Disease of genetic or genomic mechanism

This early-phase trial tests whether a bone marrow transplant from a partially matched relative can safely cure chronic granulomatous disease (CGD), a condition that causes severe infections and inflammation. Four people aged 4 to 65 with CGD and no perfect donor will receive che…

This study tests a new drug called elafibranor in people with primary biliary cholangitis (PBC), a rare liver disease that can lead to scarring and liver failure. Participants either did not get enough help from or could not take the standard medicine. The goal is to see if elafi…

This study tests a combination of three drugs—ipilimumab, nivolumab, and brentuximab vedotin—in people with Hodgkin lymphoma that has returned or not responded to treatment. The drugs work by helping the immune system attack cancer cells and by delivering a toxin directly to them…

This study looks at the long-term safety of a daily pill called lucerastat for adults with Fabry disease, a rare genetic disorder. About 107 people who completed a previous study will take the drug and be monitored for side effects over several years. The goal is to see if lucera…

This study tests whether adding a targeted drug called ganitumab to standard chemotherapy helps people with newly diagnosed Ewing sarcoma that has spread. The trial includes 312 participants and aims to see if the combination improves survival without the cancer growing back. The…

This first-in-human study tests an experimental oral drug called IDRX-42 in 278 adults with advanced gastrointestinal stromal tumors (GIST) that have spread or cannot be removed by surgery. The main goals are to check safety, find the right dose, and see if the drug can shrink tu…

This expanded access program provides tazemetostat, a drug that blocks a protein called EZH2, to adults with serious or life-threatening solid tumors that have lost the INI1 or SMARCA4 protein. Eligible patients must have tried other treatments without success and cannot join a f…

This study tests a combination of two drugs—venetoclax and vincristine—in people with T-cell or B-cell acute lymphoblastic leukemia that has returned or not responded to prior treatment. The goal is to find the safest dose and see if the combo can lead to remission. About 40 adul…

This study follows patients who have already been taking ruxolitinib alone or with panobinostat in earlier trials and are still benefiting. Researchers will track side effects over time to see how safe these drugs are for long-term use. About 296 participants with conditions like…

This phase 3 trial is testing whether adding the immunotherapy drug atezolizumab to standard chemotherapy can help prevent cancer from coming back in people with stage III colon cancer that has a specific DNA repair defect (dMMR). About 712 participants will receive either chemo …

This phase 2 trial tests whether a daily pill called miransertib can slow or stop the abnormal tissue overgrowth seen in Proteus syndrome, a rare genetic condition. About 38 people aged 3 and older will take the drug for up to 4 years. Researchers will measure changes in foot ove…

This phase 3 trial compares two immunotherapy drugs—nivolumab (Opdivo) and brentuximab vedotin (Adcetris)—when added to standard chemotherapy for people aged 12 and older with newly diagnosed stage III or IV classic Hodgkin lymphoma. The goal is to see which combination better de…

This study tests a daily pill called elafibranor in about 18 Japanese adults with primary biliary cholangitis (PBC), a rare liver disease that can lead to scarring and liver failure. Participants had not responded well to standard treatment. The main goal is to see if the drug im…

This study tests a high-dose stem cell transplant for people with chronic granulomatous disease (CGD), a condition that weakens the immune system. Participants receive donor stem cells along with drugs to prevent rejection and graft-versus-host disease. The goal is to improve tra…

This study follows 165 children who previously took dabrafenib and/or trametinib for certain brain tumors. Researchers want to see if these drugs are safe over the long term and how they affect growth. The children continue their treatment and are monitored for side effects and d…

This Phase 2 trial tests a combination of pembrolizumab (Keytruda) and quavonlimab against other drug combos in 302 people with stage IV colorectal cancer that has a specific genetic feature (MSI-H or dMMR). The goal is to see if the new combo shrinks tumors better and for longer…

This study tests a surgical technique to restore the natural curve (lordosis) of the lower spine in people who have had a previous spinal fusion that failed or caused ongoing pain. The procedure is done through the same incision used in the prior surgery. Researchers will measure…

This study tests the long-term safety and effectiveness of an experimental drug called BBP-418 (ribitol) in people with limb-girdle muscular dystrophy type 2I/R9, a rare genetic muscle-weakening disease. Participants who completed a previous study will take BBP-418 orally twice d…

This study tests a daily medication called filgotinib (200 mg) in about 94 Korean adults with moderate-to-severe ulcerative colitis. The main goal is to see if it can bring the disease into remission (no active symptoms) by week 10 or 22. Participants take the pill once daily and…

This study tests the safety of a drug called ASP3021 (avacincaptad pegol) for people in Japan with geographic atrophy, an advanced form of age-related macular degeneration that causes vision loss. Twenty participants will receive monthly injections into the affected eye for 12 mo…

This phase 2 trial compares two standard CML drugs—imatinib (Gleevec) at two different doses and dasatinib (Sprycel)—in 406 newly diagnosed patients with chronic phase CML. The main goal is to see which treatment leads to the best molecular response after 12 months, meaning a ver…

This study tests whether elafibranor, a daily pill, can normalize alkaline phosphatase (ALP) levels in adults with primary biliary cholangitis (PBC) who did not respond well to or cannot take standard treatment. About 69 participants will receive either elafibranor or a placebo f…

This study tests a drug called triheptanoin, already approved for similar conditions, to see if it can prevent dangerously low blood sugar in people with MCADD, a rare inherited disorder. About 24 participants aged 4 and older will take the medication and be monitored for safety …

This study is testing a new hip implant called the OR3O Dual Mobility System in 177 people who need hip replacement surgery. The goal is to see if the implant is safe and lasts longer without needing another surgery. Researchers will follow participants for up to 10 years to chec…

This study tests whether MiSight 1 Day contact lenses can slow the progression of myopia (nearsightedness) in children aged 8 to 12. Participants wear either MiSight 1 Day or standard Proclear 1 day lenses for three years, then all switch to Proclear for one year to see if the ef…

This study tests whether a simpler two-drug pill (dolutegravir/lamivudine) works as well as a standard three-drug pill (bictegravir/emtricitabine/tenofovir alafenamide) for adults with HIV who have never been treated. About 473 participants will take one of the two pills daily fo…

This study tests a two-step treatment for people with relapsed or refractory B-cell Non-Hodgkin lymphoma. First, patients receive a bispecific antibody therapy (Obinutuzumab and Glofitamab) to help the immune system attack cancer cells. Then, they get an infusion of CD19-CAR T ce…

This study tests a device that uses heat to ablate (destroy) a small part of the ovary in women with polycystic ovary syndrome (PCOS) who have trouble getting pregnant. The goal is to restore ovulation without needing daily medication. About 195 women will be randomly assigned to…

This study tests a new approach for people receiving a stem cell transplant from a partially matched family donor. The donor's immune cells (T cells) are modified in the lab to include a 'suicide gene' that can be activated by a drug if the cells attack the patient's body, causin…

This early-stage trial is testing a drug called vixarelimab in 24 people with moderate to severe ulcerative colitis who have not improved with standard treatments. The study looks at how the drug affects the gut, how it moves through the body, and whether it is safe. It is too ea…

This Phase 3 trial compares the drug niraparib to the standard chemo temozolomide in 450 adults with newly diagnosed, aggressive glioblastoma that has a specific genetic marker (MGMT unmethylated). Participants take the assigned drug daily during radiation, then continue until th…

This study tests whether special spectacle lenses called DIMS (defocus incorporated multiple segment) can slow the progression of nearsightedness (myopia) in university students aged 18 to 23. Participants wear either the DIMS lenses or standard single-vision glasses for comparis…

This study investigates whether combining the medication lovastatin with intensive reading tutoring can improve reading abilities in children and young adults (ages 8-20) with Neurofibromatosis Type 1 (NF1) who have reading disabilities. Participants first receive either lovastat…

This study compares two types of implants used in spinal fusion surgery: 3D-printed cages and titanium-coated cages. The goal is to see which leads to better bone fusion and less disability for patients with herniated discs or degenerative spine disease. About 78 adults undergoin…

This study looks at the long-term safety of a drug called iptacopan for adults with a kidney disease called primary IgA nephropathy. Participants have already completed earlier studies and will continue taking the drug to monitor side effects and see if it helps slow kidney damag…

This study tests a combination of two drugs, ixazomib and rituximab, in people with slow-growing B-cell non-Hodgkin lymphoma. The goal is to see how well the drugs shrink or control the cancer. About 33 adults with different types of this lymphoma are taking part.

This pilot study tests whether injections of platelet-rich plasma (PRP) made from a patient's own blood can help preserve vision in people with retinitis pigmentosa or a related condition called EMAP. Thirty participants receive three injections over four months, and researchers …

This study is testing a new device called Virtuoso for eye surgeries like cataract removal and vitrectomy. The goal is to make sure it is safe and works well. About 205 adults who need these surgeries will take part. The study will compare how often problems happen during surgery…

This Phase 2 trial tests an experimental drug called REGN7999 in about 95 people with non-transfusion dependent beta-thalassemia who have too much iron in their bodies. The drug is given as a shot under the skin and aims to safely reduce iron levels in the liver. Researchers will…

This phase 2 trial tests whether dapagliflozin, a drug already used for diabetes and chronic kidney disease, can slow kidney damage in people with lupus nephritis. Lupus nephritis is a serious complication of lupus that causes kidney inflammation and can lead to chronic kidney di…

This trial tests whether specially modified T cells from a partially matched donor can help patients recover their immune system faster after a stem cell transplant. The T cells are engineered with a 'suicide gene' that allows doctors to destroy them if they cause graft-versus-ho…

This study tests an injectable drug called TEV-53408 in 50 adults with celiac disease who follow a gluten-free diet. Participants will be exposed to small amounts of gluten to see if the drug can prevent intestinal damage. The trial is placebo-controlled and lasts about 86 weeks,…

This phase 2 trial tested the drug ivosidenib in children and young adults (ages 1 to 21) with solid tumors, lymphomas, or histiocytic disorders that have an IDH1 mutation and have either come back after treatment or not responded. The goal was to see if the drug could shrink or …

This phase 3 trial tests a single dose of CTX001, a CRISPR-edited stem cell therapy, in 13 children with severe sickle cell disease who have not responded to standard treatments. The goal is to see if it can prevent severe pain crises for at least a year. Participants receive the…

This study investigates whether injecting platelet-rich plasma (PRP) from a person's own blood can improve mild to moderate eyebrow thinning. Participants receive either PRP or saline injections into the eyebrows, and changes are tracked through photographs over nine months. The …

This study tracks adults with hemophilia A who choose to take Jivi, a longer-lasting clotting factor that requires fewer injections. Researchers will use ultrasound to see if joint health stays the same or improves after 24 months of treatment. The goal is to learn how well Jivi …

This study tests an experimental drug called ORKA-001 in 80 adults with moderate-to-severe plaque psoriasis. Participants receive either ORKA-001 or a placebo injection, and doctors measure how much skin clears up after 16 weeks. The goal is to see if ORKA-001 is safe and effecti…

This early-phase study tests whether azelaic acid gel, a topical medication already used for acne, can help women with central centrifugal cicatricial alopecia (CCCA) — a type of scarring hair loss that mainly affects Black women. Eighteen women will use the gel on their scalp on…

This study tests a precise radiation method called total marrow and lymphoid irradiation (TMLI) given with two chemotherapy drugs (etoposide and cyclophosphamide) before a donor stem cell transplant. The goal is to safely deliver higher radiation doses to the bone marrow to wipe …

