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

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

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

Brief 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 Summary:

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 UK, and has been successful in a 3 month old infant at CHOP who engrafted with a haploidentical donor.


Sponsor: Children's Hospital of Philadelphia

Current Primary Outcome: Engraftment and Survival [ Time Frame: Post Transplant -100 days ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

Original Secondary Outcome:

Information By: Children's Hospital of Philadelphia

Dates:
Date Received: January 15, 2010
Date Started: January 2008
Date Completion: December 2018
Last Updated: December 21, 2016
Last Verified: December 2016