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 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 3
Researchers are testing a new technology called 'digital twins' to help prevent second strokes in patients with three rare brain conditions: moyamoya disease, cerebral amyloid angiopathy, and previous stroke history. A digital twin is a computer model of a patient's brain that doctors can use to predict what treatments might work best for that individual. This study will randomly assign patients to either receive care guided by this digital twin technology or standard care, and compare which approach prevents more strokes.
WHY IT MATTERSThis trial offers patients with moyamoya disease or cerebral amyloid angiopathy who have had a stroke access to personalized, AI-guided prevention strategies that could reduce their risk of future strokes.