Disease of genetic or genomic mechanism

This study tested a vaccine called VBI-1901 in 70 people with recurrent glioblastoma, an aggressive brain cancer. The goal was to find a safe dose and see if the vaccine could boost the immune system against the tumor. The trial was terminated early, so results are limited.

This trial investigates whether an oral medication called etrasimod can help people with moderate to severe Crohn's disease who have not responded well to other treatments. Participants take either etrasimod or a placebo daily for up to several years. The study measures improveme…

This study investigates whether giving a repeat course of betamethasone, a steroid, to pregnant women with preterm premature rupture of membranes (PPROM) can improve health outcomes for their premature babies. The main goal is to see if it shortens the baby's stay in the neonatal…

This phase 2 study tests whether combining magrolimab and pembrolizumab can shrink or eliminate tumors in people with classic Hodgkin lymphoma that has come back or not responded to at least two prior treatments. Magrolimab targets a 'don't eat me' signal on cancer cells, while p…

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 tests an experimental treatment called GLPG5101 for people with B-cell non-Hodgkin lymphoma that has come back or not responded to standard therapy. The treatment is a cell therapy made at the hospital. The study has two phases: first, finding the best dose with the fe…

This study tests a single injection of a drug called AGA111 given directly into the spine during back fusion surgery for people with degenerative disc disease. The goal is to see if it helps the bones heal together more successfully and improves pain and function. About 412 adult…

This early-phase trial is testing a new treatment called CHM-1101 for people with a type of brain cancer (glioblastoma) that has come back or is getting worse. The treatment uses the patient's own immune cells, which are modified in a lab to recognize and attack tumor cells. The …

This early-phase trial tested a combination of two immune-boosting drugs, relatlimab and nivolumab, in children and young adults with Hodgkin or non-Hodgkin lymphoma that had come back or not responded to treatment. The goal was to check safety, drug levels, and whether the tumor…

This early study tested a laser treatment for people with excessive sweating (hyperhidrosis). The approach used a dye to mark sweat glands, then a special laser to destroy them. Only 2 people were enrolled, and the study was terminated early, so we have very little data on whethe…

This study tested brazikumab, an experimental biologic drug, in 89 adults with moderate-to-severe Crohn's disease. Participants received either brazikumab, a placebo, or an active comparator (Humira) to see if the drug could improve symptoms and heal the gut lining. The trial was…

This study tested whether a 14-day course of hydrochlorothiazide (a water pill) could prevent high blood pressure from returning after delivery in women with preeclampsia or gestational hypertension. Over 450 women were planned, but the trial was stopped early. The main goals wer…

This study looks at the long-term safety of brazikumab, a drug given as a shot every 4 weeks, for people with moderate to severe ulcerative colitis. Participants had already taken part in an earlier study of brazikumab. The goal is to see if the drug remains safe over a longer pe…

This trial tested a vaccine called PD1-Vaxx given before surgery to people with a specific type of colorectal cancer (MSI-high). The goal was to see if the vaccine could shrink tumors, measured by how many cancer cells remained after treatment. Only 3 people took part, and the st…

This early-phase study tested an injectable drug called NP-201 acetate in healthy volunteers and people with mild-to-moderate ulcerative colitis. The goal was to check safety, how the body processes the drug, and early signs of effectiveness. The trial was terminated early, so on…

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 pilot study tested whether propranolol, a beta-blocker, could shrink or stabilize kidney angiomyolipomas in people with tuberous sclerosis. Only 2 participants were enrolled before the trial was terminated, so the results are very limited. The goal was to find a less invasiv…

This study compared two treatments to reduce bleeding after heart valve surgery: a concentrated clotting drug (PCC) versus fresh frozen plasma (FFP). The trial planned to enroll 476 adults undergoing elective valve surgery. It was terminated early, so the full comparison was not …

This phase 2 trial tested brazikumab in 242 adults with moderate-to-severe ulcerative colitis. Participants received either brazikumab or a placebo intravenously and then as injections over 54 weeks. The goal was to see if the drug could improve symptoms and heal the colon lining…

This phase 2 trial investigates whether polyethylene glycol (a laxative) can reduce intestinal inflammation in children with cystic fibrosis. The study includes children aged 4 to 17 with cystic fibrosis and pancreatic insufficiency who have elevated fecal calprotectin (a marker …

This study looks at people with inflammatory bowel disease (Crohn's disease or ulcerative colitis) who are in deep remission. It compares two methods for gradually increasing the time between infliximab infusions: one based on symptoms and one based on drug levels in the blood. T…

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 study looked at whether giving pregnant people oral antibiotics at home could delay delivery after their water broke very early (between 18 and 23 weeks). The goal was to see if more people would stay pregnant for at least 28 days after the water broke. The study was stopped…

This study tested whether an extra dose of bezlotoxumab, a drug that blocks the harmful toxin made by C. diff bacteria, could help very sick patients recover after emergency colon surgery. Only 5 people took part before the study was stopped early. The goal was to see if the drug…

This study tested a special bone graft material (MatriBONE mixed with the patient's own bone marrow) for spinal fusion surgery in people with painful degenerative back problems. The goal was to see how well the bones fused together after 12 months. However, the trial was stopped …

This study tested a low dose of the drug bevacizumab (Avastin) against standard laser treatment for retinopathy of prematurity, an eye disease that can blind premature infants. Sixteen babies with severe disease were enrolled. The goal was to see if the drug could control the dis…

This study tested an oral medication, NX-13, in 81 adults with moderate to severe ulcerative colitis. Participants received either NX-13 or a placebo for 12 weeks, with a long-term follow-up option. The trial was terminated early, so the full results are not available, but the da…

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 early-stage trial tests a one-time treatment called BE-101 for adults with severe hemophilia B. The therapy uses a person's own blood cells, modified with CRISPR gene editing, to continuously produce the missing clotting factor. The goal is to reduce or prevent bleeding epis…

This study tests a new treatment called Temferon for people with glioblastoma, a fast-growing brain cancer. The therapy uses a patient's own blood stem cells, modified to deliver interferon directly to the tumor. Up to 27 patients with a specific genetic marker (unmethylated MGMT…

This study tested an experimental drug called ARO-ANG3 in 18 people with homozygous familial hypercholesterolemia (HoFH), a rare genetic condition causing extremely high cholesterol. Participants received injections of the drug and were monitored for safety and cholesterol change…

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 trial tested a single dose of CAP-002 gene therapy in children aged 18 months to 8 years with STXBP1 encephalopathy, a rare genetic brain disorder causing seizures and developmental delays. The study aimed to check safety and whether it could reduce seizures and improve skil…

This study tested a drug called siplizumab alongside a stem cell transplant for people with severe sickle cell disease. The goal was to see if it could prevent the body from rejecting the new cells and reduce a serious side effect called graft-versus-host disease. Only one person…

This study looked at the long-term safety of soticlestat when added to standard seizure medicines for people with Dravet syndrome or Lennox-Gastaut syndrome. It included 352 children and adults who had already been in earlier studies. The trial was terminated early, so results ar…

This study looked at the long-term safety of a fixed-dose combination of two drugs (obeticholic acid and bezafibrate) for people with primary biliary cholangitis (PBC), a chronic liver disease. It enrolled 62 adults who had already been taking the combination in earlier studies. …

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 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 study tested if the beta-blocker propranolol could control the growth of infantile hemangiomas (non-dangerous blood vessel growths) in babies under 4 months old. The trial was terminated early and only enrolled 14 infants. The goal was to measure changes in hemangioma thickn…

This phase 3 trial tested whether adding asciminib to the standard drug imatinib could help people with chronic myeloid leukemia (CML) achieve a deep remission and safely stop all medication. Only 8 patients were enrolled before the study was terminated early, making it too small…

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 study looked at whether switching from a standard HIV drug combination to a different one (Delstrigo) could improve cholesterol, body fat, and blood sugar in people with HIV. The study planned to enroll 60 adults whose HIV was already well-controlled. Participants were rando…

This phase 2 study tested an experimental oral drug called BGB-45035 in 49 adults with moderate to severe rheumatoid arthritis who had not responded well to standard treatments. Participants received either the drug or a placebo to see if it could reduce joint pain and swelling. …

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 a new type of artificial lens (enVista Aspire) for people having cataract surgery. The goal was to see if it improves intermediate vision, like seeing a computer screen, better than standard lenses. The trial was stopped early and included 27 adults aged 22 and …

This study looked at the long-term safety of the drug NBI-921352 for people with a rare genetic seizure disorder called SCN8A-DEE. It was an extension of an earlier study, and participants took the drug alongside their usual seizure medications. The study was stopped early and on…

This study tested an experimental drug called AMAG-423 (Digoxin Immune Fab) in pregnant women with severe preeclampsia, a dangerous condition of high blood pressure during pregnancy. The goal was to see if the drug could reduce serious health problems in babies, such as bleeding …

This study looked at whether tesamorelin (Egrifta), a daily injection that boosts growth hormone, can improve body composition in people with HIV who have excess belly fat (lipodystrophy). Six participants received the drug for up to 12 months, with researchers measuring liver fa…

This study looked at using umbilical cord blood transplants after strong chemotherapy and radiation to treat various blood cancers like leukemia and lymphoma. It involved 200 participants and aimed to see how many were alive one year after transplant. The trial was stopped early,…

This study tested the anti-inflammatory drug naproxen sodium for treating obsessive-compulsive symptoms in children aged 6-15 with PANDAS, a condition linked to strep infections. The trial was double-blind and placebo-controlled, but it was terminated early after enrolling only 2…

This study tested different warming methods—like forced air blankets and warmed IV fluids—to prevent hypothermia in women having planned C-sections. Only 16 women participated before the trial was stopped early. The goal was to see which method best keeps mother and baby warm and…

This study tested a new drug called Vodobatinib (K0706) in people with chronic myeloid leukemia or a related leukemia who had not responded to at least three other treatments. The trial was split into parts: first in healthy volunteers to check safety, then in patients to find th…

