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

NCT01962415 · Status: RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 100

Last updated 2025-12-15

No results posted yet for this study

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.

Conditions

  • 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)

Interventions

DRUG

Hydroxyurea

Oral administration

DRUG

Alemtuzumab

Intravenous (IV) administration.

DRUG

Fludarabine

IV administration

DRUG

Melphalan

IV administration

DRUG

Thiotepa

IV administration

Sponsors & Collaborators

  • Paul Szabolcs

    lead OTHER

Principal Investigators

  • Paul Szabolcs, MD · University of Pittsburgh

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
2 Months
Max Age
55 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-02-04
Primary Completion
2026-11-30
Completion
2027-11-30
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Drugs
Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01962415 on ClinicalTrials.gov