Disorder of development or morphogenesis

This early-stage study tests a new drug, PF-08046049/SGN-BB228, in people with advanced melanoma and other solid tumors that have spread or stopped responding to standard treatments. The main goal is to check the drug's safety and find the best dose. About 41 participants will ta…

This early-phase trial tested two lab-made antibodies, magrolimab and dinutuximab, together in 12 children and young adults whose neuroblastoma or osteosarcoma had returned or stopped responding to treatment. The main goal was to find a safe dose and watch for side effects. Resea…

This study tests whether ARD-101 can safely reduce extreme hunger and food-related behaviors in people with Prader-Willi syndrome over 12 months. About 90 participants who completed a prior study will take the drug daily and visit the clinic regularly. The goal is to improve qual…

This study tested an experimental drug called SAR442501 in children with achondroplasia, a common form of dwarfism. The goal was to see if the drug was safe and could improve growth. However, the trial was stopped early after enrolling only 16 children, so we have very little inf…

This study compared two antiplatelet drug regimens after transcatheter aortic valve implantation (TAVI) for aortic stenosis. One group received ticagrelor alone, the other received aspirin plus clopidogrel. The goal was to see which caused fewer serious complications like bleedin…

This study tested whether adding timolol gel to pulsed dye laser treatment could improve results for port wine stains on children's faces. Twenty-five children aged 6 months to 18 years were enrolled, but the study was stopped early. The goal was to see if the combination led to …

This early-stage study tested a new drug called CLN-617, alone or with the immunotherapy Keytruda, in 23 adults with advanced solid tumors that had not responded to standard treatments. The goal was to check safety and find the right dose. However, the trial was terminated, so fu…

This early-stage trial tested a two-step radioimmunotherapy combining GD2-SADA and 177Lu-DOTA in patients with recurrent or metastatic solid tumors, including small cell lung cancer, high-risk neuroblastoma, sarcoma, and melanoma. The goal was to assess safety and find the right …

This study tested a new combination treatment for people with advanced melanoma that cannot be removed by surgery or has spread. The combination included an experimental drug injected into tumors (CMP-001) plus an immunotherapy given through a vein (nivolumab). It was compared to…

This early study tested a new cell therapy called LUCAR-G79 in just 3 adults with severe autoimmune diseases that did not respond to standard treatments. The therapy uses specially engineered immune cells to target and potentially calm the overactive immune system. The main goals…

This trial tests whether performing balloon aortic valvuloplasty without the blood thinner heparin reduces serious bleeding and blood vessel complications. About 94 adults with severe, symptomatic aortic stenosis will be randomly assigned to receive either heparin or a placebo du…

This study tested two different proton therapy schedules for people with large choroidal melanomas (eye cancer) that would normally require removal of the eye. The goal was to see if a gentler radiation schedule could control the tumor and avoid severe side effects, allowing pati…

This study tested a new drug called acasunlimab, given alone or with pembrolizumab, in people with advanced melanoma that had stopped responding to standard treatments. The trial was terminated early after enrolling only one participant, so no conclusions can be drawn about safet…

This early-phase trial tested a combination of two oral drugs, mirdametinib and brimarafenib, in 23 adults with advanced solid tumors that had stopped responding to standard treatments. The goal was to check safety, find the best dose, and see if the combo could shrink tumors. Th…

This study tests whether an additional dose of a COVID-19 vaccine can improve immune protection in adults and children with autoimmune diseases like rheumatoid arthritis, lupus, multiple sclerosis, and others who are taking immunosuppressive medications. Participants who had a we…

This study tested whether injecting vidutolimod directly into tumors plus giving cemiplimab through a vein could shrink cancers that have spread. The trial included 77 people with several types of advanced cancer. Unfortunately, the study was stopped early, so we have less inform…

This early-stage study tested a new drug called PF-07265807 in people with advanced or metastatic solid tumors that had spread. The main goals were to find a safe dose and check for side effects. The trial was terminated early, so results are limited.

This study tested a combination of two drugs, pembrolizumab and lenvatinib, in 18 adults with melanoma or kidney cancer that had spread to the brain. Participants had already received anti-PD-1/PD-L1 therapy. The goal was to see if the drug combo could shrink or eliminate brain t…

This early-stage study tested a new drug called PF-07284892, alone or with other medicines, in people with advanced solid tumors that had specific genetic changes. The goal was to find the safest dose and check for side effects. The study was stopped early, so results are limited…

This early-stage trial tested a combination of an experimental fusion protein (hu14.18-IL2), radiation, and two immunotherapy drugs (nivolumab and ipilimumab) in people with advanced melanoma that couldn't be surgically removed. The goal was to find the safest dose and check for …

This study tested whether adding hydroxychloroquine (a malaria drug) to standard targeted therapy (dabrafenib and trametinib) could help control advanced melanoma that had spread or could not be removed by surgery. It was designed for people with a specific gene mutation (BRAF V6…

This study tested two drugs, encorafenib and binimetinib, given before surgery to people with stage IIIB-D melanoma that had spread to lymph nodes. The goal was to see if the drugs could shrink or eliminate the cancer before removal. The trial was stopped early after enrolling on…

