Clinical trialCLINICALTRIALSApr 9
Researchers at Columbia University are looking for 106 children and young adults with Duchenne Muscular Dystrophy (DMD) and Spinal Muscular Atrophy Type 3 (SMA Type 3) to join a study. The study will use special sensors and technology to track how people walk and move in their daily lives over a long period of time. The goal is to find new ways to measure how these diseases are progressing so doctors can better understand and treat them.
WHY IT MATTERSThis trial is now actively recruiting participants with DMD and SMA Type 3, offering a chance to contribute to developing better measurement tools that could help doctors track disease progression and evaluate future treatments more accurately.
Clinical trialUNITERAREApr 9
Researchers are testing a new medicine called difelikefalin to see if it's safe and works well for teenagers on kidney dialysis who have severe itching. Itching is a common problem for dialysis patients that can really affect their quality of life. This study will check whether the medicine helps reduce itching and what side effects it might cause.
WHY IT MATTERSThis is the first trial testing difelikefalin specifically in adolescents on hemodialysis with moderate-to-severe pruritus — a condition that significantly impacts quality of life but has limited treatment options for younger patients.
Clinical trialCLINICALTRIALSApr 6
Researchers are testing a new drug called zipalertinib to see if it helps people with early-stage lung cancer that has specific genetic mutations. Patients who had surgery to remove their tumors will receive either the new drug or a placebo (fake medicine) along with standard chemotherapy. The study wants to find out if zipalertinib can prevent the cancer from coming back better than chemotherapy alone.
WHY IT MATTERSIf you have early-stage NSCLC with uncommon EGFR mutations (like exon 20 insertions) and recently had surgery, this trial offers access to a targeted therapy specifically designed for your mutation type before it becomes widely available.
Clinical trialUNITERAREApr 5
Researchers are looking for people with Down syndrome (Trisomy 21) to join a study about how their immune system works differently and ages over time. Some participants will have had their thymus gland removed, while others won't have, so scientists can compare how this affects immune health. The study will help doctors understand why people with Down syndrome often get more infections and have other immune-related health problems.
WHY IT MATTERSThis trial is now recruiting people with Down syndrome to help researchers understand immune system problems that affect this population — findings could lead to better treatments for infections and other immune conditions common in Trisomy 21.
Clinical trialUNITERAREApr 5
Researchers are testing whether children and young adults can use robotic leg braces (called exoskeletons) in their daily lives to help them walk and move around. An exoskeleton is a wearable machine that supports your legs and helps you move if you have difficulty walking. This study will see if these devices can become common tools that help young people with mobility challenges do everyday activities.
WHY IT MATTERSThis trial is recruiting children and young adults with lower limb mobility challenges — if you or your child has difficulty walking due to a neurological or muscular condition, this could offer access to cutting-edge assistive technology at no cost through a major research institution.
ResearchUNITERAREApr 5
Researchers at the National Human Genome Research Institute are starting a study to understand rare genetic diseases by studying people from countries outside the United States. The study will look at how genetic changes cause unusual disease patterns in different populations. This research could help doctors better understand and diagnose rare diseases worldwide.
WHY IT MATTERSThis trial expands rare disease research beyond US populations, which means genetic discoveries could help patients globally who have been underrepresented in previous studies.
Clinical trialUNITERAREApr 5
Researchers are looking for patients with a rare cancer called adrenocortical carcinoma that has come back after surgery. This study will test whether giving radiation therapy before surgery helps remove the cancer more effectively. The trial is just starting and will enroll patients beginning in April 2026.
WHY IT MATTERSThis is the first Phase 1 trial testing preoperative radiation for recurrent adrenocortical carcinoma, offering eligible patients access to a potentially new treatment approach before it becomes widely available.
Clinical trialUNITERAREApr 5
Researchers are testing a new drug called ManNAc to treat primary focal segmental glomerulosclerosis (FSGS), a rare kidney disease that causes scarring and can lead to kidney failure. This is a Phase 2 trial, meaning the drug has already been tested for safety in a small group and now researchers want to see if it actually works to help patients. The trial is now accepting patients and is expected to start in April 2026.
WHY IT MATTERSThis trial offers patients with primary FSGS access to an investigational treatment that may slow or stop kidney damage, with enrollment now open at sites participating in the National Human Genome Research Institute study.
Clinical trialUNITERAREApr 5
Researchers are testing a new drug called CYT107 to help people with HIV who have Kaposi Sarcoma (a type of cancer) and whose immune systems aren't responding well to treatment. CYT107 is designed to boost the immune system to help fight the cancer. This is a Phase 2 trial, meaning it's testing whether the drug works and is safe in a larger group of people. The trial is now accepting patients and will start in April 2026.
WHY IT MATTERSThis trial offers a potential new treatment option for people with HIV-related Kaposi Sarcoma who haven't responded to standard immune-boosting treatments, addressing a significant gap in care for this vulnerable population.
Clinical trialUNITERAREApr 5
Researchers are testing a drug called baricitinib to see if it is safe and works well for patients with Job syndrome who also have lupus-like disease and/or atopic dermatitis (severe itchy skin). Job syndrome is a rare immune system disorder that makes it hard for the body to fight infections and causes skin problems. This is an early-stage study that will check for side effects and how well the drug works.
