Syndromic disease

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 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 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 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 new therapy called CYNK-001, which uses natural killer cells from the placenta to fight COVID-19. It enrolled only 7 adults with moderate COVID-19. The study was terminated early, so we have limited information on safety and effectiveness.

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 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 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 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 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 pilot study tested a new drug called sEphB4-HSA combined with standard chemotherapy (azacitidine or decitabine) in 7 patients with myelodysplastic syndrome, chronic myelomonocytic leukemia, or acute myeloid leukemia that had returned or not responded to prior treatment. The …

This study tested a combination of two drugs, venetoclax and azacitidine, in 34 adults with high-risk blood cancers (MDS or CMML) that returned or didn't respond to earlier treatment. The goal was to find the safest dose and see if the combo could shrink or control the cancer. Th…

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 whether giving the heart medication metoprolol through a vein could help people with severe lung failure (ARDS) in the ICU. The goal was to see if it helped them breathe without a machine for more days. However, the trial was stopped early and only 14 people too…

This early-phase trial tested whether adding the targeted drug ivosidenib to standard FLAG chemotherapy could help people with a specific genetic form of acute myeloid leukemia (IDH1-mutant AML). The study aimed to find the best dose and check for side effects in patients whose c…

This trial tested a new drug called MT1002 in 6 people having a heart procedure (PCI) after a heart attack. The goal was to see if it could safely control blood clotting. However, the study was stopped early, so we don't have enough information to know if the drug works or is saf…

This study tested a reduced-intensity bone marrow transplant, or 'mini transplant,' for people with blood cancers like lymphoma and leukemia. The transplant used marrow from a relative and was combined with chemotherapy drugs to help the donor cells take hold. Only 15 people took…

This early-phase trial tested whether inhaling low doses of carbon monoxide could be safe for people with severe lung injury caused by sepsis. Only 5 participants were enrolled before the study was stopped early. The goal was to see if a personalized dosing formula could safely a…

This study tested a drug called vibecotamab in adults with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) whose cancer did not respond to or came back after standard treatments. The drug is a special antibody designed to help the immune system attack cancer cells.…

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 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 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 an experimental drug called HZN-1116 in 209 people with Sjögren's syndrome, an autoimmune disease causing dry eyes and mouth. The goal was to see if the drug could reduce disease activity and symptoms. The trial was terminated early, so results may not be availa…

This pilot study tested the drug ADCT-301 in 3 adults whose acute myeloid leukemia, myelodysplastic syndrome, or related blood cancer had returned after a stem cell transplant. The goal was to see if the drug could safely bring about remission. However, the study was terminated e…

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 whether hydroxychloroquine, a drug used for lupus and arthritis, could help control ANCA vasculitis—a group of autoimmune diseases that cause blood vessel inflammation. Adults with mild disease activity were randomly assigned to receive hydroxychloroquine or a p…

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 study tested a new drug called AUR103 Calcium in 27 adults with advanced solid tumors, acute myeloid leukemia, myelodysplastic syndromes, or non-Hodgkin lymphoma who had no other treatment options. The goal was to find the best dose and check safety. The study wa…

This study looked at whether hydroxychloroquine (Plaquenil) could improve artery function in people with antiphospholipid syndrome (APS), a condition that raises the risk of blood clots. Researchers planned to give either hydroxychloroquine or a placebo to 25 participants and mea…

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

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

This trial tested a smartphone app (SKI App) that uses blockchain and AI to help dialysis patients track their health data and get personalized care tips. The goal was to see if the app could reduce emergency room visits and hospital stays compared to a basic health app. The stud…

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 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 early-stage trial tested a new drug called gentulizumab in 58 people with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) that had come back or stopped responding to treatment. Gentulizumab is an antibody that helps the immune system attack cancer cells. The m…

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 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 a combination of chemotherapy drugs (cladribine, cytarabine, mitoxantrone, and G-CSF) in 53 adults with acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS) that had come back or not responded to prior treatment. The goal was to see how many …

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 drug called spesolimab in 5 adults with moderate to severe pyoderma gangrenosum, a rare autoimmune disease that causes painful skin ulcers. The goal was to see if the drug could help heal the ulcers and to understand how the body's immune system responds. The …