This study tested a combination of an immunotherapy drug (nivolumab) and low-dose chemotherapy in children and teenagers whose solid tumors had not responded to standard treatments. The goal was to see if the combination was safe and could help control the cancer. The trial was s…

This early-phase trial tested ASP1012, a virus engineered to infect and kill cancer cells while making them visible to the immune system. It was given alone or with the immunotherapy pembrolizumab to 15 adults with advanced solid tumors. The main goals were to check safety and fi…

This study tested an experimental drug called CRD-4730 in people with a rare inherited heart rhythm disorder (CPVT) that can cause dangerous fast heartbeats. Seven adults received two different doses of the drug and a placebo to check safety and how the body processes it. The stu…

This study tested whether injections of MYOBLOC (botulinum toxin type B) can safely reduce muscle tightness in the arm and hand of adults with spasticity caused by stroke, brain injury, multiple sclerosis, or other conditions. The trial planned to enroll about 300 participants bu…

This phase III trial compared two radiation strategies for cancer that had spread to the brain and returned in new spots after initial radiosurgery. One group received focused radiation only to the new tumors, while the other received whole brain radiation that avoided the memory…

This study tested a personalized treatment using immune cells taken from a patient's own tumor, called tumor-infiltrating lymphocytes (TIL). The cells were grown in a lab and given back to the patient to fight their cancer. The trial included people with advanced melanoma, head a…

This early-phase study tested a new drug called PF-08046032, given alone or with another drug (sasanlimab), in adults with advanced lymphomas or solid tumors like lung cancer and melanoma. The trial aimed to check safety and find the right dose, but it was terminated early after …

This study tested whether clemastine fumarate, an allergy medication, could improve eye movement problems in people with multiple sclerosis (MS). The trial planned to enroll 80 MS patients with a specific eye condition called internuclear ophthalmoparesis, but was terminated earl…

This study tested whether adding acetaminophen to standard ibuprofen treatment could better close a blood vessel (ductus arteriosus) that stays open too long in very premature infants. The trial was planned for preterm babies born at or before 27 weeks and 6 days who needed breat…

This early-phase trial tested the drug plinabulin combined with radiation and immunotherapy in 19 people with advanced cancers (like lung, bladder, or melanoma) that had stopped responding to standard immunotherapy. The goal was to see if the combination was safe and could shrink…

This phase II trial tested whether adding radiotherapy to the immunotherapy drug pembrolizumab could better control advanced melanoma than pembrolizumab alone. Only 17 people took part before the study was stopped early. The goal was to see if radiation could trigger an 'abscopal…

This study tested a combination of two drugs, ipilimumab and epacadostat, in people with advanced melanoma that could not be removed by surgery or had spread. The goal was to see if adding epacadostat to standard treatment improved survival. However, the study was stopped early f…

This study tested a long-acting form of glatiramer acetate (GA Depot) given as a monthly shot to people with primary progressive multiple sclerosis (PPMS). The goal was to see if it could safely slow down worsening disability. The trial was stopped early, but it involved 30 adult…

This early-stage study tested a new drug called PF-08046037, alone or with another drug (sasanlimab), in 8 people with advanced lung, pancreatic, melanoma, or head & neck cancers. The goal was to check safety and how the body handles the drugs. The trial was terminated early, so …

This early-stage trial tested an oral drug called TNO155, alone or with another drug (nazartinib), in 227 adults with advanced solid tumors like lung cancer, melanoma, and head/neck cancer. The main goal was to check safety and find the right dose. The study was terminated early,…

This study tested whether the statin drug rosuvastatin, taken daily with supplements, could improve bone density and reduce heart disease risk in adults with long-term spinal cord injury. Only 8 people enrolled before the trial was stopped early. Participants received either rosu…

This early-stage trial tested an investigational drug called TransCon TLR7/8 agonist, alone or with the immunotherapy pembrolizumab, in people with advanced solid tumors that could not be cured by surgery or radiation. The study aimed to find a safe dose and check for side effect…

This phase 2 trial tested telitacicept (RC18) in 8 people with relapsing-remitting multiple sclerosis. The drug aims to reduce relapses by targeting B cells. The study was terminated early, so we have limited data on safety and effectiveness.