This phase 3 trial tests a single dose of CTX001, a CRISPR gene therapy made from the child's own blood stem cells, for children with transfusion-dependent beta-thalassemia. The goal is to help them become transfusion-free for at least 12 months. Sixteen children will receive the…

This trial investigates a new type of immunotherapy called CAR T-cell therapy that targets two proteins, CD19 and CD22, found on B-cell leukemia cells. The therapy is given along with chemotherapy to children and young adults whose leukemia has come back or has not responded to s…

This study tests whether a stem cell transplant from a matched sibling, unrelated donor, or half-matched parent can fix the immune system in people with DOCK8 deficiency, a genetic condition that causes severe infections and cancer. Participants aged 4 to 35 receive chemotherapy …

This early-stage study tests a new lab-made antibody called VH4527079 in healthy adults and people with HIV. The goal is to check if it is safe and how the body processes it when given as a single shot or multiple infusions. About 102 participants aged 18-55 will take part. This …

This study looks at the long-term safety and effectiveness of the drug combination Trikafta (elexacaftor/tezacaftor/ivacaftor) in people with cystic fibrosis who have certain non-F508del gene mutations. About 297 participants who completed a prior study will continue taking the m…

This study tests a new oral solution called NPI-001 in people with cystinosis, a rare disease that causes cystine buildup in cells. Researchers want to see if NPI-001 is safe and works better than the current drug cysteamine. About 12 participants aged 10 and older will stop thei…

This study tests a gene therapy called PBKR03 for babies with early infantile Krabbe disease, a severe genetic disorder that damages the brain and nerves. The treatment delivers a working copy of the GALC gene to the brain and body. Researchers will check safety and find the best…

This early-stage trial tests a single injection of RB001, a gene therapy, directly into the fluid around the brain of children with Phelan-McDermid syndrome caused by SHANK3 gene changes. The study includes up to 8 children aged 3 to 17 and primarily checks whether the treatment …

This study tests the long-term safety and effectiveness of an experimental drug called VX-670 in adults with myotonic dystrophy type 1 (DM1). Participants who completed a previous VX-670 study can join. The drug is given through a vein, and researchers will monitor side effects a…

This Phase 3 trial tests whether navenibart, a drug given as a shot under the skin, can prevent attacks in people with hereditary angioedema (HAE). About 145 adults and teens with HAE type 1 or 2 will receive either navenibart or a placebo for 6 months. The main goal is to see if…

This study tests a single dose of PBGM01, a gene therapy delivered directly into the fluid around the brain and spinal cord, in children with GM1 gangliosidosis. The therapy uses a harmless virus to carry a working copy of the GLB1 gene, which is missing or faulty in these patien…

This study follows 176 adults aged 21-80 who are already scheduled for a single-level lower back fusion surgery using screws and cages. The goal is to collect real-world data on pain, mobility, and safety over 24 months. It does not test a new treatment but observes standard care…

This phase 2 trial tests whether allopregnanolone, a natural hormone, can safely help repair the brain in people with early Alzheimer's disease. About 100 volunteers aged 55 to 80 will receive weekly infusions of the drug or a placebo for several months. Researchers will measure …

This study tests whether hydroxyurea, a drug already used for sickle cell disease in other parts of the world, is safe and effective for children with sickle cell anemia in Africa. Children aged 1 to 10 years receive daily doses of hydroxyurea, with careful monitoring for side ef…

This early-phase trial is testing whether combining three drugs—tazemetostat, zanubrutinib, and an anti-CD20 antibody (rituximab or obinutuzumab)—is safe and effective for people with certain types of lymphoma that have come back or not responded to treatment. The study involves …

This early-stage study tests a new drug called PIT565 in about 54 people with active lupus who have not gotten better with standard treatments. The main goal is to see if the drug is safe and how the body processes it. Participants will receive increasing doses of the drug and be…

This pilot study tests whether adalimumab, an anti-inflammatory drug, can help preserve vision in people with retinitis pigmentosa or a related condition called EMAP. Thirty participants will receive three injections into the eye over four months. The main goal is to see if the t…

This study tests deep brain stimulation (DBS) surgery for people with severe focal hand dystonia, including musician's dystonia, that hasn't improved with botulinum toxin. Five participants will have electrodes placed in brain areas that control movement, connected to a stimulato…

This study tests whether a new drug called zanidatamab, combined with chemotherapy and sometimes another drug (tislelizumab), works better than the current standard treatment for advanced HER2-positive stomach and esophageal cancers. About 920 adults whose cancer has spread or ca…

This study aimed to test a finger prosthetic system called Point Mini in 5 children aged 5-15 with partial hand loss. The device uses ratcheting mechanical digits to help with gripping and lifting tasks. However, the trial was withdrawn before any participants were enrolled, so n…

This phase 2 trial tests whether the supplement fisetin can improve lung disease in people with common variable immunodeficiency (CVID). Twenty participants will receive either fisetin or a placebo on four days over a month. The study measures changes in immune cells and lung fun…

This study offers NRX-101, a combination of two drugs, to adults aged 18-65 with treatment-resistant bipolar depression and suicidal thoughts. Participants receive the medication from their own psychiatrist and undergo regular check-ins on mood, suicidal ideation, and side effect…

This study tests a new device called MOTUS, which replaces a worn-out joint in the lower back with an artificial one. The goal is to see if it works as well as the standard surgery (spinal fusion) for reducing pain and improving function. About 158 adults aged 21-80 with single-l…

This early-stage trial tests a single injection of ARO-ATXN2 in 36 adults with spinocerebellar ataxia type 2, a rare genetic disorder that affects movement and coordination. The main goal is to see if the drug is safe and how the body processes it. Participants are randomly assig…

This phase 2 study tests the drug nirogacestat in 20 Japanese adults with desmoid tumors that are growing. The goal is to see if the drug can shrink tumors or stop them from getting worse. Participants take nirogacestat as an oral tablet, and researchers will monitor tumor respon…

This study is testing three long-acting antibodies, alone or in combination, in 645 people with ulcerative colitis. The goal is to see if they are safe and can improve bowel inflammation over 48 weeks. Participants are joining from a previous study and may have already seen some …

This study tests whether giving a higher radiation dose to a brain area called the subventricular zone (where stem cells live) can delay glioblastoma from coming back. About 60 adults with newly diagnosed glioblastoma will receive standard radiation and chemo, but half will get e…

This study tests whether anakinra (Kineret), a daily injection, can reduce monthly attacks in Chinese patients with Familial Mediterranean Fever (FMF) who do not respond to standard colchicine treatment. Three patients aged 2 years and older will receive anakinra for 6 months, wi…

This study tests whether metformin, a widely used diabetes drug, can safely slow vision loss in people with ABCA4 retinopathy (Stargardt disease). Fifty-five participants aged 12 and older will take metformin by mouth for 24 months, with regular eye exams to track changes. The go…

This early-stage study tests two drugs together—linvoseltamab and dupilumab—in just 6 adults with severe food allergies. The goal is to see if the combination is safe and can lower IgE antibodies that trigger allergic reactions. Participants must have a history of anaphylaxis to …

This phase 3 trial tests EYP-1901, a new eye injection that may last longer than current treatments for wet age-related macular degeneration (AMD). About 400 people with wet AMD will receive either EYP-1901 or standard aflibercept injections over 2 years. The main goal is to see …

This phase 3 trial tests EYP-1901, a new eye injection that may last longer than current treatments for wet age-related macular degeneration (AMD). About 400 participants will receive either EYP-1901 or the standard drug aflibercept. The goal is to see if EYP-1901 can maintain or…

This phase 3 trial tests whether a new drug combination (MK-7684A) plus chemoradiotherapy helps people with stage III non-small cell lung cancer live longer than the current standard treatment. About 611 participants will receive either the new combo or durvalumab. The study is a…

This early study tests a new drug called ABBV-101 in people with several types of non-Hodgkin lymphoma that have come back or not responded to other treatments. The drug is a pill that targets a protein (BTK) involved in cancer growth. The main goals are to find a safe dose and s…

This trial tests a combination of donor immune cells (targeting a common virus) and the drug pembrolizumab (Keytruda) in 58 people with glioblastoma, an aggressive brain cancer. The goal is to see if this approach can shrink tumors and help patients live longer. The study has two…

This study tests two doses of K-808 (Pemafibrate) in 46 adults with primary biliary cholangitis (PBC) who haven't responded well to standard treatments. The main goal is to see if the drug lowers alkaline phosphatase (ALP) levels, a key marker of liver damage, after 12 weeks. Par…

This study is checking the long-term safety of a drug called dapirolizumab pegol for people with systemic lupus erythematosus (lupus). It involves 760 participants who have already completed earlier studies of the drug. Researchers will monitor side effects over time to see if th…

This study tests a virus called AdAPT-001 that is injected directly into tumors to kill cancer cells. It is for adults with advanced solid tumors that have not responded to standard treatments. The trial will check if the virus is safe and whether it works better when combined wi…

This early-stage trial tests an experimental drug called BI 3000202 in 16 adults with rare type 1 interferonopathies, such as Aicardi-Goutières syndrome. Participants take a low dose for 4 weeks, then a higher dose for 36 weeks. The main goal is to see if the drug is safe and how…

This study tests whether adding three drugs (vincristine, irinotecan, and regorafenib) to standard chemotherapy can help people with Ewing sarcoma that has spread. About 437 participants will receive either the new combo or standard treatment. The goal is to see if the new approa…

This study tests a drug called litifilimab for people with lupus-related skin disease that didn't get better with standard antimalarial pills. About 450 adults with active skin lesions will receive either the drug or a placebo every 4 weeks for 24 weeks, then everyone gets the dr…

This phase 2 trial tests the safety and effectiveness of avelumab, an immunotherapy drug, in people with thymoma or thymic carcinoma that has progressed after platinum-based chemotherapy. About 56 participants will receive avelumab intravenously every two weeks. The study aims to…

This phase 3 study tests an oral drug called JNJ-77242113 in 311 adults with plaque psoriasis affecting special areas like the scalp, genitals, or palms and soles. Participants receive either the drug or a placebo for 16 weeks. The main goal is to see if the drug clears or nearly…

This study tests whether a robotic exoskeleton called ReWalk can help people with complete spinal cord injury below T4 walk and improve their health. Fifty participants will either use the exoskeleton or receive standard physical therapy. The goal is to see if the device improves…

This study tests a special drilling technique called osseodensification to widen narrow jawbone so dental implants can be placed without extra bone grafting. Thirty adults with healed tooth sockets and narrow ridges will have their bone width measured before and after the procedu…

This study tests whether dual focus contact lenses (MiSight) can slow the progression of nearsightedness (myopia) in university students aged 18 to 21. Participants wear either the test lenses or standard single vision lenses for at least 10 hours a day. Researchers measure chang…

This phase 3 trial tests the drug mitapivat in children aged 1 to 18 with pyruvate kinase deficiency, a rare genetic disorder that causes red blood cells to break down too quickly. These children need regular blood transfusions. The study compares mitapivat to a placebo to see if…

This study tests whether weekly infusions of Alpha-1 MP (a protein replacement) can slow lung damage in 345 people with emphysema caused by Alpha-1 antitrypsin deficiency. Participants receive one of two doses or a placebo for three years, with lung CT scans tracking changes. The…

This phase 2 trial tests whether prasinezumab, given as an IV infusion every 4 weeks, can slow motor decline in people with early Parkinson's disease. The study includes 586 participants who are already on stable Parkinson's medication. Researchers are measuring how long it takes…

