Assessing long-term effects of curative therapies for sickle cell disease
U01 Cooperative Assessment of Late Effects for Sickle Cell Disease Curative Therapies
This study is testing the long-term effects of different curative treatments for sickle cell disease to see how they impact the health of both children and adults.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 750 (estimated) |
| Ages | 4 Years to 65 Years |
| Sex | All |
| Sponsor | Vanderbilt University Medical Center Academic / other |
| Locations | 5 sites (Washington D.C., District of Columbia and 4 other locations) |
| Trial ID | NCT05153967 on ClinicalTrials.gov |
What this trial studies
This study aims to evaluate the long-term health outcomes of various curative therapies for sickle cell disease (SCD) in both children and adults. It focuses on understanding the risks and benefits associated with treatments such as myeloablative and nonmyeloablative hematopoietic stem cell transplants and gene therapies. By analyzing health outcomes related to lung, heart, and kidney function, the study seeks to inform personalized treatment choices for patients with SCD. The multicenter approach allows for a comprehensive assessment of these therapies across different populations.
Who should consider this trial
Good fit: Ideal candidates include individuals with a confirmed diagnosis of sickle cell disease who can provide pre- and post-therapy data.
Not a fit: Patients with a history of non-compliance may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to improved long-term health outcomes and personalized treatment strategies for patients with sickle cell disease.
How similar studies have performed: Other studies have shown success in evaluating long-term outcomes of curative therapies for sickle cell disease, but this study aims to provide a more personalized approach.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria * Confirmed laboratory diagnosis of SCD * Ability to give informed consent * Ability to provide pre- and post-curative therapy data * Treated with either one HSCT or with standard disease-modifying therapy Exclusion Criteria •History of non-compliance
Where this trial is running
Washington D.C., District of Columbia and 4 other locations
- Children's National Medical Center — Washington D.C., District of Columbia, United States (Not_yet_recruiting)
- Emory University School of Medicine — Atlanta, Georgia, United States (Not_yet_recruiting)
- Johns Hopkins Hospital — Baltimore, Maryland, United States (Not_yet_recruiting)
- National Institutes of Health Clinical Center — Bethesda, Maryland, United States (Not_yet_recruiting)
- Vanderbilt University Medical Center — Nashville, Tennessee, United States (Recruiting)
Study contacts
- Principal investigator: Michael R DeBaun, MD, MPH — Vanderbilt University Medical Center
- Study coordinator: Leshana Saint Jean, PhD
- Email: leshana.saint.jean@vumc.org
- Phone: 6158751992
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.