This study tested a treatment called Chondron, made from a patient's own cartilage cells, for repairing ankle cartilage defects. The trial aimed to see if the treatment could improve cartilage quality and ankle function over 18 months. However, the study was terminated early and …

This early-stage study tested a new drug combination (MGN1703 plus ipilimumab) in people with advanced solid tumors that had stopped responding to standard treatments. The main goal was to find the safest dose of MGN1703 when given with ipilimumab. The study was stopped early, bu…

This study tested a drug called vatiquinone in 68 people with mitochondrial disease and epilepsy that doesn't respond to standard treatments. Participants were randomly assigned to receive either vatiquinone or a placebo for 24 weeks to see if the drug could reduce the number of …

This was a very early Phase 1 study testing the safety of a drug called bryostatin in just 4 people with multiple sclerosis. Participants received multiple doses over 26 weeks. The study was terminated, meaning it stopped early, so we don't have clear results on whether the drug …

This early-phase trial tested a new drug called nanrilkefusp alfa, which helps activate immune cells, both alone and combined with the immunotherapy pembrolizumab. It included 115 people with various advanced solid tumors that had stopped responding to standard treatments. The st…

This early-phase study tested a new drug called PF-07921585, given alone or with another drug (sasanlimab), in adults with advanced solid tumors like lung, bladder, kidney, melanoma, head & neck, or colorectal cancer. The goal was to check safety and find the right dose. Only 4 p…

This early-phase study tested a treatment using donated natural killer (NK) cells from cord blood, combined with chemotherapy, for children and young adults with solid tumors that returned or didn't respond to standard care. The goal was to find the safest dose and check for side…

This early-stage study tested a new drug called PF-08046031 in 11 adults with advanced melanoma or other solid tumors that had spread or had no standard treatment options. The main goal was to check the drug's safety and side effects. The trial was terminated early, so its findin…

This study tested a combination of two drugs, bintrafusp alfa and pimasertib, in people whose cancer had spread to the brain. The goal was to find the safest dose and see if the treatment could slow or stop the cancer from growing. The trial was stopped early after enrolling only…

This study looked at two types of implantable cardioverter defibrillator (ICD) leads in children and patients with congenital heart disease. The goal was to see which lead type lasts longer without breaking and is easier to remove if needed. The trial was terminated early, so res…

This phase II trial tested the drug imatinib in 13 adults with metastatic ocular melanoma, a rare eye cancer that has spread. The goal was to see if imatinib could stop tumor growth for at least 3 months. The trial was terminated early, so the results are limited and it is unclea…

This study tested whether a three-drug combination (encorafenib, binimetinib, and pembrolizumab) works better than a two-drug combo (ipilimumab and nivolumab) for people with advanced melanoma that has a BRAF mutation and has already been treated with anti-PD-1 therapy. The trial…

This trial tested a personalized approach for children with relapsed or hard-to-treat neuroblastoma. Doctors used a tumor biopsy to find genetic changes and then assigned a combination of targeted drugs (ceritinib and ribociclib) to match those changes. The study was terminated e…

This early-phase trial tested an oral drug called DCC-3084, alone or with other cancer treatments, in 29 people with advanced solid tumors that have MAPK pathway mutations (like certain lung, pancreatic, or skin cancers). The study aimed to find safe doses and see if tumors shrin…

This early-stage trial tested a new drug called DFF332, which targets a protein (HIF2α) that helps some cancers grow. The study included 40 adults with advanced clear cell renal cell carcinoma or other cancers with specific gene changes. DFF332 was tested alone and in combination…

This study tested a drug called BVD-523 (Ulixertinib) in 13 people with advanced uveal melanoma, a rare eye cancer that has spread. The drug works by blocking certain proteins that help cancer grow. The trial was terminated early, so results are limited and it's unclear how well …

This study tested a surgical method to prevent lymphedema (arm swelling) in people having lymph nodes removed for breast cancer or melanoma. The technique, called axillary reverse mapping with lympho-venous bypass, aims to preserve arm drainage while removing cancer-affected node…

This early-stage study tested a new drug called PF-07826390, given alone or with another drug (sasanlimab), in people with advanced solid tumors like lung, kidney, and colorectal cancers. The goal was to check safety and find the right dose. Only 10 people took part, and the stud…

This study tested a biosimilar (JPB898) against the original immunotherapy Opdivo, both combined with Yervoy, in 52 adults with advanced melanoma that could not be surgically removed. The goal was to see if the biosimilar works just as well. However, the trial was terminated earl…

This phase II trial tested a mix of a personalized dendritic cell vaccine and three immune-boosting drugs in people with advanced melanoma that no longer responded to PD-1/PD-L1 checkpoint inhibitors. The goal was to see if the combination could shrink tumors or slow their growth…

This study tested whether adding melatonin to the standard drug ocrelizumab could safely slow disability in people with progressive primary multiple sclerosis. The trial was terminated early, so results are limited. Participants were adults aged 18–65 with this type of MS.

This phase 2 trial tested the drug guadecitabine (SGI-110) in people aged 12 and older with rare cancers like wild-type GIST, pheochromocytoma, paraganglioma, and HLRCC-related kidney cancer that had not responded to other treatments. Participants received daily injections for 5 …

This study tested an experimental drug called vobramitamab duocarmazine in people with advanced prostate cancer and other solid tumors that stopped responding to standard treatments. The goal was to see if the drug could shrink tumors or slow cancer growth. The trial was terminat…

This early-phase study tested a radioactive drug called 225Ac-SSO110 combined with standard immune checkpoint inhibitors for people with advanced small cell lung cancer or Merkel cell carcinoma. The goal was to check safety and find the right dose. However, the trial was terminat…

