Gene Therapy for Chronic Granulomatous Disease in Korea

September 23, 2025 updated by: Helixmith Co., Ltd.

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

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.

Study Overview

Status

Withdrawn

Conditions

Intervention / Treatment

Study Type

Interventional

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • South Korea
      • Seoul, South Korea, 110-744
        • Seoul National University Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: VM106
Drug: VM106
Autologous hematopoietic stem cells with MT-gp91 retroviral vector

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
The incidence of adverse events through 1 year
Time Frame: 1 year
1 year

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Helixmith Co., Ltd.

Investigators

  • Principal Investigator: Joong Gon Kim, MD, PhD, Seoul National University Hospital

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

January 1, 2007

Primary Completion (Estimated)

October 1, 2008

Study Completion (Estimated)

October 1, 2010

Study Registration Dates

First Submitted

October 21, 2008

First Submitted That Met QC Criteria

October 23, 2008

First Posted (Estimated)

October 24, 2008

Study Record Updates

Last Update Posted (Estimated)

September 25, 2025

Last Update Submitted That Met QC Criteria

September 23, 2025

Last Verified

August 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • VM106-KR-01

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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