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Recruiting
NCT03645486
Lentiviral Gene Therapy for CGD
Conditions: Chronic Granulomatous Disease
Sex: All
Healthy volunteers: No
Phase: NA
Enrollment: 10
Sponsor: Shenzhen Geno-Immune Medical Institute
Location: Shenzhen Geno-immune Medical Institute Shenzhen Guangdong
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 Criteria
Inclusion Criteria:
1. CGD patients \>= 0 years of age
2. Molecular diagnosis confirmed by DNA sequencing and supported by laboratory evidence for absent or significantly reduced biochemical activities of the NADPH-oxidase
3. Karnofsky-Index \> =70%
4. At least one prior, ongoing or refractory severe infection and/or inflammatory complications requiring hospitalization despite drug intervention
5. Written informed consent for adult patient, and assent for pediatric subjects seven years or older
Exclusion Criteria:
1. Contraindication for leukapheresis (anaemia Hb \
Source: ClinicalTrials.gov (NCT03645486). StuddyBuddy aggregates publicly available trial information.