NCT03645486

Lentiviral Gene Therapy for CGD

Lentiviral Gene Therapy for Chronic Granulomatous Disease (CGD)

• Status: Unknown
• Phase: N/A
• Study type: Interventional
• Enrollment: 10 (estimated)
• Sponsor: Shenzhen Geno-Immune Medical Institute · Academic / Other
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

This is a Phase I/II clinical trial of gene therapy for treating Chronic Granulomatous Disease using a high-safety, high-efficiency, self-inactivating lentiviral vector TYF to functionally correct the defective gene. The objectives are to evaluate the safety and efficacy of the TYF-CGD gene transfer clinical protocol.

Detailed description

Chronic granulomatous disease (CGD) is a rare disorder caused by inherited defects in the NADPH oxidase multienzyme complex. It is associated with severe and life-threatening bacterial and fungal infections. Approximately two-thirds of all CGD cases result from mutations within the X-linked gp91phox gene (CYBB), followed by the autosomal recessive forms of CGD, with defects in the gene coding for p47phox (NCF1) accounting for 10-30% of all CGD cases. The primary objectives are to evaluate the safety of the advanced self-inactivating lentiviral vector TYF-CYBB and TYF-NCF1, the ex-vivo gene transfer clinical protocol and the efficacy of immune reconstitution in patients overcoming frequent infections present at the time of treatment, assessment of vector integration sites, and finally the long-term correction of immune dysfunctions.

Conditions

• Chronic Granulomatous Disease

Interventions

Type	Name	Description
GENETIC	Infusion of lentiviral TYF-CGD-modified autologous stem cells	Infusion of lentiviral TYF-modified autologous stem cells at 1\~10x10\^6 gene-modified cells per kg body weight

Timeline

• Start date: 2018-07-01
• Primary completion: 2021-06-30
• Completion: 2021-12-31
• First posted: 2018-08-24
• Last updated: 2019-09-19

Locations

1 site across 1 country: China