Status:
WITHDRAWN
Gene Therapy for Chronic Granulomatous Disease in Korea
Lead Sponsor:
Helixmith Co., Ltd.
Conditions:
Chronic Granulomatous Disease
Eligibility:
MALE
Phase:
PHASE1
PHASE2
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 g...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
January 1 2007
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 1 2010
Estimated Enrollment :
Patients enrolled
Trial Details
Trial ID
NCT00778882
Start Date
January 1 2007
End Date
October 1 2010
Last Update
September 25 2025
Active Locations (1)
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1
Seoul National University Hospital
Seoul, South Korea, 110-744