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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 \

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View on ClinicalTrials.gov

Source: ClinicalTrials.gov (NCT03645486). StuddyBuddy aggregates publicly available trial information.