NCT01050855

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

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 75 (estimated)
• Sponsor: Children's Hospital of Philadelphia · Academic / Other
• Sex: All
• Age: 6 Months – 25 Years
• Healthy volunteers: Not accepted

Summary

This is a Phase II pilot study to evaluate engraftment and toxicity of patients with non-malignant diseases using a reduced intensity conditioning regimen in the setting of allogeneic transplant for non malignant diseases. Bone Marrow or cord blood will be acceptable as a stem cell source. Recently, reduced intensity conditioning (RIC) regimens have been used for both adult patients with leukemias and pediatric patients with non-malignant diseases. These regimens are better tolerated, resulting in less transplant related morbidity and mortality. Stable mixed chimerism, while insufficient for eradication of leukemias, may be sufficient to cure patients with non-malignant diseases.

Detailed description

There are two conditioning regimens in this protocol for children \>6 months. Alemtuzumab (Campath), Fludarabine and Melphalan are used. The regimens differ by the timing and dosing of Alemtuzumab (Campath). The two timings are distal and intermediate. * Distal campath is initiated 22 days prior to the allogeneic transplant. * Intermediate campath is initiated 14 days prior to allogeneic transplant. The conditioning regimen for children with immunodeficiencies \<6 months omits melphalan, and substitutes two days of busulfan. This regimen is successfully used in the United Kingdom, and has been successful in a 3 month old infant at the Children's Hospital of Philadelphia (CHOP) who engrafted with a haploidentical donor.

Conditions

• Non Malignant Diseases
• Immunodeficiencies
• Hemoglobinopathies

Interventions

Type	Name	Description
DRUG	RIC: Distal Campath	Campath, Fludarabine, Melphalan, Cyclosporine, Cellcept (MMF)
DRUG	RIC:Intermediate Campath	Campath, Fludarabine, Melphalan, Cyclosporine, Cellcept (MMF)
DRUG	RIC: Mini Busulfan	Campath, Fludarabine, Busulfan, Cyclosporine, Cellcept (MMF)

Timeline

• Start date: 2008-01-01
• Primary completion: 2025-12-01
• Completion: 2026-12-01
• First posted: 2010-01-18
• Last updated: 2024-07-25

Locations

1 site across 1 country: United States