Chronic Granulomatous DiseasePhase 1/2NCT00778882
Gene Therapy for Chronic Granulomatous Disease in Korea
A Phase 1/2 study testing VM106 in people with Chronic Granulomatous Disease, run by Helixmith Co., Ltd. at 1 site in South Korea.
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. Read more about this study
- Who is it for?
- Any age, men only
- Conditions the study is for
- Chronic Granulomatous Disease
- What is being tested?
- VM106
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteSouth Korea
- Study dates
- Jan 2007to Oct 2010
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
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- 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)
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
About this study
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
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.
- Conditions
- Chronic Granulomatous Disease
- Being tested
- VM106
- Keywords
- Chronic Granulomatous Disease, Retroviral Vector, Gene Therapy
- Medical subject headings
- Granulomatous Disease, Chronic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VM106Drug
Autologous hematopoietic stem cells with MT-gp91 retroviral vector
| Study group | Role | What they receive |
|---|---|---|
| VM106 | Experimental | Not described Receives: Drug: VM106 |
What is being measured
The primary outcome is the main question the study is designed to answer.
The incidence of adverse events through 1 year
Measured: 1 year
Secondary outcomes (4)
RCR, insertional mutagenesis, immune response against normal gp91 protein
Measured: 1 year
Safety and efficacy of fludarabine/busulfan conditioning
Measured: 1 year
Functional reconstitution of respiratory burst
Measured: 1 year
Presence of vector-positive cells
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Oct 2008 (estimated)
- Study startJan 2007 (estimated)
- Primary completionOct 2008 (estimated)
- Study completionOct 2010 (estimated)
Why it stopped: This Korean Phase 1 study was Withdrawn more than 15 years ago and no data is available.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Chronic Granulomatous DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Helixmith Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Joong Gon Kim, MD, PhD, Principal investigator, Seoul National University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00778882
- Sponsor's protocol ID
- VM106-KR-01
- Status last verified
- Aug 2025
- First submitted
- 21 Oct 2008
- Last updated
- 25 Sep 2025
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT00778882
- Acronym / sponsor ID
- VM106-KR-01
- Conditions
- Chronic Granulomatous Disease
- Keywords
- Chronic Granulomatous Disease, Retroviral Vector, Gene Therapy
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: This Korean Phase 1 study was Withdrawn more than 15 years ago and no data is available.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Helixmith Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- Jan 2007 (estimated)
- Primary completion
- Oct 2008 (estimated)
- Study completion
- Oct 2010 (estimated)
- First posted
- 24 Oct 2008 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| VM106 | Experimental | Drug: VM106 |
Who may qualify
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (10 listed)
- 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.
Key exclusion criteria (6 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VM106Autologous hematopoietic stem cells with MT-gp91 retroviral vector | Drug | VM106 |
Primary outcome
The incidence of adverse events through 1 year
Time frame: 1 year
4 secondary outcomes
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
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Sep 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Joong Gon Kim, MD, PhD principal investigator
Seoul National University Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing VM106 in people with Chronic Granulomatous Disease, run by Helixmith Co., Ltd. at 1 site in South Korea.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 25 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in South Korea. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The incidence of adverse events through 1 year (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Sep 2025.