This phase 2 trial tested two different drug combinations to prevent graft-versus-host disease (GVHD) in 150 people with blood cancers receiving a stem cell transplant from an unrelated donor. Both combinations used cyclosporine and sirolimus, plus either mycophenolate mofetil or…

This study compared a new device called miCOR, which uses low energy to break up cataracts, to standard cataract surgery. About 402 adults with cataracts were planned to take part. The study was terminated early, so we don't have full results on whether the new device is better o…

This study looked at adding a targeted drug called venetoclax to low-intensity chemotherapy for adults with acute lymphoblastic leukemia (ALL) that came back or didn't respond to treatment. The goal was to find the safest dose and see if the combination could help control the dis…

This study tested whether obeticholic acid, a drug already used for liver disease, can safely reduce the number of polyps in the small bowel and colon in people with familial adenomatous polyposis (FAP). FAP is a rare genetic condition that greatly raises the risk of intestinal c…

This phase II trial tested the drug niraparib (Zejula) in 11 men with high-risk prostate cancer that had not spread. All men had specific DNA repair gene mutations. They took niraparib before having their prostate removed surgically. The goal was to see if the drug could eliminat…

This early-stage trial tested a drug called DS-2325a in 9 adults with Netherton syndrome, a rare genetic condition causing severe skin redness, scaling, and allergies. The study aimed to check safety and whether the drug could help control the disease. However, the trial was term…

This early-phase trial tested combinations of the drugs bevacizumab and temsirolimus, sometimes with valproic acid or cetuximab, in 154 people with advanced cancers or certain non-cancerous but progressive diseases. The main goal was to find safe doses and look for any signs that…

This study tested a new drug called exicorilant (CORT125281) combined with enzalutamide in men with metastatic castration-resistant prostate cancer. The goal was to find a safe dose and check for side effects. The trial was terminated early, so results are limited. It involved 39…

This study tested a drug called KL130008 in 114 adults with severe alopecia areata, a condition causing major hair loss. The goal was to see if the drug could safely regrow hair, measured by how many people achieved 80% or more scalp coverage. However, the trial was terminated ea…

This study tracked 350 people with GERD who received a device that electrically stimulates the muscle that keeps stomach acid from rising. The goal was to see how safe the device is over 5 years and whether it improves symptoms and quality of life. The registry included both new …

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 early-stage study tested a new drug, PF-08046044/SGN-35C, in people with advanced lymphomas (Hodgkin and non-Hodgkin types) who had no other treatment options. The main goal was to check safety and find the right dose. The study was terminated early, so results are limited.

This study offered continued treatment with EryDex to 101 people with ataxia telangiectasia (A-T) who had finished a previous trial. The main goal was to monitor safety, including side effects and serious events. The study was terminated early, and it did not aim to cure the dise…

This study tested a combination of two drugs, ibrutinib and ixazomib, in 21 people with Waldenström macroglobulinemia, a rare blood cancer. The goal was to see if the combo could shrink or control the cancer. The trial was stopped early, so results are limited, but the approach a…

This phase 2 trial tested KVD824, an oral pill, to prevent swelling attacks in people with hereditary angioedema types I and II. The study planned to compare three doses against a placebo over several months. However, the trial was terminated early with only 33 participants, so t…

This study tested the drug fenfluramine (FINTEPLA) in adults with Dravet syndrome whose seizures were not controlled by other medications. The trial was open-label and added fenfluramine to existing treatments. It aimed to see if the drug could reduce monthly seizures by at least…

This study tested an mRNA therapy called mRNA-3705 in 18 people with a rare genetic condition called methylmalonic acidemia, which causes harmful acid buildup. The therapy aimed to help the body produce a missing enzyme to lower acid levels. The trial was terminated early, so fin…

This study tested whether giving extremely preterm babies (born before 27 weeks) a gentle saline enema twice a day could help them reach full feeding sooner and prevent necrotizing enterocolitis (NEC), a severe gut infection. The trial planned to include 200 infants but was termi…

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 study tested an experimental daily tablet called VTX002 in people with moderate to severe ulcerative colitis, a chronic inflammatory bowel disease. About 213 participants were randomly assigned to receive one of two doses of VTX002 or a placebo for 13 weeks, with the option …

This early-phase trial tested a combination of two drugs—ruxolitinib and CPX-351—in 12 people with advanced myeloproliferative neoplasms (MPNs), a group of rare blood cancers. The goal was to find the best dose and see if the combo could shrink cancer. However, the study was stop…

This phase 2 trial tested two doses of an oral drug called fidrisertib (IPN60130) in children and adults with fibrodysplasia ossificans progressiva (FOP), a rare condition where soft tissues turn into bone. The goal was to see if the drug could reduce new abnormal bone growth com…

This early-stage trial tested the safety of an intravenous drug called talineuren (liposomal GM1) in 22 people with Parkinson's disease. The study was open-label and designed to find a safe dose, but it was terminated before completion. No conclusions about effectiveness can be d…

This study tested an experimental drug called KRRO-110 in healthy volunteers and people with Alpha-1 Antitrypsin Deficiency (AATD), a genetic condition that can cause lung and liver damage. The goal was to check safety and how the drug moves through the body. However, the trial w…

This early-phase trial tested whether glycerol phenylbutyrate (Ravicti), a drug already used for urea cycle disorders, could help restore chloride transport in the nasal cells of adults with cystic fibrosis. The study enrolled 16 participants and compared low-dose Ravicti to a pl…

This early-stage study tested an oral drug called AUR104 in 9 adults with certain blood cancers (lymphomas and leukemia) that had come back or stopped responding to treatment. The main goal was to check the drug's safety and how the body processes it. The trial was terminated ear…

This study tested a single dose of an inhaled gene therapy called BI 3720931 in adults with cystic fibrosis who cannot take standard CFTR modulator drugs. The trial was in two phases and included only 5 participants. It was terminated early, so we do not have full results on safe…

This study tested whether stem cells from donated bone marrow could heal fistulas (abnormal tunnels) in people with Crohn's disease who had a pouch after colon removal. The trial planned to enroll 24 adults but was terminated early, so we don't have clear results on safety or hea…

This study tested whether stem cells from donated bone marrow could safely heal rectovaginal fistulas in women with Crohn's disease. These fistulas are abnormal connections between the rectum and vagina that cause pain, infections, and poor quality of life. The trial planned to e…

This early-phase study tested whether injecting stem cells from donated bone marrow could help heal perianal fistulas in teenagers with Crohn's disease. Six patients aged 13-17 received injections of 75 million stem cells directly into the fistula tract, with a possible second do…

This early-phase study tested whether injecting donor stem cells directly into the colon could help people with severe Crohn's colitis that didn't respond to standard treatments. Only 7 people took part before the study was stopped early. The main goal was to check safety, but be…

This early-stage study tested whether injecting donor stem cells (remestemcel-L) into the colon could help people with severe ulcerative colitis that hadn't responded to other treatments. Only 7 adults took part before the trial was stopped early. The main goal was to check safet…

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 early-stage study tested whether injecting donor stem cells directly into fistulas (abnormal tunnels) could help people with Crohn's disease. The trial planned to enroll 25 adults but was terminated early. The goal was to check safety and see if the treatment could lead to c…

This study compared two surgical methods for removing diseased intestine in people with Crohn's disease: one that cuts the main artery high up, and one that spares the mesentery (the tissue holding the intestine). The goal was to see which technique better prevents the disease fr…

This study tested whether adding the targeted drug inotuzumab ozogamicin before and after a stem cell transplant could help control leukemia and lymphoma. It included 15 people aged 12 to 75 with certain blood cancers. The study was stopped early, but researchers looked at safety…

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 study tested a drug called XmAb23104 in people with advanced sarcoma, a rare cancer. The goal was to see if the drug could shrink tumors and if it was safe. Only 6 people took part before the study was ended early.

This study tested a drug called IgPro20 (HIZENTRA) to see if it could help people with post-COVID POTS, a condition that causes a rapid heart rate and dizziness when standing. The trial was stopped early after enrolling only 16 adults. The goal was to see if the drug could reduce…

This early-stage trial tested a new drug called LY3885125 in people with high cholesterol (dyslipidemia) and non-alcoholic fatty liver disease (NAFLD). The main goal was to check safety and how the body processes the drug. The study was terminated, meaning it stopped early, so it…

This early-phase trial tested a gene therapy for X-linked chronic granulomatous disease (X-CGD), a rare genetic disorder that leaves boys unable to fight off bacteria and fungi. The treatment involved taking the patient's own blood stem cells, adding a corrected gene, and infusin…

This trial tested a gene therapy for X-linked chronic granulomatous disease (X-CGD), a rare genetic disorder that leaves boys unable to fight off infections. The treatment involved taking the patient's own blood stem cells, adding a working copy of the faulty gene, and returning …

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 study tested a drug called TPN-101 (censavudine) in people with Aicardi-Goutières syndrome, a rare genetic disorder that causes severe brain inflammation. The trial enrolled only 4 participants and aimed to see if the drug could reduce immune system overactivity and check fo…

This study tested a daily pill called deucravacitinib in 3 adults with palmoplantar pustulosis (PPP), a painful skin condition causing blisters on palms and soles. The goal was to see if the drug could reduce disease severity by at least 50% over 24 weeks. However, the trial was …

This study tested a new drug called belcesiran in people with a genetic condition called alpha-1 antitrypsin deficiency that causes liver damage. The goal was to see if the drug is safe and can lower harmful protein levels in the liver. The trial was stopped early, so results are…

This study tested whether switching to a two-drug HIV treatment (raltegravir plus maraviroc) could reduce belly fat (lipohypertrophy) in people whose HIV was already well-controlled. It included 48 adults on stable HIV therapy for at least 5 years. The study was stopped early, so…

This study looked at two ways of giving growth hormone to short children who were born small for their age. The goal was to see which dosing method works better for growth and has fewer side effects on metabolism. Only 10 children took part before the study was stopped early.

