Gene Therapy for Chronic Granulomatous Disease (CGD)

The recruitment status of this study is unknown because the information has not been verified recently.
Verified November 2007 by Johann Wolfgang Goethe University Hospitals.
Recruitment status was  Active, not recruiting
German Federal Ministry of Education and Research
Information provided by:
Johann Wolfgang Goethe University Hospitals Identifier:
First received: November 26, 2007
Last updated: November 27, 2007
Last verified: November 2007

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)patients 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
Granulomatous Disease, Chronic
Drug: 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
Official Title: Phase I/II Gene Therapy Study for X-Linked Chronic Granulomatous Disease

Resource links provided by NLM:

Further study details as provided by Johann Wolfgang Goethe University Hospitals:

Primary Outcome Measures:
  • safety, toxicity and feasibility [ Time Frame: 2 years ]

Secondary Outcome Measures:
  • Engraftment of gene corrected stem cells, functional reconstitution of respiratory burst, clinical benefit [ Time Frame: 2 years ]

Enrollment: 2
Study Start Date: January 2004
Estimated Study Completion Date: December 2008
Intervention Details:
    Drug: retroviral SF71-gp91phox transduced CD34+ cells
    autologous ex-vivo retroviral transduced (SF71-gp91phox) CD34+ cells

Ages Eligible for Study:   18 Years and older
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 non-related donor
  • therapy resistent life threatening infections/organ dysfunction
  • no other treatment options e.g. BMT

Exclusion Criteria:

  • < 18 years of age
  • HIV infection
  • life expectancy > 2 years
  • infections treatable by conventional therapy (antibiotics, allogeneic granulocytes)
  Contacts and Locations
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Please refer to this study by its identifier: NCT00564759

University Hospital, Hematology
Frankfurt, Germany, 60596
Sponsors and Collaborators
Johann Wolfgang Goethe University Hospitals
German Federal Ministry of Education and Research
Principal Investigator: Dieter Hoelzer, MD, PhD University Hospital, Frankfurt
  More Information

Publications: Identifier: NCT00564759     History of Changes
Other Study ID Numbers: 58/59, DeReG 31, KSG 31
Study First Received: November 26, 2007
Last Updated: November 27, 2007
Health Authority: Germany: Paul-Ehrlich-Institut

Keywords provided by Johann Wolfgang Goethe University Hospitals:

Additional relevant MeSH terms:
Granulomatous Disease, Chronic
Chronic Disease
Disease Attributes
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 October 07, 2015