This early-phase study tested whether a fecal transplant from a healthy donor could be safe and affect the immune system in people with relapsing-remitting multiple sclerosis. Only 5 participants were enrolled before the study was stopped. The goal was to see if changing gut bact…

This early-phase trial tested taselisib, a drug that blocks a faulty protein causing excessive growth in people with PIK3CA-related overgrowth. Nineteen participants took the drug daily for four weeks to see if it was safe and tolerable. The study was stopped early, so we have li…

This early-phase trial tested a new drug (SAR444245) combined with an existing immunotherapy (cemiplimab) in 46 adults with advanced melanoma or squamous cell skin cancer that could not be surgically removed. The goal was to see if the combination shrank tumors. However, the stud…

This early-phase trial tested a new therapeutic vaccine (EO2401) combined with the immunotherapy drug nivolumab in 70 people with advanced adrenocortical carcinoma or malignant pheochromocytoma/paraganglioma. The goal was to see if the combination is safe and can shrink tumors or…

This early-phase trial tested the safety and dosing of a new drug called modakafusp alfa, given alone or with pembrolizumab, in adults with advanced solid tumors or melanoma. The study aimed to find the highest safe dose and check for side effects. It was terminated early, so res…

This study tested a combination of two drugs, cabozantinib and pembrolizumab, as a first treatment for people with advanced melanoma that had spread. The goal was to see if the combination was safe and could shrink tumors. The trial was stopped early, but the results help guide f…

This Phase 3 trial tested an oral drug called venglustat in 75 adults and children with late-onset Tay-Sachs or Sandhoff disease, rare genetic disorders that cause progressive nerve damage. The drug aimed to lower toxic fat buildup in the brain and slow disease worsening. However…

This early-stage trial tested a modified virus (VSV-IFNbetaTYRP1) in 12 patients with advanced melanoma that had spread or could not be removed by surgery. The virus was designed to infect and kill melanoma cells while also boosting the immune system's attack. The main goals were…

This study tested leflunomide, a drug used for rheumatoid arthritis, in 3 adults with MEN1 syndrome—a genetic condition that raises the risk of multiple tumors. The goal was to see if 6 months of daily leflunomide could shrink or stabilize tumors. However, the trial was terminate…

This early-stage study tested a combination of two drugs, niraparib and dostarlimab, in children and teens (ages 6 months to 17 years) with solid tumors that had come back or were not responding to treatment. The goal was to find a safe dose and see if the drugs could shrink tumo…

This early-phase trial tested an experimental drug called OC-001, given alone or with the immunotherapy avelumab, in 73 people with various advanced or metastatic cancers (like lung, breast, and bladder cancer). The main goals were to check safety and how the drug moves through t…

This study tested whether combining two immunotherapy drugs (nivolumab and relatlimab) works better than nivolumab alone to keep melanoma from returning after surgical removal. It involved over 1,000 adults and teens with stage III or IV melanoma that had been completely removed.…

This early-phase trial tested a radioactive drug designed to seek out and destroy cancer cells in people with advanced solid tumours that had stopped responding to other treatments. The drug targets a protein called IGF-1R found on many cancer cells. The study aimed to find a saf…

This study tested a new drug called CX-072 combined with other cancer treatments for adults with solid tumors or advanced melanoma. The goal was to see if the combination could shrink tumors. However, the study was stopped early and only 3 people took part, so we don't have enoug…

This early-phase study tested a new drug called KZR-261 in 61 people with advanced solid tumors that had stopped responding to standard treatments. The main goals were to check safety, find the right dose, and see if the drug could shrink tumors. The trial was terminated, but the…

This early study tested a new procedure called SESAME that uses a wire threaded through blood vessels to slice away excess heart muscle in people with hypertrophic cardiomyopathy. Only one adult participated before the study was terminated. The goal was to see if the technique co…

This study tested a combination of two drugs—duvelisib (a pill) and nivolumab (an IV infusion)—in 13 people with advanced melanoma that had stopped responding to standard immunotherapy. The goal was to find a safe dose and see if the combination could shrink tumors or slow the di…

This study tested whether taking a pill containing gut bacteria (EDP1503) could help the immunotherapy drug pembrolizumab (Keytruda) work better in people with advanced melanoma. The trial included two groups: those who had never received anti-PD1 therapy and those whose cancer h…

This early-phase trial tested whether giving the immunotherapy drug STI-3031 directly into the lymph nodes or vessels (using a special device) is safe for people with in-transit melanoma, a type of skin cancer that spreads through lymph channels. Only 3 people took part before th…

This study tested the drug alemtuzumab in children aged 10 to 18 with relapsing-remitting multiple sclerosis (MS) whose disease was still active despite other treatments. The goal was to see if the drug could reduce new brain lesions and improve quality of life. However, the tria…

This study tested the drug natalizumab in 21 Japanese adults with relapsing-remitting multiple sclerosis (MS). The goal was to see if injections under the skin every 4 weeks could reduce new brain lesions over 24 weeks, and to check safety for up to 48 weeks. The study was termin…

