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Reduced Intensity Conditioning (RIC) Regimen for Patients With Non-malignant Disorders
Sponsor: Children's Hospital of Philadelphia
Summary
This Phase II pilot study evaluates the safety and effectiveness of reduced-intensity conditioning (RIC) regimens prior to allogeneic hematopoietic stem cell transplantation (HSCT) in pediatric and young adult patients with non-malignant disorders, including immunodeficiencies and hemoglobinopathies. The study examines different conditioning approaches that vary in the timing and dosing of alemtuzumab (Campath), as well as disease-specific modifications for beta thalassemia major and infants. The primary objective is to assess donor engraftment and event-free survival at one year following transplant. The study aims to determine whether RIC regimens can reduce toxicity while maintaining successful engraftment and disease control.
Key Details
Gender
All
Age Range
6 Months - 25 Years
Study Type
INTERVENTIONAL
Enrollment
56
Start Date
2008-01
Completion Date
2025-06
Last Updated
2026-07-21
Healthy Volunteers
No
Interventions
Reduced-Intensity Conditioning
A pre-transplant conditioning approach designed to provide sufficient immunosuppression to enable donor cell engraftment while minimizing regimen-related toxicity compared to myeloablative conditioning. Regimens are adapted based on patient age and underlying disease characteristics using the following agents: Campath, Fludarabine, Melphalan, Cyclosporine, Cellcept (MMF).
Locations (1)
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States