Clinical trialCLINICALTRIALSApr 17
Researchers are testing a new treatment called IFx-Hu2.0 combined with a cancer drug called pembrolizumab for people with Merkel cell carcinoma, a rare and aggressive skin cancer. In this study, some patients will receive the new treatment while others receive a placebo (fake treatment) to see which works better. The trial is looking for 118 adults to participate and is currently accepting new patients.
WHY IT MATTERSThis is the first Phase 2/3 trial testing IFx-Hu2.0 as an add-on therapy for Merkel cell carcinoma, offering checkpoint inhibitor-naïve patients a potential new treatment option beyond standard pembrolizumab alone.
Clinical trialCLINICALTRIALSApr 15
Researchers are testing a new AI-powered tool called IntelliWell that can find sperm cells in testicular tissue samples that appeared to have no sperm when checked the traditional way. If the tool successfully finds sperm, those cells could be used to help men with infertility have biological children through a procedure called ICSI. The study is enrolling 20 participants at Brigham and Women's Hospital.
WHY IT MATTERSFor men with azoospermia (no sperm in ejaculate) who were told their testicular tissue had no usable sperm, this AI tool could recover sperm that was missed by standard testing, potentially making fertility treatment possible when it seemed impossible before.
Clinical trialCLINICALTRIALSApr 15
Researchers are looking for 300 patients with Duchenne muscular dystrophy (DMD) to join a study about a medicine called givinostat. The study will track how safe the medicine is and how well it works for patients who are just starting to take it or have been taking it for less than 6 months. Patients will be followed for at least 2 years, with some being tracked for up to 5 years total.
WHY IT MATTERSThis real-world study will show how givinostat actually performs in everyday clinical practice for DMD patients in the US, providing practical safety and effectiveness data beyond what controlled trials reveal.
Clinical trialCLINICALTRIALSApr 15
Researchers are testing a cancer drug called nivolumab in patients with rare tumors that have a specific marker called PD-L1. This is a Phase 2 trial that will include up to 28 patients with many different types of rare cancers who haven't responded well to standard treatments. The study will last up to 12 months and measure how well the drug works.
WHY IT MATTERSIf you have one of the 43 rare tumor types listed and your cancer has high PD-L1 expression, this trial offers access to an immunotherapy that may work regardless of where your cancer started.
Clinical trialUNITERAREApr 15
Researchers are looking for pregnant women with NMOSD (a rare disease that affects the nerves in the eyes and spinal cord) who have taken or are taking a medicine called UPLIZNA to join a safety study. The study will track what happens to these women and their babies to make sure the medicine is safe during pregnancy. This information will help doctors understand whether UPLIZNA can be used safely by pregnant patients with NMOSD.
WHY IT MATTERSThis trial is recruiting pregnant women with NMOSD who have been exposed to UPLIZNA — currently there is limited safety data on this drug during pregnancy, so this study directly addresses a critical gap for women of childbearing age managing this serious neurological condition.
Clinical trialUNITERAREApr 15
Researchers are testing a new imaging scan called 18F-mFBG that can take pictures of the heart in people with Lewy body dementia, a brain disease that causes movement problems and thinking difficulties. This Phase 2 trial is now accepting patients and aims to see if this special scan can help doctors better understand and diagnose the disease. The scan uses a safe radioactive tracer that shows how well the heart's nerve endings are working.
WHY IT MATTERSThis trial is now actively recruiting patients with Lewy body dementia — if you have this diagnosis, you may be eligible to participate in a study that could help develop better diagnostic tools for your condition.
Clinical trialUNITERAREApr 15
Researchers are looking for patients with a specific type of blood cancer called Philadelphia chromosome positive acute lymphoblastic leukemia to test a new treatment combination. The treatment uses chemotherapy drugs (EPOCH), sometimes combined with rituximab (a protein therapy), plus a targeted drug called ponatinib. This is a Phase 2 trial, meaning it's testing whether the treatment works and is safe in a larger group of patients.
WHY IT MATTERSThis trial is now actively recruiting patients with newly-diagnosed Ph+ ALL/lymphoma and offers access to ponatinib, a third-generation tyrosine kinase inhibitor that may improve outcomes for this aggressive blood cancer.
Clinical trialUNITERAREApr 15
Researchers are testing a new cancer treatment that combines two approaches: a chemotherapy drug called temozolomide and a vaccine called SurVaxM that trains the immune system to fight cancer cells. This trial is for patients with neuroendocrine carcinomas (rare cancers in hormone-producing cells) that are spreading and getting worse despite other treatments. The study is now accepting patients and will run through 2026.
WHY IT MATTERSThis is one of the first trials testing an immunotherapy vaccine specifically for metastatic neuroendocrine carcinomas, offering a potential new option for patients whose cancer has progressed on standard treatments.
Clinical trialCLINICALTRIALSApr 14
Researchers are looking for young adults ages 15-25 with rare genetic disabilities to join a study about getting jobs and education. The study will follow 300 participants and examine the challenges these young people face when trying to go to school, get internships, or find work. Many young people with rare genetic diseases struggle with these opportunities because of their condition and lack of support.
WHY IT MATTERSThis trial directly addresses employment and education barriers that young adults with rare genetic disabilities face — areas where they typically have the fewest resources and support.
Clinical trialCLINICALTRIALSApr 14
Researchers tested a new gene therapy called ST-920 for Fabry disease in 36 patients. This treatment uses a modified virus to deliver instructions that help the body make an enzyme called alpha-galactosidase A, which people with Fabry disease don't produce enough of. The trial is now complete and tested whether different doses were safe and well-tolerated.
WHY IT MATTERSThis completed Phase 1/2 trial is the first human test of ST-920, meaning results could help determine if gene therapy can provide long-term relief for Fabry disease patients who currently require lifelong enzyme replacement infusions.
Clinical trialCLINICALTRIALSApr 14
Researchers at Mayo Clinic are looking for 6,000 people with rare kidney stone diseases caused by a single gene mutation to join a study. The study will identify which specific genes and genetic changes cause these kidney stones. By understanding the genetics behind these stones, scientists hope to develop better treatments in the future.
WHY IT MATTERSIf you have a rare monogenic kidney stone disease, this trial could help identify the genetic cause of your condition and accelerate development of targeted treatments — and you can enroll now.
Clinical trialCLINICALTRIALSApr 14
Researchers are testing a new medicine called pozelimab in young children ages 1 to 5 who have CHAPLE disease, a rare inherited condition that affects the stomach, intestines, heart, and blood vessels. This study is checking whether the medicine is safe and well-tolerated in this young age group. CHAPLE disease can cause serious symptoms, so finding safe treatments for young children is important.
WHY IT MATTERSThis is the first safety trial of pozelimab in very young children (ages 1-5) with CHAPLE disease, offering families of affected toddlers a potential treatment option when few alternatives exist for this life-threatening condition.
Clinical trialCLINICALTRIALSApr 14
Researchers are recruiting 7,000 cancer patients to test a new way of detecting cancer that comes back after treatment. By analyzing blood, tissue, and other body fluids for traces of cancer DNA, doctors hope to catch cancer earlier and help patients stay cancer-free longer. This study includes many types of cancer and will help doctors decide on the best treatment plans.
WHY IT MATTERSThis trial is now actively recruiting patients with various cancer types at a major Canadian cancer center, offering access to cutting-edge molecular residual disease testing that could detect cancer recurrence months before traditional imaging scans.
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 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 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.
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.