Gene Therapy for Chronic Granulomatous Disease in Korea

Chronic Granulomatous Disease Clinical Trial

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 Chronic Granulomatous Disease Patients

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT00778882
• Other study ID #: VM106-KR-01
• Status: Active, not recruiting
• Phase: Phase 1/Phase 2
• Start date: January 2007
• Est. completion date: October 2022

Study information

• Verified date: October 2019
• Source: Helixmith Co., Ltd.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 2
• Est. completion date: October 2022
• Est. primary completion date: October 2008
• Accepts healthy volunteers: No
• Gender: Male
• Age group: N/A and older

Eligibility

Inclusion Criteria:

• gp91 defective male patients with chronic granulomatous disease: confirmed by DHR

• Weigh greater than or equal to 15 kg

• History of severe infections: more than 2 times

• Performance status: ECOG 0-2

• Patients must be free of significant functional deficits in major organs, but the following eligibility criteria may be modified in individual cases.

• Heart: a shortening fraction > 28%; QTc interval < 0.44

• Liver: total bilirubin < 2 × upper limit of normal; ALT < 3 × upper limit of normal; AST < 3 x upper limit of normal

• Kidney: creatine < 2 x normal

• Blood: WBC > 2,500/uL; platelet > 100,000/uL; hematocrit > 26%

• Written informed consent obtained from patient (or guardian if patients age < 19)

Exclusion Criteria:

• Presence of a HLA-matched sibling for stem cell donation

• Evidence or history of malignant tumor

• Presence of a severe infection

• Presence of an active tuberculosis

• Uncorrectable electrolyte, Ca, P

• Unable to comply with the protocol or to cooperate fully with the Investigator or site personnel

Related Conditions & MeSH terms

• Chronic Granulomatous Disease
• Granuloma
• Granulomatous Disease, Chronic

Intervention

Drug:
• VM106
Autologous hematopoietic stem cells with MT-gp91 retroviral vector

Locations

Country	Name	City	State
Korea, Republic of	Seoul National University Hospital	Seoul

Sponsors (1)

Lead Sponsor	Collaborator
Helixmith Co., Ltd.

Country where clinical trial is conducted

Korea, Republic of, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	The incidence of adverse events through 1 year		1 year
Secondary	RCR, insertional mutagenesis, immune response against normal gp91 protein		1 year
Secondary	Safety and efficacy of fludarabine/busulfan conditioning		1 year
Secondary	Functional reconstitution of respiratory burst		1 year
Secondary	Presence of vector-positive cells		1 year