Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT
NCT01962415
This study is recruiting at 1 US site in 1 state, according to its ClinicalTrials.gov record last updated .
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.
Shortened from the sponsor's brief summary. View official record on ClinicalTrials.gov
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)
Eligibility basics and study details
- Study type
- Interventional
- Phase
- Phase 2
- Ages
- 2 months to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Sponsor
- Paul Szabolcs
- Last updated on ClinicalTrials.gov
- December 15, 2025
These are the basic criteria only. The full inclusion and exclusion criteria are on the official record, and the study team decides who can take part.
US recruiting sites
Pennsylvania · 1
- UPMC Children's Hospital of Pittsburgh · Pittsburgh, PA
Source: ClinicalTrials.gov, data processed . Recruiting US sites filtered by distance and site status; summaries shortened.