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.
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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…
Contacts

Paul Szabolcs, MD

412-692-5427

paul.szabolcs@chp.edu

CONTACT

Shawna McIntyre, RN

412-692-5552

mcintyresm@upmc.edu

CONTACT