Clinical Trial: Gene Therapy for Chronic Granulomatous Disease in Korea
Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional
Official Title: An Open-label, Uncontrolled, Single Center, Phase I/II Trial to Assess the Safety and Efficacy of Autologous Hematopoietic Stem Cells Transduced With MT-gp91 Retroviral Vector in gp91 Defective
Brief Summary: The purpose of this study is to evaluate the safety and efficacy of administration of autologous hematopoietic stem cells transduced with MT-gp91 retroviral vector for patients with X-linked chronic granulomatous disease.
Detailed Summary:
Sponsor: ViroMed Co., Ltd. dba VM BioPharma
Current Primary Outcome: The incidence of adverse events through 1 year [ Time Frame: 1 year ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- RCR, insertional mutagenesis, immune response against normal gp91 protein [ Time Frame: 1 year ]
- Safety and efficacy of fludarabine/busulfan conditioning [ Time Frame: 1 year ]
- Functional reconstitution of respiratory burst [ Time Frame: 1 year ]
- Presence of vector-positive cells [ Time Frame: 1 year ]
Original Secondary Outcome: Same as current
Information By: ViroMed Co., Ltd. dba VM BioPharma
Dates:
Date Received: October 21, 2008
Date Started: January 2007
Date Completion: October 2022
Last Updated: February 10, 2016
Last Verified: February 2016