This early-stage trial is testing a drug called BMN 351 in 18 boys aged 4 to 10 with Duchenne muscular dystrophy who have a specific genetic change. The drug is designed to help the body produce a shorter but still useful version of the muscle protein dystrophin. The main goal is…

This phase 3 study tests a drug called PTC923 (sepiapterin) in 200 people with phenylketonuria (PKU), a genetic condition that requires a strict low-protein diet. The main goal is to see if the drug is safe over the long term and whether it allows people to eat more protein witho…

This study follows 16 infants aged 6 months or younger with infantile-onset Pompe disease, a rare genetic disorder that weakens muscles and breathing. All receive alglucosidase alfa (Myozyme) as part of their routine care. Researchers track how many survive without needing a brea…

This phase 2 trial tests a drug called VK-2019 in 13 patients with Epstein-Barr virus (EBV)-positive nasopharyngeal cancer that has returned or spread and has no standard treatment. VK-2019 works by blocking a key viral protein, EBNA1, which the virus needs to survive. The main g…

This study compares two common laser eye surgeries—KLEx and FS-LASIK—for treating nearsightedness and astigmatism. Researchers will follow 80 adults aged 21 and older to see which procedure provides better vision without glasses and has fewer side effects. Both surgeries reshape …

This study offers continued access to the drug mitapivat for adults with pyruvate kinase deficiency who completed an earlier Agios-sponsored trial and cannot get the drug commercially. Only 6 participants are enrolled, and the main goal is to monitor side effects. The study does …

This phase 3 study tests whether mitapivat, an oral medication, can safely reduce the number of blood transfusions needed by adults with transfusion-dependent alpha- or beta-thalassemia. Participants receive either mitapivat or a placebo for 48 weeks. The main goal is to see if m…

This study tests an experimental drug called RO7790121 in about 21 adults with moderate to severe Crohn's disease who haven't responded well to other treatments. The goal is to see if the drug can reduce gut inflammation and improve symptoms like diarrhea and stomach pain. Partic…

This phase 3 trial tests an oral drug called JNJ-77242113 in 774 adults with moderate to severe plaque psoriasis. Participants receive either the new drug, a placebo, or an active comparator (deucravacitinib) to see how well it clears skin lesions. The main goal is to measure imp…

This phase 3 trial tests whether the drug mitapivat can improve anemia and reduce fatigue in people with alpha- or beta-thalassemia who do not need regular blood transfusions. About 194 participants received either mitapivat or a placebo daily for 24 weeks. The main goal is to se…

This study tests whether giving people different taste-based advice helps them reduce their free sugar intake to less than 5% of daily energy. Participants are adults in southern England who consume more than 10% of their energy from free sugars. They are randomly assigned to adv…

This study planned to test whether injecting botulinum toxin (Botox) into the salivary glands could reduce drooling in people with true bulbar palsy, a condition that causes swallowing problems after a brain stem injury. The trial was designed to compare the injections against st…

This early-stage trial is testing an experimental drug called APX005M in 32 children with brain tumors that have come back or are hard to treat, including a type of brain stem glioma. The drug works by activating the immune system to attack cancer cells. The main goals are to fin…

This early study tests the safety of TheraSphere PCa, tiny radioactive glass beads injected into the prostate to treat localized prostate cancer. The trial involves 36 men with favorable intermediate-risk cancer and aims to find the maximum safe radiation dose. Researchers will m…

This phase 2 trial tests whether adding a second DNA vaccine to a standard immunotherapy (pembrolizumab) can improve outcomes for men with castration-resistant metastatic prostate cancer. About 60 participants will receive either one or two DNA vaccines along with pembrolizumab f…

This study compares a new three-drug combination (NABPLAGEM) against a standard two-drug chemo regimen for people with advanced pancreatic cancer that has spread and carries a BRCA or PALB2 gene mutation. The trial enrolls 10 participants whose cancer worsened after initial treat…

This phase II trial tests whether a new drug called Briquilimab can make stem cell transplants safer for people with GATA2 deficiency, a rare genetic condition that weakens the immune system and raises the risk of infections and blood cancers. The study involves 13 participants a…

This study tests a gene therapy called laruparetigene zovaparvovec (AGTC-501) in 10 males aged 12-50 with X-linked retinitis pigmentosa, a genetic condition that leads to vision loss. The therapy is injected into both eyes to deliver a working copy of the RPGR gene. The main goal…

This study tests an experimental drug called LY2784544 in people with three types of blood cancers known as myeloproliferative neoplasms: polycythemia vera, essential thrombocythemia, and myelofibrosis. The trial enrolls patients who have not responded to, cannot tolerate, or hav…

This phase 3 study tests whether venglustat, an experimental oral drug, can slow heart thickening better than current standard treatments in 104 adults with Fabry disease. Participants are randomly assigned to venglustat or usual care (enzyme replacement or migalastat) for 18 mon…

This study tests a new device called the eShunt System for treating normal pressure hydrocephalus, a condition that causes trouble walking, thinking, and bladder control in older adults. The device drains extra fluid from the brain through a small tube. Researchers will compare i…

This study tests a drug called sparsentan for people who have protein in their urine after a kidney transplant due to IgA nephropathy or FSGS. About 20 adults will take the drug once daily for 36 weeks to see if it safely reduces protein levels and protects kidney function. The g…

This study follows 284 people with blocked leg arteries (peripheral arterial disease) who are treated with a paclitaxel-coated balloon. The balloon is inflated inside the artery to open it and release a drug that helps prevent it from narrowing again. Researchers are tracking saf…

This study looks at the long-term safety of a weekly intravenous treatment for people with emphysema caused by alpha-1 antitrypsin deficiency. About 290 adults who completed a prior study will receive the drug for 2 more years. The main goal is to monitor side effects, not to cur…

This study compares a targeted drug called selumetinib to standard chemotherapy (carboplatin/vincristine) in children aged 2 to 21 with neurofibromatosis type 1 (NF1) and low-grade glioma, a type of brain tumor. The goal is to see if selumetinib works as well or better at control…

This phase 3 trial tests a new cream (MC2-01) containing two active ingredients for plaque psoriasis in Chinese adults. About 537 participants will use either the new cream, a standard gel, or a placebo cream for 8 weeks. The main goal is to see if the new cream clears skin bette…

This study tests a drug called mitapivat in children aged 1 to 18 with pyruvate kinase deficiency, a rare genetic condition that causes red blood cells to break down too quickly, leading to anemia. The trial compares mitapivat to a placebo to see if it can raise hemoglobin levels…

This Phase 3 trial tests whether adding the targeted drug acalabrutinib to standard chemotherapy (R-CHOP) helps people with a specific, aggressive type of lymphoma (non-GCB DLBCL) live longer without their cancer getting worse. About 611 adults aged 18 to 75 who have not been tre…

This study tests a drug called mitapivat in 286 people with sickle cell disease, ages 16 and older. The goal is to see if mitapivat can raise hemoglobin levels and reduce painful sickle cell crises compared to a placebo. The trial has two phases: first to find the best dose, then…

This trial tests a new drug called Cadonilimab in 28 people with advanced colorectal cancer that has not responded to standard immunotherapy. The drug is a bispecific antibody that targets two immune checkpoints (PD-1 and CTLA-4) to potentially reactivate the immune system agains…

This study tests a drug called entrectinib in children with advanced solid tumors or brain tumors that have come back or haven't responded to other treatments. The trial has two parts: first finding the best dose, then seeing how well it works in children whose tumors have specif…

This study tests whether injecting a woman's own platelet-rich plasma (PRP) into the ovary can help restore function in women with premature ovarian insufficiency (early menopause). 35 women under 40 will receive PRP in one ovary and a saline placebo in the other. The main goal i…

This phase 3 trial tests whether the drug troriluzole can slow the progression of spinocerebellar ataxia, a rare genetic disorder that affects coordination and balance. About 300 adults with different types of SCA are randomly assigned to take either troriluzole or a placebo dail…

This study tests a new medicine called anifrolumab, given as a shot under the skin, for adults with moderate-to-severe lupus. The goal is to see if it can better control disease activity compared to a placebo, when added to standard treatments. About 367 participants are involved…

This study tests a yearly injection called Debio 4326 in 56 children with central precocious puberty, a condition where puberty starts too early (before age 8 in girls, 9 in boys). The goal is to see if it can safely suppress puberty hormones back to childhood levels for a full y…

This study tests whether dupilumab can help people with moderate to severe ulcerative colitis that has a certain type of immune cell activity (eosinophilic phenotype). About 68 adults will receive either dupilumab or a placebo for 52 weeks. The main goal is to see if symptoms and…

This study tests whether adding brentuximab vedotin (a drug that delivers cancer-killing substances directly to lymphoma cells) to standard chemotherapy works better than chemo alone for children and young adults with high-risk Hodgkin lymphoma. About 600 participants with stage …

This phase I trial tests a new approach for patients with high-risk or recurrent non-Hodgkin lymphoma. After a stem cell transplant, participants receive a single infusion of their own genetically modified T-cells designed to recognize and attack cancer cells. The study aims to f…

This phase II trial tested whether adding veliparib (a targeted drug) to standard chemotherapy (topotecan and carboplatin) helps people with advanced myeloproliferative disorders, acute myeloid leukemia, or chronic myelomonocytic leukemia. The study enrolled 25 adults whose cance…

This early-phase trial is testing a new drug called venetoclax combined with standard chemotherapy drugs for people with aggressive B-cell lymphoma that has come back or stopped responding to treatment. The main goal is to find the highest safe dose of the new combination. About …

This study is testing whether the drug spesolimab can safely and effectively treat repeated flares of generalized pustular psoriasis (GPP), a serious skin condition. Adults with GPP who have had a flare will receive a single intravenous dose of spesolimab, with a possible second …

This Phase 3 trial tests whether the oral drug remibrutinib works better than teriflunomide at reducing relapses and slowing disability in people with relapsing multiple sclerosis. About 1,000 adults aged 18–55 with active MS are taking part. The study is double-blind, meaning ne…

This study tests two types of eyeglass lenses designed to slow the progression of nearsightedness (myopia) in children aged 6 to 13. Over 6 months, researchers will measure changes in eye length and thickness to see how the lenses affect eye growth. The goal is to find a better w…

This phase 3 study tests an experimental oral drug, JNJ-77242113, in about 731 adults with moderate to severe plaque psoriasis. Participants receive either the new drug, a placebo, or an active comparator (deucravacitinib) to see how well it clears skin lesions. The main goal is …

This study tests whether combining two drugs, itacitinib and everolimus, can help people with classical Hodgkin lymphoma that has returned or not responded to prior treatments. The trial is open to adults with measurable disease and aims to see how well the combination works in s…

This early-phase study tests a new treatment called NKX019 for people with lupus, an autoimmune disease. NKX019 uses specially engineered immune cells to target and calm overactive B cells. The main goal is to check safety in about 6 participants, while also looking for signs tha…

This study investigates whether combining dry cupping therapy with muscle energy technique improves posture and reduces disability in adults with upper crossed syndrome—a condition marked by forward head posture and rounded shoulders. Participants receive either the combined ther…

This phase 3 trial tests ravulizumab, a drug that blocks part of the immune system, against a placebo in 579 adults with IgA nephropathy—a kidney disease that can lead to kidney failure. The goal is to see if ravulizumab reduces protein in the urine and preserves kidney function …

This study tests a gene therapy for people with X-linked chronic granulomatous disease (X-CGD), a genetic condition that weakens the immune system and leads to severe infections. Researchers take the patient's own stem cells, add a working copy of the faulty gene, and return the …

This study tests a one-time gene therapy called CAP-003 in adults with Parkinson's disease caused by a GBA1 gene mutation. The goal is to see if it is safe and if it can help control the disease. Participants receive a single IV infusion and are monitored for two years.

