find safe doses and see if the combination can shrink tumors or slow the disease. About 32 adults w…

This Phase III trial is testing whether adding the targeted drug vorasidenib to standard chemotherapy (temozolomide) after radiation helps people with a specific type of brain tumor (IDH-mutated grade 3 astrocytoma). About 408 participants will receive either vorasidenib or a pla…

This study tests the drug abemaciclib, typically used for cancer, in people with neurofibromatosis type 1 (NF1) who have atypical neurofibromas that cannot be surgically removed. The goal is to find a safe dose and see if it can shrink or stabilize these tumors. Participants take…

This phase 3 trial is testing whether the drug pridopidine can slow the progression of amyotrophic lateral sclerosis (ALS) in 500 adults. Participants will take either pridopidine or a placebo daily for 48 weeks, followed by an open-label extension where everyone receives the dru…

This study tests whether clemastine fumarate, a common allergy medicine, can help repair the protective coating around nerves (myelin) in people with multiple sclerosis. About 74 adults with relapsing-remitting MS will receive either the drug or a placebo, and advanced MRI scans …

This study tests a new exercise program for people with spinal cord injury. It combines arm cycling with electrical stimulation to the legs, plus high-intensity interval training, after a muscle-strengthening prep phase. The goal is to see if this program improves heart fitness, …

This study tests whether a short-term, intensive form of PTSD therapy can quickly reduce symptoms and improve thinking skills in military personnel. Participants receive either Cognitive Processing Therapy (CPT) or Eye Movement Desensitization and Reprocessing (EMDR) over a conde…

This study tests a new device called the RapidPulse Aspiration System, which uses precisely timed suction to remove blood clots from brain arteries in people having a severe stroke. The trial will enroll 170 adults aged 18 to 80 who can be treated within 8 hours of stroke symptom…

This early-stage trial tests whether giving a higher dose of focused radiation (stereotactic radiosurgery) to brain metastases is safe. About 50 people with 1 to 5 brain tumors who have never had brain radiation will receive an experimental dose on one tumor, while other tumors g…

This study tests an experimental pill called silmitasertib combined with standard chemotherapy in children and young adults (under 30) whose solid tumors (like neuroblastoma, Ewing sarcoma, or osteosarcoma) have come back or not responded to treatment. The goal is to find a safe …

This study tests a new automated method to fine-tune adaptive deep brain stimulation (aDBS) for people with Parkinson's disease. aDBS uses brain signals to adjust stimulation in real time, but setting it up is complex. The trial will compare automated aDBS to standard continuous …

This study tests a new drug called ASP2246 for adults who have trouble moving after a stroke. In the first part, small groups get increasing doses via brain surgery to check safety. In the second part, some get the drug and others get a sham surgery, with neither patient nor doct…

This study investigates whether a homemade bottle-based breathing device can strengthen the muscles used for exhaling and improve balance in people with Parkinson's disease. Parkinson's often weakens respiratory muscles, leading to breathing and coughing problems. The trial will …

This phase 3 trial tests whether adding the experimental drug MK-2870 to the standard immunotherapy pembrolizumab helps people with advanced non-small cell lung cancer live longer compared to pembrolizumab alone. The study enrolls about 614 adults whose tumors have high PD-L1 lev…

This early-stage trial tests two experimental pills (PF-07799544 and PF-07799933) taken twice daily at home by adults with advanced solid tumors that have a specific gene change called BRAF V600. The study aims to find safe doses and see if the drugs can shrink tumors. About 124 …

This phase III trial is testing whether a precise, high-dose radiation technique called stereotactic radiosurgery (SRS) is better at preserving memory and thinking than the current standard of whole brain radiation that avoids the hippocampus (the brain's memory center) plus a dr…

This study tests a drug called safusidenib in people with a type of brain tumor (glioma) that has a specific gene mutation (IDH1). The trial has three parts: one for recurrent tumors, one for high-risk tumors after standard treatment, and one for a specific tumor type after surge…

This study is checking the long-term safety of an implant called OTX-TKI (axitinib) for people with wet age-related macular degeneration (AMD). The implant is placed in the eye and slowly releases medicine to control the disease. About 850 people who already completed two years o…

This early-phase study is testing the safety and how the body handles a drug called tovorafenib in Japanese children, teens, and young adults with a type of brain tumor (low-grade glioma) that has a specific genetic change (BRAF alteration) and has come back or is growing. Six pa…

This phase 2 trial tests whether adding DOC1021, a personalized dendritic cell vaccine, to standard treatment (surgery, chemo, radiation) improves survival in adults newly diagnosed with glioblastoma. About 180 participants will be randomly assigned to receive the vaccine plus st…

This phase 2 trial tests whether NLY01, a drug similar to those used for diabetes, can slow brain and retinal shrinkage in people with multiple sclerosis. About 120 adults with stable MS will receive either NLY01 or a placebo for 96 weeks. The main goal is to see if the drug redu…

This Phase 3 study tests a drug called zorevunersen in 170 children aged 2 to 17 with Dravet syndrome, a severe form of epilepsy. The drug is given as an injection into the spine and compared to a sham procedure. The goal is to see if it reduces seizures and improves daily living…

This study tests a new virtual reality (VR) walking therapy for people who have had a stroke within the past 1 to 6 months. Participants will use either a fully immersive VR system that lets them walk over ground in realistic environments, or a semi-immersive VR system on a tread…

This phase II trial is testing whether adding the drug naxitamab (Danyelza) to standard chemotherapy improves outcomes for children newly diagnosed with high-risk neuroblastoma. About 93 participants will receive naxitamab during five cycles of induction chemo. The goal is to see…

This phase 2 trial tests a powerful mix of short-course radiation, chemotherapy (mFOLFOX6), an immunotherapy (PD-1 antibody), and a targeted drug (cetuximab or bevacizumab, depending on genetic markers) in people with high-risk rectal cancer that is mismatch repair proficient (pM…

This Phase 3 trial tests a device called SonoCloud-9, which uses ultrasound to temporarily open the blood-brain barrier, allowing more carboplatin chemotherapy to reach glioblastoma tumors that have returned after initial treatment. About 560 participants will be randomly assigne…

This study tests a one-time gene therapy called surabgene lomparvovec (Sura-vec) for diabetic retinopathy, a common eye problem from diabetes that can lead to blindness. About 576 adults with moderate-to-severe non-proliferative diabetic retinopathy will receive either the gene t…

This trial compares two types of surface-modified flow diverters—special stents used to treat unruptured brain aneurysms. The goal is to see which device causes fewer small brain injuries (seen on MRI) and fewer stroke-like symptoms after the procedure. About 196 people with unru…

This early-stage trial is testing a new oral drug, PF-07799933, in people aged 16 and older with advanced solid tumors that have a specific BRAF gene change. The drug is given alone or combined with other medicines like binimetinib or cetuximab. The study aims to find safe doses …

This study tests an experimental pill called NMD670 in 84 adults with myasthenia gravis, a condition that causes muscle weakness. Participants take the pill or a placebo twice daily for 21 days. Researchers will measure changes in muscle strength and daily activities to see if th…

This clinical trial tests an oral drug called SKY-0515 in 400 adults with Huntington's disease. The goal is to see if it can lower harmful proteins linked to the disease and improve symptoms like movement and thinking. Participants take the drug or a placebo daily for 18 months.

This early-stage study is testing a new drug called RO7121932 in 129 people with multiple sclerosis (MS). The goal is to check if the drug is safe and how the body handles it when given as a single or multiple doses, either through a vein or under the skin. Participants are not o…

This Phase 3 trial tests a single inhaled dose of alprazolam (a fast-acting sedative) to stop prolonged seizures in people aged 12 and older. The goal is to see if it can end a seizure within 90 seconds and prevent it from coming back for at least 2 hours. About 350 participants …

This phase 3 trial is testing atogepant, a daily pill already approved for adults, to prevent episodic migraines in children aged 6 to 17. About 450 participants will receive either a low or high dose of the drug or a placebo for 12 weeks. The goal is to see if it safely reduces …

This Phase 3 trial tests a product called NTX-001, a set of three solutions applied during surgery to help reconnect severed nerves in the arm. About 98 adults with severe nerve cuts will be randomly assigned to receive either standard nerve stitching plus NTX-001 or standard sti…

This study tests whether a type of talk therapy called Mentalization-Based Treatment (MBT) can help young people (ages 14–30) who are at high risk for psychosis avoid developing the condition. Participants will receive either MBT plus standard care or standard care alone for 24 w…

This study checks the long-term safety and lasting effects of a gene therapy called IPS101A in 6 people with severe Parkinson's disease who already received it in an earlier trial. Researchers will monitor side effects and measure changes in movement symptoms over time. The goal …

This early-phase trial tests whether giving two immunotherapy drugs (nivolumab and ipilimumab) before eye removal surgery is safe and feasible for people with high-risk uveal melanoma, a rare eye cancer. Fifteen participants will receive the drugs, then have their eye removed. Th…

This Phase 3 trial tests whether privosegtor, a neuroprotective drug, can improve vision recovery in people having their first episode of optic neuritis. About 210 adults aged 18 to 50 will receive either privosegtor or a placebo, both alongside standard steroid treatment. The ma…

This study tests two oral drugs, eflornithine (DFMO) and AMXT 1501, in children and young adults up to age 26 with certain cancers that have come back or not responded to treatment. The cancers include neuroblastoma, brain tumors, and sarcomas. The goal is to find a safe dose and…

This early-stage trial tests whether implanting specially grown dopamine-producing cells into the brain is safe and can help people with Parkinson's disease. Six participants receive their own lab-grown cells placed into a brain region that controls movement. The study measures c…

This Phase 3 trial tests whether adding immunotherapy and a targeted drug to short-course radiotherapy and chemotherapy improves outcomes for people with high-risk rectal cancer. About 204 participants will receive either the standard treatment or the intensified combo. The study…

This study tests an experimental drug, fosigotifator, in adults and children with Vanishing White Matter disease, a rare genetic brain disorder. The main goals are to check the drug's safety and how the body processes it. About 50 participants will take the drug for up to 201 wee…

This early-phase trial is testing a new treatment called KITE-363 for people with certain autoimmune nerve diseases that have not responded to standard therapies. KITE-363 is a type of CAR T-cell therapy that uses a patient's own immune cells, modified to target and attack faulty…

This early-stage trial tests a personalized cell therapy for children with recurrent neuroblastoma or newly diagnosed DIPG, two aggressive cancers. The treatment uses the child's own immune cells (dendritic cells and T cells) and stem cells, trained to attack their specific tumor…

