Clinical trialUNITERAREApr 3
Researchers are looking for patients with a type of blood cancer called acute lymphoblastic leukemia (ALL) to test a new treatment combination. The study will test whether adding a drug called JZP458 to standard chemotherapy works better than chemotherapy alone. This trial is just starting and will recruit patients beginning in April 2026.
WHY IT MATTERSThis Phase 2 trial is now actively recruiting newly diagnosed ALL patients without the Philadelphia chromosome, offering access to an investigational asparaginase formulation (JZP458) that may have improved tolerability compared to standard asparaginase.
Clinical trialUNITERAREApr 3
Researchers are testing a new combination treatment for advanced kidney cancer that has spread to other parts of the body. The treatment combines two immunotherapy drugs (nivolumab and ipilimumab) with an experimental live biotherapeutic product called EXL01. This Phase 1 trial is now accepting patients to see if adding EXL01 to the standard immunotherapy improves outcomes.
WHY IT MATTERSThis trial offers metastatic renal cell carcinoma patients access to a novel combination approach that may enhance the effectiveness of existing immunotherapies by incorporating a live biotherapeutic product.
Clinical trialUNITERAREApr 3
Researchers at Emory University are looking for pregnant women with sickle cell disease to join a study about blood transfusions. The study will test whether giving blood transfusions during pregnancy can help prevent serious complications for both the mother and baby. This is a Phase 4 trial, which means the treatment has already been tested before and researchers want to learn more about how well it works.
WHY IT MATTERSThis trial is now actively recruiting pregnant women with sickle cell disease — participation could help prevent pregnancy complications like stroke, organ damage, and miscarriage that occur at higher rates in this population.
Clinical trialUNITERAREApr 3
Researchers are testing a new drug called elritercept to see if it works better than a standard treatment (epoetin alfa) for anemia in adults with myelodysplastic syndromes (MDS)—a blood disorder where the bone marrow doesn't make enough healthy blood cells. People in this study have low, very low, or intermediate risk MDS and need regular blood transfusions. The trial is now accepting patients.
WHY IT MATTERSThis Phase 3 trial is actively recruiting adults with MDS who require transfusions, offering access to an investigational treatment that may reduce their dependence on blood transfusions.
Clinical trialUNITERAREApr 3
Researchers are testing a medicine called IGIV 10% to see if it helps people with CIDP, a rare nerve disease that causes weakness and numbness, usually in the legs and feet. IGIV is made from antibodies collected from donated blood. This is a Phase 3 trial, which means the medicine has already been tested in earlier studies and now doctors want to confirm it works well and is safe.
WHY IT MATTERSThis Phase 3 trial is now actively recruiting patients with CIDP — if you have confirmed CIDP diagnosis, you may be eligible to access a potential new treatment option before it becomes widely available.
Clinical trialUNITERAREApr 3
Researchers are testing a new type of cell therapy made from umbilical cord blood to treat T-cell lymphoma that has stopped responding to other treatments. The therapy uses special immune cells called NK cells that are engineered to recognize and attack cancer cells. This is an early-stage study (Phase 1) that is now accepting patients.
WHY IT MATTERSThis trial offers a potential new treatment option for patients with relapsed or refractory T-cell lymphoma who have limited alternatives after standard therapies fail, with enrollment beginning in April 2026.
Clinical trialUNITERAREApr 3
Researchers are testing a new medicine called glycerol tributyrate to see if it can help people with two rare mitochondrial diseases: MELAS (a condition that causes brain inflammation, acid buildup in the blood, and stroke-like episodes) and LHON-Plus (a condition that damages the optic nerve and causes vision loss). This is an early-stage study that will enroll patients starting in April 2026.
WHY IT MATTERSThis is the first clinical trial testing glycerol tributyrate specifically in MELAS and LHON-Plus patients, offering a potential new treatment option for these currently untreatable mitochondrial disorders.
Clinical trialUNITERAREApr 3
Researchers are looking for patients who need a procedure called a transjugular intrahepatic portosystemic shunt (TIPS) to help study how blood clotting works in patients with liver disease. This trial will examine blood clotting patterns before and after the procedure to better understand how TIPS affects the body's ability to form and break down blood clots. The study is recruiting patients starting in April 2026 and is being run by hospitals in Paris.
WHY IT MATTERSThis trial is now recruiting patients undergoing TIPS placement — if you have advanced liver disease with portal hypertension and are scheduled for this procedure, you may be eligible to contribute to research that could improve how doctors manage bleeding risks in liver disease patients.
Clinical trialUNITERAREApr 3
Researchers at King's College London are testing whether probiotics (helpful bacteria) can reduce skin inflammation that some people experience during perimenopause (the years leading up to menopause). This is an early-stage study to see if this approach might work before larger trials are done. The study is now accepting participants.
WHY IT MATTERSIf probiotics prove effective for perimenopause-related skin inflammation, this could offer a non-hormonal treatment option for people experiencing this symptom during the menopausal transition.