This study tested high doses of Ambroxol, a common cough medicine, in 12 children aged 3 to 18 with Type 3 Gaucher disease, a rare genetic disorder affecting the brain and body. The goal was to see if it is safe and can improve movement problems like ataxia. The trial took place …

This study tested a drug called PRV-3279 to see if it could prevent lupus flares in people with active systemic lupus erythematosus (SLE). The trial included 28 adults with moderate to severe lupus who had improved after steroid treatment. The study was terminated early, so resul…

This study looked at whether taking UCB0599 early or later could slow brain changes in people with Parkinson's disease. It involved 428 adults who had already completed an earlier study. The trial was stopped early, but researchers measured brain scans and medication use over 18 …

This study tested two different drug combinations given before a stem cell transplant for people with myeloid blood cancers like leukemia. One group got fludarabine and busulfan, the other got those drugs plus low-dose total body irradiation. The goal was to see which regimen led…

This study tested the safety of golodirsen (Vyondys 53) in boys and men with Duchenne muscular dystrophy who can no longer walk. Only 2 people took part before the trial was stopped early. Participants received weekly IV infusions for up to 96 weeks, with extra follow-up. The goa…

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 study tested a way to improve stem cell transplants for children with combined immunodeficiency (CID), a condition where the immune system doesn't work properly. The approach removed certain naive cells from the donor stem cells to try to prevent graft-versus-host disease (a…

This study was designed to see if brazikumab is safe and tolerable over the long term for people with moderate to severe Crohn's disease. It enrolled 18 participants who had previously been in a related study. The trial was terminated early, meaning it did not reach its planned e…

This study tested a drug called OMS721 in 31 people with certain kidney diseases, including IgA nephropathy and lupus nephritis. The main goal was to check if the drug is safe and if it reduces protein in the urine, a sign of kidney damage. The study was stopped early, so results…

This study tested the long-term safety and effectiveness of a drug called CDZ173 in people with APDS/PASLI, a rare genetic condition that causes a weak immune system and swollen lymph nodes. The 37 participants had already taken CDZ173 or a similar drug in a previous study. The t…

This study tested a drug called imsidolimab for adults with generalized pustular psoriasis (GPP), a rare and painful skin condition. The trial aimed to see if the drug is safe and works over the long term. However, the study was terminated early, so we have less information than …

This study tested whether BHV-7000, taken alongside other medications, could reduce generalized tonic-clonic seizures in people with idiopathic generalized epilepsy. The trial was terminated early and enrolled only 27 adults aged 18-75. The main goal was to see how long it took f…

This Phase II trial tested a drug called N-Acetyl-L-Leucine (IB1001) in 17 people with Ataxia-Telangiectasia, a rare genetic disease that affects movement and immunity. The study aimed to see if the drug could improve symptoms and slow the disease over time. The trial was termina…

This study looked at the long-term safety of the drug osivelotor in people with sickle cell disease who had already taken it in an earlier trial. The goal was to track side effects and blood changes over time. However, the study was terminated early, so the full safety picture is…

This study tested a new way to manage blood pressure in new mothers with severe postpartum preeclampsia. Participants received standard blood pressure medications, but one group had personalized blood pressure targets based on real-time brain monitoring. The goal was to keep bloo…

This study looked at the long-term safety of soticlestat, an experimental drug, in children and adults with severe forms of epilepsy like Dravet syndrome and Lennox-Gastaut syndrome. Participants took soticlestat twice a day along with their usual seizure medicines. The study was…

This study looked at whether a plant-based diet and lifestyle changes could help people with type 2 diabetes, prediabetes, or obesity improve their health. Researchers aimed for diabetes remission (normal blood sugar without medication for at least 3 months) or significant weight…

This study tested a device called the ELITA System to reduce or eliminate nearsightedness (myopia) and astigmatism. It involved 96 adults aged 22 and older who received a lenticule removal procedure in one or both eyes. The trial was terminated early, so the planned results on sa…

This study tested an experimental drug called PF-06823859 for adults with lupus that affects the skin. The goal was to see if the drug could reduce skin inflammation and improve symptoms. The trial was stopped early and only enrolled 8 people instead of the planned 48, so results…

This study looked at whether using a combination of weight-loss medications could help people with obesity lose weight and improve the health of their blood vessels. It involved 128 adults aged 40-75 who were obese and had at least one heart risk factor. The study was stopped ear…

This trial tested the drug deucravacitinib in people with moderate-to-severe palmoplantar pustulosis, a skin condition causing painful pustules on the palms and soles. The study planned to enroll 25 participants who would take the drug daily for 16 weeks, but it was terminated ea…

This study tested a medicine called spesolimab for people with Netherton syndrome, a rare genetic skin condition causing severe redness and scaling. About 43 people aged 12 and older took part, receiving either the drug or a placebo. The goal was to see if spesolimab could reduce…

This study tested a new amino acid formula called PKU GOLIKE in people aged 16 and older with phenylketonuria (PKU). The goal was to see if it could better control daily swings in blood phenylalanine levels compared to standard treatment. The study was stopped early, so results a…

This trial tested whether a computer program that customizes infliximab doses based on blood tests can help children and young adults with Crohn's disease reach better drug levels than standard dosing. Only 6 participants were enrolled before the study was stopped early. The goal…

This study tested whether a bile acid supplement called UDCA could reduce inflammation and improve quality of life in people with pouchitis, a complication after ulcerative colitis surgery. Only 2 participants were enrolled before the study was terminated. The goal was to see if …

This study tested an experimental drug called KAN-101 in people with celiac disease. The goal was to see if it could safely reduce the immune response to gluten. The trial included 128 participants and was split into phases to check safety and how the body processes the drug. How…

This study tested a daily pill called voxelotor in children aged 2 to 14 with sickle cell disease who had slightly abnormal blood flow in the brain. The goal was to see if the drug could improve that blood flow and reduce stroke risk. The trial was stopped early, but the results …

This phase 2 study tested a treatment called brexucabtagene autoleucel, a CAR T-cell therapy made from a patient's own immune cells, for four rare B-cell cancers: Waldenstrom macroglobulinemia, Richter transformation, Burkitt lymphoma, and hairy cell leukemia. The trial enrolled …

This phase II trial tested adding asciminib (ABL001) to standard tyrosine kinase inhibitor therapy in 7 people with chronic myeloid leukemia who still had traces of cancer. The goal was to see if the combination could eliminate remaining cancer cells. The study was terminated ear…

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 tislelizumab, which helps the immune system fight cancer, against standard chemotherapy in people with Hodgkin lymphoma that returned or didn't respond to treatment. Only 3 people took part before the study was stopped early. Because of this, we do…

This study tested the antifungal drug posaconazole in people with active Crohn's disease who carry a specific gene variant (CARD9 S12N). The goal was to see if reducing a type of fungus in the gut could lower inflammation and improve symptoms. The trial was stopped early and incl…

This study looked at the long-term safety of a drug called troriluzole for people with obsessive-compulsive disorder (OCD) who hadn't improved enough with standard treatments. The trial planned to enroll 772 participants but was terminated early. The main goal was to check for si…

This study tested an experimental oral drug, PF-07054894, in adults aged 18-75 with mild to severe ulcerative colitis (UC). The goal was to see if the drug could safely help control the disease and reduce symptoms like diarrhea and rectal bleeding. The trial was terminated early,…

This study tested a drug called vixarelimab in 79 people with moderate to severe ulcerative colitis (UC) who had not responded well to other treatments. The goal was to see if the drug could help control the disease and reduce symptoms like diarrhea and bleeding. The study was st…

This early-stage study tested a drug called ION269 in adults with Down syndrome who also showed signs of Alzheimer's disease. The goal was to check if the drug was safe and tolerable when injected into the spinal fluid. Only one person took part, and the study was stopped early, …

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 phase 2 trial tested inotuzumab ozogamicin (Besponsa) in children with B-cell acute lymphoblastic leukemia who had low levels of remaining cancer cells (MRD) despite treatment. The drug targets CD22, a protein on leukemia cells, and delivers a potent toxin. Only 5 children w…

This study looked at whether screening people for high platelet reactivity (a condition where blood clots easily) before leg artery procedures could improve outcomes. It involved 31 adults with peripheral artery disease who were scheduled for angioplasty or stenting. The trial wa…

This study looked at the safety and effectiveness of the drug ozanimod for people with moderate to severe ulcerative colitis. It aimed to see if the drug could improve symptoms like diarrhea and rectal bleeding, and how it affected quality of life. The study was terminated early,…

This study tested a new approach using a virus that attacks cancer cells (oncolytic immunotherapy) along with two standard drugs (atezolizumab and bevacizumab) in people with advanced colorectal cancer that had stopped responding to other treatments. The trial was stopped early a…

This early-phase study tested two drugs, AZD1775 and belinostat, together in 20 adults with blood cancers like acute myeloid leukemia that had come back or not responded to treatment. The main goal was to find the safest dose and understand side effects. The study was stopped ear…

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-stage trial tested a drug called nomlabofusp (CTI-1601) in 18 adolescents and children with Friedreich's ataxia, a rare genetic disease that affects movement and coordination. The goal was to check safety and how the body processes the drug. However, the study was term…

This study tested a single injection of a gene therapy called GT005 in 98 people with geographic atrophy, an advanced form of dry age-related macular degeneration that causes vision loss. The goal was to see if the treatment could slow the growth of damaged areas in the eye and c…

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 study tested a single injection of GT005, a gene therapy, in 255 people with geographic atrophy from dry age-related macular degeneration. The goal was to see if it could slow the growth of blind spots in the eye. The trial was terminated early, so we have less data than hop…

This early-phase trial tested a single infusion of IMPT-514, a personalized CAR T cell therapy, in 3 adults with severe lupus, vasculitis, or inflammatory myopathy that did not respond to standard treatments. The goal was to check safety and see if the therapy could reset the imm…

This small pilot study tested whether imatinib, a drug used for certain cancers, could reduce painful blockages in blood vessels (vaso-occlusive crises) in people with sickle cell anemia. Seven participants aged 18–25 took the drug for 6 months while researchers measured changes …

This early-stage trial tested a gene therapy called GT005 for dry age-related macular degeneration (AMD), a leading cause of vision loss. The therapy delivers a working gene to the retina via a single injection. The study enrolled 56 people and focused on safety and dosing, but w…

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 study tested a new drug, PF-08046045, in 22 adults with advanced lymphomas (cancers of the immune system). The goal was to find a safe dose and check for side effects. The trial was stopped early, so results are limited.

