Reduced Intensity Conditioning (RIC) Regimen for Patients With Non-malignant Disorders

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.

NCT Number	NCT01050855
Lead Sponsor	Children's Hospital of Philadelphia
Conditions	Non Malignant Diseases, Immunodeficiencies, Hemoglobinopathies
Enrollment	56 participants
Start Date	2008-01
Primary Completion	2024-07 (estimated)
Study Completion	2025-06 (estimated)
Updated on ClinicalTrials.gov	2026-07-21