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Low Intensity Preparative Regimen Followed by HLA-Matched, Mobilized Peripheral Blood Stem Cell Transplantation for Chronic Granulomatous Disease

Phase 1
Not Enrolling
Chronic Granulomatous Disease

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Trial Information

Low Intensity Preparative Regimen Followed by HLA-Matched, Mobilized Peripheral Blood Stem Cell Transplantation for Chronic Granulomatous Disease

Chronic Granulomatous Disease (CGD) is an inherited disorder of neutrophil function.
Patients are profoundly immunocompromised, and are plagued early in life with recurrent, and
life threatening infections. Allogeneic stem cell transplantation is the only cure for CGD.

The goal of this phase I/II study is to investigate the safety and efficacy of a novel
approach to allogeneic stem cell transplantation aimed at decreasing transplant related
morbidity and mortality. We will treat patients with CGD with an allogeneic, G-CSF
mobilized peripheral blood stem cell transplant from an HLA identical family member. The
graft will be T-cell depleted which will decrease the incidence of acute graft vs. host
disease. Donor T-cells will be infused post-transplant if engraftment of donor stem cells
is unsatisfactory. The preparative regimen utilized will provide intense immunosuppression
without myeloablation. It is designed to allow donor stem cell engraftment while minimizing
serious transplant related toxicity.

The end points of this study are engraftment, degree of donor-host chimerism, incidence of
acute and chronic GVHD, transplant related morbidity and mortality as well as overall

Inclusion Criteria



Ages 1 to 55 years

DHR proven Chronic Granulomatous Disease: Includes gp91phox, p47phox, p22phox and p67phox

Free of active infection.

Patient has experienced 2 or more prior infections requiring treatment with intravenous
anti-bacterial or anti-fungal therapy.

HIV negative.

No major organ dysfunction precluding transplantation.

HLA identical sibling or parent compatible at all 6 of the HLA A, B, and DR antigens by
serotyping or DNA typing techniques.

Left ventricular ejection fraction: greater than 40% predicted.

ECOG performance status of 0 or 1.


HLA identical sibling donor or parent donor.

Fit to receive G-CSF and give peripheral blood stem cells (normal blood count,
normotensive, no history of stroke, no history of severe heart disease).

Female x-linked CGD carriers must have greater than 30% normal neutrophils.


Patient or donor pregnant.

Age greater than 55 years.

ECOG performance status of 2 or more.

Evidence of rapid deterioration due to progressive infection and/or organ damage.

Left ventricular ejection fraction: greater than or equal to 35 percent predicted.

Creatinine Clearance greater than or equal to 50.

Serum bilirubin greater than or equal to 4 mg/dl, transaminases greater than or equal to
3x upper limit or normal.

HIV negative. Donors who are positive for HBV, HCV or HTLV will be used at the discretion
of the investigator.

Malignant diseases liable to relapse or progress within 5 years.

Donors unfit to receive G-CSF and undergo apheresis. (Uncontrolled hypertension, history
of stroke, history of heart disease, thrombocytopenia.)

Type of Study:


Study Design:

Endpoint Classification: Safety Study, Primary Purpose: Treatment


United States: Federal Government

Study ID:




Start Date:

April 1998

Completion Date:

February 2005

Related Keywords:

  • Chronic Granulomatous Disease
  • Engraftment
  • Minitransplant
  • Fludarabine
  • Neutrophil
  • Allogenic
  • Donor Apheresis
  • Anti-Thymocyte Globin
  • Cyclophosphamide
  • Nonmyeloablative Bone Marrow Transplantation
  • Graft-versus-host Disease
  • Chronic Granulomatous Disease
  • Granulomatous Disease, Chronic
  • Granuloma



National Institute of Allergy and Infectious Diseases (NIAID) Bethesda, Maryland  20892