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

This study has been completed.

Sponsor:

Collaborator:

Goethe University

Information provided by (Responsible Party):

University of Zurich

ClinicalTrials.gov Identifier:

NCT00927134

First received: June 22, 2009

Last updated: September 26, 2011

Last verified: September 2011

History of Changes

Tracking Information

First Received Date ^ICMJE

June 22, 2009

Last Updated Date

September 26, 2011

Start Date ^ICMJE

June 2004

Primary Completion Date

December 2010 (final data collection date for primary outcome measure)

Current Primary Outcome Measures ^ICMJE

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

Original Primary Outcome Measures ^ICMJE

Same as current

Change History

Complete list of historical versions of study NCT00927134 on ClinicalTrials.gov Archive Site

Current Secondary Outcome Measures ^ICMJE

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

Original Secondary Outcome Measures ^ICMJE

Same as current

Current Other Outcome Measures ^ICMJE

Not Provided

Original Other Outcome Measures ^ICMJE

Not Provided

Descriptive Information

Brief Title ^ICMJE

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

Official Title ^ICMJE

Phase I/II Gene Therapy Study for X-linked Chronic Granulomatous Disease in Children

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) 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.

Detailed Description

Not Provided

Study Type ^ICMJE

Interventional

Study Phase

Phase 1
Phase 2

Study Design ^ICMJE

Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment

Condition ^ICMJE

Chronic Granulomatous Disease

Intervention ^ICMJE

Genetic: retroviral SF71-gp91phox transduced CD34+ cells

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

Study Arm (s)

Not Provided

Publications *

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.

* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.

Recruitment Information

Recruitment Status ^ICMJE

Completed

Enrollment ^ICMJE

Completion Date

September 2011

Primary Completion Date

December 2010 (final data collection date for primary outcome measure)

Eligibility Criteria ^ICMJE

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)

Gender

Male

Ages

1 Year to 18 Years

Accepts Healthy Volunteers

Contacts ^ICMJE

Contact information is only displayed when the study is recruiting subjects

Location Countries ^ICMJE

Switzerland

Administrative Information

NCT Number ^ICMJE

NCT00927134

Other Study ID Numbers ^ICMJE

PedsZürich_GT05

Has Data Monitoring Committee

Responsible Party

University of Zurich

Study Sponsor ^ICMJE

University of Zurich

Collaborators ^ICMJE

Goethe University

Investigators ^ICMJE

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.

Information Provided By

University of Zurich

Verification Date

September 2011

^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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