This phase 1 trial tested a gene therapy called AVXS-101 (Zolgensma) in 32 children with spinal muscular atrophy (SMA) who could sit but not stand or walk. The therapy delivers a working SMN gene via a spinal injection to help improve muscle function. The study focused on safety …

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 Phase 2 study tested the drug ravulizumab (Ultomiris) in 123 adults with lupus nephritis or IgA nephropathy, two conditions that cause kidney inflammation. Participants received either ravulizumab or a placebo alongside standard care. The study aimed to see if the drug could…

This study tested a combination of two drugs (regorafenib and pembrolizumab) for people with a specific type of advanced colorectal cancer called MSI-H. The goal was to see if the combination worked better than pembrolizumab alone. The trial was stopped early after enrolling only…

This was a first-in-human study testing a single dose of an experimental drug called GSK4172239D in 11 people with sickle cell disease. The main goals were to check safety and how the drug moves through the body. The study was terminated early, so results are limited.

This study tested the long-term safety and effectiveness of a stem cell treatment called darvadstrocel (Alofisel) for people with Crohn's disease and complex anal fistulas. Researchers planned to give one injection of the stem cells and follow 53 participants over several visits,…

This study tested the safety of giving a second dose of an experimental virus (C134) directly into the brain tumor of patients whose malignant glioma had returned. Only one patient was enrolled before the study was stopped. The goal was to see if repeat dosing was safe and tolera…

This early-phase trial tested an experimental drug called TAK-500, alone or with the immunotherapy pembrolizumab, in 61 adults with advanced solid tumors like pancreatic, liver, and lung cancer. The main goals were to check safety and see if the drug could shrink tumors. The stud…

This early-phase trial tested two immunotherapy drugs, ipilimumab and nivolumab, in people with HIV who had advanced solid tumors or Hodgkin lymphoma that had spread or come back. The goal was to find the safest dose and see if the combination could shrink tumors. The study was t…

This study was designed to let people who had already taken latozinemab in an earlier study continue receiving the drug. Only 17 people took part, and the study was stopped early. The goal was to track safety and how long people stayed on treatment, not to test if the drug could …

This phase 3 study tested a drug called AL001 in 119 people who carry a mutation in the progranulin gene, putting them at high risk for or already diagnosed with frontotemporal dementia. The goal was to see if AL001 could slow the worsening of memory, behavior, and language probl…

This study aimed to see if a new medicine (efanesoctocog alfa) helps people with moderate or severe hemophilia A reach their personal goals and be more physically active. It planned to enroll 35 people aged 12 to 50, but was stopped early after only 3 joined. The medicine is used…

This study tested a new type of artificial lens (TECNIS DEN00V) for people having cataract surgery. It compared the new lens to a standard lens in 243 adults with cataracts. The goal was to see if the new lens improves vision at arm's length (66 cm) while keeping good distance vi…

This early-stage trial tested a new antibody drug (BB-TL1A-VIAL-HLE) in 16 healthy volunteers and people with moderate-to-severe ulcerative colitis. The goal was to check safety and see if it could improve colon inflammation. However, the study was terminated early, so we have li…

This study tested a gene therapy called SRP-9003 for people with limb-girdle muscular dystrophy type 2E (LGMD2E), a rare genetic disease that causes muscle weakness. The treatment aimed to deliver a working gene to muscle cells to help them produce a missing protein. Only 6 peopl…

This trial tested the drug Cayston (aztreonam lysine) in adults with bronchiectasis, a chronic lung disease causing cough and infections. The goal was to see if it could safely delay or reduce lung flare-ups. However, the study was stopped early and only enrolled 34 people, so we…

This study tested a new drug called efavaleukin alfa in 221 adults with moderate to severe ulcerative colitis. The goal was to see if it could help patients achieve remission after 12 weeks of treatment. The trial was terminated early, so final results are limited, but the data m…

This early-phase study tested a drug called ARC-AAT in healthy volunteers and people with Alpha-1 Antitrypsin Deficiency (AATD), a genetic condition that can lead to lung and liver damage. The main goal was to check safety and how the drug affects levels of a key protein in the b…

This study tested a weekly injection called telitacicept against a placebo in 91 people with moderate-to-severe lupus. The goal was to see if the drug could reduce disease activity and allow lower steroid doses. However, the trial was terminated early, so we have less information…

This early-phase trial tested a gene-edited stem cell product called OTQ923 in 4 people with severe sickle cell disease. The goal was to boost fetal hemoglobin to reduce painful crises and other complications. The study was terminated early, so we have limited data on safety and …

This study followed 265 people with sickle cell disease who were taking Oxbryta (voxelotor) as part of their normal care. Researchers wanted to see how the drug affected hemoglobin levels, sickle cell complications, and overall health over up to 5 years. The study was observation…

This study tested a high-flow nasal cannula device (Airvo) in COPD patients with high carbon dioxide levels who could not or would not use standard non-invasive ventilation. The goal was to see if the device improved blood gas levels and reduced flare-ups over one year. Only 3 pa…

This study tested a new drug called SAR444245, alone or with pembrolizumab, in 14 people aged 12 and older with B cell lymphoma that came back or didn't respond to treatment. The goal was to see if the drug could shrink tumors. The study was terminated early, so results are limit…

This study looked at a disc replacement device called CerPass for people with a damaged disc in their neck. It aimed to see if the device could reduce pain and improve movement without needing further surgery. Only 2 people joined before the study was stopped early, so results ar…

This phase 3 trial compared AVT16, a proposed biosimilar, to the approved drug Entyvio (vedolizumab) in 301 adults with moderate to severe ulcerative colitis. Participants received IV infusions over nearly a year. The study aimed to see if AVT16 works as well and is as safe as En…

This study tested an experimental drug called tildacerfont in 67 children aged 2 to 17 with congenital adrenal hyperplasia (CAH), a genetic condition that disrupts hormone production. The goal was to see if adding this once-daily pill to standard steroid treatment could improve d…

This early-stage study tested a radioactive drug called 177Lu-FF58 in 24 people with advanced pancreatic, gastroesophageal, or brain cancer. The drug seeks out and attaches to specific proteins on cancer cells to deliver radiation directly. The main goals were to check safety, fi…

This study tested a gene therapy for retinitis pigmentosa, an inherited eye disease that causes gradual vision loss. The treatment involved injecting a corrected gene under the retina in one eye. The trial included 19 people aged 13 and older with a specific genetic defect. The m…

This study looked at how well the drug tofacitinib (Xeljanz) works for people with moderate to severe ulcerative colitis in everyday medical practice in Switzerland. It measured symptom improvement and gut inflammation using stool samples. The study was small with 18 participants…

This study looked at whether an anti-inflammatory whole-food diet (rich in vegetables, fruits, and omega-3s, low in processed foods) could improve symptoms in people with mild to moderate Crohn's disease. It enrolled 28 adults and compared the diet to regular eating habits. The t…

This study tested a drug called saroglitazar in women with polycystic ovary syndrome (PCOS) who also have nonalcoholic fatty liver disease (NAFLD). The goal was to see if the drug could safely reduce liver fat over 24 weeks compared to a placebo. The trial was planned for 60 wome…

This study aimed to see if the drug SAGE-718 is safe for people with Huntington's disease when taken over a long period. It was an open-label study, meaning everyone knew they were getting the drug. The study was terminated early, so results are limited.

This study tested an experimental oral treatment called VE202 for people with mild-to-moderate ulcerative colitis, a chronic inflammatory bowel disease. The treatment uses live bacteria to help restore gut balance. The trial was terminated early, so results are limited. Participa…

This study tested an experimental drug called ARO-ENaC in healthy volunteers and people with cystic fibrosis. The goal was to see if it is safe and helps lung function. The trial was stopped early, so results are limited.

This study tested three different antibiotic regimens for people with recurrent Clostridium difficile infection (C. diff), a serious gut infection that keeps coming back. The goal was to see if newer drugs like fidaxomicin or a tapered/pulsed course of vancomycin work better than…

This study tested a new drug called VRG50635 in 54 people with ALS (Lou Gehrig's disease). The main goal was to check if the drug is safe and how the body processes it. The trial was stopped early, but researchers were looking for side effects and changes in disease progression.

This study looks at the long-term safety of inclacumab in people with sickle cell disease who have already taken the drug in a previous study. It tracks side effects and how often pain crises occur. The goal is to see if long-term use is safe and helps control the disease.

This study looked at whether voxelotor, a daily pill for sickle cell anemia, can improve blood flow and oxygen supply to the brain in children. The trial planned to enroll 22 participants aged 4 to 30 years with a severe form of sickle cell disease. However, the study was termina…

This study tested a drug called basmisanil in children aged 2 to 14 with Dup15q syndrome, a rare genetic condition that affects development. The goal was to see if the drug could improve daily living skills like communication and movement. The trial was stopped early after enroll…

This study tested a drug called ADCT-602 in 37 people with a type of blood cancer (B-cell acute lymphoblastic leukemia) that had returned or stopped responding to other treatments. The drug is a lab-made antibody that targets a protein (CD22) on cancer cells to stop them from gro…

This phase II trial tested an experimental drug called roscovitine in 36 adults with cystic fibrosis who have a common genetic mutation and a chronic lung infection. The goal was to check the safety of different doses given in short cycles. The study was terminated early, so its …

This study tested whether anti-VEGF injections into the eye could help treat Coats disease, a rare condition that causes abnormal blood vessels and fluid buildup in the retina, potentially leading to blindness. The trial included 18 people with early-stage Coats disease who had n…

This study tested whether taking vitamin B2 (riboflavin) and getting natural sunlight could strengthen the cornea in people with keratoconus, a condition that causes the cornea to thin and bulge. The approach aimed to be a simpler, cheaper alternative to the standard cross-linkin…

This phase 3 trial tested whether adding the immunotherapy drug pembrolizumab (Keytruda) to precise, high-dose radiation (SBRT) could help prevent early-stage non-small cell lung cancer from returning. The study enrolled 448 people with stage I or II lung cancer who could not hav…

This study tested a drug called narsoplimab (OMS721) for atypical hemolytic uremic syndrome (aHUS), a rare condition that causes blood clots and organ damage. The trial aimed to see if the drug could improve platelet counts and was safe for adults and adolescents. However, the st…

This study tested a drug called OMS721 for people with IgA nephropathy, a kidney disease that can lead to kidney failure. The goal was to see if the drug could reduce protein in the urine, a sign of kidney damage. The trial was stopped early, so results are limited.

