Gene Therapy for Chronic Granulomatous Disease in Korea

Phase 1

Withdrawn

• Conditions: Chronic Granulomatous Disease
• Interventions: Drug: VM106

• Registration Number: NCT00778882
• Lead Sponsor: Helixmith Co., Ltd.

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 Description

Not available

Study Timeline

• Study Start: 2007-01
• Primary Completion: 2008-10
• Study First Submitted: 2008-10-21
• Study First Submitted QC: 2008-10-23
• Study First Posted: 2008-10-24
• Study Completion: 2010-10
• Status Verified: 2025-03
• Last Update Submitted: 2025-03-21
• Last Update Posted: 2025-03-24

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: Male
• Target Recruitment: 2

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
VM106	VM106	-

Primary Outcome Measures

Name	Time	Method
The incidence of adverse events through 1 year	1 year

Secondary Outcome Measures

Name	Time	Method
RCR, insertional mutagenesis, immune response against normal gp91 protein	1 year
Safety and efficacy of fludarabine/busulfan conditioning	1 year
Functional reconstitution of respiratory burst	1 year
Presence of vector-positive cells	1 year

Trial Locations

Locations (1)

Seoul National University Hospital; 🇰🇷 Seoul, Korea, Republic of