Observational study on rare diseases and their outcomes
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
This study is trying to gather and organize health information from people with rare diseases to better understand their outcomes and improve care.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 1000 (estimated) |
| Sex | All |
| Sponsor | xCures Industry-sponsored |
| Locations | 1 site (Los Altos, California) |
| Trial ID | NCT06539169 on ClinicalTrials.gov |
What this trial studies
FLOWER is a nationwide, virtual observational study aimed at collecting and standardizing clinical data for various rare diseases. Patients participate by providing electronic consent and allowing access to their medical records, which are collected from multiple sources including electronic medical records and genomic profiling. The study employs advanced data structuring techniques to ensure that the information is machine-readable and can be analyzed effectively. By integrating clinical, imaging, and patient-reported data, FLOWER seeks to create a comprehensive and real-time documentation of rare diseases.
Who should consider this trial
Good fit: Ideal candidates include individuals with known or suspected rare diseases affecting fewer than 200,000 individuals nationwide.
Not a fit: Patients who do not have a rare disease or are unable to provide informed consent may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could enhance understanding and management of rare diseases, leading to improved patient outcomes.
How similar studies have performed: While traditional rare disease registries have been established, this approach of centralized data collection and analysis is relatively novel and untested.
Eligibility criteria
Show full inclusion / exclusion 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.
Where this trial is running
Los Altos, California
- xCures — Los Altos, California, United States (Recruiting)
Study contacts
- Study coordinator: Mark Shapiro, MS
- Email: expandedaccess@xcures.com
- Phone: 707-641-4475
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.