Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders

Recruiting Phase 1 Phase 2 Interventional Study
Severe Sickle Cell Disease Bone Marrow Failure Syndromes Metabolic Disorders Immunologic Disorders Hemoglobinopathies Non-malignant Disorders
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Prior Safety Data This treatment has already been tested in at least one earlier human trial.
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At a Glance
Age
1 Day – 21
Sex
Any
Trial phase
Phase 1/2
Study type
Interventional
Purpose
Treatment
Participants needed
29 (estimated)
Sponsor
Washington University School of Medicine · Other
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About This Trial

This study is designed to estimate the efficacy and toxicity of familial HLA mismatched bone marrow transplants in patients with non-malignant disease who are less than 21 years of age and could benefit from the procedure.

Trial Locations
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Eligibility Criteria
Inclusion Criteria: * Nonmalignant disorder requiring bone marrow transplant including bone marrow failure syndromes, metabolic disorders, immunologic disorders, or hemoglobinopathy * For patients with sickle cell disease, must have one of the following severe manifestations: 1. Overt or silent …
Contacts

Shalini Shenoy, MD

314-454-6018

shalinishenoy@wustl.edu

CONTACT

Ian Snyder, BS, CCRP

314-273-5953

ian.s@wustl.edu

CONTACT