This study tested the drug ponatinib in 51 adults with a fast-growing form of chronic myeloid leukemia (CML) who had little or no prior treatment. The goal was to see if ponatinib could control the disease by blocking a protein that drives leukemia growth. However, the drug can c…

This study looked at whether laser eye surgery (PRK) could improve vision in children aged 2 to 17 with lazy eye (amblyopia) caused by large differences in prescription between eyes or very high nearsightedness, farsightedness, or astigmatism. These children had not improved afte…

This study tested a drug called zetomipzomib for people with active lupus nephritis, a kidney disease caused by lupus. The goal was to see if the drug could improve kidney function better than a placebo over 52 weeks. The trial was terminated early, so results are limited. Partic…

This trial tested a special dialysis device (MEX-CD1) designed to remove extra copper from the blood of people with Wilson's disease, a genetic condition that causes copper buildup. The study planned to give 5 to 10 treatments over consecutive days and check if the device worked …

This early-phase trial tested the drug AR-42 in 7 adults with vestibular schwannoma or meningioma brain tumors who were already scheduled for surgery. Participants took AR-42 pills for about 3 weeks before their operation. The main goal was to see if the drug could lower a protei…

This small Phase 1 trial tested growth hormone in 5 adults with cystic fibrosis who were underweight. The goal was to see if the hormone could help them gain weight and muscle, improve their quality of life, and monitor effects on lung function and diabetes. The study was termina…

This study tested an experimental drug called losmapimod in 14 adults with FSHD1, a rare genetic condition that causes progressive muscle weakness. The main goal was to check safety and tolerability, and to see if the drug affects certain biological markers. The trial was termina…

This phase 2 study tested a drug called PGN-EDO51 in 7 people with Duchenne muscular dystrophy whose genetic mutation can be corrected by skipping exon 51. The drug was given by IV infusion to see if it is safe and tolerable. The trial was terminated, so results are limited.

This early-stage trial tested an experimental drug called XmAb27564 in 23 people with mild-to-severe plaque psoriasis or moderate-to-severe atopic dermatitis. Participants received four doses of the drug or a placebo every two weeks. The main goal was to check safety and tolerabi…

This study tested a sirolimus cream (0.2% and 0.4%) applied daily for 12 weeks to treat facial angiofibromas in children aged 2-21 with tuberous sclerosis complex. The goal was to see if the cream could safely reduce the size and redness of these skin growths. The trial was termi…

This study aimed to compare a man-made bone graft material called Virtuos against using the patient's own bone for lumbar spine fusion surgery in people with degenerative disc disease. The goal was to see if Virtuos could achieve similar fusion rates at 12 and 24 months. However,…

This study tested a drug called bertilimumab in 32 adults with moderate to severe ulcerative colitis. The goal was to see if it could reduce disease activity compared to a placebo. The trial was terminated early, so results are limited and no clear benefit was established.

This study tested whether statins, a common cholesterol drug, could change the gut bacteria in people with ulcerative colitis or Crohn's disease to a healthier type. The goal was to see if this change could reduce disease activity. The study was terminated early, so results are l…

This study tested a gene therapy (delandistrogene moxeparvovec) combined with a drug called imlifidase to see if it could safely deliver the therapy to boys with Duchenne muscular dystrophy who had antibodies that might block the treatment. Only 5 participants were planned, but t…

This study tested whether adding low-dose methotrexate to standard anti-TNF therapy could improve and extend the response in adults with moderate to severe psoriasis. The trial planned to enroll 78 participants but was terminated early. The main goal was to see how long patients …

This study tested a new surgical technique for macular holes that remained open after standard treatment. The procedure involves moving a thin membrane from the eye to cover the hole. Only 16 people were enrolled, and the study was stopped early, so we don't yet know if this appr…

This study tested whether combining two drugs, obeticholic acid and bezafibrate, could improve liver health in people with primary biliary cholangitis (PBC) who did not respond well to or could not take the standard medicine. The trial enrolled 75 adults and measured changes in a…

This study looked at the long-term safety of voxelotor (a daily pill) in 179 people with sickle cell disease who had already taken it in a previous trial. Participants continued taking the drug and were monitored for side effects and complications like pain crises. The trial was …

This study tested a new drug called REC-3964 to see if it could prevent repeat Clostridioides difficile (C. diff) infections in adults. Only 3 people were enrolled before the trial was stopped early. The goal was to check safety and whether the drug helped people stay infection-f…

This early-stage trial tests two experimental vaccines, HB-502 and HB-501, designed to teach the immune system to recognize and attack HIV. The study includes 30 people with HIV who are already on suppressive antiretroviral therapy. Participants receive injections every 8 weeks f…

This early-stage trial tested a new drug called FAZ053, alone or with another drug (PDR001), in 154 adults with advanced solid tumors. The goal was to check safety and find the right dose by boosting the immune system to attack cancer. The study was terminated early, so full resu…

This study tested a treatment called darvadstrocel for children with Crohn's disease who have complex perianal fistulas (abnormal tunnels near the anus). Seven participants received an injection of the treatment near the fistula after a cleaning procedure. The goal was to see if …

This study looked at the long-term safety of the drug voxelotor in 162 people with sickle cell disease who had already taken it in earlier trials. Everyone received voxelotor once daily. The main goal was to track side effects. The trial was terminated early, so the full safety p…

This phase 3 study tested an inhaled form of the antibiotic clofazimine added to standard therapy for people with MAC lung disease that did not respond to previous treatment. The goal was to see if the drug could clear the infection from sputum cultures and improve quality of lif…

This study looked at whether weekly injections of PRO 140, taken with standard HIV medications, could keep the virus under control over the long term. It enrolled 43 people who had already completed an earlier PRO 140 study and had undetectable virus levels. The study was termina…

This study tested a bone marrow transplant using a partially matched family donor for people with blood cancers like leukemia. The goal was to see if a milder conditioning regimen and a drug called cyclophosphamide could help patients survive at least 180 days after transplant. T…

This study tested a drug called losmapimod for people with a rare muscle-weakening disease called FSHD. The goal was to see if the drug could slow muscle loss and improve arm function over 48 weeks. About 260 adults with FSHD were randomly assigned to receive either losmapimod or…

This study tested whether adding rapamycin to high-dose chemotherapy could help people with aggressive blood cancers (like certain leukemias and lymphomas) that had returned or not responded to prior treatment. Only 4 adults aged 18–65 took part before the study was stopped early…

This early-phase study tested a drug called abiraterone acetate (normally used for prostate cancer) in 4 children with congenital adrenal hyperplasia (CAH). The goal was to see if it could lower high male hormone levels and reduce the need for strong steroid medications that can …

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 tested an oral medication called ozanimod to see if it could help people with moderate to severe Crohn's disease feel better. The trial planned to include 625 adults who had not responded well to other treatments. However, the study was terminated early, so the full re…

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 study tested a new drug called INCB059872 in people with advanced solid tumors or blood cancers that had not responded to standard treatments. The goal was to find a safe dose and see if it could control the disease. The trial was stopped early, so results are limited.

This early-phase study tested the safety of an oral drug called LY3405105 in 54 people with advanced solid tumors. The trial was divided into two parts to find the right dose and to see if the drug could shrink tumors. However, the study was terminated early, meaning the drug did…

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 an oral drug called CX-4945 (silmitasertib) in 21 children with recurrent SHH medulloblastoma, a rare brain tumor. The trial aimed to find the best dose and see if the drug could shrink tumors. However, the study was stopped early, so we don't have clear answers…

This study tested a drug called ruxolitinib in 73 people with two types of blood cancer—polycythemia vera or essential thrombocythemia—that were not helped by standard treatment. The goal was to see if the drug could safely reduce high blood cell counts and symptoms like an enlar…

This early-phase trial tested a new immunotherapy approach for children with DIPG, a rare and aggressive brain stem tumor. After standard radiation and chemotherapy, patients received special vaccines and immune cells designed to attack the tumor. The study was small (11 particip…

This study tested an experimental drug called VENT-02 in 29 people with mild to moderate Parkinson's disease. The drug aims to reduce brain inflammation by blocking a protein called NLRP3. The trial was terminated early, so we don't have clear results on whether it helps symptoms…

This study tested a new treatment called CTX110 for people with B-cell blood cancers that came back or didn't respond to standard therapy. CTX110 uses donor immune cells that are edited with CRISPR technology to target and attack cancer cells. The trial enrolled 93 adults and aim…

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 study tested a program to help Virginia Medicaid enrollees with HIV fill their antiretroviral prescriptions on time. The program offered support from providers, pharmacies, and community groups, with extra help for those who were very late refilling. Only 4 people enrolled b…

This Phase 2 trial tested a single dose of gene therapy (fordadistrogene movaparvovec) in 10 boys with early-stage Duchenne muscular dystrophy. The goal was to check safety and whether the therapy could help muscles produce a mini-dystrophin protein. The study was terminated earl…

This study looked at the long-term safety of efgartigimod in adults with post-COVID postural orthostatic tachycardia syndrome (POTS), a condition causing rapid heart rate and dizziness upon standing. The trial was an open-label extension for people who completed a previous study.…

This early-phase trial tested an experimental drug called INCB059872 in 25 people with Ewing sarcoma that had returned or stopped responding to standard treatments. The goal was to check the drug's safety and see if it could shrink tumors. The study was terminated early, so full …