This study followed one person with microtia (a small or missing outer ear) who had already received an AUR-201 implant — a 3D-printed ear made from their own cartilage cells. Researchers checked how well the implant kept its shape and whether any side effects appeared up to 96 w…

This trial tested a new ear implant called AUR-201 for people with a rare birth defect called microtia, where one ear is small or missing. The implant was custom-made using the patient's own cartilage cells and a 3D-printed scaffold. Only 3 people were enrolled before the study w…

This early-phase trial tested a new treatment called BT-001, a virus designed to infect and kill cancer cells, given alone or with the immunotherapy drug pembrolizumab. The study involved 31 people with advanced solid tumors like melanoma, lung cancer, or sarcoma that had spread.…

This study tested a new drug combination for people with metastatic melanoma (skin cancer that has spread). The goal was to find a safe dose of an experimental drug (APX005M) injected directly into tumors, combined with an approved immunotherapy (pembrolizumab) given through the …

This study tested a device called Leaflex™ Performer, which is threaded through a blood vessel to the heart to score and loosen calcium buildup on stiff aortic valves. It was designed for people with severe aortic stenosis who cannot have standard valve surgery or replacement. Ho…

This study tested an experimental implant called AUR-201 to rebuild the outer ear in people with unilateral microtia (one ear that is small or missing). Only 2 people enrolled before the study was stopped early. The goal was to check safety and see if the implant improved ear app…

This early-stage study tested a new drug called BDC-3042, alone or with another drug (cemiplimab), in 17 people with advanced cancers like breast, kidney, ovarian, head and neck, colorectal, lung, or melanoma. The main goal was to check safety and find the right dose. The study w…

This study tested Duobrii, a prescription cream that combines a strong steroid with a retinoid, for treating early-mild acne keloidalis nuchae (AKN) — a chronic scalp condition causing bumps and scarring, mainly in men of African descent. The trial planned to enroll 15 adults wit…

This study looked at whether adding an immunotherapy drug (pembrolizumab) to a targeted therapy (pemigatinib) works better than the targeted therapy alone or standard care for people with advanced bladder cancer that has a specific gene change (FGFR3). The study was stopped early…

This phase 2 study tested the drug pemigatinib in 111 people with advanced solid tumors that have specific FGFR gene mutations or translocations. The goal was to see if the drug could shrink tumors. The trial was terminated early, so the full results are not available.

This early-stage trial tested a gene therapy called SBT101 for adrenomyeloneuropathy (AMN), a rare nerve disease that causes walking difficulties. Eight adults received either the therapy or a sham procedure. The study was terminated early, so we have limited data on safety and e…

This study tested a drug called niraparib in 22 adults with advanced solid tumors (like breast, lung, or pancreatic cancer) that have a specific genetic change called a PALB2 mutation. The goal was to see if the drug could shrink tumors or slow cancer growth. The study was stoppe…

This early-phase trial tested a new drug called INCB062079 in 25 people with advanced liver cancer and other solid tumors. The main goal was to check safety and find the best dose. The study was terminated early, so results are limited.

This early-stage study tested a new drug called HFB301001 in 31 adults with advanced solid tumors (like sarcoma, kidney cancer, or melanoma) that had not responded to prior treatments. The main goal was to check the drug's safety and find the best dose. The study was terminated, …

This study tested a combination of three drugs (azacitidine, pembrolizumab, and epacadostat) in 70 people with advanced solid tumors, including lung and colorectal cancers. The goal was to see if the combination was safe and could shrink tumors. However, the trial was terminated …

This study tested whether adding an immune-boosting drug (NT-I7) to a standard immunotherapy (atezolizumab) could help people with advanced skin cancers like melanoma, Merkel cell carcinoma, and squamous cell carcinoma. The trial included 31 adults whose cancer had not responded …

This study tested a new drug called SD-101, given directly into the liver through a special pressure-enabled infusion, alone or with standard immunotherapy drugs. It was for adults with a rare eye cancer (uveal melanoma) that had spread mainly to the liver. The main goals were to…

This early-phase study tested a new drug called PF-07284890, alone or with another drug (binimetinib), in 65 people with advanced cancers that have a specific genetic change (BRAF V600 mutation), including cancers that spread to the brain. The trial was terminated early, but rese…

This early-stage study tested an experimental drug called INCB099318 in people with advanced solid tumors that had stopped responding to standard treatments. The main goal was to check the drug's safety and how the body processes it. The study was terminated early, so results are…

This early-stage study tested a new drug called ACTM-838 in 10 people with advanced solid tumors that no longer responded to standard treatments. The main goals were to check safety and find the right dose. The study was stopped early, so results are limited.

