Gene Therapy for X-CGD

X-linked Chronic Granulomatous Disease Clinical Trial

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

Status Recruiting

Clinical Trial 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.

Clinical Trial Description

n/a

Study Design

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

Related Conditions & MeSH terms

• Granuloma
• Granulomatous Disease, Chronic
• X-linked Chronic Granulomatous Disease

• NCT number: NCT01906541
• Study type: Interventional
• Source: Johann Wolfgang Goethe University Hospitals
• Contact: Hubert Serve, Prof., MD
• Phone: 0049/69/6301
• Email: serve@em.uni-frankfurt.de
• Status: Recruiting
• Phase: Phase 1/Phase 2
• Start date: July 2013
• Completion date: December 2019