Lentiviral Gene Therapy for CGD
ConditionChronic Granulomatous Disease
Trial statusNot yet recruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexAll
AgeNot listed
SponsorShenzhen Geno-Immune Medical Institute
About this trial
This is a Phase I/II clinical trial of gene therapy for treating Chronic Granulomatous Disease using a high-safety, high-efficiency, self-inactivating lentiviral vector TYF to functionally correct the defective gene. The objectives are to evaluate the safety and efficacy of the TYF-CGD gene transfer clinical protocol.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
CGD patients >= 0 years of age
Molecular diagnosis confirmed by DNA sequencing and supported by laboratory evidence for absent or significantly reduced biochemical activities of the NADPH-oxidase
Karnofsky-Index > =70%
At least one prior, ongoing or refractory severe infection and/or inflammatory complications requiring hospitalization despite drug intervention
Disqualifiers
Contraindication for leukapheresis (anaemia Hb <8g/dl, cardiovascular instability, severe coagulopathy) or for administration of conditioning medication
Female patients who are pregnant or lactating as determined by history and/or positive pregnancy test
Trial design
Design model
Single group
Treatments tested in this trial
Infusion of lentiviral TYF-CGD-modified autologous stem cells
GeneticInfusion of lentiviral TYF-modified autologous stem cells at 1\~10x10\^6 gene-modified cells per kg body weight
Treatment groups
10 Participants
are divided into 1 treatment groupGroup A: Lentiviral TYF-CGD-modified autologous stem cellsExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Overall survival
Patient will be monitored for overall health condition, including immune cell assessments, blood biochemistry and metabolitic activities, metabolic detoxification.
Time frame
15 year follow up2
Gene marking in bone marrow cells
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.
Time frame
15 year follow upSecondary outcomes
1
Change in infection frequency
Time frame
1 year after treatment by clinical history, complete physical examination, haematological and microbiological tests2
Recovery of immune function
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.
Time frame
1 year follow upSponsors and contacts
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Source ClinicalTrials.gov
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