This study tests fitusiran, a monthly injection, in boys aged 1 to under 12 with severe hemophilia A or B who have developed inhibitors (antibodies that block standard treatment). The goal is to find the right dose to safely lower a protein called antithrombin, which helps preven…

This phase 3 trial tests an experimental drug called ALXN1850 in 124 adolescents and adults with hypophosphatasia, a rare genetic bone disease. Participants receive either the drug or a placebo by injection under the skin. The main goal is to see if the drug improves walking dist…

This study looks at the long-term safety and effects of atacicept in people with IgA nephropathy, a kidney disease. Participants have already completed a previous trial with atacicept. Researchers will monitor side effects and measure kidney function over time to see if the drug …

This early-phase study tests a new drug called BGB-11417 (sonrotoclax) in people with B-cell blood cancers that have come back or not responded to treatment. The goal is to find the safest dose and see how well it works alone or combined with other drugs. About 437 adults will ta…

This Phase 2 trial tests whether N-Acetylcysteine (NAC), an antioxidant, can help people with ME/CFS (chronic fatigue syndrome). Researchers will give 95 participants different doses of NAC or a placebo for four weeks, then measure brain glutathione levels and markers of oxidativ…

This study tests a drug called rogaratinib in about 48 people with advanced sarcoma or a rare type of GIST that has certain genetic changes. The drug works by blocking proteins that help cancer cells grow. The goal is to see if it can shrink tumors or slow the disease, while moni…

This study tests a drug called certolizumab pegol (Cimzia) in children aged 6 to 17 with moderate to severe plaque psoriasis. The goal is to see how the drug moves through the body and whether it is safe. Some children will get the drug, others a placebo. The study is active but …

This phase 2 trial tests the drug atezolizumab in 27 people with chondrosarcoma or clear cell sarcoma that cannot be removed by surgery or has spread. Atezolizumab is an immunotherapy that helps the body's immune system attack cancer cells. The main goal is to see if the drug can…

This study tests two drug combinations (belinostat plus either guadecitabine or ASTX727) in 19 adults with a rare bone cancer called chondrosarcoma that has spread or cannot be removed surgically. The goal is to see if the drugs can shrink tumors or stop them from growing. Partic…

This trial tests a fracture liaison service (FLS) — a coordinated care program for people aged 50 and older who have had a hip or spine fracture. The program follows best-practice standards to ensure patients receive bone density tests and appropriate osteoporosis management. Res…

This study tests a new device called VCFix for treating a broken bone in the spine (vertebral compression fracture). Ten adults with a single recent fracture will receive the implant to see if the procedure is safe and works as intended. The goal is to gather early data to plan a…

This phase 2 trial tests the drug selpercatinib in children and young adults (ages 1 to 21) with advanced cancers that have a specific genetic change called a RET alteration. The goal is to see if the drug can shrink tumors or stop them from growing. Only one participant has been…

This study tests a single injection of ZVS101e, a gene therapy that delivers a working copy of the CYP4V2 gene directly into the retina. It is for adults with Bietti's crystalline dystrophy, a rare inherited condition that leads to progressive vision loss. The trial will check wh…

This phase 2 trial tests whether empagliflozin, a diabetes drug also known as Jardiance, can help people with glycogen storage disease type Ib (GSD-1b). GSD-1b causes low neutrophil counts, leading to frequent infections and bowel inflammation. The study gives empagliflozin orall…

This early-stage trial tests whether a single infusion of donated mesenchymal stem cells is safe for children with cerebral adrenoleukodystrophy (cALD), a rare and life-threatening brain disease. The stem cells are given as a temporary bridge to buy time before a more definitive …

This study tests a daily injection called concizumab in children under 12 with hemophilia A or B, with or without inhibitors. The goal is to see if it safely prevents bleeding episodes. Participants receive daily shots under the skin for at least 2 years, and the study tracks how…

This study tests a donor stem cell transplant for people with severe immune system problems, like SCID and Wiskott-Aldrich syndrome. The goal is to help the body make healthy blood cells and fight infections. Participants receive donated stem cells to rebuild their immune system.…

This study looks at the long-term safety of a drug called litifilimab for people with active lupus. About 864 adults who finished earlier studies will continue taking the drug for up to 3 years, along with their usual treatments. The goal is to see how safe it is and how well it …

This study tests a stem cell transplant method for people with acquired or inherited bone marrow failure. Donor stem cells are specially processed to remove certain immune cells, which may lower the risk of graft rejection and graft-versus-host disease. The goal is to see if this…

This Phase 3 trial tests whether the drug Afimkibart can help people with moderate-to-severe ulcerative colitis achieve remission. About 400 adults who have not responded well to other treatments will receive either Afimkibart or a placebo. The study measures remission at 12 and …

This phase 2 study is testing a risk-adapted approach for children and young adults with classical Hodgkin lymphoma. Depending on their risk level, patients receive different combinations of chemotherapy drugs, with the goal of improving response while reducing side effects. The …

This study tests a new device called SNIP designed to treat tongue-tie in infants. The device is used to perform a frenectomy, a procedure that releases the tight band under the tongue. The study includes 6 infants with anterior tongue-tie and focuses on how easy the device is fo…

This long-term study tests the safety and effectiveness of bitopertin (DISC-1459) in people with erythropoietic protoporphyria (EPP) or X-linked protoporphyria (XLP), rare conditions that cause severe pain from sunlight exposure. About 230 participants who previously took bitoper…

This registry collects long-term data from hundreds of people in Germany who have undergone corneal cross linking for keratoconus, a condition where the cornea thins and bulges, causing vision loss. The procedure uses riboflavin (vitamin B2) eye drops and UV light to stiffen the …

This study is tracking the long-term safety and effectiveness of the drug Saphnelo (anifrolumab) in over 1,600 people with systemic lupus erythematosus who haven't responded well to current treatments. Researchers are monitoring for side effects like serious infections, shingles,…

This phase II trial tests a combination of three drugs—cabozantinib, nivolumab, and ipilimumab—in 11 patients with advanced thyroid cancer that no longer responds to radioactive iodine or prior targeted therapy. The goal is to see if this mix can shrink tumors or stop them from g…

This Phase 2 trial tests a drug called RO7204239 in 51 adults with facioscapulohumeral muscular dystrophy (FSHD), a genetic condition that causes progressive muscle weakness. The drug is an antibody that blocks myostatin, a protein that normally limits muscle growth, to help pres…

This large phase 3 study tests whether adding the drug blinatumomab to standard chemotherapy can improve outcomes for children and young adults with newly diagnosed B-cell acute lymphoblastic leukemia or lymphoma, including those with Down syndrome. The trial enrolls over 6,700 p…

This study compares two types of contact lenses for slowing myopia (nearsightedness) in children aged 8 to 14. Over three years, researchers will measure changes in eye length and vision to see if the new lens works as well as the existing one. The goal is to help kids maintain b…

This study tests whether switching to avalglucosidase alfa is safe and more effective for people with late-onset Pompe disease whose condition has worsened on the standard treatment, alglucosidase alfa. Participants receive biweekly infusions of the new drug and are monitored for…

This phase II trial tests whether intensive blood pressure management (targeting systolic BP below 120 mmHg) is better than standard care (below 140 mmHg) for patients with advanced kidney or thyroid cancer starting anti-angiogenic TKI therapy. The study enrolls 61 participants a…

This early-phase trial tests a gene therapy called MVX-220 in 12 children and adults with Angelman syndrome. The therapy delivers a working copy of the UBE3A gene via a single injection into the fluid around the brain. Researchers are primarily checking safety, but also looking f…

This study tests a precise type of proton radiation (IMPT) for people with chordoma or chondrosarcoma in the spine, sacrum, or skull base. The goal is to control the tumor while reducing damage to healthy tissue. Some participants may also have surgery. The study includes 64 adul…

This phase 2 trial is testing a combination of two immunotherapy drugs, nivolumab and ipilimumab, in 798 people with over 50 types of rare cancers. The goal is to see if these drugs can shrink tumors by helping the immune system attack cancer cells. Participants receive the drugs…

This study tests special spectacle films designed to slow down myopia (nearsightedness) in children. About 210 kids aged 6 to 14 are taking part. The films use a new technology called S.T.O.P.® that creates changing visual cues, which may help control how fast the eye grows and p…

This phase 2 trial tests the immunotherapy drug atezolizumab, alone or with bevacizumab, in 63 people with advanced alveolar soft part sarcoma that cannot be surgically removed. Atezolizumab helps the immune system attack cancer cells, while bevacizumab cuts off the tumor's blood…

This study tests a single injection of ZVS101e gene therapy for people with Bietti's crystalline dystrophy, a rare inherited condition that leads to progressive vision loss. The therapy delivers a working copy of the CYP4V2 gene directly to the retina. Researchers will measure wh…

This early-stage study tested the safety and best dose of vorinostat when given with the chemotherapy drug temozolomide in 83 adults with aggressive brain tumors called malignant gliomas. The goal was to see if adding vorinostat could make the tumor cells more sensitive to chemot…

This study tests two different tendon grafts (kneecap tendon vs. quadriceps tendon) for ACL reconstruction, with or without an extra stabilizing procedure called lateral extra-articular tenodesis (LET). The goal is to see which approach best prevents graft failure in 1,272 young,…

This study tests a drug called ulixertinib in children and teens whose advanced cancers have a specific genetic change in the MAPK pathway. The goal is to see if the drug can shrink or control the tumor. About 20 participants with various solid tumors, lymphomas, or related disor…

This program allows eligible patients to receive RGX-202, a gene therapy, on a single-patient basis outside of a clinical trial. It is designed for those with serious conditions who have no other treatment options. Currently, the program is temporarily not available.

