Lentiviral Gene Therapy for CGD
Recruiting
N/A
Interventional Study
Chronic Granulomatous Disease
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
At a Glance
- Sex
- Any
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 10 (estimated)
- Sponsor
- Shenzhen Geno-Immune Medical Institute · Other
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About This Trial
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.
Trial Locations
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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 seve…
Contacts