Lentiviral Gene Therapy for CGD
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|ClinicalTrials.gov Identifier: NCT03645486|
Recruitment Status : Recruiting
First Posted : August 24, 2018
Last Update Posted : September 19, 2019
|Condition or disease||Intervention/treatment||Phase|
|Chronic Granulomatous Disease||Genetic: Infusion of lentiviral TYF-CGD-modified autologous stem cells||Not Applicable|
Chronic granulomatous disease (CGD) is a rare disorder caused by inherited defects in the NADPH oxidase multienzyme complex. It is associated with severe and life-threatening bacterial and fungal infections. Approximately two-thirds of all CGD cases result from mutations within the X-linked gp91phox gene (CYBB), followed by the autosomal recessive forms of CGD, with defects in the gene coding for p47phox (NCF1) accounting for 10-30% of all CGD cases.
The primary objectives are to evaluate the safety of the advanced self-inactivating lentiviral vector TYF-CYBB and TYF-NCF1, the ex-vivo gene transfer clinical protocol and the efficacy of immune reconstitution in patients overcoming frequent infections present at the time of treatment, assessment of vector integration sites, and finally the long-term correction of immune dysfunctions.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||10 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Lentiviral Gene Therapy for Chronic Granulomatous Disease (CGD)|
|Actual Study Start Date :||July 1, 2018|
|Estimated Primary Completion Date :||June 30, 2021|
|Estimated Study Completion Date :||December 31, 2021|
Experimental: Lentiviral TYF-CGD-modified autologous stem cells
Autologous hematopoietic stem cells transduced with lentiviral TYF vector carrying the functional gene
Genetic: Infusion of lentiviral TYF-CGD-modified autologous stem cells
Infusion of lentiviral TYF-modified autologous stem cells at 1~10x10^6 gene-modified cells per kg body weight
- Overall survival [ Time Frame: 15 year follow up ]Patient will be monitored for overall health condition, including immune cell assessments, blood biochemistry and metabolitic activities, metabolic detoxification.
- Gene marking in bone marrow cells [ Time Frame: 15 year follow up ]Gene-modified cells in the bone marrow will be measured by vector-specific quantitative PCR of colony-forming cells. Patient overall survival will be followed up for 15 years.
- Change in infection frequency [ Time Frame: 1 year after treatment by clinical history, complete physical examination, haematological and microbiological tests ]
- Recovery of immune function [ Time Frame: 1 year follow up ]Whole blood cell counts (WBC), including CD3+ CD4, CD8 T cells, CD19+ B cells and CD16/CD56 NK cells, and absolute neutrophil counts (ANC), the percentage of NADPH oxidase positive cells, and the kinetics of transduced cells as determined by dihydrorhodamine (DHR) assay, will be measured.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03645486
|Contact: Lung-Ji Chang, Ph.Dfirstname.lastname@example.org|
|Shenzhen Geno-immune Medical Institute||Recruiting|
|Shenzhen, Guangdong, China, China|
|Contact: Lung-Ji Chang, Ph.D 86-0755-86725195 email@example.com|