NCT06539169 · RECRUITING
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
FLOWER is a virtual observational study — no treatment involved — that collects and organizes medical records from people living with rare diseases. Researchers use electronic health records, faxed documents, and lab data to build a standardized database. There is no assigned intervention. This is not a phase-numbered trial; it is a data registry designed to improve how rare disease outcomes are tracked over time.
Eligibility criteria
Inclusion Criteria: * Any person with a known or suspected rare disease, defined by their prevalence of fewer than 200,000 individuals nationwide. Diseases include but are not limited to: Alpha- or Beta- Thalassemia Amyloidosis Amyotrophic Lateral Sclerosis (ALS) Creutzfeldt-Jakob disease (CJD) Cystic Fibrosis (CF) Duchenne Muscular Dystrophy (DMD) Early-onset Alzheimer's Disease Ehlers-Danlos Syndrome (EDS) Huntington's Disease (HD) Gaucher Disease GM1 Gangliosidosis Myasthenia Gravis Pompe Disease Sickle Cell Disease Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Transthyretin Amyloid Polyneuropathy (ATTR-PN) - Patients or their legally-authorized representative must be willing and able to provide informed consent (and assent, if applicable). Deceased persons may participate via consent of their legally-authorized representative in accordance with applicable Federal and state laws Exclusion Criteria: * Patient or LAR is unable to provide informed consent. * Patient resides in a country other than the United States and is unable to provide access to medical records.
The sponsor's own eligibility wording, lightly reformatted. The study team makes the final eligibility decision — worth discussing with your doctor.
Eligibility criteria as of 2024-11-14