Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT
Recruiting
Phase 2
Interventional Study
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)
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.
At a Glance
- Age
- 2 Months – 55
- Sex
- Any
- Trial phase
- Phase 2
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 100 (estimated)
- Sponsor
- Paul Szabolcs · Other
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About This Trial
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). …
Trial Locations
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Eligibility Criteria
Inclusion:
1. A 4/6, 5/6 or 6/6 HLA matched related or unrelated UCB unit available that will deliver a pre-cryopreservation total nucleated cell dose of ≥ 3 x 10e7 cells/kg, or double unit grafts, each cord blood unit delivering at least 2 x 10e7 cells/kg OR an 8 of 8 or 7 of 8 HLA allele level ma…
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