This early-stage trial tests a new treatment called CC-97540, which uses a patient's own immune cells engineered to target and destroy harmful immune cells. The study includes 120 people with relapsing or progressive multiple sclerosis or refractory myasthenia gravis. The main go…

This study tests whether rimegepant, a dissolving tablet, can prevent migraines in children and adolescents ages 6 to under 18. About 640 participants will receive either the drug or a placebo for 12 weeks. The main goal is to see if the drug reduces the number of migraine days p…

This study tests a single injection into the eye of a new drug (SAR446597) for people with geographic atrophy, a progressive form of age-related macular degeneration that causes vision loss. About 104 participants aged 60 and older will be followed for 2 years, with an optional 3…

This study tests a new treatment called LB-DTK-CMV for people with cytomegalovirus (CMV) retinitis that has not responded to standard antiviral medicines. The treatment uses a patient's own immune cells, specially trained to attack CMV, and is given as two infusions. The trial ai…

This study tests two different doses of an intravenous immune globulin (IGIV 10%) as maintenance therapy for adults with chronic inflammatory demyelinating polyneuropathy (CIDP), a nerve disorder causing weakness and numbness. About 161 participants will receive either a higher o…

This study is testing an experimental drug called DNL628 in people with early-stage Alzheimer's disease. The main goal is to see if the drug is safe and how the body processes it. About 68 participants will receive either DNL628 or a placebo. This is an early-phase trial, so it f…

This study tests whether starting a special exercise program soon after a stroke can protect the brain from memory loss and dementia. Researchers will enroll 120 people who had a stroke in the past 6 months. Participants will do a mix of exercises, and the study will measure chan…

This Phase 2 trial tests an oral drug called NEU-411 in 150 adults aged 40-80 with early Parkinson's disease who have a specific genetic marker (LRRK2-driven). The study is double-blind and placebo-controlled, meaning neither patients nor doctors know who gets the real drug. Rese…

This study tests whether the antibiotic minocycline can improve recovery in people having an ischemic stroke. About 934 adults will receive either minocycline or a placebo, in addition to standard clot-busting medication. The goal is to see if minocycline leads to better function…

This study tests an implanted device that stimulates nerves at the back of the head to prevent chronic cluster headaches. Ten adults with difficult-to-treat cluster headaches will receive the implant and use it daily for 48 weeks. Researchers will track side effects and whether t…

This study tests a device called Artisse, which is placed inside a brain aneurysm to block blood flow and prevent rupture. It involves 300 people aged 18-75 with wide-neck aneurysms that are either unruptured or recently ruptured. The main goal is to see if the device safely and …

This early-stage trial tests a combination of four drugs—lenalidomide, nivolumab, rituximab, and methotrexate—in people with primary CNS lymphoma, a rare cancer of the brain or spinal cord. The goal is to find the safest dose and see if adding lenalidomide and nivolumab to standa…

This study tests whether giving a second dose of the clot-busting drug prourokinase can help stroke patients who have a blocked medium or large artery and don't show improvement one hour after the first dose. About 122 adults will be enrolled within 4.5 hours of stroke onset. The…

This Phase 3 trial tests whether DYNE-101 can improve muscle function and daily life in 150 adults with myotonic dystrophy type 1 (DM1). Participants receive either the drug or a placebo by IV every few weeks for 48 weeks. The study measures how quickly people can stand from a ch…

This early-stage study tests a new oral drug, DEG6498, in about 100 adults with advanced solid tumors that have not responded to standard treatments. The main goals are to find a safe dose and understand side effects. Participants take the drug once daily and are closely monitore…

This study tests a new drug called Ulotaront (SEP-363856) to see if it can reduce symptoms like hallucinations and delusions in people with schizophrenia. About 522 adults experiencing an acute episode will receive either the drug or a placebo for 6 weeks. The main goal is to mea…

This early-stage trial tests a new gene therapy called SKY-GJB2 for children aged 9 months to 7 years with hearing loss caused by changes in the GJB2 gene. The therapy is given as a single injection into the inner ear using a special delivery device. The study will enroll 10 chil…

This study tests a drug called tivozanib in people with bile duct cancer that cannot be removed by surgery and has already been treated with chemotherapy. The goal is to find the safest dose and see if the drug can shrink tumors or slow the cancer. Participants take tivozanib by …

This study tests whether adding an antibody drug called hu14.18K322A to standard chemotherapy helps children with high-risk neuroblastoma, a rare but aggressive childhood cancer. About 144 children and teens aged 18 months to 18 years will receive the combination over up to 12 cy…

This study tests whether a bivalent vaccine, taken with a sugar called beta-glucan and a protein (GM-CSF), can help prevent high-risk neuroblastoma from returning in children who are currently in remission. The treatment works by training the immune system to recognize and attack…

This study tests a new medicine called tiespectus (EYE201) for wet age-related macular degeneration, a leading cause of vision loss. About 960 people will receive either tiespectus or the standard treatment aflibercept via eye injections. The goal is to see if tiespectus can impr…

This study tests a one-time gene therapy called RGX-202 in boys with Duchenne muscular dystrophy (DMD). The therapy delivers a mini version of the missing dystrophin protein to muscle cells. Researchers will check safety and whether it improves muscle function, like standing and …

This study tests whether HBS-301 (pitolisant) can reduce excessive daytime sleepiness and cataplexy (sudden muscle weakness) in adults with narcolepsy. Participants receive either the drug or a placebo for several weeks, followed by an open-label phase where everyone gets the dru…

This early-phase study tests the drug TURALIO (pexidartinib) in people aged 3 to 35 whose cancer has not responded to standard treatments. The goal is to find a safe dose and see if it can shrink tumors or control leukemia. Participants take a daily capsule for up to 2 years, wit…

This study uses botulinum toxin (Botox) injections to treat movement disorders such as dystonia, tremor, and spasticity. Up to 2,000 participants aged 2 and older will receive tailored injections, with doctors trained in proper techniques. The goal is to manage symptoms and build…

This early-stage trial tests a single dose of TSHA-105, a gene therapy injected into the spinal fluid, in 8 people aged 2 to 20 with SLC13A5 citrate transporter disorder. The goal is to see if it is safe and whether it can improve motor and thinking skills. Because the trial is v…

This study looks at how safe and effective temozolomide (brand name Amitzo) is for people with aggressive brain tumors (grade III and IV gliomas). Researchers are tracking 100 patients to see how often liver injury occurs and to monitor other side effects. The goal is to better u…

This study is testing whether it is safe and effective to perform two heart procedures at the same time in people with atrial fibrillation (AF). The first procedure uses pulsed field ablation to correct the heart's rhythm, and the second closes off a small pouch in the heart to r…

This study tests whether the drug ubrogepant can prevent menstrual migraines when taken daily for a week around each period. About 496 women with regular cycles and a history of menstrual migraines will receive either ubrogepant or a placebo for three cycles, then all may receive…

This Phase 3 trial tests an oral drug called orelabrutinib against a placebo in about 990 people with non-active secondary progressive multiple sclerosis (MS). The goal is to see if the drug can delay worsening disability over 24 to 60 months. Participants are randomly assigned t…

This phase 3 trial tests whether the drug tebentafusp can prevent uveal melanoma from coming back in high-risk patients after their initial treatment. About 290 people with a specific genetic marker (HLA-A*02:01) will either receive weekly infusions of tebentafusp or be observed …

This study tests a new drug called tiespectus (MK-8748) against the current standard treatment, aflibercept, for people with wet age-related macular degeneration (AMD). About 960 participants will receive injections into the eye and be followed for a year to see if tiespectus can…

This phase 3 trial is testing whether the drug KarXT can safely and effectively treat schizophrenia in adolescents aged 13 to 17. About 166 teens will receive either KarXT or a placebo, and researchers will track changes in symptoms like delusions and social withdrawal. The goal …

This study looks at the long-term safety and tolerability of the drug TAK-360 in people with narcolepsy (type 1 or 2) or idiopathic hypersomnia — conditions that cause severe daytime sleepiness. Participants who completed a previous TAK-360 study can join and will continue taking…

This study tests a special stem cell transplant for children, teens, and young adults (ages 6 months to 25 years) with solid tumors that have come back or not responded to treatment. The transplant uses cells from a donor, with certain immune cells removed to reduce side effects,…

This study tests an experimental drug called EPX-100 (clemizole) in 150 children and adults with Dravet syndrome, a severe form of epilepsy. The goal is to see if adding EPX-100 to current treatments can safely reduce the number of motor seizures. Participants will be randomly as…

This trial tests a product called NTX-001, which is applied during surgery to repair completely severed nerves in the arm or face. The product combines three solutions and a device, and is given only once. The study includes people with fresh nerve injuries (within 48 hours) that…

This study follows 200 people with acromegaly who are using octreotide subcutaneous depot, a medication that helps control hormone levels. Researchers will monitor safety and effectiveness for up to two years in everyday clinical practice, not a controlled lab setting. The goal i…

This study tests whether adding team-building tools—like team charters, communication handoffs, and performance monitoring—to an existing school-based ADHD program (the Collaborative Life Skills Program) makes it work better. Researchers will compare the enhanced program to the s…

This early-phase trial tests the safety and best dose of the drug triapine when given together with radiation therapy for people whose glioblastoma or astrocytoma has come back. Triapine works by blocking enzymes that cancer cells need to grow, while radiation kills cancer cells …

This early-stage study tests a new drug called RO7812653 in 50 people with early Alzheimer's disease. The main goal is to check if the drug is safe and how the body handles it. Participants receive a single dose injected into the spine and are closely monitored for side effects.

