Clinical trialUNITERAREApr 3
Researchers at the University of Wisconsin are testing a new combination of two cancer treatments called Hepzato Kit and Opdualag for patients with melanoma (a type of skin cancer) that has spread to the liver. This is an early-stage study that started in 2026 and is currently looking for patients to participate. The goal is to see if this combination can help people whose cancer has spread to multiple parts of their body.
WHY IT MATTERSThis trial offers patients with metastatic melanoma and liver involvement access to a novel combination therapy at a major academic medical center, potentially providing treatment options for a particularly aggressive cancer presentation.
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 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 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 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 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
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 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 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.
ResearchCLINICALTRIALSMar 30
This study is looking for 2,800 people to help researchers understand how being a caregiver affects a person's health and stress levels over time. The study includes caregivers of people with rare diseases like Batten disease, Tay-Sachs disease, and other inherited metabolic disorders, as well as caregivers of people with undiagnosed diseases. By learning more about caregiver stress, researchers hope to find ways to better support the millions of unpaid family caregivers in the U.S.
WHY IT MATTERSIf you or a family member care for someone with a rare inherited metabolic disorder, Batten disease, Tay-Sachs, or an undiagnosed disease, this study directly addresses the health challenges caregivers face and could lead to better support resources.
ResearchCLINICALTRIALSMar 27
Researchers are looking for 63 participants to help them understand how DNA changes in babies before birth compare to DNA changes after birth. They want to collect DNA from amniotic fluid (the fluid around a baby in the womb) or from tissue samples after delivery to create a reference guide. This guide will help doctors better diagnose rare genetic diseases that start before birth.
WHY IT MATTERSThis trial could improve how doctors diagnose rare genetic diseases in fetuses and newborns by establishing what normal DNA patterns look like before birth, which is currently unknown.
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.