This study tested an oral drug called SAR443820 in 174 adults with multiple sclerosis (MS) to see if it could lower a marker of nerve damage in the blood. The trial was stopped early because the drug did not meet its main goal of reducing that marker. Participants were followed f…

This early-phase trial tested a combination of three drugs—ribociclib, topotecan, and temozolomide—in children with relapsed or refractory neuroblastoma and other solid tumors. The goal was to find a safe dose and see if the combination could shrink tumors. Only 12 children took …

This phase 2 trial tested the drug tovorafenib (DAY101) in 23 people aged 12 and older with recurrent or progressive solid tumors that have specific genetic changes in the MAPK pathway. The goal was to see how many patients' tumors shrank or disappeared. The study was terminated …

This phase II trial tested a combination of precise radiation therapy followed by injections of aflibercept (Eylea) in people with uveal melanoma, a rare eye cancer. The goal was to see if this approach could control the tumor while reducing side effects like swelling or damage t…

This early study tested an experimental oral drug called IK-595 in people with advanced solid tumors that have specific RAS or RAF gene mutations. The goal was to check safety, dosing, and early signs of tumor shrinkage. The study was terminated before completion, so full results…

This study compared two ways of doing endoscopic strip craniectomy (a keyhole surgery to remove a fused skull seam) in infants under 6 months with sagittal craniosynostosis. The goal was to see which method leads to better head shape and fewer complications. Only 6 babies were en…

This phase 3 study tested whether adding an immunotherapy drug (spartalizumab) to two targeted therapy drugs (dabrafenib and trametinib) works better than the targeted drugs alone for people with advanced BRAF V600 mutant melanoma. The trial enrolled 568 participants but was term…

This early-stage trial tested a new drug, PF-07820435, alone or with another immunotherapy (sasanlimab) in 9 people with advanced solid tumors like lung, skin, or kidney cancer. The main goals were to check safety and find the right dose. The study was terminated early, so result…

This study tested a radioactive drug called 67Cu-SARTATE in 21 children with high-risk neuroblastoma that had relapsed or not responded to treatment. The drug is designed to deliver radiation directly to tumor cells. The goal was to find the safest dose and see if it shrinks tumo…

This early-stage trial tested a single dose of a drug called SAR439459 in 16 adults with osteogenesis imperfecta (brittle bone disease). The drug aims to block a protein that may weaken bones. The study focused on safety and how the drug moves through the body, but it was termina…

This early-stage trial tested an experimental drug called SAR441000, injected directly into tumors, either alone or with another drug (cemiplimab) given through a vein. The study aimed to find a safe dose and see if the combination could shrink tumors in people with advanced soli…

This phase 2 trial tested two stronger treatment plans for children with very high risk neuroblastoma whose cancer did not respond well to initial chemotherapy. The treatments combined radioactive therapy (mIBG), chemotherapy drugs (topotecan and thiotepa), and a stem cell transp…

This trial investigates whether a new, ultra-high-frequency ultrasound device can accurately measure the depth of skin melanomas before surgery. Currently, doctors must remove the tumor, measure its depth under a microscope, and then often perform a second surgery to remove more …

This study tests whether a tear sample can diagnose primary progressive multiple sclerosis (MS) as accurately as a spinal tap. Currently, doctors need to collect spinal fluid to confirm this type of MS, which is invasive and uncomfortable. Researchers will compare proteins in tea…

This early-phase trial tested a radioactive imaging agent called 68GaNOTA-Anti-MMR-VHH2 to see if it could safely highlight tumors on PET scans. It involved 13 adults with advanced solid tumors, breast cancer, or melanoma. The study was terminated early, so we have limited inform…

This study tested three portable imaging systems (MoleMapper, Visiomed, and confocal microscopy) to see how well they could detect melanoma compared to a dermatologist's exam. Seventeen people with suspicious moles participated. The goal was to find out if these devices could acc…

This study tested whether a device called the Sklip System, which uses artificial intelligence, could help people at home decide if a mole might be skin cancer. Only one person took part before the study was stopped early. The goal was to see if the AI could match or beat standar…

This study tested a wearable device that sits on the neck to detect swallowing problems in people with conditions like stroke, Parkinson's, multiple sclerosis, Alzheimer's, or dementia. The device was compared to a standard X-ray test. Unfortunately, the device did not meet the a…

This phase 3 trial tested a vaccine called POL-103A (Seviprotimut-L) in 504 people with high-risk melanoma who had just had surgery. The goal was to see if the vaccine could prevent the cancer from coming back. However, the study was terminated early, so it is not known whether t…

This phase 3 trial tests whether ARD-101 can reduce the intense, constant hunger (hyperphagia) seen in Prader-Willi syndrome. About 90 participants will take either ARD-101 or a placebo daily for 12 weeks. Caregivers will track changes in hunger-related behaviors using a standard…

This study looked at whether blowing a fan with aromatherapy on the face can help reduce breathlessness in people with terminal illness. The trial planned to enroll 26 adults who were near the end of life and had trouble breathing. Participants were randomly assigned to receive a…

This study tested whether propranolol, a beta-blocker usually used for heart conditions, can reduce nosebleeds in people with hereditary hemorrhagic telangiectasia (HHT), a genetic disorder that causes abnormal blood vessels. Fifteen adults with HHT took either propranolol or a p…

This small study tested a special laser (violet and green light) to see if it could tighten loose skin on the belly. Only 9 people signed up, and the study was stopped early. Researchers planned to compare before-and-after photos to judge improvement, but the tiny size and early …