This study tests whether a special probiotic supplement can change fat-related molecules in the blood of people with ALS and frontotemporal dementia (FTD). Researchers will compare those taking the probiotic to those taking a placebo over 24 weeks. The goal is to see if the probi…

This early-phase trial tests a single dose of YTB323, a CAR-T cell therapy, in 28 adults with non-active progressive multiple sclerosis. The study aims to check safety and track disability changes over time. Participants must be 18–60 and able to undergo lumbar punctures and MRIs…

This study follows 300 adults with Parkinson's disease, essential tremor, or dystonia who receive deep brain stimulation (DBS). DBS uses a device implanted in the chest to send electrical pulses to brain areas controlling movement. Researchers will track symptom severity, medicat…

This early-stage trial tests a gene therapy called RC001 in children aged 2 to 18 with Dravet syndrome, a severe form of epilepsy caused by a genetic mutation. The therapy is given as a single injection into the spinal fluid. The main goal is to check safety and how the body proc…

This phase 3 study tests whether KarXT, a new drug, can safely reduce schizophrenia symptoms in 250 Japanese adults experiencing acute psychosis. Participants first receive either KarXT or a placebo for 5 weeks, then all may receive KarXT for a year. The goal is to see if KarXT i…

This phase 3 trial tests whether a new drug (MK-2870), alone or with another drug (pembrolizumab), works better than standard treatments for people with a common type of advanced breast cancer (HR+/HER2-). About 1,200 participants whose cancer has worsened after hormone therapy w…

This early-stage study is testing a single injection of a long-acting form of the drug brexpiprazole in healthy volunteers and people with schizophrenia. The main goal is to check if the injection is safe and tolerable, and how the body processes the drug. The trial involves 56 p…

This program provides access to an experimental drug, AOC 1044, for people with Duchenne muscular dystrophy (DMD) whose genetic mutation can be treated by skipping exon 44. The goal is to help control the disease and improve muscle function. Participants must be at least 2 years …

This Phase 2 study tests whether a single dose of AAV2-GDNF gene therapy, delivered directly into the brain, can improve motor function in adults with moderate Parkinson's disease. About 127 participants will receive either the gene therapy or a sham surgery. The main goal is to …

This study tests a single dose of GB221, a gene therapy that delivers a working SMN1 gene, in infants aged 2 weeks to under 12 months with spinal muscular atrophy (SMA) Type 1. The goal is to see if it is safe and helps improve motor function. The trial includes both symptomatic …

This study tests a new device called CLAAS that is placed in the heart to prevent strokes in people with atrial fibrillation (an irregular heartbeat). About 1600 participants will be randomly assigned to get either the new device or one of two already-approved devices. The goal i…

This study tracks how well the drug Koselugo (selumetinib) works and what side effects it causes in people with Neurofibromatosis Type 1 and painful nerve tumors. About 200 patients in South Korea will be followed during their normal doctor visits. The goal is to confirm the drug…

This early-phase study tests whether adding melatonin to standard cooling therapy can improve brain outcomes in newborns with hypoxic-ischemic encephalopathy (HIE), a condition caused by oxygen loss at birth. About 70 full-term infants will receive different doses of melatonin to…

This early-phase trial tests whether a combination of two drugs (loncastuximab tesirine and rituximab) given after precise radiation to the brain is safe and effective for central nervous system lymphoma. The study enrolls 12 adults whose lymphoma has returned or who cannot recei…

This study is testing a device called ExAblate 4000 that uses focused ultrasound waves to treat non-cancerous brain tumors in children and young adults. The goal is to see if the treatment is safe and can shrink tumors without open surgery. About 20 participants will be enrolled,…

This early study tests whether a smart brain implant can help people with spinocerebellar ataxia type 6, a condition that causes trouble with balance and coordination. Five adults will get the implant, which records brain signals and adjusts stimulation automatically. The goal is…

This study tests whether giving the drug milrinone directly into the brain's arteries, followed by a 72-hour IV drip, can prevent brain damage in people who develop artery spasms after a bleeding stroke from a burst aneurysm. The trial will enroll 306 adults across multiple hospi…

This study tests a mobile health app called uMAT-R, designed to help Black adults recover from opioid or stimulant use and manage depression or anxiety. Participants are recruited from community treatment centers and use the app to track substance use and mental health symptoms. …

This study tests whether injecting a person's own bone marrow stem cells into or near the eye can help treat various retinal and optic nerve diseases, including age-related macular degeneration, retinitis pigmentosa, and glaucoma. Participants receive stem cell injections via dif…

This phase 3 trial tests whether the oral drug orelabrutinib can slow disability progression in people with primary progressive multiple sclerosis (PPMS). About 705 adults aged 18–60 with PPMS will receive either orelabrutinib or a placebo for up to 5 years. The study measures ti…

This study tests a new radioactive drug, [212Pb]VMT-alpha-NET, in people with advanced gastrointestinal neuroendocrine tumors or pheochromocytoma/paragangliomas that have already been treated with radiation therapy. The drug is designed to attach to a protein on the surface of th…

This study tests whether a device called MyokinE100, which sends electrical signals to the thigh muscles, is safe and practical for use in the ICU. It includes 50 critically ill patients with sepsis who are at risk of muscle weakness. Half will receive standard care plus daily 60…

This study compares three oral medications used to prevent migraines: atogepant (a newer drug), topiramate, and propranolol (both older). About 1,335 adults with migraine will be randomly assigned to one of the three drugs for 12 weeks. Researchers will track headache frequency a…

This study tests a single intravenous dose of a gene therapy called GNR-097 in boys aged 4 to 9 with Duchenne muscular dystrophy (DMD). The therapy uses a harmless virus to deliver a shortened version of the dystrophin gene, which is missing or faulty in DMD. The trial aims to se…

This early-stage trial tests a new medicine called EYC-0305, given as an eye injection every 24 weeks, in 30 people aged 50 and older with wet age-related macular degeneration (AMD). The main goal is to check if the treatment is safe and tolerable. Researchers will also measure h…

This study tests whether taking probiotics daily for 3 months can reduce symptoms of anxiety and depression in 60 adults with mild-to-moderate conditions. Participants will receive either probiotics, a placebo, or the antidepressant escitalopram. Researchers will measure changes …

This study tests whether ublituximab, given as an infusion, can help adults with myasthenia gravis maintain symptom control after initial treatment with efgartigimod. Participants will receive either ublituximab or a placebo, and researchers will track how long it takes for sympt…

This trial compares two rehabilitation techniques—constraint-induced movement therapy (CIMT) and proprioceptive neuromuscular facilitation (PNF)—to see which better improves hand dexterity in children with cerebral palsy. The study includes children aged 8 to 13 with moderate men…

This study tests a gene therapy called ETX101 for children with Dravet syndrome, a severe form of epilepsy caused by a genetic change. The treatment uses a harmless virus to deliver a working copy of the gene to help control seizures. The trial includes infants and children up to…

This study tests a new device and technique (ADAPT 2.0) for removing blood clots in people having an acute ischemic stroke. The goal is to see if it can restore blood flow more completely and safely than current methods. About 750 adults who are already scheduled for clot removal…

This study tests whether a new drug, sacituzumab tirumotecan (Sac-TMT), given alone or with pembrolizumab, helps people with advanced triple-negative breast cancer live longer or keep their cancer from growing. About 1,000 adults whose cancer has spread and who have not had prior…

This early-stage trial compares a new investigational drug, CG-0255, to the established blood thinner Plavix in 136 healthy adults aged 18-55. The goal is to see if CG-0255 is processed by the body similarly to Plavix and has the same effect on preventing blood clots. Participant…

This study tests whether deep brain stimulation (DBS) can help people with severe depression that hasn't improved with multiple treatments. Researchers will implant a device that sends electrical pulses to a specific brain area. The trial includes 100 adults and compares active s…

This phase II trial tests whether vortioxetine, a drug currently used for depression, can help people with newly diagnosed glioblastoma. The study will enroll 78 adults and measure if the drug extends the time before the cancer worsens. Vortioxetine was selected because lab tests…

This study tests whether starting virtual reality-based rehabilitation within 24 hours of an acute ischemic stroke can improve arm muscle strength, thinking skills, and motivation to exercise. One hundred adults recently diagnosed with stroke will be randomly assigned to either v…

This study tests a combination of chemotherapy and focused radiation in children newly diagnosed with a rare brain tumor called ETMR. About 70 children will receive standard treatments to see if this approach helps keep the tumor from growing for at least 6 months. The goal is to…

This study tests a gene therapy called ABBV-RGX-314 for people with wet age-related macular degeneration (AMD), a leading cause of vision loss. The goal is to see if a single treatment can reduce the need for frequent eye injections while keeping vision stable. About 561 adults a…

This early-stage study tests a new drug called RG6496 in people who carry the gene for Huntington's disease. The main goal is to check if the drug is safe and how the body handles it. About 40 participants will receive a single dose injected into the spine, with some getting a pl…

This phase 1 trial tests a new type of immunotherapy for people with recurrent or progressive high-grade gliomas, including glioblastoma. The treatment uses the patient's own T cells, which are genetically modified in the lab to target a protein called IL13Rα2 found on tumor cell…

This early-phase study tests the drug lorlatinib in children and young adults (ages 1 to 21) with newly diagnosed high-grade glioma, a fast-growing brain tumor, that has a specific genetic change (ALK or ROS1 fusion). The goal is to see if the drug can shrink or control the tumor…

This study tests an experimental drug called povetacicept in adults with generalized myasthenia gravis, a condition that causes muscle weakness. The goal is to see if the drug safely reduces harmful immune proteins. About 30 participants will receive either the drug or a placebo,…

This trial evaluates the safety of a single intravenous dose of DON, an experimental drug, in healthy adults, adults with uncomplicated malaria, and children aged 12 months to 14 years with cerebral malaria. Researchers will monitor side effects and measure how the drug moves thr…

This pilot study explores whether taking high-dose cocoa extract supplements is feasible and acceptable for people with episodic migraine. Researchers will compare two doses of cocoa extract to a placebo, tracking how many participants stick with the daily pills for 12 weeks. The…

This study tests a drug called radiprodil in people with GRIN-related neurodevelopmental disorder, a rare genetic condition that often causes seizures and developmental delays. The trial includes two groups: one with frequent seizures and one with fewer or no seizures. Participan…

This trial tests whether removing a brain bleed with a minimally invasive scope within hours of symptoms improves recovery. 300 adults with a specific type of brain bleed will be randomly assigned to either get the surgery plus standard care, or standard care alone. The study wil…

This phase 3 trial is testing a new oral form of cladribine, a drug that calms the immune system, in 264 adults with generalized myasthenia gravis. Participants receive either low-dose cladribine, high-dose cladribine, or a placebo in two courses four weeks apart. The main goal i…

This study tests a structured problem-solving approach called CO-OP (Cognitive Orientation to daily Occupational Performance) for adults who have had a stroke or traumatic brain injury. Participants learn a 'Goal-Plan-Do-Check' strategy to tackle everyday tasks like dressing or c…

This early-stage study tests a new medicine called PYC-001, given as an injection into the eye, for people with a rare genetic eye disease (autosomal dominant optic atrophy) caused by a change in the OPA1 gene. The main goal is to check the safety of different doses and schedules…

This trial tests a new clothing-like robotic arm that detects muscle signals to help people who have had a stroke move their arm and hand. The device supports coordinated movements of the shoulder, elbow, wrist, and fingers during 20 one-hour training sessions. The study includes…

