Gene Therapy for Chronic Granulomatous Disease in Korea | NCT00778882

Studies
Gene Therapy for Chronic Granulomatous Disease in Korea

NCT00778882

Sponsor

Helixmith Co., Ltd.

Status

Withdrawn

Last Update Posted

Sep 25, 2025Actual

Enrollment

0Actual

Phase

Phase 1Phase 2

Conditions

Chronic Granulomatous Disease

Interventions

VM106

Countries

South Korea

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Aug 2025

Overall Recruitment Status or Expanded Access Status

Withdrawn

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

This Korean Phase 1 study was Withdrawn more than 15 years ago and no data is available.

Expanded Access Info

Start Date

Jan 2007Estimated

Primary Completion Date

Oct 2008Estimated

Completion Date

Oct 2010Estimated

First Submitted Date

Oct 21, 2008

First Submission Date that Met QC Criteria

Oct 23, 2008

First Posted Date

Oct 24, 2008Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Sep 23, 2025

Last Update Posted Date

Sep 25, 2025Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 1Phase 2

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

0Actual

Label	Type	Description	Intervention Names
VM106	Experimental		Drug: VM106

Name	Type	Description	Arm Group Labels	Other Names
VM106	Drug	Autologous hematopoietic stem cells with MT-gp91 retroviral vector	VM106

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
The incidence of adverse events through 1 year		1 year

Secondary Outcomes

Measure	Description	Time Frame
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

Other Outcomes

Not provided

Eligibility Module

Eligibility 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

Accepts Healthy Volunteers

Sex

Male

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

Not provided

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

ID	Term
D010585	Phagocyte Bactericidal Dysfunction
D007960	Leukocyte Disorders
D006402	Hematologic Diseases
D006425	Hemic and Lymphatic Diseases