NCT01906541

Gene Therapy for X-CGD

A Phase I/II Gene Therapy Trial for X-CGD With a SIN Gammaretroviral Vector

• Status: Unknown
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 5 (estimated)
• Sponsor: Hubert Serve, Prof., MD · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

X-linked chronic granulomatous disease (X-CGD) is a rare inherited immune defect, which is caused by the inability of phagocytic cells to produce reactive oxygen species due to a defect in the gp91phox subunit of the NADPH oxidase complex. X-CGD patients suffer from recurrent and life-threatening infections and severe hyperinflammatory complications. The only curative treatment for X-CGD is allogenic hematopoietic stem cell transplantation, but this procedure implies severe risks and many patients lack an appropriate donor. Therefore alternative curative approaches are urgently needed. In this study, patients will be treated with gene-corrected autologous CD34+ cells, using a SIN gammaretroviral vector for ex-vivo gene-therapy.

Conditions

• X-linked Chronic Granulomatous Disease

Interventions

Type	Name	Description
GENETIC	ex-vivo gene-therapy	transplantation autologous CD34+ cells, transduced with a SIN gammaretroviral vector

Timeline

• Start date: 2013-07-01
• Primary completion: 2013-12-01
• Completion: 2019-12-01
• First posted: 2013-07-24
• Last updated: 2013-08-02

Locations

1 site across 1 country: Germany