This phase 2 study tests whether the sleep medication suvorexant can reduce insomnia in 76 adults aged 21–65 who have both alcohol use disorder and posttraumatic stress disorder. Participants take either suvorexant or a placebo for 14 days while trying to stop drinking. The study…

This study tests a new approach using a mix of gene-modified immune cells (CAR-T cells) that target multiple proteins found on B cell lymphoma cells. The goal is to see if this combination is safe and can shrink tumors in patients whose cancer has not responded to standard treatm…

This study compares Tai Chi and Qi-Gong, two gentle movement therapies, to see which better improves balance and motor function in people with stage I Parkinson's disease. Fifty participants aged 50 to 65 will receive either Tai Chi or Qi-Gong alongside standard physical therapy …

This study investigates whether ketamine, given as four intravenous infusions over two weeks, can reduce depression and alcohol cravings in adults who have both conditions. Participants are already in inpatient addiction therapy and are randomly assigned to receive either ketamin…

This study explores whether a robotic device that amplifies a person's own walking movements can help improve balance in people who have had a stroke. Participants with chronic stroke (more than 6 months ago) will do two treadmill walking sessions—one in a normal environment and …

This trial tests a gene therapy for metachromatic leukodystrophy (MLD), a rare and life-threatening genetic disorder that damages the nervous system. The treatment uses a lentivirus to deliver a working copy of the ARSA gene directly into the spinal fluid and bloodstream. Up to 1…

This study investigates whether a wrist-worn sensor (KinesiaU) can accurately measure how long people with Parkinson's disease experience 'good on-time' — periods when medication works well and symptoms are controlled without troublesome movements. Researchers are testing IPX-203…

This study tests an experimental oral drug called KDS2010 in people with mild cognitive impairment or mild dementia due to Alzheimer's disease. Participants take either a low dose, a higher dose, or a placebo daily for 24 weeks. Researchers measure changes in memory, thinking, an…

This study tests an investigational drug called LTX-002 in people with amyotrophic lateral sclerosis (ALS). The drug is designed to target a specific genetic message linked to the disease. The main goal is to check the drug's safety and tolerability, and to see how it moves throu…

This clinical trial tests a new type of immunotherapy called 4SCAR-T, which uses a patient's own immune cells engineered to recognize and attack three different markers (GD2, PSMA, and CD276) on neuroblastoma cancer cells. The study enrolls children and adults (ages 1 to 65) with…

This pilot study explores whether computer-assisted music remediation can improve memory, attention, and thinking in young people aged 18 to 30 who have had a first psychotic episode. Participants attend weekly group sessions using music software to practice memory and executive …

This phase 1 trial tests an experimental drug, DXC006, in people with advanced solid tumors (like small cell lung cancer and neuroblastoma) or blood cancers (like multiple myeloma) who have not responded to standard treatments. DXC006 is an antibody-drug conjugate designed to del…

This study tests whether adding sodium benzoate—a substance that affects brain glutamate—to standard antipsychotic medication can improve symptoms and thinking in people with treatment-resistant schizophrenia. About 90 adults who have not responded well to at least two antipsycho…

This study evaluates PathFinder 1.0, a software that analyzes OCT images of the retina to detect macular abnormalities such as epiretinal membrane, dry age-related macular degeneration, and other retinal conditions. About 400 adults with and without macular issues will undergo st…

This study looks at whether measuring certain proteins in the blood (p-tau217 and neurofilament light) can help doctors diagnose Alzheimer's disease more quickly and confidently in people with memory problems. About 550 participants visiting a memory clinic will have their blood …

This study tests whether using an AI-driven retinal camera during regular diabetes checkups can increase the number of young people screened for diabetic retinopathy, a leading cause of blindness. Researchers will enroll 500 youth with type 1, type 2, or cystic fibrosis-related d…

This pilot study tests a special PET/MRI scan that uses a radioactive tracer to find HER2+ breast cancer that has spread to the brain. The scan aims to show whether the cancer cells take up the tracer, which might predict if they will respond to the standard drug trastuzumab deru…

This study aims to find better ways to diagnose Parkinson's disease in its earliest stages. Researchers will use wearable devices to track movement and analyze blood samples for specific markers from nerve cells. By combining these digital and biological clues, they hope to creat…

This study tests whether a portable, low-field MRI machine can detect brain injuries in children who are on ECMO (a heart-lung bypass machine). The MRI is brought to the bedside, avoiding the need to move critically ill children. Researchers will scan children at several time poi…

This study tests a new device called nOCT that acts like a tiny camera on a flexible wire. It slides into brain blood vessels during standard aneurysm treatment to take detailed pictures of the vessel wall and any implanted coils or stents. The goal is to see if these images help…

This study tests a new radioactive tracer called 18F-mFBG to see if it can detect nerve damage in the heart caused by Lewy body diseases like Parkinson's and Lewy body dementia. About 20 adults will get a PET scan after an injection of the tracer. The goal is to see if the scan c…

This early-stage trial tests a fluorescent dye called panitumumab-IRDye800 that attaches to cancer cells. Before surgery, 30 adults with brain tumors will receive a single infusion of the dye. During their operation, a special camera will detect the dye's glow, helping surgeons d…

This early-stage study tests a new radioactive tracer called SST001 that lights up on PET scans. Researchers want to see if it is safe and can help tell the difference between multiple system atrophy (MSA) and Parkinson's disease. The study will include 30 healthy volunteers and …

This study enrolls about 4,000 adults aged 60 and older with heart failure who are at high risk for ATTR amyloidosis, a rare disease that stiffens the heart. Doctors receive special training on how to spot and diagnose the condition using standard tests. The goal is to see if thi…

This study tests a special dye that makes brain tumor cells glow during surgery. The dye is given before surgery, and a special camera helps surgeons see the glowing tumor tissue. The goal is to help surgeons remove more of the tumor while protecting healthy brain tissue. The stu…

This trial tests a wearable bracelet called Stroke Alarm that detects when a stroke causes arm weakness. People aged 50 and older with certain high-risk conditions (like recent mini-stroke, atrial fibrillation, or artery disease) wear the device for 3 months. The goal is to see i…

This study investigates whether adding RNA sequencing to standard DNA sequencing can help identify genetic causes of neurodevelopmental disorders that also involve physical birth defects. Many children with these conditions go years without a clear diagnosis. The research compare…

This study aims to create a blood test that helps doctors quickly diagnose traumatic brain injury (TBI) and predict how patients will recover. Researchers will collect blood samples from 2,000 adults with suspected TBI in emergency rooms and hospitals. The goal is to find specifi…

This study tests a special dye that makes brain tumor cells glow under a camera during surgery. The goal is to help surgeons see and remove more of the tumor while sparing healthy brain tissue. About 46 adults with malignant glioma or vestibular schwannoma will receive the dye be…

This Phase 2 study tests whether a daily pill called EVO756 can prevent migraines in adults who have not had success with other preventive treatments. About 330 participants will receive either EVO756 or a placebo, and researchers will track how many migraine days they have each …

This study tests a laser treatment called OSC/SS to prevent retinal detachment in people with Stickler Syndrome, a genetic condition that raises the risk of vision loss. About 500 children and adults will receive the procedure in one or both eyes and be followed for 5 years. Rese…

This study tests whether a non-invasive brain stimulation technique called TMS, given within two weeks of a traumatic event, can prevent chronic PTSD. Researchers will measure changes in brain activity linked to fear and threat. The goal is to see if early intervention can reduce…

This study tests whether taking the sleep medication suvorexant (Belsomra) nightly for two years can slow the buildup of amyloid plaques in the brain, a hallmark of Alzheimer's disease. Researchers will enroll 200 healthy adults aged 65 and older with no signs of dementia. The go…

This phase 3 trial tests whether acoramidis can prevent or delay transthyretin amyloidosis (ATTR) in 587 adults who carry a faulty gene but have no symptoms yet. ATTR causes sticky plaques to build up in the heart and nerves, leading to heart failure and nerve damage. Acoramidis …

This study tests whether adding folic acid to iodized salt can raise folate levels in women of childbearing age in Zambia, a country without mandatory folic acid fortification. Two hundred fifty non-pregnant, non-lactating women aged 18–45 will use the fortified salt instead of r…

This study tests a new tablet called ALKS 2680 to see if it can reduce daytime sleepiness in adults with narcolepsy type 2. About 176 participants will receive either the study drug or a placebo for 12 weeks. The main goal is to measure how well they can stay awake using a standa…

This study compares two types of nerve blocks (paravertebral block and external oblique intercostal block) to standard pain medicine for people having gallbladder removal surgery. About 147 adults will be randomly assigned to one of three groups to see which approach reduces pain…

This study from Yale University will test whether 20 minutes of moderate cycling can reduce obsessive thoughts, compulsive urges, and improve thinking flexibility in adults with OCD. Fifty participants will be randomly assigned to either moderate or low-intensity cycling. Researc…

This study tests a smartphone app designed to help teenagers aged 14-18 who self-harm and are not currently in therapy. The app teaches coping skills and connects them with support through a friendly chatbot. The goal is to see if the app is easy to use and helps reduce self-inju…

This study tests an experimental oral drug called CYB003 in 220 adults with major depressive disorder. Participants receive either CYB003 or a placebo alongside their usual treatment. The goal is to see if CYB003 safely reduces depression symptoms better than placebo.

