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Gene Therapy for Chronic Granulomatous Disease (CGD)

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ClinicalTrials.gov Identifier: NCT00564759
Recruitment Status : Unknown
Verified November 2007 by Johann Wolfgang Goethe University Hospital.
Recruitment status was:  Active, not recruiting
First Posted : November 28, 2007
Last Update Posted : November 28, 2007
Sponsor:
Collaborator:
Information provided by:

Study Description
Brief Summary:
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 or disease Intervention/treatment Phase
Granulomatous Disease, Chronic Drug: retroviral SF71-gp91phox transduced CD34+ cells Phase 1 Phase 2

Study Design

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 2 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Official Title: Phase I/II Gene Therapy Study for X-Linked Chronic Granulomatous Disease
Study Start Date : January 2004
Estimated Study Completion Date : December 2008


Arms and Interventions

Intervention Details:
    Drug: retroviral SF71-gp91phox transduced CD34+ cells
    autologous ex-vivo retroviral transduced (SF71-gp91phox) CD34+ cells

Outcome Measures

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

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

Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00564759


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

Publications:
ClinicalTrials.gov Identifier: NCT00564759     History of Changes
Other Study ID Numbers: 58/59
DeReG 31
KSG 31
First Posted: November 28, 2007    Key Record Dates
Last Update Posted: November 28, 2007
Last Verified: November 2007

Keywords provided by Johann Wolfgang Goethe University Hospital:
CGD

Additional relevant MeSH terms:
Granulomatous Disease, Chronic
Chronic Disease
Granuloma
Disease Attributes
Pathologic Processes
Lymphoproliferative Disorders
Lymphatic Diseases
Phagocyte Bactericidal Dysfunction
Leukocyte Disorders
Hematologic Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Immunologic Deficiency Syndromes
Immune System Diseases