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

Part of paid clinical trials in Pittsburgh, Pennsylvania.

Sponsor
Paul Szabolcs
Study ID
NCT01962415
Phase
PHASE2
Status
Recruiting
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Conditions

  • Congenital Bone Marrow Failure Syndromes
  • Hereditary Anemias
  • Inflammatory Conditions
  • Inherited Metabolic Disorders (IMD)
  • Juvenile Rheumatoid Arthritis (JRA)
  • Primary Immunodeficiency (PID)
  • Systemic Juvenile Idiopathic Arthritis (sJIA)

Eligibility Criteria

Sex
ALL
Age
2 Months - 55 Years
Healthy Volunteers
Not accepted

Interventions

  • Hydroxyurea — DRUG
    Oral administration
  • Alemtuzumab — DRUG
    Intravenous (IV) administration.
  • Fludarabine — DRUG
    IV administration
  • Melphalan — DRUG
    IV administration
  • Thiotepa — DRUG
    IV administration

Study Details

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 Dates

Start date
Feb 4, 2014
Status verified
Dec 2025
Primary completion
Nov 30, 2026
Completion
Nov 30, 2027

Study Design

Enrollment
100 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: UCBT:transfusion dependent anemias or increased rejection risk
    Alemtuzumab, Hydroxyurea, Fludarabine, Melphalan, and Thiotepa conditioning regimen prior to allogenic HSCT.
  • Experimental: BMT, PBSCT and not transfusion dependent UCBT
    Alemtuzumab, Hydroxyurea, Fludarabine, Melphalan, and Thiotepa conditioning regimen prior to allogenic HSCT.

Primary Outcome Measure

Post-transplant treatment-related mortality (TRM) [ Time Frame: 1 year post-transplant ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
UPMC Children's Hospital of PittsburghPittsburghPennsylvania15224
Shawna McIntyre, RN
[email protected]
412-692-5552
Paulina Horvei, MD (SUB_INVESTIGATOR)
Randy Windreich, MD (SUB_INVESTIGATOR)
Archana Ramgopal, DO (SUB_INVESTIGATOR)

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By research site
UPMC Children's Hospital of Pittsburgh· Pittsburgh, PA

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