This early-stage study tested an experimental drug called INKmune in people with myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML). The goal was to find a safe dose and check for side effects. However, the study was stopped early after only 2 people enrolled, so no c…

This early-stage study tested a new oral drug, ASP5502, for primary Sjögren's syndrome, an autoimmune condition causing dry eyes and mouth. It involved 116 healthy adults and patients to check safety and how the body processes the drug. The study was terminated, but results may g…

This study tested whether breathing in nitric oxide gas could keep mild-to-moderate COVID-19 from becoming severe. Researchers enrolled 62 hospitalized adults who were breathing on their own. The goal was to see if the gas reduced the need for a breathing tube. The trial was stop…

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

This early-phase trial tested an experimental drug called SL-172154, given alongside standard chemotherapy drugs, for people with higher-risk myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML). The main goals were to check safety and find the right dose. 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 early-phase trial tested a new treatment for adults with acute myeloid leukemia that had come back or not responded to standard therapy. The treatment involved giving patients natural killer cells grown from donated umbilical cord blood, along with a drug called IL-2 to help…

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 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 phase II trial tested a combination of two drugs, venetoclax and decitabine, in 235 patients with certain blood cancers like acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (MDS) that had come back or not responded to treatment, as well as in older patien…

This study tested whether using a urine biomarker test (NephroCheck) to guide treatment could reduce kidney injury, dialysis, or death in sepsis patients. The trial planned to enroll many participants but was stopped early after only 19 people joined. The idea was to catch kidney…

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 early-phase study tested two chemotherapy drugs, pevonedistat and belinostat, in 18 adults with acute myeloid leukemia or myelodysplastic syndrome that returned or didn't respond to prior treatment. The main goal was to find the safest dose and understand side effects. The s…

This study tested a drug called MBG453 in 10 people with a type of bone marrow disorder called lower-risk myelodysplastic syndromes (MDS). The goal was to see if the drug could improve blood cell counts and reduce the need for transfusions. The trial was terminated early, so the …

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

This study tested a combination of two drugs, venetoclax and azacitidine, for people with a high-risk type of bone marrow cancer called myelodysplastic syndrome (MDS) that either came back or didn't respond to treatment. The goal was to find the best dose and see how well it work…

This study looked at the long-term safety of a drug called NBI-827104 in children with a rare epilepsy that causes seizures during sleep. The trial was stopped early and only enrolled 19 children, so the results are limited. The main goal was to see how many children had serious …

This early-phase study tested whether a special type of cell (interferon gamma-primed mesenchymal stromal cells) could be safely given to prevent graft-versus-host disease, a common and serious complication after bone marrow transplant. Only 4 patients with acute leukemia or myel…

This study looked at 500 adults with septic shock and severe acute kidney injury to see if starting dialysis early (within 12 hours) or waiting (48-60 hours) affected survival at 90 days. The trial was terminated early, so the results are not conclusive. The goal was to find the …

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 study tested whether a sedative drug called dexmedetomidine could help restore blood pressure control in patients with severe septic shock that does not respond to standard treatments. The trial involved 32 adults in intensive care. The goal was to see if the drug could impr…

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-stage trial tested a new drug called NC525 in people with advanced myeloid neoplasms, including relapsed or refractory acute myeloid leukemia. The main goal was to check safety and tolerability, with a secondary focus on whether the drug could shrink tumors or improve …

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 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 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 study tested whether adding sabatolimab to standard chemotherapy (azacitidine) helps people with high-risk myelodysplastic syndrome (MDS) or a related leukemia (CMML-2). The trial aimed to see if the combination improved survival. However, the study was stopped early, so we …

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 adding an experimental drug called MBG453 to standard chemotherapy for people with a serious bone marrow disorder called myelodysplastic syndrome (MDS). The trial included 127 adults with high-risk MDS who could not have a stem cell transplant. The goal was to s…

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 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 looked at the long-term safety of dexpramipexole in adults with severe eosinophilic asthma. Participants had already completed earlier phase 3 trials. The goal was to track side effects and asthma control over time. However, the study was terminated early, which may li…

This study tested a daily pill called dexpramipexole in 654 people with severe eosinophilic asthma, a type of asthma linked to high levels of certain white blood cells. The goal was to see if the drug could reduce severe asthma attacks over a year. However, the trial was terminat…

This study tested a new drug called SAR443579 in adults and children with certain blood cancers that had come back or not responded to treatment. The goal was to see if the drug was safe and could shrink or control the cancer. The trial was stopped early, so results are limited.