This phase II trial tests a personalized cell therapy called tisagenlecleucel (Kymriah) in children and young adults with high-risk B-cell acute lymphoblastic leukemia who still have detectable cancer cells after initial chemotherapy. The treatment involves collecting the patient…

This study provides ZVS101e, a gene therapy, to 32 people with Bietti crystalline corneoretinal dystrophy (BCD) who were in the control group of a previous Phase III trial. The therapy uses a harmless virus to deliver a working gene to the eye, aiming to slow vision loss. The mai…

This early-stage study tests whether a mix of three drugs—tacrolimus, nivolumab, and ipilimumab—can shrink or control certain skin cancers in people who have had a kidney transplant. The main goal is to see if the cancer responds without causing the body to reject the transplante…

This study tests a drug called selumetinib in children and young adults (ages 3-18) with neurofibromatosis type 1 (NF1) who have nerve tumors that cannot be removed by surgery. The goal is to see if the drug can shrink or slow the growth of these tumors. Participants take the dru…

This phase II trial is studying a combination of chemotherapy (Hyper-CVAD) and the targeted drug dasatinib, with or without a donor stem cell transplant, in 97 adults with Philadelphia chromosome-positive acute lymphoblastic leukemia (ALL). The goal is to see how well this approa…

This study tests a service designed to help people with osteoporosis take their medications as prescribed. Participants receive education, reminders, and regular follow-up calls. The goal is to see if this support improves medication adherence and reduces fracture risk over 18 mo…

This study aimed to see if the drug clomiphene citrate could help men with a condition called non-obstructive azoospermia, where the body produces little to no sperm. The plan was to give the drug or a placebo for nine months, then check if sperm could be found in the semen or th…

This study tests whether adding the immunotherapy drug pembrolizumab to standard tyrosine kinase inhibitors (TKIs) can eliminate tiny amounts of leftover leukemia cells in people with chronic myeloid leukemia (CML). About 40 adults with CML who still have detectable cancer marker…

This clinical trial is testing whether dimethyl fumarate, a drug already used for multiple sclerosis, can improve balance and walking in adults with adrenomyeloneuropathy (AMN), a rare nerve disease. Forty participants will take either the drug or a placebo daily for 36 months, w…

This study tests a new treatment called CHT105 for people with lupus nephritis that hasn't responded to standard therapies. CHT105 uses specially engineered immune cells from healthy donors to target and destroy the faulty immune cells causing kidney damage. Fourteen adults will …

This was a planned early-stage trial to test whether anti-CD19 CAR-T cells could safely help children with multi-drug resistant nephrotic syndrome, a serious kidney condition. The study was withdrawn before any patients were enrolled, so no data on safety or effectiveness were co…

This phase 3 trial tests whether an oral antioxidant called N-acetylcysteine (NAC) can slow the loss of vision in people with retinitis pigmentosa, an inherited eye disease that causes gradual blindness. About 485 adults aged 18 to 65 are taking NAC or a placebo for several years…

This early-stage study tests a new drug called ADX-850 in 28 people with mild to moderate high blood pressure. The drug uses siRNA technology to target genes involved in blood pressure regulation. The main goal is to check safety and how the drug moves through the body, with a se…

This study tests an oral medication called tofacitinib in children aged 2 to 17 with moderate to severe ulcerative colitis, a chronic bowel disease causing inflammation and bleeding. The goal is to see if the drug can bring the disease into remission (no symptoms and healed gut l…

This study looks at how the drug crinecerfont works in babies under 2 years old with congenital adrenal hyperplasia (CAH), a condition where the body can't make certain hormones properly. The goal is to measure drug levels in the blood and check for side effects. Only 7 infants a…

This study is checking the long-term safety and effectiveness of the drug ESK-001 (envudeucitinib) for people with plaque psoriasis. It includes 165 adults who have already completed a previous ESK-001 study. Participants will take the drug as an oral tablet and be monitored for …

This study tests whether giving vedolizumab every 4 weeks instead of every 8 weeks can help Japanese patients with moderate to severe ulcerative colitis or Crohn's disease whose symptoms returned during standard treatment. About 56 participants will receive the drug intravenously…

This study tests whether a low dose of lithium (20 mg/day) can slow disease-related changes in the brain and blood of people with early-stage Parkinson's disease. Twenty participants will receive either lithium or a placebo for a period, and researchers will use MRI scans and blo…

This study follows 100 Polish adults with systemic lupus erythematosus (SLE) who are receiving anifrolumab as part of their routine care. Researchers will track changes in disease activity and overall response over time. The goal is to see how well the drug works outside of contr…

This study follows 30 people with hemophilia A or B (without inhibitors) in Japan who are already using Alhemo (concizumab) as part of their routine care. Researchers will monitor side effects and how well the drug works over about 2 years per person. The goal is to see if Alhemo…

This early-stage trial is testing a new treatment called KITE-363 for people with severe autoimmune diseases like lupus and scleroderma that haven't improved with standard treatments. KITE-363 is a type of cell therapy where a patient's own immune cells are modified in a lab to t…

This study tests a one-time gene therapy called UX701 for adults with Wilson disease, a genetic condition that causes copper buildup. The goal is to see if it is safe and can help the body control copper levels, possibly allowing patients to take fewer or no daily medications. Ab…

This early-stage study tests a combination of two drugs, avutometinib and defactinib, in children and young adults (ages 3 to 30) with advanced or recurrent solid tumors that have specific genetic changes. The main goal is to find the safest dose with the fewest side effects. The…

This study tests a single injection of VIA Disc NP, a processed human disc tissue, for people with long-term lower back pain from degenerated discs. About 121 participants will receive either the injection or a sham procedure. The goal is to see if the injection safely reduces pa…

This study tests a new drug called ADX-324 for hereditary angioedema (HAE), a rare genetic condition that causes sudden, severe swelling in the body. The trial has two parts: first, a small group of healthy volunteers will receive the drug to check its safety and how the body pro…

This early study at Mayo Clinic tests whether ultra-low, anti-inflammatory doses of radiation can slow Parkinson's disease. Twenty people with early-stage Parkinson's will receive five daily radiation treatments guided by special brain scans. The main goal is to see if the treatm…

This Phase 3 trial tests whether a daily injection of metreleptin (a lab-made leptin hormone) can improve blood sugar and fat levels in people with partial lipodystrophy, a rare condition where fat is distributed abnormally. About 69 participants will receive either metreleptin o…

This phase 3 study tests setrusumab, a monthly IV infusion, in 6 Japanese children with osteogenesis imperfecta (types I, III, or IV), a condition that causes fragile bones and frequent fractures. The main goal is to see if the drug lowers the number of fractures, including spine…

This early-stage study tests a gene therapy for people with severe beta-thalassemia who need regular blood transfusions. The treatment modifies the patient's own blood stem cells to produce healthy hemoglobin, aiming to reduce or eliminate the need for transfusions. Six participa…

This study tests a gene therapy called vesemnogene lantuparvovec in about 20 people with spinal muscular atrophy (SMA), a genetic condition that causes muscle weakness. The therapy delivers a working copy of the SMN1 gene to help muscles work better. The main goal is to check saf…

This study tests an experimental eye drop called ZOC2017217 in 40 people with age-related cataracts. Participants will use the drops twice a day for 24 weeks to see if it improves vision and lens clarity compared to a placebo. The goal is to find a non-surgical option for catarac…

This study tests a one-time gene therapy called BAY2599023 for adults with severe hemophilia A. The therapy uses a harmless virus to deliver a working copy of the factor VIII gene, so the body can produce its own clotting factor. The trial involves 11 participants and aims to fin…

This study tests a drug called leniolisib in children aged 1 to 6 years who have a rare genetic immune disorder called APDS. The drug aims to control the overactive immune system and reduce swollen lymph nodes. Researchers will monitor safety and how well the drug works over time…

This Phase 3 trial tests whether bitopertin, a daily pill, can help people with erythropoietic protoporphyria (EPP) or X-linked protoporphyria (XLP) spend more time in sunlight without pain. About 183 participants aged 12 and older will take bitopertin or a placebo for 6 months. …

This study tests whether infliximab, a drug used for inflammatory conditions, can treat colitis (colon inflammation) caused by immune checkpoint inhibitors in people with stage III or IV skin cancer. About 42 participants will receive either infliximab or steroids, and researcher…

This study looks at whether giving vitamin D supplements to people with newly diagnosed lymphoma or chronic lymphocytic leukemia who have low vitamin D levels can improve their survival and delay the need for treatment. About 565 participants are being given vitamin D3 or a place…

This early-stage trial is testing a drug called NS-050/NCNP-03 in 20 boys with Duchenne muscular dystrophy (DMD) who have a specific genetic mutation. The drug is designed to skip a faulty section of the dystrophin gene, allowing the body to produce a shorter but still functional…

This phase 2 trial tests whether the drug TPST-1495 can reduce the number and size of polyps in the small bowel and colon of people with familial adenomatous polyposis (FAP), an inherited condition that greatly raises colon cancer risk. About 38 participants will take the drug by…

This phase 3 trial is testing the safety and tolerability of fenfluramine (Fintepla) in 25 infants aged 1 to 2 years with Dravet syndrome, a severe form of epilepsy. The drug is given as an oral solution twice daily alongside other seizure medications. Researchers will monitor he…

This phase 2 study tests acalabrutinib, a targeted drug, in 107 people with Waldenström macroglobulinemia, a rare blood cancer. The goal is to see how well the drug shrinks tumors and controls the disease. Participants either had prior treatment or are new to therapy and cannot t…

This study was designed to test two immunotherapy drugs, nivolumab and ipilimumab, in children and young adults with cancers that returned or didn't respond to treatment and had many genetic mutations. The goal was to see if the drug combination was safe and could shrink tumors. …

This study tests if a new growth hormone medicine (somapacitan) given once a week works as well as the standard daily growth hormone (Norditropin) for children who are very short due to being born small, or having Turner syndrome, Noonan syndrome, or unknown causes. About 412 chi…

This study tests whether adding a specially trained doula, along with home blood pressure and weight monitoring, can improve blood pressure in women who had high blood pressure during pregnancy. About 454 women will be randomly assigned to usual care or the doula program. The goa…

This study looks at the long-term safety and effects of an experimental drug called DNL310 for people with Hunter syndrome (MPS II), a rare genetic disorder. About 99 participants who completed earlier studies will receive the drug for up to 5 years. Researchers will monitor side…

This phase 2 trial tests an oral drug called venglustat, given alongside the standard enzyme therapy Cerezyme, in adults with Gaucher disease type 3 (a rare genetic disorder affecting the body and brain). The study has four parts: first, it checks spinal fluid biomarkers to disti…

This study tests a combination of two drugs, avelumab and M6620, in people with advanced solid tumors that have spread or can't be removed by surgery. The tumors must have a specific DNA repair defect. The goal is to find the safest dose and see if the combination can help contro…

This early-phase trial tests whether adding special immune cells called veto cells to a stem cell transplant can help donor cells grow in the patient without causing severe graft-versus-host disease (GVHD). The study includes 16 people with various blood cancers or bone marrow fa…

This study tests two common antibiotics—erythromycin and azithromycin—in pregnant people whose water breaks too early (before 34 weeks). Both are given along with standard antibiotics. The goal is to see which combination helps delay delivery longer, giving the baby more time to …

This early-stage study tests a gene therapy called AT845 for adults with late-onset Pompe disease, a condition that weakens muscles. The treatment aims to deliver a working copy of the GAA gene to muscle cells. Eleven participants who have been on standard enzyme replacement ther…

This phase 2 trial tests the drug cabozantinib in children and young adults with rare cancers like sarcomas, Wilms tumor, and others that have come back or not responded to treatment. Cabozantinib works by blocking enzymes that help tumors grow and form blood vessels. The study a…

This study tests a drug called concizumab, given as a daily shot under the skin, to prevent bleeding in people with hemophilia A or B who have inhibitors (antibodies that block standard treatment). About 134 participants either start concizumab right away or continue their usual …

This study tests whether intravenous immunoglobulin (IVIG) can reduce symptoms of autonomic dysfunction—such as dizziness, rapid heartbeat, and fatigue—in people with long COVID. Two hundred participants will receive either IVIG or a placebo (saline) for 9 months, plus coordinate…

This study offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…

This study is testing a new, less invasive device that measures the health of the esophagus in people with acid reflux (GERD). Researchers will compare two versions of the device in 75 patients undergoing routine endoscopy. The goal is to see if this technology can diagnose GERD …

This study tests an AI tool called AIDP that analyzes MRI scans to help diagnose Parkinson's disease and two similar conditions (MSA and PSP). Researchers at 21 sites will compare the AI's diagnosis to that of movement disorder specialists. The goal is to see if the tool can impr…

This study uses special brain scans (PET imaging) to measure amyloid buildup—a protein linked to Alzheimer's—in healthy older adults, people with mild memory problems, and those with dementia. Participants receive scans every 2-3 years for up to 20 years to see how amyloid change…

