Clinical trialCLINICALTRIALSToday
Researchers are testing a new cancer drug called sutetinib maleate in people with advanced lung cancer that has specific uncommon genetic mutations. This is a Phase 2 trial, meaning the drug has already been tested in a small group and now researchers want to see if it works better and remains safe in a larger group of 66 patients. The study is currently accepting new participants.
WHY IT MATTERSIf you have metastatic or locally advanced non-small cell lung cancer with uncommon EGFR mutations, this trial offers access to a potentially new treatment option that may not yet be available outside of clinical research.
Clinical trialCLINICALTRIALS4 days ago
Williams syndrome is a rare genetic condition that affects about 1 in every 8,000 to 12,000 people. It happens when a person is missing a small section of DNA on chromosome 7, which includes the gene that makes elastin (a protein that helps blood vessels stretch). This causes distinctive facial features and heart and blood vessel problems. Researchers are now recruiting 1,099 people with Williams syndrome or a related heart condition called supravalvular aortic stenosis to donate DNA and tissue samples to help scientists better understand these conditions.
WHY IT MATTERSThis actively recruiting biobank offers Williams syndrome and SVAS patients a direct way to contribute DNA and tissue samples to NHLBI-sponsored research that could accelerate understanding of the genetic causes and lead to new treatments.
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 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 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 3
Researchers at St. Jude Children's Research Hospital are testing a drug called entrectinib to treat very young children (under 3 years old) who have brain or spinal cord tumors caused by changes in specific genes called NTRK1, NTRK2, NTRK3, or ROS1. This is a Phase 2 trial, meaning they've already tested the drug in a small group and now want to see if it works well in more patients. The trial is now accepting new patients and is expected to start in April 2026.
WHY IT MATTERSThis trial offers a targeted treatment option specifically designed for infants and toddlers with NTRK- or ROS1-fused CNS tumors, a group that has historically had very limited treatment options and poor outcomes.
NewsUNITERAREApr 3
The PAN Foundation's financial assistance fund for paroxysmal nocturnal hemoglobinuria (PNH) — a rare blood disorder where red blood cells break down too quickly — is currently closed and stopped accepting new applications as of April 2, 2026. This fund previously provided up to $12,000 per year to help patients pay for treatment costs. Patients who need financial help with their PNH medications should check the PAN Foundation website for updates on when the fund might reopen.
WHY IT MATTERSPatients with PNH who were relying on this $12,000 annual benefit to afford their medications now need to find alternative financial assistance programs immediately, as this major funding source is no longer available.
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 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.
Clinical trialCLINICALTRIALSMar 26
Researchers are testing a new drug called zipalertinib to treat advanced lung cancer in patients who have a specific genetic mutation called EGFR exon 20 insertion. This mutation makes lung cancer harder to treat with standard medications. The study is looking for 220 patients to see if zipalertinib is safe and effective for this type of cancer.
WHY IT MATTERSThis trial targets EGFR exon 20 insertion mutations in advanced NSCLC, a genetic subtype that historically has limited treatment options and poor response to standard EGFR inhibitors.
ResearchCLINICALTRIALSMar 26
Researchers at St. Jude Children's Research Hospital are recruiting 385 people—including patients, families, doctors, and scientists—to share their thoughts about ethical questions that come up when testing new treatments for rare brain and nerve diseases. The study wants to understand what different groups of people think is fair and right when designing these small, personalized treatment trials.
WHY IT MATTERSThis study directly shapes how future rare neurological disease trials will be designed and conducted, meaning your input as a patient or caregiver could influence the ethical standards that protect you in future treatment studies.
Clinical trialCLINICALTRIALSMar 26
Researchers are looking for patients with a specific type of lung cancer (non-small-cell lung cancer with EGFR PACC mutations) who have never received treatment for advanced disease. This study will test whether a new drug called firmonertinib works better and is safer than two existing drugs (osimertinib or afatinib) that doctors currently use. About 480 patients will be enrolled worldwide.
WHY IT MATTERSThis trial is actively recruiting patients with EGFR PACC mutations—a rare subset of lung cancer—and offers access to firmonertinib, a potentially more effective treatment option compared to standard first-line therapies.