Trial Now Recruiting: Longitudinal Studies of Patient With FPDMM (NCT03854318)
WHY IT MATTERS
This is an active recruiting trial sponsored by the National Human Genome Research Institute seeking 1,000 participants with RUNX1-variant FPDMM — participation could directly advance understanding of diagnosis and treatment for this rare inherited bleeding disorder.
Researchers are looking for 1,000 people of any age who have a rare blood disorder called FPDMM, which is caused by a change in the RUNX1 gene. People with this condition may bleed easily and for longer than normal when injured. This study will help doctors better understand the disease, diagnose it more accurately, and find better ways to treat it.
NCT ID: NCT03854318 Status: RECRUITING Conditions: Inherited Hematological Diseases, Rare Diseases, FPDMM Enrollment: 1000 Sponsor: National Human Genome Research Institute (NHGRI) Summary: Background: Genes tell the body and its cells how to work. Familial platelet disease (FPD) or FPD with associated malignancies (FPDMM) is caused by a variant in the gene RUNX1. People with this disease may have problems with their blood and bleed for a long time when they are injured. Researchers want to learn more about RUNX1 variants and FPD. Objective: To learn more about FPD in people with RUNX1 variants to lead to better diagnosis, monitoring, and treatment. Eligibility: People any age
YOU CAN ACT ON THIS
If you or a family member has been diagnosed with FPDMM or FPD with a RUNX1 variant, contact the study team through clinicaltrials.gov (NCT03854318) to learn if you qualify and how to enroll.