RecruitingNCT06539169

FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases

Sponsor

xCures

Enrollment

1,000 participants

Start Date

Jun 10, 2024

Study Type

OBSERVATIONAL

Conditions

Myasthenia Gravis Amyloidosis Alpha-Thalassemia Beta-Thalassemia Amyotrophic Lateral Sclerosis Creutzfeld-Jakob Disease Cystic Fibrosis Duchenne Muscular Dystrophy Early-Onset Alzheimer Disease Ehlers-Danlos Syndrome Huntington Disease Gaucher Disease GM1 Gangliosidosis Pompe Disease Sickle Cell Disease Transthyretin Amyloid Cardiomyopathy Rare Diseases

Summary

FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases. Patients participate in the study by electronic consent (eConsent) and sign a medical records release to permit data collection. Medical records are accessed from institutions directly via eFax or paper fax, online from patient electronic medical record (EMR) portals, direct from DNA/RNA sequencing and molecular profiling vendors, and via electronic health information exchanges. Patients and their treating physicians may also optionally provide medical records. Medical records are received in or converted to electronic/digitized formats (CCDA, FHIR, PDF), sorted by medical record type (clinic visit, in-patient hospital, out-patient clinic, infusion and out-patient pharmacies, etc.) and made machine-readable to support data annotation, full text searches, and natural language processing (NLP) algorithms to further facilitate feature identification.

Eligibility

Plain Language Summary

Simplified for easier understanding

This study is building a long-term registry of people living with rare diseases to understand how these conditions progress over time and how they impact daily life. Rare diseases are those affecting fewer than 200,000 people nationwide. The goal is to gather real-world data that can help researchers better understand and treat these conditions. **You may be eligible if...** - You have a known or suspected rare disease (examples include ALS, cystic fibrosis, Huntington's disease, sickle cell disease, Duchenne muscular dystrophy, Ehlers-Danlos syndrome, and many others) - You or a legally authorized representative (such as a family member) can give informed consent - Deceased individuals may also be enrolled through family consent **You may NOT be eligible if...** - You do not have a known or suspected rare disease - You are unwilling to provide consent or have it provided on your behalf Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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