Gene Therapy for X-linked Chronic Granulomatous Disease (CGD) in Children (XCGDinChildren)

This study has been completed.
Goethe University
Information provided by (Responsible Party):
University of Zurich Identifier:
First received: June 22, 2009
Last updated: September 26, 2011
Last verified: September 2011
The aim of the study is to evaluate the side effects and risks after infusion of retroviral gene corrected autologous CD34+ cells of the peripheral blood of chemotherapy conditioned (busulphan) children with chronic granulomatous disease (CGD). Also gene corrected and functional active granulocytes in the peripheral blood and the engraftment in the bone marrow of the patients will be monitored an documented.

Condition Intervention Phase
Chronic Granulomatous Disease
Genetic: retroviral SF71-gp91phox transduced CD34+ cells
Phase 1
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Phase I/II Gene Therapy Study for X-linked Chronic Granulomatous Disease in Children

Resource links provided by NLM:

Further study details as provided by University of Zurich:

Primary Outcome Measures:
  • eradication of pre-existing therapy refractory bacterial and/or fungal infections [ Time Frame: 6 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Reconstitution of ROS production by peripheral blood cells [ Time Frame: 1 month ] [ Designated as safety issue: No ]

Enrollment: 2
Study Start Date: June 2004
Study Completion Date: September 2011
Primary Completion Date: December 2010 (Final data collection date for primary outcome measure)
Intervention Details:
    Genetic: retroviral SF71-gp91phox transduced CD34+ cells
    autologous ex-vivo transduced (SF71-gp91phox)CD34+ cells

Ages Eligible for Study:   1 Year to 18 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • x-linked Chronic Granulomatous Disease
  • history of life-threatening severe infections
  • no HLA-matched related or unrelated donor
  • therapy resistent life threatening infections/organ dysfunction
  • no other treatment options e.g. HSCT

Exclusion Criteria:

  • > 18 years of age
  • HIV infection
  • life expectancy > 2 years
  • infections treatable by conventional therapy (antibiotics, antimycotics, allogeneic granulocytes)
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Please refer to this study by its identifier: NCT00927134

University Children's Hospital
Zürich, Switzerland, CH-8032
Sponsors and Collaborators
University of Zurich
Goethe University
Principal Investigator: Reinhard Seger, Prof Dr med University Children's Hospital Zürich
Study Chair: Janine Reichenbach, PD Dr med University Children's Hospital Zürich
Study Chair: Ulrich Siler, Dr rer nat University Children's Hospital Zürich
Study Chair: Manuel Grez, Dr rer nat Georg Speyer Research Institute, Frankfurt a.M.
  More Information

Responsible Party: University of Zurich Identifier: NCT00927134     History of Changes
Other Study ID Numbers: PedsZürich_GT05 
Study First Received: June 22, 2009
Last Updated: September 26, 2011
Health Authority: Switzerland: Federal Office of Public Health

Keywords provided by University of Zurich:
Chronic Granulomatous Disease in children (=or< 18 years)

Additional relevant MeSH terms:
Granulomatous Disease, Chronic
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Hematologic Diseases
Immune System Diseases
Immunologic Deficiency Syndromes
Leukocyte Disorders
Lymphatic Diseases
Lymphoproliferative Disorders
Pathologic Processes
Phagocyte Bactericidal Dysfunction processed this record on May 30, 2016