MedTrace Logo

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

NCT01050855 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 56

Last updated 2026-07-21

Study results available
· View outcomes & findings →

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.

Conditions

  • Non Malignant Diseases
  • Immunodeficiencies
  • Hemoglobinopathies

Interventions

DRUG

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).

Sponsors & Collaborators

Principal Investigators

  • Timothy J Olson, MD · Children's Hospital of Philadelphia

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
6 Months
Max Age
25 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2008-01-31
Primary Completion
2024-07-31
Completion
2025-06-30

Countries

  • United States

Study Locations

More Related Trials

Entities

Companies

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 NCT01050855 on ClinicalTrials.gov