Lentiviral Gene Therapy for CGD

Plain-language summary

This study examines a gene therapy in which the patient's own stem cells are genetically modified with a lentiviral approach and returned, in patients with chronic granulomatous disease. Eligible participants have a diagnosis confirmed by DNA sequencing and at least one severe infection or inflammatory complication requiring hospitalization. It is being conducted as a study without a formal phase and is currently recruiting participants.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

Not Applicable

Status

Recruiting

Sponsor

Shenzhen Geno-Immune Medical Institute

Intervention

Infusion of lentiviral TYF-CGD-modified autologous stem cells

Start date

7/1/2025

Estimated completion

12/31/2029

Countries

China

Related genes

Locations

• Shenzhen, China

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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 \<8g/dl, cardiovascular instability, severe coagulopathy) or for administration of conditioning medication
2. Female patients who are pregnant or lactating as determined by history and/or positive pregnancy test