This study evaluates RDX-Aneurysm, a computer-assisted detection software designed to help radiologists find saccular intracranial aneurysms (bulging blood vessels in the brain) on MRI scans. Researchers will analyze 550 past scans—half with confirmed aneurysms and half without—t…

This study tests whether a special PET scan using a radioactive form of methionine (a natural amino acid) can help doctors see tumors in children and young adults with various cancers, including brain tumors and sarcomas. About 503 participants will be scanned to see if the metho…

This study tests whether combining a special MRI analysis (called Habitat Risk Score) with blood and urine tests can more accurately rule out aggressive prostate cancer than the standard MRI reading. About 289 men with elevated PSA or abnormal exams will be enrolled. The goal is …

This study tests a drug called triheptanoin in 8 adults with MCADD, a rare condition that can cause dangerously low blood sugar. The goal is to see if the drug is safe and can prevent hypoglycemia during fasting. Participants will stay overnight at a hospital for monitoring and b…

This study looks at how to keep a youth-friendly HIV self-testing program called 4YBY running in Nigeria. It will involve over 1,200 young people aged 14-24 who are at risk for HIV. Researchers will compare standard program delivery with an enhanced version that includes extra su…

This trial investigates whether a daily effervescent supplement containing calcium, magnesium, and citrate can prevent bone loss, magnesium deficiency, and kidney damage in adults who take proton pump inhibitors (PPIs) long-term for conditions like heartburn or GERD. Participants…

This study will test a telehealth group prenatal care program called Centering HER in 72 low-income, predominantly Black pregnant women in Kansas who are at moderate or high risk for preeclampsia. Participants will receive routine care plus seven doula-led virtual group sessions,…

This phase IIb trial tests whether a vaccine called Tri-Ad5, combined with an immune booster N-803, can prevent colon cancer in people with Lynch syndrome. The vaccine targets proteins found in precancer and cancer cells, aiming to train the immune system to destroy them. 186 par…

This study looks at the long-term safety and effectiveness of odevixibat in 70 people with Alagille syndrome, a rare genetic condition that affects the liver and other organs. The drug aims to reduce severe itching and improve bile flow. Participants who completed a prior 24-week…

This study tests a special bed called SuPerSense that senses how a person lies on it and gives feedback to help improve posture and balance. People with Parkinson's disease who have trouble with balance or posture will either do standard rehab exercises or use the smart bed plus …

This study tests whether a specific ultrasound-guided nerve block can reduce pain after elective lower back surgery better than the usual method of injecting pain medicine into the wound. Adults aged 18-65 undergoing surgery for conditions like a herniated disc or spinal stenosis…

This study looks at whether pramipexole, a drug used for restless leg syndrome, can ease leg symptoms in women with X-linked adrenoleukodystrophy (ALD). About 24 women will take the drug or a placebo for two months. The goal is to see if it improves sleep, walking, and quality of…

This study tests a new functional drink made with chicory inulin, B-vitamins, and vitamin D to see if it can improve constipation and overall well-being in people with Parkinson's disease. About 56 participants will drink either the active supplement or a placebo twice daily for …

This study tests whether smelling essential oils during skin surgery can reduce the unpleasant smell of burnt flesh from electrocautery and lower patient anxiety. About 111 adults having same-day skin procedures will be randomly assigned to receive either a real essential oil inh…

This study explores whether a new type of group therapy, based on Eastern cultural practices, can help people with anxiety disorders. Researchers will enroll 60 participants aged 15 to 55 to test the therapy's effects on anxiety symptoms. The goal is to see if this approach is bo…

This study investigates whether whole-body vibration (WBV) therapy, added to standard exercise, can improve balance, walking, thinking, and the ability to do two things at once in people with Parkinson's disease. Participants will be randomly assigned to receive either low-freque…

This study tests whether alternating between high and low frequencies of deep brain stimulation (DBS) can improve walking and balance in people with Parkinson's disease. Twelve participants who already have DBS implants will try different stimulation patterns while their gait and…

This study tests whether a smartphone app called AIM+ can help juvenile justice officers and other professionals who work with youth in the legal system manage chronic stress, anxiety, and burnout. Over 30 days, participants use the app for short guided meditations and videos. Th…

This study tests a program called PRISM that teaches teens with HIV how to manage stress, set goals, and build resilience. It involves three one-on-one sessions with a nurse. The goal is to see if the program is feasible and acceptable, and whether it reduces psychological distre…

This study tests whether microfocused ultrasound can safely improve facial pigmentary diseases (like melasma) and rosacea. Two hundred adults will be randomly assigned to receive either real ultrasound or a sham (fake) treatment once a month for six months. Researchers will track…

This phase 2 trial tests whether fasudil, a drug already used in Japan for blood vessel spasms, is safe and can help symptoms in people with early Parkinson's disease. 75 participants will take either fasudil or a placebo for 3 weeks. The study focuses on safety and tolerability,…

This study tests whether an injection of hyaluronidase can reduce muscle stiffness and spasticity in the arm after a stroke. 56 adults with moderate-to-severe stiffness will receive either the study drug or a placebo, and later switch to the other option. The goal is to improve r…

This study tests an internet-based cognitive behavioral therapy (CBT) program designed to help autistic adolescents aged 11-17 manage anxiety. The program, called LUNA-Adolescent, is parent-led and delivered online, making it easier for families who cannot access in-person therap…

This study looks at two ways to lower anxiety in children aged 3 to 10 who need surgical pins removed from an elbow fracture. One approach removes the pins right after the cast comes off, before an X-ray, instead of after. The other uses a virtual reality headset during the pin r…

This study tests whether a virtual reality (VR) program can help people with inflammatory bowel disease (IBD) manage pain and stress. Forty adults with IBD will use a VR headset at home for 4 weeks, doing activities like breathing exercises, meditation, and interactive games. The…

This study looks at whether a peer support program is acceptable and helpful for people with mild depression or anxiety who are starting or receiving hormone care. One hundred participants are randomly assigned to either the peer support group or usual care. The program includes …

This study looks at whether a structured physiotherapy and exercise program can improve pain, strength, balance, and quality of life in one person with melorheostosis, a rare bone condition. The patient will do exercises three times a week for eight weeks, along with education ab…

This study tests whether a simple ear acupressure technique, called auricular point sticking therapy, can reduce anxiety and improve sleep in women undergoing IVF. 178 women aged 20-45 with anxiety or insomnia will either receive standard IVF care or standard care plus ear acupre…

This study looks at whether writing about their experiences can improve the health of Asian American breast cancer survivors. About 192 people who finished cancer treatment in the last 5 years will take part. They will write and report how they feel, and researchers will track an…

This study tests whether playing an immersive virtual reality exergame (Beat Saber) for 12 weeks can improve physical fitness, cognitive skills, and quality of life in 20 people with Down syndrome. Participants will do two supervised sessions per week. The goal is to see if gamif…

This study is testing whether a 6-month program of online exercise classes, guided meditation, and nutrition advice can improve thinking, sleep, and overall health in people with Parkinson's disease. 60 participants aged 40 to 85 will take part, with some starting the program rig…

This study tests whether an injection made from human fat cells (Tience®) can safely reduce moderate to severe acne scars. Twenty adults aged 18-40 with scars on their cheeks will receive three treatments on one side of the face over three months. Doctors and participants will tr…

This study tests the long-term safety of linerixibat, a drug for severe itching caused by primary biliary cholangitis (PBC). All 242 participants receive the drug openly. The goal is to see if it remains safe over time and improves quality of life.

This study looks at the long-term safety of valbenazine for people with Huntington disease who have involuntary jerking movements (chorea). It includes 7 participants from Canada who were in a previous valbenazine study. Everyone gets the drug, and researchers track side effects …

This study tests whether a safe, painless brain stimulation technique called tDCS can improve verbal learning in people with Alzheimer's disease. Researchers will compare active stimulation to a sham (fake) version in 90 participants with typical or language-based Alzheimer's. Th…

This study compares two types of talk therapy—Acceptance and Commitment Therapy (ACT) and Cognitive Behavioral Therapy (CBT)—for HIV/AIDS patients experiencing psychological distress. 72 adults on antiretroviral therapy will receive 8 weekly sessions of either therapy or standard…

This study tests a single dose of an experimental drug called REGN7544 in 82 people with Postural Orthostatic Tachycardia Syndrome (POTS). The goal is to see if the drug safely reduces the rapid heart rate that happens when standing up. Researchers will also check for side effect…

This study looks at whether auto-captioning glasses are easy and helpful for adults with NF2-related schwannomatosis who have hearing loss. Eighteen participants will use the glasses for 12 weeks and report their experience through surveys and interviews. The goal is to see if th…

This study tests whether adding cognitive training to balance and gait exercises helps people with Parkinson's disease improve their balance, walking, and thinking. Fourteen participants will undergo 12 sessions of combined training. The goal is to see if this approach eases move…

This study tests whether a unique music therapy, based on Traditional Chinese Medicine's five-element theory, can help men with depression and anxiety when added to their usual medication. One hundred male inpatients will receive either standard drugs alone, drugs plus regular mu…

This study tests a new nurse-led counselling program designed to help people with stable COPD manage emotional distress. Fifteen outpatients will receive ten counselling sessions over three months. The main goal is to see if the program is feasible, not to prove it works. Researc…

This study tests whether a single injection of rexlemestrocel-L (a type of stem cell) combined with hyaluronic acid can reduce chronic low back pain for at least 12 months. It involves 300 adults with moderate to severe pain from a worn spinal disc who haven't improved with stand…

This study tests whether using telemedicine and remote monitoring can help people with advanced digestive cancers manage chemotherapy side effects at home, reducing the need for emergency room visits and hospital stays. About 750 patients and their caregivers will receive enhance…

This study aimed to see if a web-based self-help program called Deprexis could help orthopedic patients with major depressive disorder. The plan was to offer the 8-week online program to patients with depression and see if they would use it and if their mood and physical function…

This study tests a pill called deucrictibant for quickly treating swelling attacks in people with hereditary angioedema (HAE), including life-threatening throat swelling. About 150 adults who have had HAE attacks before will take the drug as needed and be monitored for safety and…

This study compares two different remote support programs to see which one helps women feel better after surgery for suspected gynecological cancer. About 160 women will take part, and researchers will measure pain, mood, and sleep quality. The goal is to find simple, at-home way…

This study looks at whether speech therapy can reduce air-related symptoms like belching and bloating in people with GERD. Researchers will compare symptoms before and after therapy in 100 adults. The goal is to see if speech therapy can help patients avoid antireflux surgery, wh…

This study tests a 7-session, peer-led self-management program for 234 cancer survivors who recently finished treatment with curative intent. The program aims to reduce anxiety and depression by teaching symptom management skills in a group setting. Participants are from central …

This study tests whether a probiotic supplement called Neuralli® MP can help adults aged 18 to 50 who have autistic traits. Participants take two capsules daily and set their own personal goals to track progress. The trial is small and open-label, meaning everyone knows they are …

This study looks back at the medical records of 500 children and adults with cancer who received immunotherapy at the National Cancer Institute. The goal is to understand the side effects, like inflammation and immune reactions, that can happen with these treatments. By learning …

This study collects small skin samples from people with primary immunodeficiency or immune regulation disorders, as well as from healthy volunteers. Researchers will use these cells to create cell lines for laboratory research. The goal is to better understand immune diseases and…

This study tests a special needle with a tiny tracking device inside, like a mini GPS, to help doctors find tumors that are hard to see on CT scans. About 3,900 adults with certain cancers or growths will have their biopsy or ablation procedure done using this smart needle. The g…

This study looks at a rare liver disease called PFIC, which causes severe itching and liver damage. Researchers compare patients who took the drug odevixibat to those who did not, using data from past studies and a patient registry. The goal is to see if odevixibat helps avoid li…

This study looks at whether teenagers and young adults with Polycystic Ovary Syndrome (PCOS) have more fat in their liver than those without PCOS. Researchers will use advanced imaging and tests to measure liver fat, insulin resistance, and cholesterol problems in 80 participants…

This study collects and analyzes blood, DNA, and tissue samples from 315 adults with pulmonary fibrosis, their relatives, and healthy volunteers. The goal is to learn more about the disease's causes, not to test a new treatment. Researchers hope the findings will guide future the…

This study observes 80 children with Marfan syndrome to understand how the condition impacts their quality of life, self-image, and ability to manage their health. Participants fill out questionnaires about their well-being and medication habits. The goal is to identify who might…

This study explores how low-level laser therapy (LLLT) affects skin stiffness and thickness. Researchers will use a special imaging system to track tiny skin movements in 20 healthy adults. The goal is to find the best laser settings for future wound healing treatments.

