NCT05153967 Cooperative Assessment of Late Effects for SCD Curative Therapies
| NCT ID | NCT05153967 |
| Status | Recruiting |
| Phase | — |
| Sponsor | Vanderbilt University Medical Center |
| Condition | Sickle Cell Disease |
| Study Type | OBSERVATIONAL |
| Enrollment | 750 participants |
| Start Date | 2022-07-12 |
| Primary Completion | 2026-06 |
Eligibility & Interventions
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 750 participants in total. It began in 2022-07-12 with a primary completion date of 2026-06.
⚠ 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
Sickle Cell Disease is one of the most common genetic diseases in the United States, occurring in approximately 1 in 400 births. Approximately 100,000 individuals are diagnosed with SCD in the United States. Mortality for children with SCD has decreased substantially over the past 4 decades, with \>99% of those born in high resource settings, including the United States, France, and England, now surviving to 18 years of age. However, the life expectancy of adults with SCD is severely shortened. Dysfunction of the heart, lung, and kidney is directly associated with decreased life expectancy. With the variety of curative therapies that are now available for SCD, long-term health outcomes studies are time-sensitive. As of now, efforts to determine long-term health outcomes following curative therapies for SCD have been limited. Though curative therapies initially should provide a cure for symptoms of SCD, there is the risk of late health outcomes to consider. Defining health outcomes following curative therapy is essential to improve personalized decision-making when considering curative versus disease-modifying therapeutic options. The primary goal of this study is to determine whether curative therapies for individuals with SCD will result in improved or worsening heart, lung, and kidney damage when compared to individuals with SCD receiving standard therapy. The investigators will also explore whether certain genes are associated with a good or bad outcome after curative therapy for SCD.
Eligibility 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
Contact & Investigator
Michael R DeBaun, MD, MPH
PRINCIPAL INVESTIGATOR
Vanderbilt University Medical Center
Frequently Asked Questions
Who can join the NCT05153967 clinical trial?
This trial is open to participants of all sexes, aged 4 Years or older, up to 65 Years, studying Sickle Cell Disease. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT05153967 currently recruiting?
Yes, NCT05153967 is actively recruiting participants. Contact the research team at leshana.saint.jean@vumc.org for enrollment information.
Where is the NCT05153967 trial being conducted?
This trial is being conducted at Washington D.C., United States, Atlanta, United States, Baltimore, United States, Bethesda, United States and 1 additional location.
Who is sponsoring the NCT05153967 clinical trial?
NCT05153967 is sponsored by Vanderbilt University Medical Center. The principal investigator is Michael R DeBaun, MD, MPH at Vanderbilt University Medical Center. The trial plans to enroll 750 participants.
Related Trials
Related Intelligence Guides
In-depth guides covering this condition's trials, eligibility, and what to expect.