This phase 3 trial tested whether adding parsaclisib to the standard drug ruxolitinib could better shrink the spleen and ease symptoms in people with myelofibrosis. The study enrolled 252 participants but was terminated early, so the full results are not available. The goal was t…

This study follows 7 people with Duchenne muscular dystrophy who previously received an experimental gene therapy called fordadistrogene movaparvovec. Researchers will monitor them for 10 years to check for side effects and see if the treatment continues to help with movement. Th…

This study looks at the long-term safety of spesolimab for people with palmoplantar pustulosis, a chronic skin condition causing painful pustules on hands and feet. Participants who benefited from spesolimab in earlier trials can continue treatment for up to 5 years, with monthly…

This study tested an experimental drug called NBI-921352 in people aged 2 to 21 with a rare, severe form of epilepsy caused by a change in the SCN8A gene. The goal was to see if adding this drug to their current seizure medicines could reduce how often they had seizures. The tria…

This study tested a special contact lens that slowly releases the glaucoma drug latanoprost, aiming to replace daily eye drops. Only one person enrolled before the study was stopped early. The goal was to check safety and whether the lens could lower eye pressure, but no conclusi…

This study tested a drug called ALXN2050 in people with kidney inflammation caused by lupus (lupus nephritis) or IgA nephropathy. The goal was to see if it could reduce protein in the urine, a sign of kidney damage. About 100 adults were planned to take the drug or a placebo alon…

This phase 2 trial tested a drug called REGN4461 (mibavademab) in 20 people with familial partial lipodystrophy, a rare condition causing abnormal fat distribution and metabolic problems. The study aimed to see if the drug could lower high triglycerides and improve blood sugar co…

This study tested a drug called efavaleukin alfa in people with moderate to severe ulcerative colitis who had already completed a previous study. The goal was to see if the drug is safe and works over a longer period, up to 104 weeks. The study was terminated early and enrolled o…

This study tested whether the oral drug ozanimod can help people with moderate to severe Crohn's disease maintain symptom control and reduce gut inflammation over 52 weeks. About 550 participants who had already responded to an initial treatment were randomly assigned to receive …

This study tested a drug called tildacerfont in 27 women with polycystic ovary syndrome (PCOS) who had high levels of adrenal androgens (hormones). The goal was to see if the drug could safely lower these hormones. The trial was stopped early, so results are limited.

This study tested a drug called REC-2282 in people aged 12 and older with progressive meningiomas (brain tumors) linked to NF2 gene mutations. Participants took the drug or a placebo three times a week in cycles. The goal was to see if the drug could slow tumor growth. The trial …

This study tested whether lorcaserin, when added to current medications, could reduce convulsive seizures in people with Dravet syndrome, a severe form of epilepsy. The trial planned to enroll about 22 participants aged 2 and older, but it was terminated early. The main goal was …

This study looked at whether measuring oxygen levels in the brain and gut using a light-based device (NIRS) could help doctors diagnose necrotizing enterocolitis (NEC) earlier in newborns with symptoms like bloody stool or a swollen belly. The study planned to include 45 newborns…

This study tested a new blood test that looks for fetal cells in the mother's blood to diagnose genetic diseases in the baby. It aimed to replace invasive tests like amniocentesis, which carry a small risk of miscarriage. The trial enrolled 18 pregnant women but was terminated ea…

This study tested a new imaging tool called multispectral optoacoustic tomography (MSOT) to see if it could safely detect inflammation in people with graft-versus-host disease, Crohn's disease, or colitis. The device uses light and sound to create images of inflamed areas. The tr…

This study tested a new oral drink (GS3-007a) to diagnose adult growth hormone deficiency (AGHD). It compared the drink to the standard insulin tolerance test in 120 adults suspected of having AGHD and healthy volunteers. The goal was to see if the oral test works as well as the …

This study tested a smartphone app called CRADLE that uses a photo to detect leukocoria (a white glow in the pupil), which can be a sign of retinoblastoma, cataracts, or glaucoma. Researchers planned to compare the app's accuracy to a standard eye exam in children with these cond…

This study aimed to see if the CONNEQT Pulse blood pressure monitor gives accurate readings in pregnant women, including those with high blood pressure or pre-eclampsia. Researchers compared it to a standard blood pressure cuff in 41 pregnant volunteers. The study was stopped ear…

This study tested whether special MRI sequences can spot leftover brain arteriovenous malformations (AVMs) after embolization treatment. Researchers compared the MRI results to standard angiography in 53 adults. The study was terminated early, so its findings are limited.

This study looked at whether a third-trimester ultrasound can accurately find bowel rotation issues in fetuses that raise the risk of a life-threatening twist (volvulus). Researchers compared ultrasound results with fetal MRI, the current best method. The study included 77 pregna…

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 study tested whether wearing a small, waterproof heart monitor (ePatch) for 7 days finds more treatable heart rhythm problems than the usual 24-hour Holter monitor in people who have fainted. About 55 adults with fainting symptoms were randomly assigned to either the 7-day e…

This early-phase trial tested a radioactive imaging agent called 68Ga-FF58 in 14 adults with certain advanced solid tumors (glioblastoma, brain metastases from breast cancer, gastroesophageal cancer, or pancreatic cancer). The goal was to see if the agent could help detect these …

This study looked at whether eating corn rich in zeaxanthin (a natural pigment) could increase the density of the macular pigment in the eye, which may help prevent age-related macular degeneration (AMD), a leading cause of blindness. Nine healthy volunteers aged 20-35 ate a dail…

This pilot study tested the blood pressure drug telmisartan (Micardis) to prevent acute graft-versus-host disease (GVHD) in people receiving a stem cell transplant for blood cancers. The trial aimed to enroll 60 participants but was terminated early with 32. Researchers tracked s…

This study tested a drug called RLYB212 in pregnant women at high risk for a condition where the mother's immune system attacks the baby's platelets, causing dangerous bleeding. The goal was to see if the drug was safe and how it moved through the body. Only one woman enrolled be…

This pilot study tested whether placing a special mesh during ileostomy closure surgery could prevent hernias in people with inflammatory bowel disease. Only 8 patients were enrolled before the study was stopped. The main goal was to check safety, like infections or wound problem…

This study tested whether a non-invasive breathing machine (ventilation) could prevent acute chest syndrome, a serious lung complication, in children with sickle cell disease who were hospitalized for severe pain. The standard prevention is spirometry, which requires active deep …

This study tested a new monthly pill (islatravir) to see if it could prevent HIV infection better than a daily pill in 730 women at high risk. The trial was stopped early, so results are limited. The goal was to offer a more convenient prevention option.

This small phase 2 trial tested whether the drug Panhematin could prevent acute attacks in people with certain types of porphyria. Only 13 participants were enrolled before the study was terminated early. Researchers tracked attacks and serious side effects, but the limited data …

This study tested whether the cholesterol-lowering drug pravastatin could prevent preeclampsia in women who had a previous early preterm delivery due to the condition. The trial planned to enroll 1,550 women but was terminated early with only 102 participants. Because it stopped …

This study tested whether a combination of antibodies (casirivimab+imdevimab) could prevent COVID-19 in people with weakened immune systems, such as transplant recipients or cancer patients. Participants received either the antibodies or a placebo as a shot under the skin. The tr…

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 compares two treatments for transformed migraine (chronic daily headache): a sphenopalatine ganglion nerve block using lidocaine gel applied inside the nose, versus daily oral amitriptyline (Elavil), an antidepressant often used for migraine prevention. Ten adults aged…

This study compared a new, minimally invasive ultrasound-guided procedure to standard surgery for carpal tunnel syndrome. The goal was to see if the new technique could provide similar relief with a smaller incision and faster recovery. Only 7 people were enrolled before the stud…

This study compared two ways to drain a pilonidal abscess: a simple needle puncture (aspiration) versus the standard incision. The goal was to see if puncture leads to faster healing, lower cost, and less time off work. Only 5 adults took part before the trial was stopped early, …

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 study tested a nasal spray containing a strong painkiller (sufentanil) for adults with sickle cell disease experiencing a painful crisis. The goal was to see if it could provide faster relief than the standard gas mixture (EMONO) before giving morphine through an IV. The tri…

This small Phase 2 trial tested whether the antibiotic rifaximin could reduce bloating in people with diabetic gastroparesis, a condition where the stomach empties slowly. Only 5 participants were enrolled before the study was terminated early. The main goal was to measure change…

This study tested whether injecting methotrexate directly into the nail area could improve nail psoriasis symptoms like pitting and discoloration. Only one person enrolled before the trial was stopped early. The goal was to measure changes in nail appearance using a severity scor…

This study tests whether the drug pregabalin, alone or with a rehabilitation program, can reduce chronic fatigue in people with post-COVID syndrome. Participants are adults aged 18 to 65 who had COVID-19 at least six months ago and still feel very tired. The trial compares four g…

This study tested whether N-acetylcysteine (NAC), an antioxidant, could help reduce impulse control disorders caused by Parkinson's medications. Only 14 people with mild-to-moderate symptoms were enrolled before the trial was stopped early. The goal was to see if 10 weeks of NAC …

This study looked at whether acupressure, a non-invasive technique, can reduce nausea and vomiting after the NUSS procedure for pectus excavatum. The trial planned to enroll 40 people aged 8 to 40 but was terminated early. The goal was to see if this simple method could improve r…

This study tested whether smelling lavender essential oil before and during an office hysteroscopy (a procedure to look inside the uterus) could lower women's anxiety and pain. Twenty-two women were randomly assigned to inhale either lavender oil or distilled water (placebo). The…

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 looked at whether adding high-dose steroid pulses to the usual oral steroid course could help people with moderate ulcerative colitis flares achieve remission without needing other treatments. The trial was terminated early and enrolled 75 adults with left-sided or ext…

This study tested whether a fractional carbon dioxide laser can improve moderate to severe acne in adults aged 18 to 40. Participants received up to 3 laser treatments and were followed for changes in blemish count and skin severity. The trial was terminated early and only enroll…