This study follows 68 people aged 12 and older who have ABCA4 gene mutations, which can cause vision loss from diseases like Stargardt disease. Over 10 years, researchers will collect blood and skin samples and perform detailed eye exams to understand how the disease progresses. …

This study aims to better understand fatigue by comparing people who have felt tired for more than a month with those who are not fatigued. Researchers will use questionnaires, physical tests, and optional brain scans to identify different types of fatigue. The goal is to gather …

This pilot study looks at biomarkers in the blood of people with fragile sarcolemmal muscular dystrophy, a condition that makes muscle membranes weak. Researchers will collect blood samples at rest and after exercise during four 5-day hospital stays. The goal is to better underst…

This study looks for biological markers in the blood and tissue of adults with Marfan syndrome that could signal when an aortic aneurysm is dangerous enough to require surgery. Researchers will compare three groups of Marfan patients: those without aortic aneurysms, those with st…

This study follows 110 people who previously took part in the AREDS or AREDS2 trials to see how age-related macular degeneration (AMD) and cataracts change over time. Participants get a yearly eye exam, including vision tests and eye photos, and may give a blood sample. The goal …

This study investigates whether advanced eye imaging and artificial intelligence can predict proliferative vitreoretinopathy (PVR), a condition that can cause retinal detachment surgery to fail. Researchers will analyze scans from 100 patients with retinal detachment, some with P…

This study aims to identify genetic, biochemical, and clinical factors linked to disease severity in people with Gaucher disease and other lysosomal storage disorders. Researchers will evaluate up to 1,000 participants, including patients, carriers, and healthy controls, to bette…

This study follows 25 people with spinocerebellar ataxia type 7 (SCA7) for up to 5 years to learn how the disease changes vision, coordination, and thinking. Participants will have yearly eye exams, brain scans, and neurological tests. No treatment is given; the goal is to gather…

This study looks at the genetic makeup of a rare stomach cancer called gastrointestinal stromal tumor (GIST) in patients whose tumors lack common mutations. Researchers will analyze tumor samples from 50 people in Taiwan to find other genetic changes that might be treatable. The …

This study explores whether a new type of resistance exercise is practical and safe for people with low bone mass, including those with osteopenia, osteoporosis, or sarcopenia. Over two months, one group performs progressive muscle-strengthening exercises while a control group co…

This study follows 125 people with Parkinson's disease or related conditions over two years. Researchers use special brain scans to measure changes in a chemical called acetylcholine, which is linked to walking problems, falls, and thinking skills. The goal is to understand how t…

This study looks at how the drug Jivi (damoctocog alfa pegol) works for people with hemophilia A in real-world settings, not just in controlled trials. Researchers are tracking bleeding episodes, side effects, and how satisfied patients and doctors are with the treatment. About 3…

This study investigates whether people with systemic lupus erythematosus (SLE) show early signs of retinal damage before they notice any vision problems. Researchers will use advanced eye imaging and vision tests to compare lupus patients with healthy volunteers. The goal is to f…

This pilot study is testing whether it's possible to run a larger study that focuses on heart disease in women. Researchers will gather information from 176 women aged 25-55 in Ontario who have had certain female-specific health issues like pre-eclampsia, gestational diabetes, or…

This study follows 62 people with hemophilia A who are already using Jivi (a long-acting clotting factor) to prevent bleeding. Researchers will track safety events like side effects and lab changes over at least 4 years during routine doctor visits. The goal is to better understa…

This observational study will examine how the drug mitapivat changes red blood cells in 6 people with sickle cell disease. Researchers will measure oxygen binding and cell survival markers from blood samples and muscle oxygen tests. The goal is to understand the drug's effects, n…

This study looks at whether a genetic test can predict who will develop Paget's disease of the bone, a condition that can cause pain, deformity, and hearing loss. Researchers are studying 135 people aged 45 and older who have a parent or sibling with Paget's but do not have the d…

This study follows 35 children with CLN2 disease (a rare, severe brain disorder) who are taking cerliponase alfa (Brineura). Researchers will monitor for side effects and serious reactions over time. The goal is to better understand the long-term safety of this enzyme replacement…

This study looks at how silicone oil, used to repair detached retinas, affects the cells on the inner surface of the cornea. Researchers will measure cell density and shape changes in 40 patients at 1 and 3 months after surgery. They also want to see if any changes go away after …

This observational study will follow 2,500 adults with vision loss from nAMD or DME who are already prescribed aflibercept 8 mg by their doctors. Researchers will collect data from routine visits over up to 24 months to see how vision changes. The goal is to understand how well t…

This study looks at how HIV infection and aging impact the body's response to the Prevnar 13 vaccine, which protects against pneumococcal bacteria. Researchers will measure antibody levels in the lungs, nasal passages, and blood of participants aged 21-45 or 55-75, comparing thos…

This study looked at how sounds played during sleep affect brain waves and memory in children with Rolandic epilepsy. Researchers used brain scans (MRI, MEG/EEG) to understand which brain circuits are involved. The goal was to find new treatment ideas for this type of childhood e…

This study tests whether offering online educational videos and mailed saliva genetic testing kits helps more first-degree relatives of people with BRCA mutations get tested, compared to standard care with a family letter. About 820 participants will be enrolled across several me…

This study collects blood and other samples from people with sickle cell disease and healthy volunteers to help researchers understand how inflammation affects blood vessels. Up to 2,000 adults aged 18 and older will take part. The samples will be used in lab tests to support fut…

This study tests a new 'red flag' classification system to predict which women might faint or feel faint during office hysteroscopy, a procedure where a small camera is inserted into the uterus. Researchers will monitor 100 women for signs of distress and pain during the procedur…

This study tests whether combining standard monitoring tools with new light-based sensors can better detect a dangerous narrowing of the aorta in newborns. Infants under one month old with suspected aortic arch obstruction will be monitored using resonance Raman spectroscopy, pul…

This study follows 154 people taking Epidiolex (cannabidiol) for seizures caused by Lennox-Gastaut syndrome, Dravet syndrome, or tuberous sclerosis complex. Researchers will monitor for signs of chronic liver injury and fibrosis over time using blood tests and liver scans. The go…

This study examines health data from about 800,000 Korean adults to see how often conditions like obesity, type 2 diabetes, heart disease, chronic kidney disease, and fatty liver occur together. Researchers will analyze national survey and insurance records from 2013 to 2023. No …

This observational study collects extra tumor tissue from 416 people with various cancers (bladder, kidney, colorectal, head and neck, liver, lung, skin, endometrial) during routine biopsies. The goal is to develop and train a 'live tumor diagnostic platform' that can predict whe…

This study aimed to see if how well a person can fix their gaze relates to signs of active disease on eye scans in patients with age-related macular degeneration (ARMD) treated with antiVEGF injections. Researchers planned to follow patients for two years, measuring fixation qual…

This study is a national registry in China that will track 500 women who experience an aortic dissection (a tear in the main artery) during pregnancy or within 12 weeks after giving birth. Researchers will collect information on treatments and outcomes for both mothers and babies…

This study looks back at medical records of 500 adults with aggressive B-cell lymphoma that has a MYC gene change. Researchers want to see if a stronger chemotherapy plan called CARMEN leads to better survival and remission rates compared to other treatments. Since it's a review …

This study follows 25 adults with inactive lupus to see if blood samples can predict when a flare will happen. Researchers will analyze genes and other molecules to find patterns linked to flare risk. The goal is to develop tools that help doctors personalize treatment and preven…

This study was designed to look back at the health of people with Cushing's disease in Western France between 1990 and 2015. The goal was to find out how many patients went into remission and how many had their disease come back after their first surgery. However, the study was w…

This study looks at how a brain procedure called embolization affects the immune system. Researchers will take blood samples from 78 patients before and after the procedure to measure immune markers. They want to understand why patients with brain arteriovenous malformations (AVM…

This Phase 2 trial tests whether dichloroacetate (DCA), a drug that affects cell energy use, can alter tumor metabolism in people with recurrent glioblastoma. Forty patients scheduled for surgery will either receive DCA or no drug for one week before their operation. Researchers …

This study aims to describe French gamblers who are at risk or have a pathological gambling problem. Researchers will collect information on their gambling history, personality, and other health issues. The goal is to compare this data with studies from other countries and to tes…

This study aimed to see if Alzheimer's disease affects the nerves in the gut, similar to what happens in Parkinson's disease. Researchers planned to take extra colon biopsies during routine colonoscopies from people with Alzheimer's, Parkinson's, and healthy individuals to compar…

This study is building a large gene bank by collecting blood samples and health information from nearly 10,000 people who have had heart catheterization or cardiac CT scans. The goal is to identify genetic and other factors that contribute to coronary artery disease. Participants…

This study explores whether artificial intelligence can predict which children with a type of heart hole (perimembranous ventricular septal defect) will later develop complications like valve problems or blockages. Researchers will analyze past echocardiogram reports and medical …

This study looks at whether regular text message check-ins and the ability to text your IBD care team can improve disease management and quality of life. About 900 people with Crohn's disease or ulcerative colitis will take part. Some will get extra symptom surveys and texting ac…

This study tests a short guide called the 'Jumpstart Guide' to help doctors have better conversations with dementia patients and their families about healthcare goals. Over 1,800 patients and their caregivers are taking part. Researchers will compare whether those whose doctors u…

This study looks at whether two blood markers, zonulin and LBP, can help detect liver damage in people with both ulcerative colitis (UC) and fatty liver disease. Researchers will measure these markers and use a special ultrasound to check liver stiffness in 100 adults with UC who…

This study follows 200 adults aged 30-55 who smoke or have early COPD to learn how the disease progresses. Researchers will use breathing tests, symptom surveys, and CT scans to find early warning signs. The goal is to better understand risk factors and improve future care for CO…

This study investigates how the brain's emotion network works by recording brain activity directly from the surface of the brain in people with epilepsy who are already undergoing surgery for seizure monitoring. Participants watch short video clips designed to trigger emotions li…

This study was designed to compare electrical and mechanical activation of the right ventricle in adults who had surgery for Tetralogy of Fallot, a congenital heart condition. Researchers planned to use 3D mapping and imaging to see if electrical signals match the heart's physica…