This Phase 3 trial tested dexpramipexole, an oral drug, in over 1,000 people with severe eosinophilic asthma that was not well controlled by standard treatments. The goal was to see if it could reduce severe asthma attacks over a year. However, the study was terminated early, so …

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 device called Efferon LPS that filters blood during heart surgery when a heart-lung machine is used. The goal was to see if it could prevent multiple organ failure, especially kidney damage. Only 14 people took part before the study was stopped, so the results…

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 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 study tested a procedure called renal denervation, which uses a catheter to calm overactive nerves in the kidneys, in people with chronic heart failure. The goal was to see if it is safe and feasible, and whether it could improve symptoms like walking distance and heart stra…

This study tested whether the drug maraviroc could reduce inflammation in the lungs of people with active sarcoidosis. Participants took the drug for 6 weeks and had two lung exams (bronchoscopy) to measure immune cells before and after treatment. Only 3 people enrolled, and the …

This study tested two oral drugs, eltanexor and Inqovi, in adults with high-risk myelodysplastic syndromes (MDS) that did not improve with standard treatment. MDS is a bone marrow disorder that can lead to leukemia. The goal was to find a safe dose and see if the combination help…

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 early-phase study tested an experimental drug called BR108 in 8 adults with advanced blood cancers (lymphoma, leukemia, or myelodysplastic syndrome) that had not responded to prior treatments. The drug was given by IV infusion twice per cycle. The main goals were to check sa…

This early-stage trial tested a new drug called DSP107 combined with standard chemotherapy drugs (azacitidine and venetoclax) in 25 people with relapsed or hard-to-treat acute myeloid leukemia, myelodysplastic syndrome, or chronic myelomonocytic leukemia. The study aimed to check…

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 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 trial tested a combination of two drugs, omacetaxine and venetoclax, in 24 adults with relapsed or refractory acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) that had a specific genetic change called RUNX1. The goal was to find the best dose and se…

This study tested a drug called vilobelimab for people with pyoderma gangrenosum, a rare condition causing painful skin ulcers. The trial aimed to see if the drug could completely close the ulcers compared to a placebo. It included 54 adults and was stopped early, so the results …

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 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 phase 2 trial tested a daily pill called etavopivat in 17 adults with very low, low, or intermediate risk MDS who had anemia. The goal was to see if the drug could improve red blood cell counts and reduce the need for transfusions. The study was terminated early, so the full…

This study tested a daily pill called INCB000928 for people with anemia caused by myelodysplastic syndromes (MDS) or multiple myeloma. The goal was to see if it was safe and could raise red blood cell levels, reducing the need for transfusions. The trial was terminated early afte…

This early-phase trial tested whether adding the drug itacitinib to standard immune-suppressing medications could help prevent graft-versus-host disease (GVHD) in people receiving stem cell transplants for blood cancers. The study enrolled 84 participants with leukemia, lymphoma,…

This phase 2 study tested adding sabatolimab, an experimental immunotherapy, to standard chemotherapy drugs (azacitidine, decitabine, or oral decitabine) in 39 adults with intermediate to very high risk myelodysplastic syndrome (MDS). The main goal was to check safety, and the tr…

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 treatment for pyoderma gangrenosum, a rare skin condition that causes painful, hard-to-heal ulcers. Researchers used a special wound covering made from donated placenta tissue (EpiFix) followed by skin grafts. The study was stopped early after only 5 people en…

This early-stage trial tested two new drug combinations in 52 adults with acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS). One group received HDM201 plus MBG453, the other HDM201 plus venetoclax. The main goal was to check safety and find the right dose. …

This early-phase study tested combinations of three antibody drugs (sabatolimab, NIS793, and canakinumab) in 33 adults with lower-risk myelodysplastic syndrome (MDS), a blood disorder. The main goal was to check safety and find the right doses. The study was terminated early, so …