This study tested a fast-dissolving film (BXCL501) placed under the tongue to quickly reduce severe agitation in older adults with dementia. The trial aimed to enroll many participants but was stopped early after only 5 people joined. Because it ended so soon, we cannot draw reli…

This study tested a small device worn on the ear that uses mild electrical pulses to help control pain after a liver transplant. The goal was to see if it could reduce the need for strong painkillers like opioids. The study was stopped early and included 27 adults waiting for a l…

This study tested a new way to treat hand tremors caused by essential tremor or Parkinson's disease using a linear accelerator, a common radiation machine. Instead of one large dose, the radiation was given in three smaller sessions to protect healthy brain tissue. The goal was t…

This study tested a new, short therapy module for people with obsessive-compulsive disorder (OCD) who struggle with compulsive checking. The therapy focuses on fixing unhelpful beliefs about memory that drive the urge to check. Researchers aimed to see if this approach reduces ch…

This study tested a smartphone app that teaches mindfulness meditation to help Black and Latino adults with anxiety during the COVID-19 pandemic. About 400 participants were assigned to one of two mindfulness programs or a waitlist. The trial was terminated early, so we don't hav…

This study tested a drug called AVP-786 to see if it could safely reduce disinhibition—impulsive or inappropriate behavior—in people with neurodegenerative disorders like Alzheimer's or frontotemporal dementia. The trial planned to include many participants but was terminated ear…

This study tested a personalized touchscreen device (My PATI) to help people with Alzheimer's or related dementias communicate their care preferences. The goal was to improve quality of life for both patients and their caregivers. The study was terminated early, so results are li…

This study tested whether a personalized exercise program could reduce fatigue in people with chronic fatigue syndrome. About 101 participants were planned, but the trial was terminated early. The approach involved tailoring aerobic and resistance exercises based on weekly fatigu…

This study looked at whether a brief, noninvasive brain stimulation technique called tDCS could immediately reduce pain and improve function in people with fibromyalgia or chronic fatigue syndrome. About 20 participants received one session, and researchers measured changes in pa…

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 robotic device called the G-EO Gait Trainer to help people with Parkinson's disease improve their walking. The device supports the body and guides leg movements to make walking practice safer and more effective. The trial was terminated early, so only limited …

This study looked at whether moderate physical activity three times a week could reduce chronic fatigue in people with inflammatory bowel disease (IBD). Fatigue affects about 40% of IBD patients and can lower quality of life. The trial planned to compare a group that added extra …

This study aimed to compare two blood pressure medications—oral nifedipine (a pill) and intravenous labetalol (given through a vein)—for treating dangerously high blood pressure in women who have just given birth. The goal was to see which drug lowers blood pressure faster. Howev…

This pilot study tested whether MitoQ, an antioxidant supplement, could improve bladder symptoms like urgency and frequency in women aged 50 and older with metabolic syndrome. Twenty participants took either MitoQ or a placebo for 4 months, tracking their symptoms through questio…

This study looked at whether using a lower fluid pressure (60 mmHg) during hysteroscopy, a procedure to remove fibroids, works as well as the standard pressure (80 mmHg). The goal was to see if it improves surgery time, fluid use, and surgeon's view. The study was stopped early a…

This study looked at whether adding platelet-rich plasma (PRP) to standard carpal tunnel release surgery helps people with severe carpal tunnel syndrome. The trial planned to enroll 15 adults with severe nerve damage. It compared surgery with PRP to surgery alone. The study was t…

This study aimed to see if acceptance and commitment therapy (ACT), a type of talk therapy, could help HIV-positive men with mental health issues like stress and anxiety. Only one person enrolled before the study was stopped early. Because of this, no meaningful results were obta…

This study tested the drug deucravacitinib (SOTYKTU) in 130 people with psoriasis on the palms, soles, and genitals. The goal was to see if it improves skin clearance and reduces symptoms. The trial was terminated early, so final results are limited.

This study tested the drug guanfacine to see if it could reduce hyperactivity and impulsivity in children aged 6 to 12 with Down syndrome. The plan was to give the drug or a placebo for 8 weeks and measure behavior changes. However, the trial was stopped early after only 3 childr…

This study tested whether a mindfulness program (including meditation, body scans, and gentle yoga) could help people with implanted heart devices like ICDs or pacemakers feel less anxious, stressed, and depressed. The study planned to enroll 34 adults who had recently received s…

This study compared two types of non-invasive breathing support for people with sudden breathing failure who are obese or have sleep apnea. One machine automatically adjusts pressure and volume, while the other uses a fixed pressure setting. The goal was to see which method clear…

This study looked at whether using walking poles during an 8-week independent walking program could improve walking and activity levels in people with Parkinson's disease. Eleven participants were assigned to walk with or without poles at least three times a week. The trial was t…

This study tested a drug called CVL-871 to see if it is safe and can help reduce apathy (lack of motivation or interest) in people with dementia. The trial included 41 participants with mild to moderate dementia and clinically significant apathy. The main goal was to check for si…

This study looked at whether the drug safinamide (Xadago) could improve sleep quality in people with Parkinson's disease. Eleven participants took safinamide for 12 weeks while their sleep was measured. The trial was terminated early, so the results are not conclusive.

This study tested whether a low dose of fentanyl, a strong painkiller, placed under the tongue could help people with severe COPD feel less breathless during exercise. The trial planned to include 40 participants but was stopped early. Because it was terminated, we cannot draw cl…

This study tested whether a gentle, non-invasive brain stimulation technique (tDCS) done at home, along with computer-based brain games, could improve thinking and language skills in people with primary progressive aphasia, mild cognitive impairment, or dementia. Participants use…

This study tested a redesigned lacrimal stent, called the Slit Stent II, for people with severe tearing (epiphora) caused by a blocked tear duct. The stent has slits to allow tears to drain while it is still in place after DCR surgery. The trial planned to enroll 5 people to chec…

This phase 2 pilot study tested a new dual bronchodilator inhaler called PT003 in 4 people with stable moderate-to-severe COPD. Researchers measured breathing patterns and lung function right after the first dose and again after 4 weeks of treatment. The goal was to see if PT003 …

This study tested whether istradefylline, a drug already approved for motor symptoms in Parkinson's, could also improve apathy and motivation. Only 8 people enrolled before the trial was terminated early. The results are too limited to draw any conclusions.

This study tested whether deep brain stimulation (DBS) could help people with severe Tourette syndrome whose symptoms don't improve with medication. Researchers placed tiny electrodes in a part of the brain called the thalamus to try to calm abnormal signals that cause tics. Only…

This study tested a device called CoolStat that is placed in the nose to cool down brain-injured patients with fever in the ICU. The goal was to see if it could safely and quickly bring their temperature to normal. Only 9 patients were enrolled before the study was stopped early,…

This study gave free eyeglasses to over 1,800 students aged 9-20 in Sierra Leone who had trouble seeing. Researchers wanted to find out if wearing glasses would improve their math and reading skills, and also help with anxiety and social well-being. The study was planned to last …

This study looked at whether a gentle electrical stimulation device (TENS) placed on the skin could help people with COPD exercise longer after a serious flare-up. The plan was to compare exercise time with and without TENS in a small group of patients. However, the study was sto…

This study tested whether a stepped-care approach using internet-based cognitive behavioral therapy (ICBT) works as well as group therapy or standard medical care for adults with obsessive-compulsive disorder (OCD). 46 adults in China received one of the three treatments for 6 we…

This study looked at whether having a peer mentor—an older teen or young adult who also has IBD—can help youth ages 10-17 better manage their disease and improve their quality of life. Participants were randomly assigned to either a year-long mentoring program or educational acti…

This study tested whether a high-dose magnetic stimulation (TMS) targeting both motor and cognitive brain areas could better reduce freezing of gait in people with Parkinson's disease compared to standard low-dose stimulation of just the motor area. The trial planned to enroll 64…

This study tested a school-based program called Proud & Empowered, designed to help LGBTQ students cope with minority stress and improve mental health. About 228 high school students took part. The program was led by school counselors and social workers. The study was terminated …

This study looked at whether using the Calm meditation app could help medical residents and fellows at Banner University Medical Center Phoenix feel less stressed, anxious, or burned out during the COVID-19 pandemic. Only 7 people signed up, and the study was stopped early, so we…

This pilot study explored whether adding acupuncture to standard pulmonary rehabilitation can improve lung function, exercise tolerance, and quality of life in people with COPD. Thirteen adults took part, receiving either electro-acupuncture at specific points or a control acupun…

This study tested a cannabidiol (CBD) oral solution in children under 2 years old with tuberous sclerosis complex, Dravet syndrome, or Lennox-Gastaut syndrome who had uncontrolled seizures. The goal was to see if CBD is safe and can reduce seizures. However, the study was termina…

This study tested an inhaled form of apomorphine (AZ-009) to quickly treat 'OFF' episodes in people with Parkinson's disease—times when symptoms like stiffness or trouble moving return between regular medication doses. The trial was planned for more participants but was terminate…

This study looks at how the drug luspatercept is used in real-world clinics for patients with lower-risk myelodysplastic syndromes (MDS) or beta-thalassemia. Researchers will track patient characteristics, treatments, and outcomes like reduced need for blood transfusions. The goa…

This trial investigates whether using 3D image fusion during iliac angioplasty reduces the amount of radiation patients receive compared to standard practice. The study involves 37 adults with iliac artery narrowing who need angioplasty. Researchers will measure radiation exposur…

This early-stage study aimed to compare how a tablet form of tafamidis is absorbed in the body compared to the existing capsule form. It involved 24 healthy adults who took a single dose of each form under fed conditions. The study was terminated early, so results may be limited.

This study aimed to collect real-world data on Netherton Syndrome, a rare genetic skin disorder. Researchers planned to follow 4 participants over 52 weeks, measuring skin severity and other symptoms. The study was terminated early, so results are limited.