This study tested whether closing a small hole in the heart (called a PFO) with a device could reduce migraine headaches. It planned to compare the real procedure to a sham (fake) procedure in adults aged 18-55 with frequent migraines. However, the study was stopped early after e…

This study tested two different ways of using a breathing machine (ventilator) in 12 premature infants with severe bronchopulmonary dysplasia (BPD), a serious lung condition. One mode, called NAVA, uses the baby's own diaphragm signals to trigger breaths, while the other, CMV, us…

This study tested whether a cannabis-derived compound called CBDV could safely reduce irritability in children and young adults with Prader-Willi syndrome. Only 6 people enrolled before the study was stopped early. The goal was to see if CBDV helped with mood and behavior problem…

This study tested a traditional Chinese herbal formula called Qingwei Huazhuo Decoction in 94 girls with mild early puberty (breast development starting too soon). The goal was to see if the herbs could slow breast growth and bone age. However, the trial was terminated early, so …

This early-stage trial tested whether adding a device that creates electric fields (NovoTTF-200M) to standard radiation and immunotherapy is safe for people with melanoma that has spread to the brain. Only one person took part before the study was stopped early. The main goal was…

This study was designed to track side effects and quality of life in people aged 70 and older with advanced melanoma or non-small cell lung cancer who are starting immunotherapy. Researchers planned to monitor 40 participants for serious side effects and changes in daily well-bei…

This study tested a special pacifier that plays music or a mother's voice when a baby sucks correctly. It aimed to help premature infants with chronic lung disease learn to feed by mouth more quickly. The trial enrolled 33 babies but was stopped early, so the results are not conc…

This study tested a rehabilitation program for hand sensitivity problems in people with multiple sclerosis. The program used TENS (a mild electrical stimulation device) and hand exercises to improve the ability to feel and recognize objects. The goal was to improve quality of lif…

This study aimed to see if the drug ozanimod could help reduce fatigue in people with multiple sclerosis (MS). Researchers planned to follow 26 adults with MS for three months, measuring fatigue using special questionnaires and brain scans. The study was terminated early, so resu…

This pilot study looked at whether an interactive storyteller called LUNII could lower anxiety in children aged 4 to 6 with cleft lip and palate before their bone graft surgery. The goal was to see if using the device before the operation would also reduce pain and shorten hospit…

This study tested whether combining a computer-based speed-of-processing training with a gentle electrical brain stimulation (alpha-tACS) could improve how quickly people with mild cognitive impairment process visual information. The trial planned to enroll 40 adults aged 65 and …

This study used a special MRI technique to look at tiny brain changes in people who recently had a first episode of symptoms that could lead to multiple sclerosis (MS). Researchers compared 84 participants (patients and healthy controls) to see if early memory problems are linked…

This study explores whether the community of bacteria living on the skin (the skin microbiome) plays a role in vitiligo—a condition that causes white patches—and in side effects from anti-PD-1 immunotherapy for advanced melanoma. Researchers will collect skin swabs from people wi…

This study aimed to find out how common sperm problems are in men with Fabry disease. Researchers planned to check sperm samples from 22 men aged 18 to 65. The study was stopped early, so results are limited.

This study aimed to understand why people with autism or schizophrenia sometimes struggle to recognize emotions on faces. Researchers used brain wave recordings (EEG) and eye-tracking to see how participants processed facial expressions. The study included people with autism, sch…

This study collects small skin samples from children aged 2 to 17 with Costello syndrome or a related condition. Researchers will analyze the cells to understand how a mutation in the HRAS gene affects energy use and mitochondria. The goal is to learn more about the disease's und…

This study tested whether special insoles with pressure sensors (FEETME®) can accurately measure walking speed in people with CIDP, a nerve condition that causes walking difficulties. Researchers compared the insoles to a standard walkway system in 18 adults aged 18-75 with CIDP.…

This early-stage study tested whether inhaling the cancer drug afatinib works as well as taking it by mouth. Four healthy adult smokers received both forms in separate sessions to compare drug levels in the blood and lungs. The study was terminated early, so the findings are limi…

This study aimed to see if orthognathic (jaw) surgery improves quality of life for people born with cleft lip and palate. Researchers planned to compare questionnaire scores before and 6 months after surgery in about 10 participants. The study was terminated early, so results are…

This small, early-terminated study explored whether measuring certain receptors on immune cells could predict disease activity and rebound risk in multiple sclerosis patients taking or stopping ozanimod. Researchers analyzed blood samples from 23 adults with relapsing-remitting M…

This pilot study aims to understand why people with pseudohypoparathyroidism type 1A (PHP1A) have a higher risk of type 2 diabetes. Researchers will measure insulin sensitivity and beta-cell function in 14 participants with PHP1A or related conditions, comparing them to matched h…

This study aimed to help children aged 6-8 from low-income families learn how to avoid foods they are allergic to. The program used fun, hands-on activities to teach safety skills. The study was stopped early, so we don't have clear results on whether it worked.