This phase 2 trial tested a new drug called sabatolimab, combined with two standard chemotherapy drugs (azacitidine and venetoclax), in 20 adults with high or very high risk myelodysplastic syndromes (MDS) who could not have intensive chemotherapy or a stem cell transplant. The g…

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 aimed to see how two antipsychotic drugs, aripiprazole and ziprasidone, affect heart risk in people with schizophrenia, schizoaffective disorder, or bipolar disorder. Researchers measured blood fats and heart electrical changes in 48 adults over 12 months. The trial wa…

This early-phase trial tested a new drug called BMS-986253, alone or with standard chemotherapy, for people with myelodysplastic syndromes (MDS), a group of bone marrow disorders. The study aimed to find a safe dose and see if the drug could shrink cancer. However, the trial was …

This study tested whether setting a ventilator based on each patient's lung volume could improve outcomes for people with moderate-to-severe acute respiratory distress syndrome (ARDS). The approach used a bedside measurement to tailor the pressure applied during breathing. Howeve…

This study tested a treatment called TCB008, made from donated immune cells, for adults with acute myeloid leukemia (AML) that came back or didn't respond to standard therapy. The goal was to see if it could reduce cancer cells in the bone marrow. The trial was stopped early, but…

This study looked at a new way to prevent severe graft-versus-host disease (GVHD) in children who had a bone marrow transplant for blood cancers. The goal was to see if stopping certain immune-suppressing drugs after day 60 could lower the risk of severe GVHD. Only 3 children too…

This phase 3 study tested the drug ganaxolone as an add-on treatment for seizures in children and adults with tuberous sclerosis complex (TSC). The trial was open-label, meaning everyone received the drug, and it included 117 people who had previously been in related studies. The…

This study tested a targeted cancer drug called ponatinib combined with standard chemotherapy in children and young adults up to age 21 with Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) that had come back or stopped responding to other treatments. The g…

This study tested whether the oral drug midodrine could help people with septic shock stop needing IV blood pressure support sooner. The trial planned to enroll many patients but was stopped early with only 10 participants. Because it was so small, we cannot draw firm conclusions…

This early-stage study tested a new drug called CC-95251, alone or with other cancer drugs, in people with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS) that had come back or not responded to treatment. The main goals were to check safety and find the right dose…

This early-stage trial tested an experimental oral drug called SGR-2921 in adults with relapsed or refractory acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS). The main goals were to check safety and find the right dose. However, the study was terminated e…

This study looked at whether using a positive airway pressure (PAP) machine at night can reduce the return of atrial fibrillation (an irregular heartbeat) in people who also have obstructive sleep apnea. About 129 participants with both conditions were assigned to receive either …

This phase 2 trial tested a weekly injection of lademirsen in 43 adults with Alport syndrome, a genetic condition that damages kidneys. The goal was to see if the drug could slow the decline in kidney function. The study was terminated early, so the results are limited and it's u…

This study tested namilumab, an experimental drug, in 107 people with chronic pulmonary sarcoidosis, a condition causing lung inflammation. The trial was randomized and placebo-controlled, but it was terminated early. The goal was to see if namilumab could reduce the need for res…

This study tested an experimental drug called VX-864 in 14 people with a severe form of alpha-1 antitrypsin deficiency (PiZZ genotype). The goal was to see if the pill could raise protective protein levels in the blood and reduce harmful buildup in the liver. The trial was termin…

This study tested a new drug called VAL-083 in people with glioblastoma, an aggressive brain cancer that had returned after standard treatments. The trial aimed to see if VAL-083 could help patients live longer. However, the study was stopped early after only 2 people enrolled, s…

This Phase II study tested a radioactive tracer called 68GaNOTA-Anti-MMR-VHH2 to see if it can light up certain immune cells (macrophages) on PET scans. The trial included 29 people with head and neck cancer, lymphoma, atherosclerosis, or sarcoidosis. The goal was to see if the t…

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 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 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 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 tested whether osteopathic care (hands-on manipulation) can help reduce pain in people with fibromyalgia. 32 participants received osteopathic treatments over 8 months, with a follow-up 3 months later. The goal was to see if this approach could improve pain and quality…