This study compares an online program called Veteran ACT for Chronic Pain (VACT-CP) to another online pain program, Online Pain School, for veterans with chronic pain. The goal is to see which program better helps veterans improve their daily functioning, manage pain, and enhance…

This study tests whether adding music-like sounds (sonification) to standard walking rehabilitation can improve gait, balance, and quality of life. 120 adults with Parkinson's disease, stroke, or multiple sclerosis will be randomly assigned to standard rehab or rehab with sonific…

This study looks at how sensory sensitivity affects sleep and emotions in autistic children ages 6-10. Researchers will test a simple bedtime routine called Power Down, where caregivers give their child a gentle massage to help them relax before sleep. The goal is to see if this …

This study investigates whether high-power laser therapy can reduce pain, improve nerve signals, and restore daily function in people with cervical radiculopathy—a condition where a nerve in the neck is compressed. Sixty adults aged 40 to 60 with confirmed nerve compression will …

This phase 3 trial tests whether a daily tablet called ALKS 2680 can reduce daytime sleepiness and cataplexy (sudden muscle weakness) in 150 adults with narcolepsy type 1. Participants will take the drug or a placebo for 12 weeks, and researchers will measure changes in sleep tes…

This study tests a program called CAPABLE Transplant, where an occupational therapist, nurse, and handy worker visit people at home over 4 months. The goal is to help kidney transplant candidates who are currently inactive on the waitlist become active again, and to improve their…

This study tests whether a home-based telerehabilitation program, using games, exercises, and video calls with therapists, can improve arm function and reduce disability in people who had a stroke 90-150 days ago. 202 participants will be randomly assigned to receive telerehabili…

This study tests two digital mindfulness programs—one live over Zoom and one self-paced app—to see if they can reduce depression in younger breast cancer survivors. It includes 402 women diagnosed at or before age 50 who finished cancer treatment at least 6 months ago and have el…

This study looks at whether daily growth hormone shots for 6 months can improve quality of life in veterans who have both mild traumatic brain injury and adult growth hormone deficiency. About 172 veterans aged 21-55 will receive either the hormone or a placebo. The main goal is …

This study tests whether spermidine, a natural substance found in foods like wheat germ, can improve sleep quality and memory in older adults with mild cognitive impairment (MCI), a condition that raises dementia risk. 76 participants aged 55-70 will take spermidine or a placebo …

This study tests a new way to treat post-traumatic stress disorder (PTSD) using personalized brain stimulation. Researchers use an EEG to find the right frequency for each person, then apply transcranial magnetic stimulation (TMS) to the frontal lobe. The trial includes 20 vetera…

This study tests a group therapy program called the Unified Protocol for adults with emotional disorders such as anxiety, depression, and related conditions. The program teaches skills like emotional awareness, flexible thinking, and facing fears. Researchers want to see if it im…

This study tests whether a four-session mindfulness class (Primary Care Brief Mindfulness Training) can help veterans reduce symptoms of anxiety, depression, and PTSD compared to a problem-solving class. The classes are designed for primary care settings and focus on meditation, …

This study tests a wearable device that sends gentle, low-frequency electromagnetic fields to the brain to help people who had a stroke more than 6 months ago recover arm movement and reduce disability. About 25 adults with chronic stroke will use the device in a clinic to see if…

This phase 2 trial tests whether injecting lidocaine and methylprednisolone into the middle meningeal artery can reduce severe headaches after a subarachnoid hemorrhage (a type of brain bleed). The procedure is done during a routine angiogram and aims to lower pain and opioid use…

This study tests whether a non-invasive device that stimulates a nerve in the ear can help people with fibromyalgia. Participants use the device for 30 minutes daily over four weeks. The goal is to see if it reduces pain, fatigue, and other symptoms by calming the body's stress r…

This study tests a new drug, elismetrep, to see if it can quickly stop migraine pain and bothersome symptoms like nausea or light sensitivity. About 1800 adults with frequent migraines will receive either the drug or a placebo. The main goal is to measure how many people are pain…

This study is testing whether a specially designed amino acid supplement can improve nutrition, reduce oxidative stress, and boost physical and mental health in people with Parkinson's disease. Thirty adults aged 60-80 with Parkinson's will take either the supplement or a placebo…

This study tests a medicated skin patch (dexmedetomidine) to see if it can reduce agitation in people with Alzheimer's dementia. About 150 adults living in care facilities will receive either the patch or a placebo for 96 hours. The goal is to measure changes in agitated behavior…

This study tests whether exercise paired with simple mental tasks (like counting backwards) can improve walking, balance, and thinking in people with multiple sclerosis. Forty adults with MS will do supervised workouts twice a week for 8 weeks. The goal is to find better rehab st…

This phase 3 trial tests whether the drug neridronate, given as four IV infusions over 10 days, can reduce pain and other symptoms of complex regional pain syndrome type 1 (CRPS-1). About 270 adults with CRPS in one limb will receive either the drug or a placebo. The main goal is…

This study tests the long-term safety of rimegepant, a migraine medication taken as a dissolving tablet, in children and adolescents aged 6 to 18. About 600 participants with a history of migraine will take the drug as needed for up to one year. The main goal is to check for side…

This pilot study tests whether a gentle, non-invasive electrical current applied to the brain can improve how people with tunnel vision (from retinitis pigmentosa, rod-cone dystrophy, or advanced glaucoma) navigate obstacles. Twenty adults will receive both real and sham stimulat…

This study tests whether a program that addresses chronic pain can help teens with obesity stay in weight management treatment. Fifty adolescents aged 12-18 with both obesity and musculoskeletal pain will either receive the Pain and Weight Treatment (PAW) plus standard care, or p…

This study is testing the long-term safety of a combination of two drugs, KarXT and KarX-EC, for treating agitation in people with Alzheimer's disease. It is an open-label extension, meaning everyone knows they are getting the treatment. The study enrolls 600 participants who hav…

This trial tests whether starting PTSD treatment with one type of talk therapy and then switching to another based on early progress leads to better symptom relief. About 430 adults receiving care at federally qualified health centers will take part. The study compares different …

This study looks at whether taking a second dose of the migraine medication rimegepant is safe and helpful for adults whose migraine pain isn't fully relieved by the first dose. About 400 people aged 18 and older with a history of migraines will take part. Researchers will track …

This study looks at whether the position of a neck muscle before dry needling changes how well the treatment works for chronic neck pain. About 80 adults with neck pain lasting over 3 months will be randomly assigned to have the muscle stretched or shortened during two needling s…

This study investigates whether wearing a virtual reality headset during epidural injections can lower pain and anxiety in people with nerve root pain (radiculopathy). Participants receive two injections, each with the headset, and researchers measure anxiety levels and pain scor…

This study tests whether a drug called MDMA, given alongside therapy, can help veterans with PTSD feel better. About 52 veterans will receive a dose of MDMA during therapy sessions, either alone or in a group. The goal is to see if this approach is safe and reduces PTSD symptoms.

This study tests whether a virtual reality training program can help people with cerebellar ataxia improve their reaching movements. Participants will use an Oculus Rift headset to practice reaching to virtual targets over several weeks. The goal is to see if this type of trainin…

This study tests three approaches—oxygen therapy, the drug trazodone, and brief low-oxygen episodes—to see if they can improve sleep apnea in people with chronic cervical spinal cord injury. Researchers will measure changes in breathing and sleep quality in 100 participants. The …

This study tests whether a brief form of talk therapy, called Prolonged Exposure for Primary Care (PE-PC), can reduce PTSD symptoms in first responders and frontline healthcare workers. Participants receive 4-6 weekly 30-minute sessions either by telehealth or in person, delivere…

This study tests whether a gentle movement program called Body Awareness Therapy (BAT) can help young adults (ages 20-30) who feel very stressed. Over 8 weeks, one group does BAT twice a week while another group does nothing new. Researchers will measure changes in balance, sleep…

This study tests whether yoga-based exercises can help veterans with schizophrenia, schizoaffective disorder, or bipolar 1 disorder improve their ability to function in daily life—like socializing, working, and managing leisure activities. About 112 veterans will be randomly assi…

This study tests an experimental drug called cleminorexton in 222 adults with narcolepsy type 1 or 2. The drug mimics a natural brain protein that helps keep people awake. The goal is to see if it safely reduces daytime sleepiness and improves daily functioning. Participants must…

This study tests whether a drug called psilocybin (the active ingredient in magic mushrooms) combined with therapy can help women with PTSD from sexual assault. About 70 women will receive the treatment in a controlled setting. The goal is to see if it reduces PTSD symptoms and i…

This study tests whether rimegepant, a fast-dissolving tablet, can safely relieve migraine pain in children and adolescents aged 6 to 18. About 2,100 participants will receive either the drug or a placebo during a migraine attack. The main goal is to see if more kids become pain-…

This study tests whether a mobile app with short weekly exercises can help first-time parents manage emotions and reduce depression during pregnancy. About 572 expectant mothers and fathers in Singapore will use the app or receive standard care. Participants will also wear a Fitb…

This phase 3 trial tests whether a combination of two drugs, KarXT and KarX-EC, can safely reduce agitation in people with Alzheimer's disease. About 352 adults will receive either the drug combo or a placebo for 14 weeks. The main goal is to see if agitation symptoms improve, as…

This study tests whether a smartphone app that teaches mindfulness can help reduce stress in people who have finished cancer treatment. About 345 cancer survivors will be randomly assigned to use the app right away or wait three months. The goal is to see if the app makes a real …

This trial tests whether adding either dexmedetomidine or magnesium sulphate to the standard numbing medicine bupivacaine improves pain control after chest surgery. About 60 adults with cancer undergoing thoracotomy will receive one of three nerve-block combinations. The main goa…

This study tests whether a non-invasive brain stimulation technique called HD-tDCS can improve visual and thinking abilities in people with posterior cortical atrophy (PCA), a rare condition often linked to Alzheimer's. Fifty participants will receive either real or sham stimulat…

This study tests whether a specially designed bright light can improve cognitive problems like memory and attention in people with major depressive disorder. 120 adults with depression and cognitive issues will receive either the bright light or a dim red placebo light for four w…

This trial tests a new anti-fog suction device that keeps the camera lens clear during nose-to-pituitary tumor surgery. Twenty adults with pituitary or craniopharyngioma tumors will either get the device or the usual water-squirt method. The goal is to see if the device reduces f…

This study tests a wearable neuroprosthesis that uses mild electrical stimulation to help stroke survivors open their hand. Two participants will use the device at home for one year to see if it improves daily activities and quality of life. The goal is to understand how well the…

This study tests whether the experimental drug ACP-204 can reduce psychosis symptoms like hallucinations and delusions in people with Lewy body dementia. About 180 adults aged 55 to 85 will take either ACP-204 or a placebo daily for 6 weeks. The goal is to see if the drug safely …

This study compares two types of nerve blocks for pain relief in women undergoing breast cancer surgery. One group receives a single-level block, while another receives a two-level block, with some patients also getting the medication dexmedetomidine. The goal is to see which app…

This study tests a wearable device that uses mild nerve stimulation to reduce symptoms of restless legs syndrome (RLS). Twenty adults with RLS will use the device at bedtime to see if it decreases nighttime awakenings and improves symptom severity. The goal is to find a safe, non…

This trial tests whether giving the painkiller parecoxib before a CT-guided wire placement can reduce pain during lung nodule localization for surgery. About 150 adults with lung cancer scheduled for video-assisted thoracoscopic surgery will receive either parecoxib or a placebo …

This study tests whether using the soundBrilliance app—which combines enhanced music, natural visuals, and spoken guidance—can help people aged 13 and older manage anxiety, depression, and stress. Over 12 weeks, participants use the app at least 4 days per week and report their m…