This study tried to create a score that doctors could use before birth to predict whether a newborn would die after the mother's water broke very early (between 22 and 28 weeks of pregnancy). The researchers looked back at medical records of 34 women to find factors that might pr…

This study looked at how patients with hemophilia A or those on blood thinners feel about monitoring their blood clotting at home. Researchers interviewed 19 people to understand their needs and hopes for a new device that could measure clotting factors. The goal was to gather in…

This study aimed to see if certain substances in stool and blood can predict whether the gut lining has healed in people with moderate to severe ulcerative colitis. Researchers planned to measure these markers in 62 adults before and after treatment. The study was terminated earl…

This early-stage study is testing a new drug called pociredir in 24 adults with sickle cell disease. Researchers want to see how the drug moves through the body and whether taking it with food changes its effects. The goal is to gather safety and dosing information, not to treat …

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 looks at the tiny hair-like structures (cilia) inside the fallopian tubes of women who have had surgery to remove a tube or uterus. Researchers want to see if differences in these cilia help explain why some women have ectopic pregnancies (when a fertilized egg grows o…

This study reviewed medical records of 42 people with familial adenomatous polyposis (FAP) who had previously taken the drug erlotinib. Researchers wanted to see how quickly the disease progressed after the drug was stopped. The goal was to better understand what happens when tre…

This study looked at how anesthesia changes brain activity in people with Parkinson's disease or essential tremor who were already scheduled for deep brain stimulation (DBS) surgery. Researchers recorded brain signals during a short period of anesthesia to understand which circui…

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 aimed to measure how eye volume changes (a sign of pressure fluctuation) before and after cataract surgery in patients with exfoliative glaucoma, a type of glaucoma common in Brittany. Only 6 adults were enrolled before the study was terminated. The goal was to see if …

This study looked at whether MRI scans can detect changes in the lungs of people with cystic fibrosis as well as CT scans do. CT scans use radiation, so finding a reliable alternative like MRI could mean safer monitoring over time. The study included 212 patients aged 8 and older…

This study aimed to compare the types of Candida yeast found in patients with two forms of autoimmune polyendocrinopathy, a rare condition where the immune system attacks multiple glands. Researchers planned to collect samples from the mouth and urine of 7 participants to see how…

This study looks at adults with phenylketonuria (PKU) who were diagnosed as newborns and treated with a special diet. Researchers want to see if how long and how strictly they followed the diet as children affects their intelligence (IQ) as adults. The goal is to use this informa…

This study looked at people with cone disorders, a type of inherited eye disease that affects color vision and sharp sight. Researchers analyzed genetic changes of unknown significance to see if they cause disease. The goal was to improve genetic diagnosis, not to test a treatmen…

This study looked at whether a weak immune response to bacteria in COPD patients leads to more lung infections and flare-ups. Researchers collected sputum, blood, and nasal swabs from 23 hospitalized COPD patients during a flare-up and again 8 to 16 weeks later. The goal was to m…

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 study looked at the VERTICALE Cervical System, a device used to stabilize the spine during surgery. Researchers planned to track neck disability in patients with conditions like degenerative disc disease or trauma. Only 4 patients were enrolled before the study was terminate…

This study looked at how the mpox virus spreads and how the immune system responds in people with advanced HIV. Researchers tracked the virus in different body samples and measured immune markers to better understand severe illness. The goal is to use these findings to improve tr…

This study aimed to find biomarkers (measurable substances in the blood) that could help doctors diagnose bradykinin angioedema and predict how severe attacks might be. The condition causes sudden, unpredictable swelling, and current tests don't work well for all types. Researche…

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 looked at whether two simple blood test results—hemoglobin and red blood cell distribution width—could predict if a person with polycythemia vera would stop responding to the drug hydroxyurea. Researchers planned to follow 76 adults starting hydroxyurea for the first t…

This study tested a web-based program to help Black women in Texas use HIV and STI home tests and connect to treatment or PrEP. The program included five sessions with goal-setting and action plans. Only 31 women enrolled before the study was stopped early, so the findings are li…

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 whether a liquid biopsy—testing the fluid around the brain and spinal cord—can find tumor DNA in children and young adults with brain tumors. The goal was to see if this safer, non-surgical test could replace risky tissue biopsies for diagnosis and monitoring…

This study looked at whether peeling a thin membrane inside the eye (the internal limiting membrane) during surgery for a detached retina helps patients see better and prevents scar tissue. The trial was stopped early after enrolling only 21 people, so the results are not strong …

This study tested whether two gut hormones, GIP and GLP-2, could reduce bone breakdown in children with spinal muscular atrophy, cerebral palsy, or Duchenne muscular dystrophy who use wheelchairs. Participants received a liquid meal and then either a hormone injection or a placeb…

This study tested a new, non-invasive eye imaging method called hyperspectral imaging. It takes pictures of the back of the eye using many different colors of light to find details not visible with standard cameras. About 679 adults with healthy eyes or eye diseases like diabetic…

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 aimed to compare the gut bacteria of children newly diagnosed with juvenile idiopathic arthritis, type 1 diabetes, or inflammatory bowel disease with those of their healthy siblings. Researchers wanted to see if differences in gut microbes and diet could explain why so…

This study aimed to find out how common non-tuberculous mycobacteria (NTM) infections are in people with cystic fibrosis in France. Researchers collected sputum and blood samples from 848 participants to test for NTM using culture and a new blood test. The study was terminated ea…

This study looked at whether using a CPAP machine at night could reduce abnormal acid reflux and lung inflammation in people with idiopathic pulmonary fibrosis (IPF) who also have sleep apnea. Only 7 people enrolled before the study was terminated early. The goal was to measure c…

This study looked at how a gentle brain stimulation technique called tDCS affects thinking and behavior in children aged 10-17 with obsessive-compulsive disorder (OCD). The goal was to understand brain processes linked to OCD, not to treat the condition. Only 6 participants were …

This study aimed to understand why some people taking the cancer drug ibrutinib develop heart issues like atrial fibrillation (irregular heartbeat) and high blood pressure. Researchers planned to follow 8 patients with blood cancers, using heart tests and blood markers to try to …

This study looked at how insulin works in the skin of people with plaque psoriasis who are also overweight or have prediabetes. Researchers took small skin samples from both psoriasis patches and healthy skin to measure insulin activity. The goal was to understand if insulin acts…

This study tested a new lab method (RAD51 assay) to see if it could identify ovarian cancer patients whose tumors are vulnerable to the drug olaparib, even if they don't have BRCA gene mutations. The goal was to find a larger group of patients who might benefit from this treatmen…

This study tested whether a 16-week program of yoga and lifestyle changes could affect gene activity (micro RNA) and heart disease risk factors in 16 adults with coronary artery disease. Participants had either undergone a heart procedure or had non-obstructive heart disease with…

This study aimed to find brain and movement markers in people with a type of depression that doesn't respond to standard antidepressants, called anhedonic depression. Researchers measured brain waves (P300), walking speed, and hand movements in 36 participants. The goal was to id…

This registry tracked 336 adults who had surgery for a large hiatal hernia (over 3 cm) at least two years earlier. Some received a LINX magnetic implant to strengthen the valve between the stomach and esophagus, while others had a fundoplication (wrapping the stomach around the e…

This study is for people with cystic fibrosis and their family members. Its goal is to collect medical information and biological samples during regular clinic visits. These will be stored and used by researchers to learn more about how cystic fibrosis affects the body and how it…

This study aimed to validate a scoring system that predicts whether patients will have good vision one year after combined cataract and epiretinal membrane surgery. The score uses factors like age, symptom duration, and initial vision. Researchers planned to enroll 164 adults age…

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 for new genes that might increase the risk of developing differentiated thyroid cancer. Researchers planned to analyze the complete DNA of people with thyroid cancer and their family members. The study was stopped early and involved 34 participants.

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 early-phase trial tested whether posaconazole, a drug used for fungal infections, can reach brain tumors in high enough amounts to stop cancer cells from dividing. Seven adults with glioblastoma took the drug before surgery, and researchers measured drug levels in their spin…

This study looked at how moderate or severe liver problems change the amount of the cancer drug oral azacitidine in the blood. It was designed for adults with certain blood cancers. The study was stopped early after only 2 people enrolled, so the results are very limited.

This early-phase trial tested whether ketoconazole, a drug normally used for fungal infections, can enter brain tumors in high enough amounts to stop tumor cells from dividing. Only 1 person was enrolled before the study was terminated. The goal was to measure drug levels in the …

This study aimed to understand how the COVID-19 vaccine impacts the immune system in people with lupus, a chronic autoimmune disease. Researchers planned to enroll lupus patients to measure their immune response to the vaccine and see if lupus disease activity or treatments affec…

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 looked at whether high levels of male hormones (hyperandrogenism) lower the chance of IVF success in women with PCOS. Researchers planned to track 32 women aged 18–39, but the study was terminated early. The goal was to see if these hormones affect pregnancy rates afte…

This study looked at over 100 people with inherited retinal dystrophies, a group of rare eye diseases that can cause vision loss. Researchers collected genetic and eye exam data to find links between specific gene mutations and symptoms. The goal was to better understand these di…

This study compared two methods of removing the clear outer layer of the eye (the epithelium) during laser vision correction for nearsightedness and astigmatism. One method uses a laser, the other uses alcohol and manual scraping. The goal was to see which approach leads to less …

This study tested a new questionnaire called Prolac-10 to see if it can accurately measure quality of life in people with prolactinoma (a type of pituitary tumor) who are starting medical treatment. Only 8 people took part before the study was stopped early. The goal was to check…

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 tested whether artificial intelligence can detect and classify abnormal lung sounds in people with cystic fibrosis, COPD, or pulmonary fibrosis. Researchers recorded lung sounds with an electronic stethoscope during routine checkups and analyzed them using AI. The goal…

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 spraying water on the body could help older adults with COPD stay cool during heat waves. Researchers planned to measure body temperature and lung function in a climate chamber. However, only one person took part before the study was stopped, so no mean…

This study was a registry that planned to follow 234 adults undergoing spine fusion surgery for conditions like spinal stenosis and degenerative disc disease. Researchers aimed to compare different surgical techniques and track long-term outcomes, including pain, function, and wh…