Clinical trialUNITERAREApr 3
Researchers at New York State Institute for Basic Research are testing a new treatment called transcranial direct current stimulation (tDCS) in children with autism. This treatment uses mild electrical currents applied to the scalp to potentially help with autism symptoms. The study is now accepting children to participate and will begin in April 2026.
WHY IT MATTERSThis trial offers families with autistic children a chance to explore a non-medication brain stimulation approach that may help with core autism symptoms, with enrollment actively opening at a major research institution.
Clinical trialUNITERAREApr 3
Researchers are looking for people with diabetes to test a new way to treat bad breath caused by gum disease. The treatment combines regular gum cleaning with a special light therapy that kills bacteria in the mouth. This study will help doctors understand if this combination works better than standard gum treatment alone.
WHY IT MATTERSIf you have diabetes and struggle with bad breath despite good oral hygiene, this trial offers access to an innovative combination treatment that may address both the gum disease and halitosis that often accompany diabetes.
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 trialCLINICALTRIALSApr 1
Researchers are looking for 88 people with Fragile X Syndrome or Creatine Transporter Deficiency to test a new imaging technology that can see how the brain works. Both of these are genetic conditions that affect how the brain develops and cause intellectual disability. This study might help doctors better understand and diagnose these conditions in the future.
WHY IT MATTERSThis trial is recruiting patients with two of the most common X-linked intellectual disability disorders and may provide a new diagnostic tool that could improve how these conditions are identified and monitored.
Clinical trialCLINICALTRIALSApr 1
Researchers are testing a new technology called SmartMatch that helps doctors find the best cancer medicines for patients with rare brain and spinal cord tumors. The technology works by taking a small piece of tumor removed during surgery and testing it against different drugs to see which ones work best. The study wants to see if SmartMatch can complete this analysis and give doctors a report within 3 weeks of surgery. This trial is looking for 80 patients with various types of brain tumors.
WHY IT MATTERSThis trial offers patients with recurrent or advanced brain tumors access to personalized drug testing that could identify the most effective treatment for their specific tumor type within weeks of surgery.
Clinical trialCLINICALTRIALSApr 1
Researchers are looking for 3,100 people to join a study about why patients don't take their medications as prescribed. The study will test a new approach to help people stick to their treatment plans, which could improve their health and reduce unnecessary hospital visits. About half of all patients don't take their medicines the right way, and this problem gets worse when treatment also requires lifestyle changes.
WHY IT MATTERSThis trial directly addresses medication non-adherence across multiple rare disease categories, meaning patients with rare conditions could benefit from proven strategies to better manage their treatments and health outcomes.
Clinical trialCLINICALTRIALSApr 1
Researchers are testing a combination of two cancer drugs called dabrafenib and trametinib in patients with rare tumors that have a specific genetic change called BRAF V600E. This is a phase IV study, which means the drugs have already been approved and doctors are gathering more information about how well they work. The study will include children (age 1 and up) and adults, and is currently looking for 40 patients to participate.
WHY IT MATTERSIf you have a rare solid tumor with a BRAF V600E mutation and your doctor has already recommended dabrafenib plus trametinib treatment, this trial offers an opportunity to participate in research that could help future patients while receiving the same treatment your doctor planned for you anyway.
Clinical trialCLINICALTRIALSMar 31
Researchers are building a large international database to collect information and tissue samples from children, teens, and young adults with rare brain tumors. By gathering this data from 5,800 patients, scientists hope to better understand these uncommon cancers and develop better treatments. The study is currently looking for patients to participate.
WHY IT MATTERSThis registry directly addresses rare brain tumors like astroblastoma and BCOR ITD sarcoma that have limited research data—participating patients contribute to the only large-scale international effort to understand these specific tumor types.
Clinical trialCLINICALTRIALSMar 27
Researchers in France are recruiting 5,000 newborns to test a new way of screening for rare diseases using genome sequencing—a complete reading of a baby's DNA. Instead of the current blood spot tests that check for only a few dozen conditions, this study will see if reading a baby's entire genome can safely and effectively find many more rare genetic diseases at birth. This is one of the first major studies in Europe to test this approach.
WHY IT MATTERSThis trial could expand newborn screening in France to detect dozens of additional rare genetic diseases at birth, potentially allowing earlier treatment and better health outcomes for babies with conditions that currently go undiagnosed until symptoms appear.
Clinical trialCLINICALTRIALSMar 27
Researchers are looking for 1,000 people of any age who have a rare blood disorder called FPDMM, which is caused by a change in the RUNX1 gene. People with this condition may bleed easily and for longer than normal when injured. This study will help doctors better understand the disease, diagnose it more accurately, and find better ways to treat it.
WHY IT MATTERSThis is an active recruiting trial sponsored by the National Human Genome Research Institute seeking 1,000 participants with RUNX1-variant FPDMM — participation could directly advance understanding of diagnosis and treatment for this rare inherited bleeding disorder.
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.