Clinical trialCLINICALTRIALSToday
Researchers at Memorial Sloan Kettering Cancer Center are testing a smartphone app designed to help breast cancer and rare cancer survivors deal with the fear that their cancer might come back. The app uses a technique called Attention and Interpretation Modification (AIM) to help people change how they think about cancer recurrence. The study has enrolled 252 people and is no longer recruiting new participants, but the results could help many cancer survivors in the future.
WHY IT MATTERSThis trial addresses a common but often overlooked mental health challenge for cancer survivors—fear of recurrence—by testing a personalized, accessible mobile app intervention that could be widely available if proven effective.
Clinical trialRSS2 days ago
A new drug called masitinib helped ALS patients live longer than expected. In a clinical trial, over 40% of patients treated with masitinib survived for at least 5 years from when their disease started. This is much better than the typical outcome, where fewer than 25% of ALS patients usually live that long.
WHY IT MATTERSIf masitinib gains approval, ALS patients may have access to a treatment that could significantly extend their survival time compared to current standard care.
Clinical trialRSS3 days ago
A new medication called Vyalev helped people with Parkinson's disease stop experiencing unwanted, uncontrolled movements (called dyskinesia) when they switched to it from other Parkinson's drugs. In two clinical trials, most people saw these troublesome movements go away within a few months of starting Vyalev. This is good news because dyskinesia is a common side effect that happens after taking Parkinson's medications for a long time.
WHY IT MATTERSIf you have Parkinson's disease and experience dyskinesia from your current levodopa treatment, switching to Vyalev could eliminate these involuntary movements within months rather than managing them as a permanent side effect.
Clinical trialRSS3 days ago
A new clinical trial has started testing a drug called IKT-001 in people with pulmonary arterial hypertension (PAH), a rare condition where blood vessels in the lungs become narrowed and make it hard for the heart to pump blood. This is a large Phase 3 trial, which is a late-stage test that happens before a drug might be approved. Nearly 500 adults will participate to see if this drug works better than current treatments.
WHY IT MATTERSIf you have PAH and are already on stable treatment, you may be eligible to join the IMPROVE-PAH trial and potentially access IKT-001 before it becomes widely available.
Clinical trialRSS3 days ago
The FDA has approved a clinical trial for a new cell therapy called remestemcel-L-rknd made by Mesoblast. The therapy will be tested in children ages 5-9 who have Duchenne muscular dystrophy (DMD), a serious muscle disease. About 76 children will participate in the trial and receive either the therapy or a placebo while continuing their regular DMD treatments.
WHY IT MATTERSThis trial represents a new treatment approach for DMD in young children who are still in a critical window for muscle development, offering families a potential alternative or complement to existing therapies.
Clinical trialRSS3 days ago
AB Science is preparing to test an experimental drug called masitinib for ALS (a disease that affects nerve cells that control muscles). To protect their investment in this large clinical trial, the company bought special insurance that would reimburse them if the trial doesn't go as planned or costs more than expected.
WHY IT MATTERSThis insurance arrangement signals that AB Science is confident enough in masitinib to move forward with Phase 3 testing in ALS patients, which is a critical step toward potentially bringing a new treatment option to people living with this progressive disease.
Clinical trialCLINICALTRIALSApr 17
Researchers are testing a new gene therapy called BBP-812 to treat Canavan disease, a rare brain disorder that affects children. The therapy uses a modified virus to deliver a healthy copy of a gene that's missing or broken in people with this disease. This early-stage trial will check if the treatment is safe and whether it helps patients.
WHY IT MATTERSThis trial is now actively recruiting children with Canavan disease — if your child has been diagnosed, you may be eligible to participate in one of the first human tests of this gene therapy approach.
Clinical trialCLINICALTRIALSApr 8
Researchers completed a small study testing whether a drug called Adalimumab (brand name Humira) could help people with Netherton syndrome, a rare skin condition that causes severe itching, rashes, and inflammation. Eleven patients received the drug for 3 months and were then followed for another 3 months to see if it was safe and if it improved their symptoms, including pain and itching.
