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 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 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
This is a continuation study for patients who are already taking the cancer drugs dabrafenib and/or trametinib and have done well on them. If your doctor thinks you're still benefiting from these medications after your original trial ends, you may be able to keep taking them through this new study. The study is looking for about 100 patients with various types of cancer including melanoma, lung cancer, and brain tumors.
WHY IT MATTERSThis rollover study allows patients whose cancers are responding well to dabrafenib and/or trametinib to continue access to these drugs after their original trial ends, rather than losing treatment.
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 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 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 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 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 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 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 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.
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 26
Researchers are testing a combination of two immunotherapy drugs in patients with four types of rare cancers: neuroendocrine tumors, biliary tract cancers, ovarian clear cell carcinoma, and tumors with high microsatellite instability. This Phase 2 trial involves 240 patients and is based on earlier research showing these cancers may respond well to this type of treatment.
WHY IT MATTERSPatients with these rare cancers now have access to a targeted immunotherapy combination that showed promise in earlier studies, potentially offering a new treatment option for cancers that historically have limited therapeutic choices.
Clinical trialCLINICALTRIALSMar 26
Researchers are testing a new cancer treatment called CART19 for children and young adults with specific types of blood cancer (B-ALL). The treatment uses the patient's own immune cells that are modified in a lab to fight cancer cells. This trial is looking for patients with rare, high-risk forms of this cancer, including some cases that have come back after previous treatment.
WHY IT MATTERSThis trial is actively recruiting children and young adults with hard-to-treat B-ALL subtypes (hypodiploid, t(17;19), KMT2A, and CNS relapse cases) — these patients have limited treatment options and this study offers access to an advanced cell therapy approach.
Clinical trialCLINICALTRIALSMar 26
Researchers are looking for pregnant women and breastfeeding mothers who have taken or are taking a medication called odevixibat to join a safety study. The study will track the health of these women and their babies to make sure the medication is safe to use during pregnancy and while breastfeeding. About 20 people will participate, and doctors will collect information from the mothers, babies, and healthcare providers involved in their care.
WHY IT MATTERSThis surveillance program is recruiting pregnant and lactating women exposed to odevixibat to establish safety data for a medication used in rare bile acid disorders, filling a critical gap in pregnancy and lactation safety information.
Clinical trialCLINICALTRIALSMar 26
Researchers are looking for 1,500 people with inherited eye diseases caused by rare genetic changes to join a study. The study has two parts: first, they'll collect genetic information and eye health data from participants, and second, they'll follow some participants over time to understand how these eye diseases progress. This international study is now accepting new participants.
WHY IT MATTERSIf you have an inherited retinal disease with a rare genetic variant, enrolling in this registry could help researchers understand your condition better and speed up development of future treatments while contributing to a global database of genetic information.
Clinical trialCLINICALTRIALSMar 26
Researchers at Emory University are testing a new combination of medicines for children whose cancer has come back or stopped responding to previous treatments. The study combines three drugs: sirolimus (which slows cell growth), celecoxib (an anti-inflammatory), and two chemotherapy drugs given in low doses. The goal is to see if this combination works better than standard treatments for solid tumors and brain cancers in children.
WHY IT MATTERSThis trial offers a potential new treatment option for children with recurrent or refractory cancers who have limited alternatives, though enrollment is currently closed.
Clinical trialCLINICALTRIALSMar 26
Researchers are testing a new treatment for calciphylaxis, a serious condition where calcium builds up in blood vessels and skin tissue, causing painful sores. The treatment uses special cells from amniotic fluid (the fluid around a baby during pregnancy) to see if they can help heal the damage. This is an early-stage trial with a small group of 9 patients to check if the treatment is safe and works.
WHY IT MATTERSCalciphylaxis has very few treatment options and high mortality rates — this trial offers patients with chronic kidney disease a chance to access an experimental stem cell therapy that could reduce tissue damage and improve survival.