New bone marrow transplant trial offers hope for kids with severe blood disorders

NCT ID NCT03128996

Recruiting now Disease control Sponsor: Washington University School of Medicine Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a bone marrow transplant from a family donor for children under 21 with severe non-cancer blood disorders like sickle cell disease, bone marrow failure, or immune problems. The goal is to see if the transplant can replace the diseased cells with healthy donor cells and control the disease. Participants will receive a reduced-intensity conditioning regimen before the transplant to help their body accept the new cells.

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Conditions

The condition(s) this trial relates to.

Bone Marrow Failure Disorders bone marrow failure syndrome disease hemoglobinopathy immune system disorder inherited hemoglobinopathy metabolic disease sickle cell disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

BONE MARROW FAILURE SYNDROMES HEMOGLOBINOPATHIES IMMUNOLOGIC DISORDERS METABOLIC DISORDERS NON MALIGNANT DISORDERS SEVERE SICKLE CELL DISEASE

Contacts and locations

Locations

Helen DeVos Children's Hospital
RECRUITING

Grand Rapids, Michigan, 49503, United States
Nemours Children's Health
RECRUITING

Wilmington, Delaware, 19803, United States
Washington University School of Medicine
RECRUITING

St Louis, Missouri, 63110, United States
Yale School of Medicine
RECRUITING

New Haven, Connecticut, 06510, United States

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