Actively Recruiting
U01 Cooperative Assessment of Late Effects for Sickle Cell Disease Curative Therapies
Led by Vanderbilt University Medical Center · Updated on 2026-01-30
750
Participants Needed
5
Research Sites
35 weeks
Total Duration
On this page
Sponsors
V
Vanderbilt University Medical Center
Lead Sponsor
J
Johns Hopkins University
Collaborating Sponsor
AI-Summary
What this Trial Is About
Sickle Cell Disease (SCD) is a common genetic condition affecting approximately 100,000 people in the United States. While childhood survival rates have improved greatly, adults with SCD face significantly reduced life expectancy due to heart, lung, and kidney problems. This study aims to evaluate whether curative treatments for SCD improve or worsen these organ functions compared to standard therapies, with a focus on personalized treatment decisions and genetic factors linked to outcomes. The study observes groups of children and adults with SCD receiving either curative therapies such as myeloablative or nonmyeloablative hematopoietic stem cell transplant (HSCT) or standard disease-modifying treatments. Children aged 4 to 17 undergoing myeloablative HSCT or standard therapy and adults aged 18 to 65 undergoing nonmyeloablative HSCT or standard therapy are included. Researchers will compare lung and kidney function, heart measurements, and risks of blood-related complications between these groups over time. Participants will be monitored for an average of four years, with regular assessments of lung function (FEV1, FVC, FEV1/FVC ratio), kidney function (eGFR, albuminuria), and heart function (tricuspid regurgitant jet velocity, blood pressure). Data will be collected before and after treatment to track long-term health changes. The study also evaluates the accuracy of health data reported by patients and families compared to clinical records, aiming to better understand late effects of curative therapies.
CONDITIONS
Brief Title
Cooperative Assessment of Late Effects for SCD Curative Therapies
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Confirmed laboratory diagnosis of sickle cell disease
- Ability to give informed consent
- Ability to provide pre- and post-curative therapy data
- Treated with either one hematopoietic stem cell transplant or with standard disease-modifying therapy
- Age between 4 and 65 years
You will not qualify if you...
- History of non-compliance
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)
Duration - Up to 4 years
Participants who have undergone or are scheduled to undergo curative therapies or standard therapy for sickle cell disease are observed to evaluate long-term health outcomes including lung, heart, and kidney function over time.
Periodic visits over the course of up to 4 years
Trial Site Locations
Total: 5 locations
1
Children's National Medical Center
Washington D.C., District of Columbia, United States, 20010
Not Yet Recruiting
2
Emory University School of Medicine
Atlanta, Georgia, United States, 30322
Not Yet Recruiting
3
Johns Hopkins Hospital
Baltimore, Maryland, United States, 21287
Not Yet Recruiting
4
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20814
Not Yet Recruiting
5
Vanderbilt University Medical Center
Nashville, Tennessee, United States, 37232-9000
Actively Recruiting
Research Team
L
Leshana Saint Jean, PhD
K
Kristin Wuichet, PhD
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
4
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