Lentiviral Gene Therapy for CGD

Recruiting now · Not applicable

Conditions studied: Chronic Granulomatous Disease

In brief

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.

Key facts

Study ID

NCT03645486

Run by

Shenzhen Geno-Immune Medical Institute

People needed

10

Starts

2025-07-01

Expected to finish

2029-12-31

Last updated by the study team

2026-06-22

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• Shenzhen Geno-immune Medical Institute — Shenzhen, Guangdong, China (enrolling)

Full record on ClinicalTrials.gov

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