Gene Therapy for Chronic Granulomatous Disease in Korea

NCT ID: NCT00778882

Last Updated: 2025-09-25

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

WITHDRAWN

Clinical Phase

PHASE1/PHASE2

Study Classification

INTERVENTIONAL

Study Start Date

2007-01-31

Study Completion Date

2010-10-31

Brief Summary

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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

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Conditions

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Chronic Granulomatous Disease

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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VM106

Group Type EXPERIMENTAL

VM106

Intervention Type DRUG

Autologous hematopoietic stem cells with MT-gp91 retroviral vector

Interventions

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VM106

Autologous hematopoietic stem cells with MT-gp91 retroviral vector

Intervention Type DRUG

Eligibility Criteria

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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
Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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Helixmith Co., Ltd.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Joong Gon Kim, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Seoul National University Hospital

Locations

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Seoul National University Hospital

Seoul, , South Korea

Site Status

Countries

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South Korea

Other Identifiers

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VM106-KR-01

Identifier Type: -

Identifier Source: org_study_id

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