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Search / Trial NCT05153967

Cooperative Assessment of Late Effects for SCD Curative Therapies

Launched by VANDERBILT UNIVERSITY MEDICAL CENTER · Nov 29, 2021

Trial Information

Current as of June 27, 2025

Recruiting

Keywords

Myeloablative Autologous Gene Editing Myeloablative Autologous Gene Therapy Myeloablative Allo Hsct Nonmyeloablative Allo Hsct Disease Modifying Therapy

ClinConnect Summary

This clinical trial is studying the long-term effects of curative therapies for Sickle Cell Disease (SCD), a condition that affects the blood and can lead to serious complications in the heart, lungs, and kidneys. The goal is to find out if these new therapies improve or worsen health problems in these organs compared to standard treatments. Researchers also want to see if certain genes might influence how well patients do after receiving curative therapies.

To be eligible for this trial, participants must have a confirmed diagnosis of SCD and be able to provide information about their health before and after treatment. They should be between the ages of 14 and 23 and can be of any gender. Participants will be asked to share their experiences and health data, which will help doctors understand the best treatment options for individuals with SCD. It's important to know that the trial is currently recruiting participants, and those who join will contribute valuable information that could lead to better care for future patients.

Gender

ALL

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

About Vanderbilt University Medical Center

Vanderbilt University Medical Center (VUMC) is a leading academic medical center located in Nashville, Tennessee, renowned for its commitment to advancing healthcare through innovative research and clinical excellence. As a prominent sponsor of clinical trials, VUMC integrates cutting-edge scientific inquiry with patient-centered care, supporting a diverse array of studies aimed at enhancing medical knowledge and improving treatment outcomes. With a robust infrastructure and a multidisciplinary team of experts, VUMC fosters collaboration across various fields, ensuring the highest standards of safety and ethical considerations in its research endeavors. Through its dedication to medical discovery and education, VUMC plays a pivotal role in shaping the future of medicine.

Locations

Atlanta, Georgia, United States

Baltimore, Maryland, United States

Washington, District Of Columbia, United States

Nashville, Tennessee, United States

Bethesda, Maryland, United States

Patients applied

0 patients applied

Trial Officials

Michael R DeBaun, MD, MPH

Principal Investigator

Vanderbilt University Medical Center

Timeline

First submit

Trial launched

Trial updated

Estimated completion

Not reported

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