Gene Therapy for Chronic Granulomatous Disease in Korea

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT00778882
• Date of registration: 21/10/2008
• Prospective Registration: No
• Primary sponsor: Helixmith Co., Ltd.
• Public title: Gene Therapy for Chronic Granulomatous Disease in Korea
• Scientific 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
• Date of first enrolment: January 2007
• Target sample size: 2
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/show/NCT00778882
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1/Phase 2

Countries of recruitment

Korea, Republic of

Contacts

• Name: Joong Gon Kim, MD, PhD
• Affiliation: Seoul National University Hospital

Key inclusion & exclusion criteria

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



Age minimum: N/A
Age maximum: N/A
Gender: Male

Health Condition(s) or Problem(s) studied

Chronic Granulomatous Disease

Intervention(s)

Drug: VM106

Primary Outcome(s)

The incidence of adverse events through 1 year [Time Frame: 1 year]

Secondary Outcome(s)

Presence of vector-positive cells [Time Frame: 1 year]

Safety and efficacy of fludarabine/busulfan conditioning [Time Frame: 1 year]

Functional reconstitution of respiratory burst [Time Frame: 1 year]

RCR, insertional mutagenesis, immune response against normal gp91 protein [Time Frame: 1 year]

Secondary ID(s)

VM106-KR-01

Source(s) of Monetary Support

Please refer to primary and secondary sponsors