Lentiviral Gene Therapy for CGD
Lentiviral Gene Therapy for Chronic Granulomatous Disease (CGD)
Shenzhen Geno-Immune Medical Institute
10 participants
Jul 1, 2025
INTERVENTIONAL
Conditions
Summary
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
Inclusion Criteria5
- 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
- Written informed consent for adult patient, and assent for pediatric subjects seven years or older
Exclusion Criteria2
- 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
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Interventions
Infusion of lentiviral TYF-modified autologous stem cells at 1\~10x10\^6 gene-modified cells per kg body weight
Locations(1)
View Full Details on ClinicalTrials.gov
For the most up-to-date information, visit the official listing.
NCT03645486