This Phase 3 study tests whether CYB003, a psilocin analog (related to the active ingredient in magic mushrooms), can safely reduce depression symptoms in 330 adults with major depressive disorder. Participants receive either one of two doses of CYB003 or a placebo, along with ps…

This study tests a new treatment called ACTIVE that combines counseling (Acceptance and Commitment Therapy) with balance exercises for people with mild traumatic brain injury. Researchers want to see if it helps reduce dizziness and anxiety better than standard balance therapy al…

This Phase 3 trial tests whether the drug combination KarXT + KarX-EC can safely reduce psychosis symptoms (like hallucinations and delusions) in people with Alzheimer's disease. About 325 participants aged 55-90 will receive either the drug or a placebo. The study is currently r…

This study tests whether clemastine, a common antihistamine, can improve brain white matter and make antidepressants work better for adults over 60 with depression. Eighty participants will receive either clemastine or a placebo alongside their antidepressant. The goal is to see …

This pilot study tests a wearable device called MedRhythms that uses shoe sensors and headphones to deliver real-time rhythmic cues (like a musical beat) to help people with incomplete spinal cord injury improve their walking. Fifteen participants will use the device during super…

This study explores whether a type of talk therapy called behavioral activation, delivered via video call, can help older adults who are involved with Adult Protective Services due to neglect or self-neglect. The therapy aims to reduce depression and apathy, and improve daily act…

This study tests whether taking photos of things related to well-being and discussing them with a therapist can help people with stress-related illness. Participants take pictures with their phone before sessions, and the therapist uses the photos to guide conversations about wel…

This study tests a program called Invest in Play (iiP) for parents of children aged 6-12 with problem behaviors. About 180 families will be randomly assigned to the program or a control group. Parents will fill out surveys to see if the program reduces challenging behaviors and i…

This pilot study tests whether a single session of breathing air with slightly higher carbon dioxide and lower oxygen can improve speech, cough, and movement in people with Parkinson's disease. Participants are randomly assigned to one of two breathing patterns, each lasting abou…

This study explores whether a mobile app called FlowMD can help adults with mild anxiety. Participants use the app for 1-5 minutes of guided breathwork each day for four weeks. Surveys measure changes in anxiety levels and how easy the app is to use. The goal is to see if this ap…

This study compares buprenorphine to a standard opioid (hydromorphone) for pain relief in adults aged 65 and older who come to the emergency department with a broken bone. Researchers want to see if buprenorphine works just as well but causes fewer side effects like nausea, confu…

This study tests an investigational drug called gemibotulinumtoxinA (a type of botulinum toxin) injected into arm muscles to reduce tremor in adults with essential tremor. About 94 participants will receive either the drug or a placebo injection. Researchers will measure changes …

This study tests whether ear acupuncture, led by village doctors and supported by telemedicine, can improve sleep in adults aged 65 and older who have both diabetes and high blood pressure. Participants will receive either real or sham ear acupuncture for 6 weeks. The goal is to …

This study tests whether wearing a headset that delivers a flickering 60Hz white light for 30 minutes a day can help ease depression symptoms. Adults with major depressive disorder will use the device at home for three weeks. Researchers will measure how well people stick with th…

This study tests whether combining home-based brain stimulation (tDCS) with virtual speech therapy can improve communication in adults with primary progressive aphasia, a language disorder often caused by Alzheimer's disease. Eighty participants will receive either active or plac…

This study tests whether osteopathic manipulative treatment (OMT) — a hands-on therapy using gentle techniques on the head, neck, and ribs — can improve sleep quality in people with Parkinson's disease. Participants are split into two groups: one receives OMT, the other a light t…

This study tests whether adding a crisis response plan (CRP) to prolonged exposure (PE) therapy helps reduce suicidal thoughts and behaviors in people with PTSD. Researchers will compare PE plus CRP to PE with standard care in 100 adults. The goal is to find a better way to suppo…

This study tests whether a safe, non-invasive brain stimulation technique called transcranial magnetic stimulation (TMS) can improve working memory in people with dementia. The stimulation is timed to each person's brain waves, measured by EEG, to help brain regions communicate b…

This study tests a new online therapy platform called Teleo, designed specifically for children with anxiety or mood disorders. Researchers want to see if kids are more engaged during therapy sessions on Teleo compared to standard video calls. About 156 children will take part, a…

This study tests a new drug, TAK-360, for adults with narcolepsy type 1, a condition causing severe daytime sleepiness and sudden muscle weakness (cataplexy). About 92 participants will receive either TAK-360 or a placebo to see if the drug is safe and well-tolerated. Participant…

This study tests whether a personalized form of transcranial magnetic stimulation (TMS) can improve motor and non-motor symptoms in people with Parkinson's disease, multiple system atrophy, or progressive supranuclear palsy. Fifty participants will receive 10 days of targeted TMS…

This study tests whether high-intensity walking therapy helps people with incomplete spinal cord injury walk better than standard rehab. About 25 adults who had a spinal injury within the past year will take part. Their walking speed, endurance, and daily activity will be measure…

This early-phase study tests whether MDMA-assisted therapy can reduce pain in 20 adults with fibromyalgia. Participants receive MDMA combined with psychotherapy and undergo brain scans to see how the drug affects pain and the bond between patient and therapist. The goal is to und…

This phase 3 study is testing an oral drug called DT120 (related to LSD) against a placebo in 165 adults with major depressive disorder. Participants will take the drug or placebo for 12 weeks, followed by a 40-week period where everyone can receive DT120. The main goal is to see…

This study tests whether a version of Dialectical Behavior Therapy (DBT) skills training, adapted for autistic adults, is feasible and helpful for those with emotion regulation difficulties. The program uses simplified language, more visuals, autism-relevant examples, and hands-o…

This study tests whether a noninvasive brain stimulation technique called tDCS can reduce depression in people with multiple sclerosis. Participants use a headband-like device at home for 30 minutes a day over 30 days, while listening to mindfulness meditation. Half receive real …

This study is testing if a non-invasive brain stimulation method called HD-tDCS can help people with ALS. The goal is to see if it can improve muscle control and breathing by calming overactive brain cells. The study will involve 80 adults with ALS and will measure changes in bra…

This study tests whether sitting in front of a bright lightbox for 60 minutes each morning can improve sleep and overall well-being in veterans who have traumatic brain injury, post-traumatic stress disorder, and chronic pain together—known as the polytrauma clinical triad. The t…

This small study tests if a non-invasive device that stimulates the brain during sleep can increase deep sleep in adults with depression. Researchers want to see if better deep sleep helps with memory, thinking, and mood. Participants wear a headband and activity tracker at home …

This study looks at whether a high-dose capsaicin patch (made from chili pepper extract) can relieve long-lasting tailbone pain that hasn't responded to other treatments. Researchers will review medical records from 106 patients treated over six years to see how well the patch wo…

This trial tests whether shining a specific type of near-infrared light on the forehead—called transcranial photobiomodulation (tPBM)—can improve attention and reduce impulsivity in adults with and without ADHD. Participants receive either active tPBM or a sham (fake) treatment f…

This study explores whether rhythmic auditory stimulation (like a metronome beat) can improve walking and brain function in people with Alzheimer's disease or mild cognitive impairment. Participants will walk while listening to rhythmic sounds, and researchers will measure change…

This study tracks about 1,000 people with migraine who are taking AJOVY (fremanezumab) as part of their regular care. The goal is to see how safe the drug is over the long term, especially regarding heart problems. Researchers will also collect information on how well it works. T…

This study investigates whether drinking plasma-activated water—water treated with light and nano gold particles to create smaller water clusters—can safely improve symptoms in people with fibromyalgia, a condition causing widespread pain. Researchers will measure changes in bloo…

This study compares two ways of delivering cognitive processing therapy (CPT) to pregnant women with posttraumatic stress disorder (PTSD). One group receives standard weekly therapy sessions for 12 weeks, while the other gets an intensive schedule of 10 sessions over just 5 days.…

This study looks at whether a person's personality before a brain injury or stroke influences how well they recover thinking and memory skills. Researchers will follow 250 adults with severe acquired brain injury or stroke, testing their cognitive abilities, daily functioning, an…

This study compares a new portable, ultra-low field MRI to standard hospital MRI for detecting brain diseases like stroke, tumors, and epilepsy. About 200 people (ages 3 and up) with neurological symptoms, plus healthy volunteers, will get both types of scans. The goal is to see …

This study investigates how fluid drains from the brain to lymph nodes in the neck, a connection that may be important for clearing waste linked to Alzheimer's disease. Researchers will inject a fluorescent dye into the meningeal space of recently deceased organ donors and track …

This study looks at how social and lifestyle factors—like education, race, and income—affect a person's thinking and memory after a stroke. Researchers will interview 450 adults who had a stroke and review their brain scans and medical records. The goal is to understand why some …

This early-stage study is testing whether a contrast agent called mangafodipir can safely highlight areas where the blood-brain barrier is broken in people with drug-resistant epilepsy. Forty adults will undergo MRI scans with mangafodipir and gadolinium to see if these scans can…

This study uses a non-invasive brain stimulation technique called transcranial magnetic stimulation (TMS) to examine how nerve pathways from the brain to the legs and trunk are affected in people with Parkinson's disease. Researchers will measure brain activity and link it to wal…

This study follows people infected with HTLV-I virus, some of whom develop a rare spinal cord condition called HAM/TSP. Researchers will track changes in symptoms, immune system markers, and virus levels over time. The goal is to better understand why only a few infected people g…

This study explores whether a small accelerometer worn on the thigh can objectively measure how well treatments work for neurogenic orthostatic hypotension—a condition causing dizziness and fainting upon standing. Participants wear the device for one week on placebo and one week …

This study at the National Institutes of Health looks at how infections can worsen symptoms in people with mitochondrial disease, a group of disorders that affect energy production in cells. Researchers will evaluate participants' immune systems through blood tests, physical exam…

This study from the National Human Genome Research Institute aims to better understand Congenital Disorders of Glycosylation (CDG), a group of rare genetic conditions that affect how the body attaches sugars to proteins and fats. Researchers will examine up to 200 participants, i…

This study aims to learn more about methylmalonic acidemia (MMA) and related disorders by observing people with these conditions over time. Researchers will track complications, perform tests like blood draws and MRIs, and look for new genetic causes. The goal is to better unders…

This study uses special PET scans to measure inflammation in the brains of people with Alzheimer's, Parkinson's, ALS, and other neurodegenerative diseases. Researchers will compare these scans to those from healthy volunteers to see if inflammation levels differ. The goal is to d…

