RecruitingNot Applicable

Lentiviral Gene Therapy for CGD

China10 participantsStarted 2025-07-01

Plain-language 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.

Who can participate

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. 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 criteria

✕. 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

What they're measuring

Overall survival

Timeframe: 15 year follow up

Gene marking in bone marrow cells

Timeframe: 15 year follow up

Trial details

NCT IDNCT03645486

SponsorShenzhen Geno-Immune Medical Institute

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2028-06-30

Contact for this trial

Lung-Ji Chang, Ph.D

86-0755-86725195 c@szgimi.org

View on ClinicalTrials.gov More Chronic Granulomatous Disease trials