Gene Therapy for Chronic Granulomatous Disease - Full Text View

Gene Therapy for Chronic Granulomatous Disease (CGD)

This study is ongoing, but not recruiting participants.

Sponsors and Collaborators:	Johann Wolfgang Goethe University Hospitals Bundesministerium für Bildung und Forschung (BMBF), Germany
Information provided by:	Johann Wolfgang Goethe University Hospitals
ClinicalTrials.gov Identifier:	NCT00564759

Purpose

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 I Phase II

Genetics Home Reference related topics: L1 syndrome

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study
Official Title:	Phase I/II Gene Therapy Study for X-Linked Chronic Granulomatous Disease

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:

autologous ex-vivo retroviral transduced (SF71-gp91phox) CD34+ cells

Eligibility

Ages Eligible for Study:	18 Years and older
Genders 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

Please refer to this study by its ClinicalTrials.gov identifier: NCT00564759

Locations

Germany
University Hospital, Hematology
Frankfurt, Germany, 60596

Sponsors and Collaborators

Johann Wolfgang Goethe University Hospitals

Bundesministerium für Bildung und Forschung (BMBF), Germany

Investigators

Principal Investigator:

Dieter Hoelzer, MD, PhD

University Hospital, Frankfurt

More Information

Publications of Results:

Ott MG, Schmidt M, Schwarzwaelder K, Stein S, Siler U, Koehl U, Glimm H, Kühlcke K, Schilz A, Kunkel H, Naundorf S, Brinkmann A, Deichmann A, Fischer M, Ball C, Pilz I, Dunbar C, Du Y, Jenkins NA, Copeland NG, Lüthi U, Hassan M, Thrasher AJ, Hoelzer D, von Kalle C, Seger R, Grez M. Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. Nat Med. 2006 Apr;12(4):401-9. Epub 2006 Apr 2.

Study ID Numbers:	58/59, DeReG 31, KSG 31
Study First Received:	November 26, 2007
Last Updated:	November 27, 2007
ClinicalTrials.gov Identifier:	NCT00564759
Health Authority:	Germany: Paul-Ehrlich-Institut

Keywords provided by Johann Wolfgang Goethe University Hospitals:

CGD

Study placed in the following topic categories:

Lymphatic Diseases
Genetic Diseases, Inborn
Hematologic Diseases
Granulomatous Disease, Chronic
Genetic Diseases, X-Linked
Leukocyte Disorders

Chronic Disease
Chronic granulomatous disease
Lymphoproliferative Disorders
Granuloma
Immunologic Deficiency Syndromes

Additional relevant MeSH terms:

Phagocyte Bactericidal Dysfunction
Disease Attributes
Pathologic Processes
Immune System Diseases

ClinicalTrials.gov processed this record on January 15, 2009