Clinical trial · Observational
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
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.
Conditions
Conditions (17)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Alpha-Thalassemia | — | UNRESOLVED | — |
| Amyloidosis | Amyloidosis | ONTOLOGY_EXACT | 0.98 |
| Amyotrophic Lateral Sclerosis | — | UNRESOLVED | — |
| Beta-Thalassemia | — | UNRESOLVED | — |
| Creutzfeld-Jakob Disease | — | UNRESOLVED | — |
| Cystic Fibrosis | — | UNRESOLVED | — |
| Duchenne Muscular Dystrophy | — | UNRESOLVED | — |
| Early-Onset Alzheimer Disease | — | UNRESOLVED | — |
| Ehlers-Danlos Syndrome | — | UNRESOLVED |
Interventions
Interventions (0)
Data not yet available
Design
Arms and outcomes
Arms (0)
[]Primary outcomes (6)
- measure
- Overall Survival (OS)
- timeFrame
- 5 Years
- measure
- Safety/tolerability of medications
- timeFrame
- 5 years
- measure
- Changes in normal development milestones
- timeFrame
- 5 years
- measure
- Changes in functional status
- timeFrame
- 5 years
- measure
- Changes in motor function
- timeFrame
- 5 years
Eligibility
Eligibility (as posted)
- Sex
- All
Show eligibility criteria text
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.
References
Publications (0)
Data not yet available