WHY IT MATTERSThis Phase 1 trial is now actively recruiting patients with Job syndrome complicated by lupus-like disease and/or atopic dermatitis — a rare combination that has limited treatment options and is being studied by the National Institute of Allergy and Infectious Diseases.
Clinical trialUNITERAREApr 5
Researchers are testing a new drug called pacritinib in people ages 12 and older who have myelodysplastic syndromes or myelodysplastic/myeloproliferative neoplasms — rare blood disorders where the bone marrow doesn't make enough healthy blood cells. The drug works by blocking several proteins that may be causing these diseases. This is an early-stage study sponsored by the National Cancer Institute that will start recruiting patients in April 2026.
WHY IT MATTERSThis trial offers patients with myelodysplastic syndromes access to a novel multi-targeted kinase inhibitor that may address disease mechanisms not covered by current standard treatments, with enrollment beginning in April 2026.
Clinical trialUNITERAREApr 5
Researchers are testing a new cancer treatment that takes a patient's own immune cells, modifies them in a lab to recognize and attack their specific cancer, and then puts them back into the body. This Phase 1 trial is for people with blood cancers like leukemia and lymphoma. The treatment is personalized—each patient's cells are customized based on their individual cancer's unique mutations.
WHY IT MATTERSThis trial offers patients with hematologic malignancies access to a cutting-edge personalized immunotherapy that targets their cancer's unique mutations, potentially offering a new treatment option for those who may have limited alternatives.
Clinical trialUNITERAREApr 5
Researchers are testing a new tool called the Esophageal String Test to help diagnose eosinophilic esophagitis, a condition where too many white blood cells build up in the food pipe and cause trouble swallowing. The test uses a small string that you swallow to collect samples from your esophagus. This study is enrolling people in Mali and the United States who have difficulty swallowing.
WHY IT MATTERSThis trial offers a potentially simpler, non-invasive screening method for eosinophilic esophagitis that could reduce the need for traditional endoscopy procedures, particularly for underserved populations in Africa and the US.
Clinical trialUNITERAREApr 4
Researchers at Dartmouth-Hitchcock Medical Center are testing a therapy called TF-CBT (Trauma-Focused Cognitive Behavioral Therapy) to see if it works well for autistic young people. This is a pilot study, which means it's a small test to see if the approach is worth studying more carefully. The study is now accepting participants and is expected to start in April 2026.
WHY IT MATTERSThis trial addresses a significant gap in mental health treatment for autistic youth, as trauma-focused therapies are often adapted without testing whether they work safely and effectively for autistic individuals.
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 at the University of Alabama at Birmingham are looking for premature babies to join a study about pulmonary hypertension—a condition where blood pressure in the lungs gets too high. The study will test different oxygen level targets to see which approach helps prevent or reduce lung problems in preterm infants. This trial is now actively recruiting participants and is expected to start in April 2026.
WHY IT MATTERSThis trial directly addresses a common complication in premature infants where improper oxygen management can cause lasting lung damage, making oxygen saturation targeting strategies critical for improving long-term outcomes in this vulnerable population.
ResearchUNITERAREApr 3
This is a research study looking for patients and families affected by rare diseases to share their thoughts and values about brain and nerve-related medical decisions. Researchers at St. Jude Children's Research Hospital want to understand what matters most to people living with rare diseases so they can make better ethical decisions in medical research and treatment. The study is now accepting participants and will start in April 2026.
WHY IT MATTERSThis study gives rare disease patients and caregivers a direct voice in shaping how medical ethics and brain-related research decisions are made — your input could influence future treatment guidelines and research practices.
Clinical trialUNITERAREApr 3
Researchers at St. Jude Children's Research Hospital are looking for people with sickle cell disease to participate in a study about egg health and fertility. The study will examine how sickle cell disease affects the ovaries and a woman's ability to have children. This research could help doctors better understand and protect fertility in women with sickle cell disease.
WHY IT MATTERSThis trial is now recruiting patients with sickle cell disease to understand how the disease affects ovarian function and fertility — information that could lead to better fertility preservation strategies for affected women.
Clinical trialUNITERAREApr 3
Researchers are testing a new treatment approach for elderly patients with a type of blood cancer (MDS or AML) that has come back or stopped responding to previous treatments. The study uses a combination of chemotherapy drugs followed by a stem cell transplant from a donor to try to help patients recover. This trial is now actively looking for patients to participate.
WHY IT MATTERSThis trial offers elderly patients with relapsed or refractory MDS/AML a potentially less toxic conditioning regimen before stem cell transplant — cladribine-bridged LABU may improve tolerability compared to standard high-dose approaches in this vulnerable population.
Clinical trialCLINICALTRIALSApr 3
Researchers are looking for 100 people with rare types of joint inflammation to better understand how these conditions develop and damage joints. Some of these inflammatory arthritides occur alongside rare autoimmune diseases like systemic sclerosis, while others develop as side effects from cancer immunotherapy drugs. By studying tissue samples and immune cells from patients, scientists hope to discover why these conditions happen and potentially develop better treatments.
WHY IT MATTERSIf you have rare inflammatory arthritis, systemic sclerosis with joint involvement, or developed joint inflammation after cancer immunotherapy, this trial could help researchers understand your condition better and may lead to more targeted treatments in the future.