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

Hemoglobinopathies Clinical Trial

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

Clinical Trial 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.

Clinical Trial 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. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT01050855
Study type Interventional
Source Children's Hospital of Philadelphia
Contact
Status Active, not recruiting
Phase Phase 2
Start date January 2008
Completion date December 2026
View Details
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