This study looked at how craniosynostosis—a condition where a baby's skull bones fuse too early—affects brain development. Researchers used special MRI scans and developmental tests to compare children with the condition before and after skull surgery to healthy children of the s…

This study looked at how stress, hormonal cycles, illness, and missed medications might affect when symptoms appear or get worse in women with relapsing-remitting multiple sclerosis (RR-MS). Researchers planned to track 80 women over time, recording daily symptom changes. The stu…

This study aimed to observe how MECP2 duplication syndrome progresses over time by measuring biological markers in spinal fluid and blood, and by tracking changes in development, behavior, and seizures. It enrolled 29 males aged 1 month to 65 years with a confirmed genetic diagno…

This study was a large registry that planned to follow 180,000 patients in Germany with aortic valve problems (leaky or narrowed valves). The goal was to compare newer, less invasive treatments like TAVI with standard open-heart surgery to see which works best and for whom. Resea…

This study looked at bone health in transgender youth aged 9-14 who were starting puberty blockers (GnRH agonists). Researchers used MRI and other scans to measure bone marrow and density changes over 24 months, comparing them to healthy peers. The goal was to understand if block…

This study looked at whether special MRI scans can detect early brain changes in people with multiple sclerosis who are starting treatment with Tysabri. Researchers planned to follow 70 patients for two years, comparing MRI images before and after treatment. The goal was to find …

This study followed 28 adults with liver, colorectal, melanoma, or kidney cancers who received high-dose radiation therapy. Researchers collected blood samples before, during, and after treatment to track immune cells and other markers. The goal was to learn how the immune system…

This study tested a method called EPR oximetry to measure oxygen levels in skin tumors. Only 3 people took part before the study was stopped early. The goal was to see if this technique could track how tumors respond to oxygen therapy and standard cancer treatments.

This study looked at a type of spinal cord stimulation (SCS) that doesn't cause tingling, called paresthesia-free SCS, in 10 adults with chronic pain. Researchers wanted to see how well it works and how it affects pain perception. The study was terminated early, so the findings a…

This study looked at women and girls aged 12 to 40 with heavy menstrual bleeding to see if they also have joint hypermobility (loose joints). Researchers wanted to find out if these two issues together might point to an underlying connective tissue disorder. The study was stopped…

This study followed 7 people with Mucolipidosis Type IV (MLIV) to learn how the disease naturally progresses. Researchers used tests like neuropsychological exams, blood and urine tests, and brain MRIs to find better ways to measure the disease. The goal was to improve future cli…

This study looked at whether people born with cytomegalovirus (CMV) infection who have hearing loss or balance problems also have a condition called endolymphatic hydrops (fluid buildup in the inner ear). Researchers used a special MRI scan with a contrast dye to check for this f…

This study looked at a specific hip implant shell (the Pinnacle Acetabular Shell) used in total hip replacement. Researchers wanted to see how much the implant moved (migrated) over two years using a special X-ray technique called RSA. They also tracked how patients felt and func…

This study tested a wearable device called Syde® in 86 people with multiple sclerosis. Participants wore the device for one month every six months over two years. The goal was to see if the device's activity data matches standard clinic tests like walking speed and disability sco…

This study looked at children with idiopathic short stature (short stature with no known cause) who had already received growth hormone therapy for at least a year. Researchers compared the genetic profiles of those who responded well to treatment versus those who did not. The go…

This early-stage study looked at how two common drugs (carbamazepine and itraconazole) change the levels of radiprodil in the blood of 30 healthy adults. The goal was to understand safety and dosing for future use in neurological conditions like tuberous sclerosis. However, the s…

This study aimed to understand how age influences serious health issues like infections, cancer, heart disease, and bone fractures in people with rheumatoid arthritis. Researchers compared patients over 65 with younger adults (18-50) who had the same disease. The study was stoppe…

This study followed 65 adult men with a rare inherited nerve disease called AMN (a form of spastic paraplegia) to understand how their symptoms change over time. Researchers collected data on walking ability and quality of life. The goal was to fill gaps in knowledge about the di…

This study aimed to see if a wireless sensor could collect health information from babies with heart defects at home, replacing wired devices. Only 2 infants were enrolled before the study was stopped. The goal was to understand if the wireless device was easy to use and worked w…

This observational registry aims to learn about the health, wellbeing, and needs of people who were diagnosed with cancer between ages 18 and 39. Participants complete surveys every 6 to 12 months about anxiety, depression, social support, and financial distress. The registry als…

This study looked at bone density in HIV-positive men to see if antiretroviral drugs cause bone thinning. Researchers compared men starting HIV treatment with those not yet on therapy over two years. The goal was to understand why bone loss is more common in HIV patients.

This study aimed to find out how often infections happen after a procedure called TEVAR, which repairs the main artery in the chest. Researchers looked at data from 457 patients who had this surgery in the past five years. They measured infection rates, fever, and lab results to …

This pilot study aimed to compare two standard surgical methods for creating an arteriovenous (AV) fistula in the arm for dialysis patients with kidney failure. The goal was to see if a single procedure or two separate procedures leads to better outcomes and quality of life. Howe…