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 adding pregabalin (Lyrica) to standard treatment could reduce pain and improve function in people with complex regional pain syndrome (CRPS) of the upper limb. Only 14 adults were enrolled before the trial was stopped early. The goal was to see if the dr…

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 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 tested whether Thealoz Total eye drops can reduce inflammation and improve symptoms in people with chronic dry eye disease. The drops contain hyaluronic acid for lubrication, plus trehalose and NAAGA to fight inflammation. The trial planned to enroll 30 adults with mod…

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 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 tested whether Acthar gel could improve symptoms and lung function in people with sarcoidosis, a disease that causes inflammation in the lungs. Researchers also checked if a blood marker called CXCL9 could predict how well the treatment works. Only 2 people joined befo…

This study compared a new prototype nasal mask (Whitsundays) to a standard mask (AirFit N30i) in 30 adults with obstructive sleep apnea. Participants used each mask for 7 nights at home. The goal was to see which mask sealed better, was more comfortable, and easier to use. The st…

This study aimed to see if a personalized physical activity program, with one face-to-face session followed by phone coaching, could improve quality of life for people with fibromyalgia compared to standard group exercise classes. The trial was terminated after enrolling only one…

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 compared two different eye drops (Systane PRO and Refresh Optive Mega-3) to see which one works better for people with dry eye. Twenty adults with moderate dry eye symptoms were enrolled, but the study was stopped early. Participants filled out questionnaires about the…

This early-stage study tested the safety of an experimental drug called Allocetra, injected directly into the joint, for people with psoriatic arthritis. Only 5 participants were enrolled. The main goal was to check for side effects, with some initial data on pain relief. The stu…

This study looked at whether a combined treatment plan—including physical therapy, behavioral health therapy, and medical procedures—could reduce symptoms of chronic bladder pain. Researchers compared this bundled approach to usual care in 26 women with interstitial cystitis or b…

This study looked at whether a preservative-free eye gel called IRIDIUM® could help relieve dry eye symptoms in people with glaucoma or high eye pressure who were already using multiple eye drop medications. The goal was to see if the gel improved comfort and quality of life. The…

This study tested whether a gas sedative (isoflurane) could help patients with septic shock wake up and get off a breathing machine faster than standard IV sedation (midazolam). The goal was to reduce complications from long-term sedation and ventilation. Only one patient was enr…

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 looked at whether desipramine, a medication that affects brain regions involved in pain processing, could help people with irritable bowel syndrome (IBS). Researchers planned to use brain scans to see how the drug changes pain signals in the brain. Only 18 people were …

This study looked at whether a treatment called acupuncture-like Transcutaneous Electrical Nerve Stimulation (TENS) could improve quality of life and bowel habits in people with irritable bowel syndrome (IBS). The study planned to enroll 30 adults with IBS, but was terminated ear…

This Phase 2 trial tested whether CIN-103, taken twice daily, could ease symptoms of irritable bowel syndrome with diarrhea (IBS-D) in adults. The study planned to enroll 421 participants and compare two doses of CIN-103 against a placebo over 12 weeks. However, the trial was ter…

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

This study tested whether giving methadone to ICU patients on breathing machines could help them get off the ventilator faster. Patients who needed strong painkillers were given either methadone or a placebo. The trial was stopped early and only included 9 people, so no firm conc…

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 compared the TearCare system, a device that applies heat to the eyelids, to standard warm compresses and lid massage for people with dry eye disease. The goal was to see if TearCare is safe and better at improving tear stability and reducing eye discomfort. The study p…

This early-phase trial tested a new eye drop called rhPRG4 in 8 people with Sjögren's syndrome-related dry eye. Participants received either the active drop or a placebo, then switched after 90 minutes. The study measured eye dryness and safety over a single day. Because the tria…

This study aimed to see if certain eye tests could predict which people with dry eye disease would get relief from over-the-counter artificial tears. Researchers planned to enroll 66 adults with dry eye, but the study was terminated early with only 36 participants. Participants u…

This study tested whether pramipexole (Mirapex), a drug for restless legs syndrome, could also reduce symptoms of opioid withdrawal in people with opioid use disorder. The trial planned to enroll 75 adults but was terminated early. Researchers measured changes in restless legs se…

This study tested a monthly solution called Tangible Boost that is meant to restore a special coating on contact lenses. The goal was to see if it could improve comfort and vision in people with severe dry eye from Stevens-Johnson syndrome, Sjogren's syndrome, or graft-versus-hos…

This study tested a single dose of danavorexton in 41 people with moderate to severe sleep apnea who were having abdominal surgery. The goal was to see if the drug could help them breathe steadily in the recovery room. The trial was terminated early, so results are incomplete.

