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Clinical trialNCT04018937Active, not recruitingPhase 2Interventional

Early Human Leukocyte Antigen (HLA) Matched Sibling Hematopoietic Stem Cell Transplantation

SponsorEmory UniversityEnrollment43Sickle Cell Disease
View the registry record on ClinicalTrials.gov

Summary

The lay summary the sponsor registered on ClinicalTrials.gov.

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.

Investigators

The overall officials registered on this study. Names that resolve to a FindMyKOL profile link to it; the rest are shown as registered.

A profile link is shown only when the registered name resolves to a person record with a confident, namesake-checked match; ambiguous names stay unlinked.

Sites

15 registered facilities, with the per-site recruitment status where the registry publishes one. ClinicalTrials.gov does not publish per-site enrollment counts.

Canada
  • Cancercare Manitoba/winnipeg Children'S Hospital

    Winnipeg, Manitoba, Canada

  • Centre Hospitalier Universitaire Sainte-Justine

    Montreal, Quebec, Canada

United States
  • Children'S Hospital of Alabama

    Birmingham, Alabama, United States

  • Phoenix Children'S

    Phoenix, Arizona, United States

  • Yale University, Yale Cancer Center

    New Haven, Connecticut, United States

  • Children'S National Hospital

    Washington D.C., District of Columbia, United States

  • Children'S Healthcare of Altanta

    Atlanta, Georgia, United States

  • University of Chicago

    Chicago, Illinois, United States

  • Riley Children'S Health/indiana University

    Indianapolis, Indiana, United States

  • Dana-Farber Cancer Institute/boston Children'S Hospital

    Boston, Massachusetts, United States

  • Washington University School of Medicine

    St Louis, Missouri, United States

  • Hackensack University Medical Center

    Hackensack, New Jersey, United States

  • University of North Carolina Medical Center

    Chapel Hill, North Carolina, United States

  • Atrium Health Levine Cancer Institute

    Charlotte, North Carolina, United States

  • Children'S Hospital of Philadelphia

    Philadelphia, Pennsylvania, United States

Site investigator names are published by ClinicalTrials.gov only while a study recruits, so completed studies list sites without them.

Drugs studied

The drug and biological interventions registered on this study, normalized to their RxNorm ingredient where possible.

Linked drugs open the directory filtered to KOLs working with them.clinicaltrials.gov ↗

Registration & key dates

Registry attributes as recorded on the ClinicalTrials.gov study record.

Start
22 Mar 2019
Primary completion
30 Nov 2026
Completion
30 Nov 2026
Last update posted
25 Nov 2024
Registered enrollment
43
Registry id
NCT04018937

Source: ClinicalTrials.gov, via the CTTI AACT database, data as of 24 Aug 2026. Registry facts are shown as registered by the study sponsor; FindMyKOL does not interpret them.