Gene Therapy for Chronic Granulomatous Disease in Korea

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

Status

Withdrawn

Phases

Phase 1Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT00778882

Enrollment

Registered

2008-10-24

Start date

2007-01-31

Completion date

2010-10-31

Last updated

2025-09-25

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Chronic Granulomatous Disease

Keywords

Chronic Granulomatous Disease, Retroviral Vector, Gene Therapy

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.

Interventions

DRUGVM106

Autologous hematopoietic stem cells with MT-gp91 retroviral vector

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

MALE

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame
The incidence of adverse events through 1 year	1 year

Secondary

Measure	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	1 year
Presence of vector-positive cells	1 year

Countries

South Korea

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026