This study aims to improve how doctors test for autonomic failure, a condition where the body's automatic functions like blood pressure control don't work properly. Researchers will use advanced imaging, sweat tests, and skin biopsies in up to 89 adults with Parkinson's disease, …

This study is a global registry that will follow about 500 people with pyruvate kinase (PK) deficiency, a rare inherited anemia, for up to 9 years. Researchers will collect medical data to better understand the disease's natural history, treatments, and complications. No new drug…

This long-running study follows nearly 100,000 French women, mostly teachers, since 1990 to understand what factors influence their risk of cancer, heart disease, diabetes, and other major illnesses. Participants fill out questionnaires every few years about their lifestyle, medi…

This study follows 30 adults aged 45-80 who are having spinal fusion surgery for spondylolisthesis. Researchers will use CT scans to measure changes in the bone graft placed between vertebrae over time. The goal is to develop an objective way to assess whether the bones have fuse…

This study looks at how adults at risk for Alzheimer's disease react after learning their amyloid and tau biomarker test results. Researchers will track changes in distress, stigma, quality of life, and future planning over time. The goal is to understand the psychological and be…

This study looks at the genetic makeup of rare blood diseases like Castleman disease and Langerhans cell histiocytosis. Researchers will analyze blood or tissue samples from 135 participants to find genetic changes that could be targeted by existing or experimental therapies. The…

This study collects blood and bone marrow samples from up to 550 people with blood cancers like leukemia, lymphoma, and myelodysplastic syndrome. Researchers will study the cells, genes, and proteins in these samples to better understand how these cancers develop and to find new …

This study looks at HIV patients over 40 (or those on HIV therapy for over 10 years) who switched to either a three-drug or a two-drug antiretroviral regimen. Researchers will measure markers of inflammation in the blood and track how many patients develop other health conditions…

This study will follow 3,000 people with COPD who have frequent flare-ups to see how the disease changes over time and what treatments they receive. Researchers will collect information from medical records and surveys to understand treatment patterns, quality of life, and caregi…

This study was designed to see if giving a short, mild electrical stimulation to compressed nerves during surgery could speed up recovery of strength and function. It planned to include people with nerve compression in the spine or limbs who were already scheduled for decompressi…

This study looks at how patients with advanced blood cancers (like acute myeloid leukemia) who are not eligible for a cure, along with their nurses and doctors, feel about the benefits of blood transfusions. Researchers will give questionnaires before each transfusion to compare …

This study follows people with advanced dry age-related macular degeneration who previously received a one-time injection of the experimental treatment VOY-101. Researchers are monitoring participants over the long term to check for any side effects related to the treatment. The …

This study looks at whether having a heart doctor check patients during a COPD flare-up helps find and treat hidden heart problems. 150 hospitalized adults with COPD are randomly assigned to see only a lung doctor or also a heart doctor. Researchers then track changes in heart tr…

This study aims to find early DNA changes in blood and esophageal tissue that could signal a higher risk of developing esophageal cancer. Researchers are studying 600 people with gastroesophageal reflux, Barrett's esophagus, or esophageal cancer who are already scheduled for an u…

This pilot study tests whether daily, at-home stimulation of a nerve in the ear can change brain activity in people with inflammatory bowel disease (IBD) who are in remission. Fifteen adults with Crohn's disease or ulcerative colitis will use a small device for 28 days and underg…

This study looks at how often new pupil differences (one pupil larger than the other or not reacting normally) appear in adult intensive care patients. Researchers will review medical records from 2013 to 2024 to see what these changes mean for diagnosis, treatment, and patient o…

This study follows 400 people with intermediate age-related macular degeneration (AMD) for about 3 years. Researchers watch how the disease advances to more severe stages and test if measuring the loss of light-sensing cells in the eye can be a useful way to track the disease. No…

This early-stage trial tests a new drug called AJU-C716 against a reference drug (C716R) in 41 healthy adults aged 19 to 55. The goal is to see how safe the drug is and how the body processes it. This study does not test if the drug works for high cholesterol—it only looks at saf…

This natural history study follows 259 children, adolescents, and adults with Neurofibromatosis Type 1 (NF1) for up to 10 years. Researchers will track tumor growth, monitor quality of life, and perform genetic testing to better understand how the disease changes over time. The g…

This study follows 250 obese adults who are having a weight-loss procedure—either surgery or an endoscopic stomach reduction. The goal is to see how often acid reflux (GERD) develops afterward, including silent reflux with no symptoms. By tracking symptoms and using tests, resear…

This study aims to find the genes that cause inherited hearing loss by analyzing DNA from 24,000 people in large Pakistani families. Researchers will compare affected and unaffected family members to identify new deafness genes. The goal is to improve genetic testing and counseli…

This study aims to understand inflammation in four chronic skin conditions: vitiligo, psoriasis, eczema (atopic dermatitis), and alopecia areata. Researchers will collect blood and small skin samples from 450 adults to analyze immune cells called T-cells. The goal is to describe …

This study tracks a protein called neurofilament light chain in the blood of 342 people who carry genetic mutations linked to frontotemporal dementia (FTD). Participants provide blood samples every three months for three years, often from home. The goal is to see if this biomarke…

This study aims to identify biomarkers—biological clues in the body—that can help distinguish between Parkinson's disease and similar conditions like progressive supranuclear palsy and multiple system atrophy. Researchers will follow people with early-stage parkinsonism and healt…

This study examines how often healthcare workers in a hospital's intensive care unit (ICU) experience physical, verbal, or sexual assaults from patients or their relatives. Researchers will review medical records of 865 adults who were in the ICU and had a reported incident of vi…

This study looks at whether a special electronic pill bottle (AdhereTech) can accurately track how often teens with sickle cell disease take their medication, hydroxyurea. About 36 teens aged 12 to 17 will use the bottle for two months. Researchers will compare the bottle's data …

This study screens children and teens with advanced solid tumors, lymphomas, or histiocytic disorders to find genetic changes in their cancer cells. Participants undergo biopsies and scans to identify mutations that can be targeted by specific drugs. The goal is to see if matchin…

This study is a registry that will follow about 500 adults with lupus (SLE) in Kazakhstan to see how the disease behaves and how doctors treat it in everyday practice. Researchers will look at disease activity, organ damage, and which medications are used. No new treatments are b…

This study surveys 500 doctors in Egypt to see how well they understand prediabetes—a condition where blood sugar is high but not yet diabetic. Doctors answer questions about risk factors, diagnostic tests, and lifestyle recommendations. The goal is to find gaps in knowledge that…

This was a planned French study to collect information on chronic myeloid leukemia (CML) in young adults aged 18 to 30 who were treated with tyrosine kinase inhibitors. The goal was to describe their diagnosis, treatment choices, side effects, and quality of life. However, the st…

This study follows 70 infants with cystic fibrosis to see if a specific germ (Porphyromonas catoniae) found in their body at 12 months old can predict whether they will get a serious lung infection with Pseudomonas aeruginosa by age 3. Researchers collect sputum, stool, and blood…

This study compares stainless steel and titanium-molybdenum alloy wires for moving canine teeth in people with braces. Sixteen participants will have one type of wire on each side of their upper jaw to see which works better. The goal is to find out which wire moves teeth more ef…

This long-term study looks at people and families with a higher chance of developing blood or lymph node cancers like leukemia or lymphoma. Researchers collect medical histories, genetic samples, and sometimes perform exams to find inherited genes or environmental triggers. The g…

This study tracks 585 patients with familial or regular high cholesterol who are already prescribed Leqvio. Researchers will monitor side effects and cholesterol levels over time to see how safe and effective the drug is in everyday use. No new treatment is given—it's purely obse…

This study follows 214 men treated for prostate cancer to see how many develop lower limb lymphedema (leg swelling) and who is most at risk. Participants attend check-ups every three months for 18 months. The goal is to better understand this side effect and its impact on quality…

This study looks at X-linked juvenile retinoschisis (XLRS), a rare inherited eye disease that causes vision loss in young males. Researchers will examine 351 participants, including affected males and female carriers, using eye exams, retinal photos, and genetic tests. The goal i…

This study follows 1,500 volunteers with normal memory, mild cognitive impairment, or Alzheimer's over several years. Researchers use brain scans, memory tests, and blood samples to track how the disease progresses. The goal is to find better ways to diagnose and treat Alzheimer'…

This study tracks the safety and effectiveness of the drug Alhemo (concizumab) in 23 people with hemophilia A or B in Japan. Participants receive the drug as part of their normal care, and researchers will monitor side effects for about 2 years. The goal is to see how well Alhemo…

This study looks at whether taking short walks throughout the day is better for kids at risk for type 2 diabetes than one longer exercise session. Researchers will measure blood sugar, insulin, and thinking skills in 188 children aged 8-11 with overweight or obesity. The goal is …

This study follows 80 people aged 8 to 50 with Stargardt disease, a genetic eye condition that causes vision loss. Researchers will monitor how the disease changes over time using eye imaging. The goal is to better understand the disease and prepare for future clinical trials.

This study is observing 217 people with ALS and related conditions like frontotemporal dementia and hereditary spastic paraplegia. Researchers aim to connect each person's genetic makeup with their symptoms and find biological markers in blood and spinal fluid. No new treatments …

This study looks at people with ALS and similar diseases like frontotemporal dementia and hereditary spastic paraplegia. Researchers want to understand how genes affect the disease and find biological markers (biomarkers) to help develop future treatments. About 708 participants,…

This study investigates how genetic, lifestyle, and biological factors relate to memory and thinking problems in people over 60 with Alzheimer's disease, Parkinson's disease, or mild cognitive impairment. Researchers will use brain scans, spinal fluid and blood tests, and data fr…

This study aimed to see if a portable device could reliably collect breath samples from people with cystic fibrosis and healthy volunteers while they walked in parks or city streets. The goal was to measure certain substances in the breath that might show how the environment affe…

This study enrolls people with ALS and related disorders, as well as healthy volunteers, to track biological markers (biomarkers) in urine, blood, and spinal fluid. The goal is to better understand how these markers change over time, which could help design more effective future …

This study looks at people and families who have a high risk of cancer to learn what genes and environmental factors might increase that risk. Over 5,000 participants are followed over time with questionnaires, medical records, and optional genetic testing. No treatment is given,…

This study at Duke University tests whether a special MRI scan using inhaled xenon gas can detect changes in lung function over time in people with progressive pulmonary fibrosis. Sixty adults with various types of lung scarring will have MRI scans before starting anti-fibrotic m…

This 24-month observational study will follow 80 people with Becker muscular dystrophy (BMD) to better understand how the disease progresses. Researchers will measure muscle strength, walking speed, breathing, and heart function using standard tests. The goal is to identify which…

This 24-month observational study follows 100 people aged 12–50 with Limb Girdle Muscular Dystrophy type R1 (LGMD R1). Researchers will test whether a motor function scale called NSAD and muscle fat measurements from MRI can reliably track disease progression. The goal is to vali…

This study tracks brain activity in 160 Parkinson's patients who have or will receive deep brain stimulation (DBS). Researchers record signals from the brain during memory tasks, with the stimulator on and off, to find patterns linked to cognitive side effects. The goal is to ide…

This study tests new radio frequency coils for MRI scanners to see if they can produce better images in children, including those with Duchenne muscular dystrophy. Researchers will check for side effects like heating and discomfort, and measure image quality. The goal is to impro…

This study follows about 35 people with dilated cardiomyopathy caused by a BAG3 gene mutation, a condition that weakens the heart muscle. Over three years, researchers will monitor heart function, symptoms, and quality of life using tests like imaging and blood work. The goal is …