This study follows 130 adults with moderate to severe traumatic brain injury to see if early problems with brain blood flow regulation are linked to thinking and memory problems one year later. Participants undergo brain scans and cognitive tests over the first year after injury.…

This study aims to find differences between organic tics (related to diagnosed disorders) and functional tics (caused by problems in brain-body communication). Researchers will use sensors to measure muscle activity and brain waves in 75 adults with tics and healthy volunteers. P…

This study investigates the sequence of early warning signs, called premonitory symptoms, that occur before a migraine headache starts. Researchers will follow 500 adults with migraine who will record their symptoms using an electronic diary for up to five attacks. The goal is to…

This study is testing whether a skin biopsy can detect abnormal proteins linked to Parkinson's disease in people with essential tremor, a common movement disorder. Researchers will follow 300 participants aged 50-85 for two years to see who develops Parkinson's. The goal is to fi…

This study aims to learn more about rare inherited disorders that affect the brain, spinal cord, muscles, and nerves. Researchers will collect medical history, perform exams, and run genetic tests on up to 3,500 participants. No new treatments are tested; the goal is to better un…

This long-term observational study uses MRI scans to track how multiple sclerosis (MS) changes over time in up to 3750 people. Researchers will compare brain images and immune system markers in untreated patients, those on approved therapies, and healthy volunteers. The goal is t…

This study uses yearly PET scans to track amyloid protein buildup in the brains of 400 adults aged 55 and older over 5 years. Participants are already part of the Baltimore Longitudinal Study of Aging. The goal is to see how these brain changes relate to memory loss and other thi…

This study aims to collect movement data from up to 7,500 people, including healthy volunteers and those with movement disorders. Participants perform tasks like walking or reaching while cameras track reflective markers on their body. The goal is to build a reference database th…

This pilot study looks at how belzutifan, a drug already used for some tumors, changes the production of adrenaline-like hormones in people with pheochromocytoma and paraganglioma (PPGL). Over 5 days, researchers measure key substances in the hormone-making process to see if belz…

This study follows 5000 people admitted to a neuro-intensive care unit with life-threatening brain conditions like bleeding in the brain or severe seizures. Researchers collect medical data and biological samples to better understand how brain injuries progress and what factors h…

This study enrolls people with endocrine tumors (such as thyroid, parathyroid, adrenal, or pancreatic tumors) who are scheduled for surgery or biopsy. Researchers collect samples of the tumor, nearby healthy tissue, blood, and urine to analyze genetic and molecular changes. The g…

This study aims to uncover genetic and environmental risk factors for hemorrhagic stroke, a type of stroke caused by bleeding in the brain. Researchers will analyze DNA, RNA, and blood samples from 1,000 adults who have had such a stroke, comparing them to healthy controls. By id…

This study follows 155 adults with CADASIL, a genetic condition that narrows small blood vessels and raises the risk of strokes and dementia, over nine years. Researchers will use brain scans, blood tests, and other exams to track how the disease changes blood vessels and thinkin…

This study looks at how people with certain types of brain damage process what they see and pay attention. Researchers will compare them to healthy volunteers using brain scans like fMRI and MEG. The goal is to better understand which brain areas are involved in visual attention.

This study looks at how stroke survivors with weakness on one side (hemiplegia) perceive internal body signals, like heartbeat, and whether that relates to their balance, touch sensation, and joint position sense. Researchers will assess 60 participants in a single session using …

This study investigates whether brain imaging can predict how well children with cochlear implants will develop language skills. Researchers are building models to forecast long-term language outcomes in English- and Spanish-speaking children. They are also testing a parent-led c…

This study works with over 126,000 adults and clinic staff to improve how primary care clinics identify and accommodate patients with communication disabilities, such as speech or hearing problems. Researchers will test tools to help clinics record these needs in electronic healt…

This study is looking for ways to predict which migraine patients will respond well to a class of drugs called anti-CGRP monoclonal antibodies. Researchers will analyze clinical, biological, and brain imaging data from 350 adults with frequent migraines. The goal is to find bioma…

This observational study is recruiting 2400 people with immune-related central nervous system diseases, such as multiple sclerosis, along with healthy volunteers for comparison. Researchers will use MRI scans, lumbar punctures, blood tests, and neurological exams to track disease…

This study uses advanced brain scans (MRI and MEG) to better locate seizure-causing areas in people with epilepsy who are considering surgery. Researchers will also create a database of normal brain images from healthy volunteers. The goal is to improve surgical outcomes by compa…

This study investigates whether the bacteria causing Lyme disease can survive antibiotic treatment and cause ongoing symptoms. Researchers will follow 700 participants, including those with chronic Lyme disease, recovered individuals, and healthy volunteers, using blood tests, sp…

This study uses PET scans to measure levels of COX-1 and COX-2, enzymes linked to inflammation, in the brains of people with major depressive disorder (MDD) during a depressive episode. Researchers will compare these levels between MDD participants and healthy volunteers. The goa…

This study follows 500 people with geographic atrophy, an advanced form of age-related macular degeneration that causes permanent vision loss. Participants receive avacincaptad pegol (IZERVAY) as part of their routine care. Researchers will collect data on treatment patterns, sid…

This study investigates how depression and antidepressants influence the way older adults update their beliefs about future events. Researchers compare 31 patients aged 65+ with major depression who are switching antidepressants to 31 healthy volunteers of the same age. Participa…

This study aims to see if people with major depressive disorder have lower levels of an enzyme called PDE4B in their brains compared to healthy volunteers. Researchers will use PET and MRI scans along with an experimental radioactive tracer to measure this enzyme. The study invol…

This international observational study will follow 50,000 people with Alzheimer's disease or related dementias for many years. Researchers will collect real-world data from routine doctor visits to track how the disease progresses and how treatments are used and tolerated. The go…

This study explores whether migraine attacks can be predicted before the headache phase begins. Researchers will monitor people with episodic migraine (10–14 migraine days per month) using a wearable device that tracks heart rate and other body signals, along with a diary of earl…

This study tests a new radioactive tracer to see if it can measure brain inflammation in people with Alzheimer's disease or mild cognitive impairment. Researchers will compare scans from 90 volunteers (patients and healthy older adults) to understand how inflammation relates to m…

This study aims to learn more about the genetics and natural history of autoinflammatory diseases, which cause repeated fevers and inflammation. Researchers will study up to 5,000 people, including patients, their relatives, and healthy volunteers. Participants provide blood and …

This study collects extra blood, urine, and tumor samples from children and adults with certain cancers to build a large tissue repository. Researchers will analyze these samples to learn more about the genetic and molecular makeup of these tumors. The goal is to support future s…

This study will carefully examine the physical and mental health of 100 adults admitted to NHS mental health wards. Researchers will review medical notes, do physical exams, and use questionnaires to measure conditions like bipolar disorder, schizophrenia, depression, and persona…

This study looks at how exercise training impacts blood vessel function in people with PTSD or generalized anxiety disorder, compared to healthy individuals. Researchers want to understand if harmful substances called oxidants play a role in blood vessel damage. The study involve…

This early-phase trial tests a special version of the immunotherapy drug nivolumab that has a fluorescent dye attached (nivo800). The dye makes the drug glow under near-infrared light, allowing researchers to see exactly where the drug goes in the tumor during surgery. About 38 a…

This study looks at whether parents of people with bipolar II disorder or major depressive disorder have certain personality traits (called Type D, which involves negative emotions and social inhibition) and lower life satisfaction. Researchers compare these parents to parents of…

This study follows adults who have recently received a cochlear implant to track how their ability to distinguish sounds and understand speech changes over time. Participants complete at-home sound tests frequently during the first three months, then return for lab tests up to on…

This study looks at how well hospitals manage severe oxygen deprivation (asphyxia) in newborns at or near full term. Researchers will review care given during labor and after birth to find where improvements can be made. The goal is to help doctors and nurses provide better, fast…

This study follows 450 babies born with serious heart defects to find early signs of developmental delays by 6 months of age. Researchers want to understand why over half of these children face learning, thinking, or behavior challenges later in life. The goal is to identify risk…

This study aims to enroll 50,000 adults aged 45 and older in France to test for a gene called APOE, which is linked to Alzheimer's risk. Participants provide a saliva sample at home and complete online questionnaires. The goal is to build a large registry of genetic and health da…

This study aims to learn more about heart and blood vessel diseases by observing people who have them or are at risk. Researchers will collect samples and perform tests like imaging and stress tests on up to 5,000 participants, including healthy volunteers and relatives of affect…

This study compares brain responses to heat, cold, brushing, and mild electric shocks in people with and without intellectual disability. Researchers use brain scans and heart rate monitors to measure pain. The goal is to better understand how pain is felt in this group, especial…

This study looks at MRI scans of 128 people who have had a brain hemorrhage (bleeding in the brain). Researchers want to see if a specific contrast pattern called a 'spot sign' can help predict which bleeds might get worse. By understanding these patterns, doctors hope to identif…

This study looks at whether anxiety or depression makes asthma harder to control and aims to build a model that predicts when a serious asthma attack might happen. Researchers will follow 256 people with asthma for up to two years, tracking their symptoms, mental health, and lung…

This study tracks the safety of Fycompa injection in people with epilepsy who have partial-onset seizures (age 4 and older) or primary generalized tonic-clonic seizures (age 12 and older). Researchers will record any side effects and measure drug levels in children aged 4 to 12. …

This study follows people with rare genetic disorders that cause harmful substances to build up in the body, leading to brain damage. Researchers will monitor up to 200 participants over time using exams, surveys, and lab tests. The goal is to better understand how these diseases…

This study investigates whether the quality of parent-adolescent communication at the start of psychiatric inpatient treatment predicts how much symptoms improve six months later. Researchers will track 60 adolescents hospitalized for depression, anxiety, or other mental disorder…

This study will test how the heart drug empagliflozin is absorbed and processed in 10 children with Duchenne muscular dystrophy, ages 8 to 18. The goal is to find the best dose for protecting the heart in this rare disease. Participants will take the drug by mouth and have blood …

This study looks at how blood pressure changes in the first year after a spinal cord injury and whether a device that stimulates the spine through the skin can help. Five people with recent injuries will be followed for a year, testing the stimulation during sessions to see if it…

This study investigates how social information—like knowing a task will be easy or hard—affects brain activity in people with epilepsy. Participants, who already have electrodes implanted in their brains for medical reasons, will rate their doctors and then complete tasks involvi…

