Overview

High Dose Peripheral Blood Stem Cell Transplantation With Post Transplant Cyclophosphamide for Patients With Chronic Granulomatous Disease

Status:
Recruiting

Trial end date:
2025-12-31

Target enrollment:

Participant gender:

Summary

Chronic granulomatous disease (CGD) affects white blood cell function. Currently, the only curative treatment is bone marrow transplant to replace the abnormal stem cells with new ones (donor cells) capable of making a normal immune system. Transplant problems include graft versus host disease (GvHD) and graft rejection. With GvHD, donor cells attack the recipient s normal tissue. Researchers want to use preparation drugs and a high cell dose to increase graft success. They want to use 2 immunosuppressive drugs (cyclophosphamide and sirolimus) to lessen the risk of GvHD.

Phase:

Phase 1/Phase 2

Details

Lead Sponsor:

National Institute of Allergy and Infectious Diseases (NIAID)

Treatments:

Alemtuzumab
Busulfan
Cyclophosphamide
Sirolimus