Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT
Recruiting now · Phase 2
Conditions studied: 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)
In brief
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.
Key facts
- Study ID
- NCT01962415
- Run by
- Paul Szabolcs
- People needed
- 100
- Starts
- 2014-02-04
- Expected to finish
- 2027-11-01
- Last updated by the study team
- 2025-12-15
Who can join
Age: 0 and older, up to 55. Sex: any. Healthy volunteers: not accepted.
Where it is running
- UPMC Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania, United States (enrolling)
Full record on ClinicalTrials.gov
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