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Stopped: This Korean Phase 1 study was Withdrawn more than 15 years ago and no data is available.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
Sex
MALE
See this in plain English?
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
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.