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

Plain-language summary

This study examines a gene therapy (retroviral SF71-gp91phox transduced CD34+ cells) for X-linked chronic granulomatous disease in children. It enrolled children with CGD who had a history of life-threatening infections and no HLA-matched donor. The study was conducted in Phase 1/Phase 2 and has been completed.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

PHASE1/PHASE2

Status

Completed

Sponsor

University of Zurich

Intervention

retroviral SF71-gp91phox transduced CD34+ cells

Start date

6/1/2004

Estimated completion

9/1/2011

Countries

Switzerland

Related genes

Locations

• Zurich, Switzerland

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine 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)