WHY IT MATTERSThis completed trial provides evidence about whether Humira—a drug already used for other inflammatory conditions—could be repurposed to treat Netherton syndrome, which currently has very limited treatment options.
Clinical trialUNITERAREApr 4
Researchers are testing a new drug called JL15003 to see if it can safely treat glioblastoma, a serious brain cancer that has come back after previous treatment. This is an early-stage study that will check both how safe the drug is and whether it helps patients live longer or feel better. The study is now accepting patients and will run starting in April 2026.
WHY IT MATTERSThis trial offers a potential new treatment option for patients with recurrent glioblastoma, a condition with very limited treatment choices after initial therapy fails.
Clinical trialUNITERAREApr 3
Researchers are testing a new combination of three drugs (RBS2418, tremelimumab, and durvalumab) to treat advanced hepatocellular carcinoma, a type of liver cancer that cannot be surgically removed. This is an early-stage clinical trial (Phase 2) that is currently accepting patients. The study aims to see if combining these drugs works better than existing treatments.
WHY IT MATTERSThis trial offers eligible patients with advanced unresectable hepatocellular carcinoma access to a novel three-drug combination that may provide a new treatment option beyond current standard therapies.
Clinical trialUNITERAREApr 3
Triple Hair Inc is recruiting patients for a Phase 3 clinical trial testing a new treatment called TH07 for androgenic alopecia, commonly known as male or female pattern baldness. This is a large-scale study to confirm whether TH07 works better than current treatments. If you have pattern hair loss, you may be eligible to join and help test this potential new therapy.
WHY IT MATTERSThis Phase 3 trial is actively recruiting now, meaning if you have androgenic alopecia, you could potentially enroll and access an investigational treatment while contributing to evidence that could lead to FDA approval.
Clinical trialUNITERAREApr 3
This is a clinical trial testing a blood clotting medicine called Nuwiq in women and girls with hemophilia A who need surgery. Hemophilia A is a rare bleeding disorder where the body doesn't make enough of a protein that helps blood clot. The trial will study how well Nuwiq works and what dose is best for these patients during surgical procedures.
WHY IT MATTERSThis trial is specifically designed for women and girls with hemophilia A undergoing surgery — a population that often receives less research attention than males with the same condition.
Clinical trialCLINICALTRIALSApr 2
Researchers in France are studying a new tool called MEKANOS that measures how strong bones are and predicts fracture risk in people with fibrous dysplasia, a rare bone disease. This clinical trial involves 20 patients and aims to solve a major problem doctors face: they currently have no good way to know which patients are at highest risk of bone breaks. The study could help doctors make better treatment decisions for this condition.
WHY IT MATTERSThis trial addresses a critical gap in fibrous dysplasia care—doctors currently lack reliable tools to predict which patients will experience fractures, making this the first systematic assessment of fracture risk in this population.
Clinical trialCLINICALTRIALSMar 27
Researchers are testing a new combination treatment for metastatic Merkel cell carcinoma, a rare type of skin cancer. The study combines a drug called avelumab with either a radioactive therapy or radiation treatment. The trial is currently enrolling patients and aims to see if this combination is safe and effective at fighting the cancer.
WHY IT MATTERSThis trial offers patients with metastatic Merkel cell carcinoma access to a novel combination therapy that may provide additional treatment options beyond standard care, though enrollment is currently closed.
Clinical trialCLINICALTRIALSMar 27
Researchers are testing a cancer drug called trastuzumab deruxtecan (T-DXd) in patients with several types of rare and uncommon cancers that have a specific protein marker called HER2. The study includes seven different cancer types in the first part, including bladder, bile duct, cervical, uterine, ovarian, and pancreatic cancers. This drug has already shown promise in breast and stomach cancers, and doctors want to see if it works in these other cancer types too.
WHY IT MATTERSThis trial offers patients with HER2-positive rare cancers like biliary tract, cervical, endometrial, ovarian, and pancreatic cancer access to a targeted therapy that has demonstrated effectiveness in other cancer types—potentially providing a new treatment option where few exist.