Actively Recruiting
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
Led by xCures · Updated on 2024-11-14
1000
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are conducting FLOWER, a fully virtual, nationwide observational study to collect and analyze clinical data from people with rare diseases. This study aims to gather detailed, standardized medical information in real time from patients with conditions affecting fewer than 200,000 individuals nationwide, including diseases like Alpha-Thalassemia, Amyloidosis, Duchenne Muscular Dystrophy, and others. The study is designed to overcome traditional research challenges by reducing burdens on patients and healthcare providers. Participants contribute by electronically consenting and allowing access to their medical records from various sources, including hospitals, clinics, pharmacies, and genetic testing vendors. Data collected include electronic medical records, imaging files, molecular and genetic information, and patient or caregiver-reported outcomes. The study staff standardize and code this information using recognized medical ontologies to create a comprehensive, longitudinal dataset without requiring data entry from treating physicians. During the study, researchers monitor several outcomes over five years, including overall survival, medication safety, developmental milestones, functional and motor abilities, and symptom changes. The study involves no direct interventions, focusing instead on gathering and analyzing real-world data. Participation is entirely remote and ongoing, with data collected continuously to provide a detailed picture of rare diseases' natural history and progression.
CONDITIONS
Brief Title
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Any person with a known or suspected rare disease affecting fewer than 200,000 individuals nationwide, including but not limited to listed rare diseases
- 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 according to applicable laws
You will not qualify if you...
- Patient or legally-authorized representative is unable to provide informed consent
- Patient resides outside the United States and cannot provide access to medical records from that location or U.S. institutions or vendors
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person or remote)
Duration - Up to 5 years
Participants who undergo routine care are observed over time with data collected from medical records, imaging, biomarker tests, and patient-reported outcome surveys to document the natural history of rare diseases.
Data collection occurs continuously through medical record aggregation and periodic surveys
Trial Site Locations
Total: 1 location
1
xCures
Los Altos, California, United States, 94022
Actively Recruiting
Research Team
M
Mark Shapiro, MS
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
0
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