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Recruiting NCT06539169

NCT06539169 FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases

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Clinical Trial Summary
NCT ID NCT06539169
Status Recruiting
Phase
Sponsor xCures
Condition Alpha-Thalassemia
Study Type OBSERVATIONAL
Enrollment 1,000 participants
Start Date 2024-06-10
Primary Completion 2026-06-10

Eligibility & Interventions

Sex All sexes
Min Age N/A
Max Age N/A
Study Type OBSERVATIONAL

Eligibility Fast-Check

Enter your details for a quick preliminary check. This does not replace medical advice.

What to Expect as a Participant

This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.

This trial targets 1,000 participants in total. It began in 2024-06-10 with a primary completion date of 2026-06-10.

⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.

Brief 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 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.

Contact & Investigator

Central Contact

Mark Shapiro, MS

✉ expandedaccess@xcures.com

📞 707-641-4475

Frequently Asked Questions

Who can join the NCT06539169 clinical trial?

This trial is open to participants of all sexes, studying Alpha-Thalassemia. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.

Is NCT06539169 currently recruiting?

Yes, NCT06539169 is actively recruiting participants. Contact the research team at expandedaccess@xcures.com for enrollment information.

Where is the NCT06539169 trial being conducted?

This trial is being conducted at Los Altos, United States.

Who is sponsoring the NCT06539169 clinical trial?

NCT06539169 is sponsored by xCures. The trial plans to enroll 1,000 participants.

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ClinicalMetric — Independent clinical trial intelligence platform. Not affiliated with NIH, ClinicalTrials.gov, the U.S. FDA, or any pharmaceutical company, hospital, or clinical research organization. Trial data is sourced from ClinicalTrials.gov for informational purposes only and does not constitute medical advice. Do not make any treatment, enrollment, or health decisions based solely on information found here — always consult a qualified healthcare professional. Full Disclaimer  ·  Last Reviewed: April 2026  ·  Data Methodology