Clinical trialRSSYesterday
A grandmother shares how a small change in how her grandson's condition was measured helped him qualify for a Duchenne muscular dystrophy (DMD) treatment trial. Researchers adjusted the scoring system by 19 points, which gave him a second chance to participate in the study. This story highlights how trial eligibility rules can sometimes be adjusted to help more patients access new treatments.
WHY IT MATTERSIf your child with DMD has been told they don't qualify for a trial, this story shows that eligibility criteria can sometimes be reconsidered or adjusted—it's worth asking your doctor whether similar flexibility might apply to your situation.
Clinical trialCLINICALTRIALSYesterday
Researchers at Memorial Sloan Kettering Cancer Center are testing a smartphone app designed to help breast cancer and rare cancer survivors deal with the fear that their cancer might come back. The app uses a technique called Attention and Interpretation Modification (AIM) to help people change how they think about cancer recurrence. The study has enrolled 252 people and is no longer recruiting new participants, but the results could help many cancer survivors in the future.
WHY IT MATTERSThis trial addresses a common but often overlooked mental health challenge for cancer survivors—fear of recurrence—by testing a personalized, accessible mobile app intervention that could be widely available if proven effective.
Clinical trialCLINICALTRIALSYesterday
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 trialRSS3 days ago
A company called BlueRock Therapeutics tested a new cell therapy called bemdaneprocel for Parkinson's disease. In a small early-stage trial, patients who received one dose of this therapy showed improvement in their movement symptoms, and these improvements lasted for up to three years. This is promising because it suggests a single treatment might help Parkinson's patients for a long time.
WHY IT MATTERSIf bemdaneprocel continues to show sustained benefits in larger trials, it could offer Parkinson's patients a one-time treatment option instead of daily medications, potentially improving quality of life and treatment adherence.
Clinical trialCLINICALTRIALS5 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 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 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 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 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.
Clinical trialUNITERAREApr 3
Researchers are testing a new experimental drug called EPI-326 in people with two types of cancer: non-small cell lung cancer and head and neck cancer, both caused by mutations in the EGFR gene. This is an early-stage study (Phase 1) to see if the drug is safe and how much patients can tolerate. The study is currently accepting new participants and is expected to start in April 2026.
WHY IT MATTERSThis trial offers access to a novel EGFR-targeted therapy for patients with EGFR-mutant lung or head and neck cancers who may have limited treatment options or resistance to current therapies.
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.
Clinical trialCLINICALTRIALSMar 26
Researchers are testing a new drug called furmonertinib in patients with advanced lung cancer that has specific genetic mutations. This is an early-stage study (Phase 1) with 160 patients to see if the drug is safe and how well it works. The drug targets uncommon mutations in EGFR and HER2 genes that some lung cancer patients have.
WHY IT MATTERSThis trial addresses treatment options for patients with advanced NSCLC carrying uncommon EGFR or HER2 mutations, which typically have fewer targeted therapy options than common mutations.
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.