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 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 looked at how diuretics (water pills) affect kidney and heart function in people who just had heart surgery. The goal was to see if removing extra fluid changes how well the kidneys and right side of the heart work. Only 9 people took part, and the study was stopped ea…

This trial tested a new way of using a breathing machine (flow-controlled ventilation) against the standard method (pressure-controlled ventilation) in 15 ICU patients with moderate to severe acute respiratory distress syndrome (ARDS). The goal was to see if the new method uses l…

This study aimed to find genetic mutations in people with idiopathic cytopenia of undetermined significance (ICUS), a condition of unexplained low blood cell counts. Researchers wanted to see if these mutations are similar to those found in myelodysplastic syndrome (MDS), a type …

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 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 compared two breathing techniques (CPAP and eSigh) in 18 patients with brain injury and acute respiratory distress syndrome (ARDS). The goal was to see which method better maintains oxygen levels in the brain without causing harm. The trial was terminated early, so fin…

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 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 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 aimed to see if a tiny camera on a thin tube (bronchoscopy) could help doctors diagnose diffuse lung diseases without needing a surgical lung biopsy. The camera takes live microscopic images of the air sacs. The study was stopped early, but it involved 38 adults with c…

This study tested whether raising the blood pressure target from 65 mmHg to 85 mmHg in patients with septic shock could help prevent kidney injury. Researchers used a drug called norepinephrine to adjust blood pressure and monitored kidney function. The trial was stopped early an…

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 very early study aimed to compare an oral (pill) version of the chemotherapy drug cladribine with the standard IV (vein) version in people with certain leukemias. Only 3 people took part before the study was stopped early. The main goal was to see how the body handles the or…

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 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 observational study aimed to understand why some septic shock patients respond better to the drug vasopressin. Researchers planned to compare blood markers, heart ultrasound images, and tissue oxygen levels between responders and non-responders. However, the study was termin…

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 looked at how mechanical ventilation (breathing machines) affects the energy centers (mitochondria) of immune cells in patients with severe lung infections and acute respiratory distress syndrome (ARDS). Researchers aimed to understand why some patients get worse despi…

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

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

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

This 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 find early signs of heart and blood vessel damage in people with obstructive sleep apnea, before serious symptoms appear. Researchers planned to follow 133 patients and check if CPAP treatment could reverse these early changes. The study was terminated early, …

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 aimed to compare two ways of setting breathing machine pressure (PEEP) in patients with severe lung injury (ARDS). The goal was to see which method better protects the lungs and heart. Only 2 patients were enrolled before the study was stopped, so no meaningful results…

This study involved 65 adults with obstructive sleep apnea who had been using CPAP therapy for at least 6 months. Participants stopped using CPAP for 2 weeks so researchers could measure changes in blood vessel function and nerve control of the heart. The goal was to see if a spe…

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 how certain immune cells, called non-conventional T cells, behave in patients with severe pneumonia and acute respiratory distress syndrome (ARDS). Researchers collected blood and airway samples from 258 intensive care unit patients to track these cells over …

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 find out how many people with Sjögren's dry eye have reduced feeling in their cornea (the clear front part of the eye). Researchers measured corneal sensitivity, tear production, and pain in 103 adults with confirmed Sjögren's syndrome and dry eye. The study w…

This study tested a special band called AirGo to monitor breathing patterns in hospitalized COVID-19 patients with respiratory failure. The goal was to see if the band could detect early signs of worsening breathing, helping doctors decide when to use a breathing tube. The study …

This study looked at whether proteins that control our body's daily rhythms (clock proteins) can predict how severe asthma will get. Researchers monitored these proteins in the blood and sputum of 50 adults with severe eosinophilic asthma before and during treatment with mepolizu…