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

Chronic Granulomatous Disease Clinical Trial

— XCGDinChildren  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00927134
• Other study ID #: PedsZürich_GT05
• Status: Completed
• Phase: Phase 1/Phase 2
• First received: June 22, 2009
• Last updated: September 26, 2011
• Start date: June 2004
• Est. completion date: September 2011

Study information

• Verified date: September 2011
• Source: University of Zurich
• Contact: n/a
• Is FDA regulated: No
• Health authority: Switzerland: Federal Office of Public Health
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 2
• Est. completion date: September 2011
• Est. primary completion date: December 2010
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 1 Year to 18 Years

Eligibility

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)

Study Design

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

Related Conditions & MeSH terms

• Chronic Granulomatous Disease
• Granuloma
• Granulomatous Disease, Chronic

Intervention

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

Locations

Country	Name	City	State
Switzerland	University Children's Hospital	Zürich

Sponsors (2)

Lead Sponsor	Collaborator
University of Zurich	Goethe University

Country where clinical trial is conducted

Switzerland, 

References & Publications (1)

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. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	eradication of pre-existing therapy refractory bacterial and/or fungal infections		6 months	No
Secondary	Reconstitution of ROS production by peripheral blood cells		1 month	No