This study will compare levels of fatty acid binding proteins and cellular aging markers in 120 cancer survivors versus people who never had cancer. Researchers want to see if these markers are higher in survivors and linked to conditions like diabetes and high blood pressure. Pa…

This study evaluates a questionnaire called the Emotional Allodynia Questionnaire (AEQ), which measures how people with fibromyalgia react emotionally to everyday social cues. Researchers want to see if the tool can reliably identify fibromyalgia and tell it apart from other type…

This study is following children and adults with olfactory neuroblastoma, a rare cancer of the nasal cavity, to learn more about how the disease behaves over time. Researchers will collect medical records, blood samples, and survey responses from 525 participants to track symptom…

This study looks at whether keeping a patient's blood pressure within their personal safe range during heart surgery can reduce brain problems like confusion, stroke, or memory issues. Researchers will monitor 500 patients having heart surgery and track their blood pressure and b…

This study creates a large registry of people with Rett syndrome, a rare genetic disorder that causes severe intellectual and physical disabilities. Researchers will collect information from doctors and caregivers to understand how the disease progresses over time. The goal is to…

This study is creating a large collection of health information and biological samples (like blood, saliva, and eye tissue) from people with and without retinal diseases. Researchers aim to use this database to better understand conditions like age-related macular degeneration an…

This study aims to understand what happens in the brain when someone thinks about or attempts suicide. Researchers will use brain scans, sleep monitoring, blood tests, and questionnaires to compare four groups: people with recent suicidal thoughts or attempts, those with past sui…

This study screens males with Duchenne muscular dystrophy (ages 0 to under 25) for antibodies against AAV8, a virus used in some gene therapies. The goal is to find out how many have these antibodies and to identify who might be eligible for future gene therapy trials. No treatme…

This study looks at how the brains of children with anxiety disorders work differently from healthy children. Researchers will use brain scans and thinking tasks to track changes over a year, before and after standard treatments like therapy or medication. The goal is to find pat…

This study aims to create a risk score that uses information from electronic medical records to predict the chance of developing dementia within 5, 9, or 13 years in older adults with depression. Researchers will analyze data from 44 participants aged 50 and older who have a depr…

This study screens 360 adults with neurodegenerative disorders like ALS, frontotemporal dementia, and progressive supranuclear palsy. Researchers will use medical history, physical exams, memory tests, movement analysis, MRI scans, and other tests to find common features and diff…

This study explores whether a computer algorithm can analyze resting-state MRI scans taken before surgery to predict how long a person with glioblastoma multiforme (a type of brain cancer) might survive. The goal is to see if the algorithm can accurately classify patients as shor…

This study will follow 1,000 people receiving Medtronic spinal or cranial devices during routine surgery. Researchers will track outcomes like fusion success, pain relief, and device safety for up to two years. The goal is to gather real-world data on how these already-approved p…

This study looks at tongue strength and endurance in people who have had part of their tongue removed due to oral tongue cancer. Researchers will use a device called the Iowa Oral Performance Instrument (IOPI) to measure tongue pressure. Some participants will also use the IOPI a…

This study at the National Institutes of Health is looking at 1,000 adults with known or suspected hormone gland (endocrine) disorders. The goal is to better understand these conditions and help train doctors. Participants may receive tests, treatments, or surgery as part of thei…

This study looks at why people with tuberous sclerosis, a rare genetic condition, develop non-cancerous skin tumors. Researchers will examine up to 400 adults, perform skin exams, and take small tissue samples (biopsies) from tumors. The goal is to identify the cells and genetic …

This study aims to understand how heart problems develop in people with Duchenne and Becker muscular dystrophy, as well as in carriers. Researchers will collect cardiac MRI scans and clinical data from 1,000 participants to build a registry. Using advanced image analysis and deep…

This study uses safe brain imaging techniques (NIRS and EEG) to see how the brain controls movement in people with and without cerebral palsy. Researchers will compare brain activity patterns and test whether biofeedback training can help people with cerebral palsy improve their …

This study tests a lab technique called pharmacoscopy that quickly checks which drugs work best on a patient's own brain metastasis tissue. About 102 adults with brain metastases and few treatment options will have their tumor samples tested. The goal is to see if using this test…

This study follows 500 adults already receiving care for depression, bipolar disorder, or suicide risk at the NIH. Researchers will conduct weekly interviews and optional tests like blood draws and brain scans to track symptoms and treatment responses. The goal is to better under…

This study explores whether planning radiation therapy to avoid key brain regions can reduce thinking and learning problems in children with brain or head and neck tumors. Participants include children receiving radiation, survivors who completed radiation at least two years ago,…

This study enrolls 600 adults with Huntington disease to examine specific genetic markers (SNPs) linked to the disease. Participants provide a blood sample and answer questions about their health and medications. The goal is to better understand the genetics of Huntington disease…

This study looks at how ongoing stress, anxiety, and poor sleep might affect the course of pancreatic cancer. Researchers will ask 320 participants to fill out simple questionnaires about their mental well-being and will also measure stress markers in leftover blood samples. The …

This study investigates how depression and cannabis use together affect brain circuits involved in reward and pain in young people living with HIV. Researchers will use MRI scans and questionnaires to measure brain activity, mood, and substance use. The goal is to understand why …

This study looks at children under 15 with neurofibromatosis type 2 (NF2), a rare genetic condition that causes nerve tumors. Researchers want to see if skin and mouth lesions can help diagnose NF2 earlier than current methods, which rely on hearing, nerve, and eye symptoms. By e…

This study is building a huge registry of information and biological samples from adults with or at risk for many types of cancer, as well as healthy volunteers. Researchers will use this resource to study what causes cancer and how to better prevent, detect, and treat it. The re…

This study will check if certain blood markers can predict how well a nerve block procedure works for people with nerve pain. Researchers will measure pain levels and blood inflammation markers before and after the procedure in 120 adults. The goal is to see if these blood tests …

This study is testing a new leg exoskeleton designed by the NIH to help children and young adults with muscle weakness from conditions like cerebral palsy or spinal cord injury. Researchers want to see how well the device works in different real-world settings, like walking on a …

This research study creates a registry of older adults with cancer to collect information about their health, treatments, and outcomes. Participants complete a geriatric assessment and allow researchers to follow their medical records. The goal is to better understand how cancer …

This study uses MRI scans to look at brain and spinal cord structure and function in people with cerebral palsy who experience chronic pain. Researchers hope to find patterns in the brain that are linked to pain and sensory issues. Participants aged 8 and older with cerebral pals…

This study investigates whether older adults with age-related hearing loss (presbycusis) can still hear and enjoy the sound of a crunchy biscuit while eating. Researchers will compare a crunchy biscuit with a soft one in 100 seniors aged 65 and older, asking them if they hear the…

This study creates a registry of medical records, scans, and surveys from adults with neurofibromatosis type 1 (NF1) who also have a glioma brain tumor. Researchers will track diagnosis, treatments, and quality of life over time to better understand the disease. The goal is to im…

This study aims to better understand disorders that cause hearing to fluctuate, such as Meniere's disease. Researchers will use advanced MRI scans and blood tests to look for biomarkers linked to hearing changes. The goal is to identify different types of these disorders and pave…

This study tests whether advanced MRI scans that map the brain's structural and functional connections can better predict neurological recovery in coma patients after cardiac arrest than standard methods. Researchers will scan 263 adults who remain unconscious at least 72 hours a…

This study examines whether a person's personality traits before they got sick are linked to the thinking and behavior problems seen in certain brain disorders. Researchers will ask caregivers about the patient's personality from the 10 years before the illness started. The goal …

This study looks at how the location of brain-stimulating electrodes affects pain relief in adults with long-term nerve pain that hasn't improved with other treatments. Researchers will use brain scans to see which areas are activated and how that relates to pain reduction. The g…

This study observes critically ill children in the ICU who are already undergoing continuous EEG monitoring to detect brain seizures. Researchers aim to identify which children are most at risk for seizures and evaluate the safety of current seizure management strategies. The goa…

This study gathers information from memory clinics across France to track how many people have Alzheimer's disease, how it progresses, and what factors affect diagnosis and care. Researchers will analyze anonymous medical records from over 500 centers to spot trends in health and…

This study explores how a technique called Temporally Interfering Electric Fields Stimulation (TIEFS) affects brain activity and thinking. TIEFS uses electrodes on the scalp to send electrical currents that can reach deep brain areas without surgery. Researchers will test it in a…

This study investigates how rehearsal (repeating information) and sleep help solidify memories in people with epilepsy. Participants learn object-location pairs, with some pairs rehearsed and others not, and their memory is tested after a short delay and again after 12 hours—eith…

This study aims to create a smartphone system that sends personalized pain management tips to adults with multiple sclerosis or spinal cord injury who have chronic pain. Researchers will interview participants to understand their preferences for the content and timing of these no…

This study investigates why some adolescents act impulsively when experiencing negative emotions, a trait called negative urgency. Researchers will measure brain cell communication using transcranial magnetic stimulation and EEG in teens aged 13-21 with depression and suicidal th…

This study follows children and adults worldwide who have a rare genetic muscle disease called TNNT1 myopathy. Researchers aim to document how the disease progresses over time, including survival and motor milestones. The goal is to identify reliable measures that can be used in …

This study looks at whether certain proteins in the blood can help doctors monitor severe traumatic brain injury and predict who might benefit from hyperbaric oxygen therapy. Researchers will analyze blood samples from 150 patients already enrolled in a larger trial testing diffe…

This study compares three types of exercise programs—traditional physical activity, virtual reality, and video game-based—to see how they affect physical fitness and attention in healthy adolescents aged 10-18. Participants will be split into three groups and follow their assigne…

This study follows 1,200 families who already have a child with autism (high-risk group) and 500 families from the general population (low-risk group) starting from early pregnancy. Researchers collect biological samples and survey data to explore how genetics, environment, diet,…

This study investigates whether the emotional distress of parents—such as depression, anxiety, or trauma—affects how well children with recurrent medulloblastoma follow their treatment plans. Researchers will follow 450 parent-child pairs across four hospitals, measuring parental…

This study follows 50 children who had a nerve-freezing procedure (cryoneurolysis) before surgery to correct a sunken chest (pectus excavatum). Researchers check for long-term nerve pain, quality of life, and sensory recovery using questionnaires. The goal is to see if the pain r…

This study investigates whether a clinical test using lorazepam (Ativan) and brain imaging can identify veterans with PTSD who might benefit from medications that boost GABA signaling. Researchers aim to validate a computational method to distinguish subgroups of PTSD patients ba…