Clinical Trial

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

Study acronym: HSCT+RIC
Recruiting Phase 2
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Summary
The objective of this study is to evaluate the efficacy of using a reduced-intensity condition (RIC) regimen with umbilical cord blood transplant (UCBT), double cord UCBT, matched unrelated donor (MUD) bone marrow transplant (BMT) or peripheral blood stem cell transplant (PBSCT) in patients with non-malignant disorders that are amenable to treatment with hematopoietic stem cell transplant (HSCT). After transplant, subjects will be followed for late effects and for ongoing graft success.
Protocol Amendment History 22 amendments
This ClinicalTrials.gov record has been amended 22 times since 2013-10-10; most recent amendment 2025-12-08.
Status change: Not Yet Recruiting → Recruiting 2014-03-25
Trial Details
NCT Number NCT01962415
Lead Sponsor Paul Szabolcs
Conditions Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions, Systemic Juvenile Idiopathic Arthritis (sJIA), Juvenile Rheumatoid Arthritis (JRA)
Enrollment 100 participants
Start Date 2014-02-04
Primary Completion 2026-11 (estimated)
Study Completion 2027-11 (estimated)
Updated on ClinicalTrials.gov 2025-12-15