Early Human Leukocyte Antigen (HLA) Matched Sibling Hematopoietic Stem Cell Transplantation (NCT04018937)
Early HLA Matched Sibling Hematopoietic Stem Cell Transplantation for Children with Sickle Cell Disease: a Sickle Transplant Advocacy and Research Alliance (STAR) Trial
🩺 Plain-English Summary (8th-Grade Level)
This study aims to enroll 58 pre-adolescent (\<13 years) pediatric participants with sickle cell disease (SCD) who have a pre-adolescent sibling bone marrow donor. All participants will go through a pre-transplant evaluation to find out if there are health problems that will keep them from being able to receive the transplant. It usually takes 2 to 3 months to complete the pre-transplant evaluation and make the arrangements for the transplant. Once they are found to be eligible for transplant, participants will be admitted to the hospital and will start transplant conditioning. Conditioning is the chemotherapy and other medicines given to prepare them to receive donor cells. It prevents the immune system from rejecting donor cells. Conditioning will start 21 days before transplant. Once they complete conditioning, participants will receive the bone marrow transplant. After the transplant, participants will stay in the hospital for 4-6 weeks. After they leave the hospital, participants will be followed closely in the clinic. Outpatient treatment and frequent clinic visits usually last 6 to 12 months. Routine medical care includes at least a yearly examination for many years after transplant by doctors and nurses familiar with sickle cell disease and transplant. The researchers will collect and study information about participants for 2 years after transplant.
Who can join: Review the eligibility criteria below.
Location: Children's Hospital of Alabama — Birmingham, Alabama
Age range: 2 Years to 13 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (15 Sites)
- Children's Hospital of Alabama — Birmingham, Alabama
- Phoenix Children's — Phoenix, Arizona
- Yale University, Yale Cancer Center — New Haven, Connecticut
- Children's National Hospital — Washington D.C., District of Columbia
- Children's Healthcare of Altanta — Atlanta, Georgia
- University of Chicago — Chicago, Illinois
- Riley Children's Health/Indiana University — Indianapolis, Indiana
- Dana-Farber Cancer Institute/Boston Children's Hospital — Boston, Massachusetts
- Washington University School of Medicine — St Louis, Missouri
- Hackensack University Medical Center — Hackensack, New Jersey
- University of North Carolina Medical Center — Chapel Hill, North Carolina
- Atrium Health Levine Cancer Institute — Charlotte, North Carolina
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania
- Cancercare Manitoba/Winnipeg Children's Hospital — Winnipeg, Manitoba
- Centre Hospitalier Universitaire Sainte-Justine — Montreal, Quebec
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Sickle Cell Disease
- Kidney Function in Sickle Cell Anemia — Chapel Hill, North Carolina
- BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease — Birmingham, Alabama
- Nonmyeloablative Haploidentical Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease — Bethesda, Maryland
- Acupressure in Patients With Sickle Cell Disease — Irvine, California
- Long-term Follow-up (LTFU) of Patients Treated With Genome-edited Autologous Hematopoietic Stem and Progenitor Cells (HSPC) — Chicago, Illinois
- Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD) — Charlotte, North Carolina
Explore by condition: All Sickle Cell Disease clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.