Study Stopped
This Korean Phase 1 study was Withdrawn more than 15 years ago and no data is available.
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
1 other identifier
interventional
N/A
1 country
1
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
Started Jan 2007
Typical duration for phase_1
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
January 1, 2007
CompletedPrimary Completion
Last participant's last visit for primary outcome
October 1, 2008
CompletedFirst Submitted
Initial submission to the registry
October 21, 2008
CompletedFirst Posted
Study publicly available on registry
October 24, 2008
CompletedStudy Completion
Last participant's last visit for all outcomes
October 1, 2010
CompletedSeptember 25, 2025
August 1, 2025
1.8 years
October 21, 2008
September 23, 2025
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
The incidence of adverse events through 1 year
1 year
Secondary Outcomes (4)
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
Study Arms (1)
VM106
EXPERIMENTALInterventions
Eligibility Criteria
You may qualify if:
- 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)
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
Seoul National University Hospital
Seoul, 110-744, South Korea
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Joong Gon Kim, MD, PhD
Seoul National University Hospital
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
October 21, 2008
First Posted
October 24, 2008
Study Start
January 1, 2007
Primary Completion
October 1, 2008
Study Completion
October 1, 2010
Last Updated
September 25, 2025
Record last verified: 2025-08