Gene Therapy for Chronic Granulomatous Disease in Korea (NCT00778882) | Clinical Trial Compass
WithdrawnPhase 1/2
Gene Therapy for Chronic Granulomatous Disease in Korea
Stopped: This Korean Phase 1 study was Withdrawn more than 15 years ago and no data is available.
South Korea0Started 2007-01
Plain-language 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.
Who can participate
SexMALE
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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