Lentiviral gene therapy for chronic granulomatous disease.
Lentiviral Gene Therapy for Chronic Granulomatous Disease (CGD)
This trial will test whether a modified lentivirus used to correct patients' own stem cells can restore immune function and reduce severe infections in people with chronic granulomatous disease.
Quick facts
| Phase | Not applicable |
|---|---|
| Study type | Interventional |
| Enrollment | 10 (estimated) |
| Sex | All |
| Sponsor | Shenzhen Geno-Immune Medical Institute Academic / other |
| Locations | 1 site (Shenzhen, Guangdong) |
| Trial ID | NCT03645486 on ClinicalTrials.gov |
What this trial studies
This Phase I/II trial uses a high-safety, self-inactivating lentiviral vector (TYF) to deliver functional CYBB or NCF1 genes into patients' autologous hematopoietic stem cells ex vivo. Collected stem cells are genetically modified with TYF-CYBB or TYF-NCF1 and then infused back into the patient after conditioning, with scheduled follow-up visits to monitor safety and clinical response. The protocol includes molecular monitoring of vector integration sites and assays of NADPH oxidase activity to track immune reconstitution. Participants are children and adults with genetically confirmed CGD and a history of severe, ongoing, or refractory infections or inflammatory complications.
Who should consider this trial
Good fit: Ideal candidates are children or adults with molecularly confirmed CGD (e.g., CYBB or NCF1 mutations), significant recurrent or refractory infections or inflammatory complications, adequate performance status (Karnofsky ≥70%), and the ability to undergo leukapheresis and conditioning.
Not a fit: Patients who cannot tolerate leukapheresis or conditioning, pregnant or lactating individuals, or those without significant clinical complications are less likely to benefit from this protocol.
Why it matters
Potential benefit: If successful, the therapy could restore NADPH oxidase function and substantially reduce life-threatening bacterial and fungal infections in people with CGD.
How similar studies have performed: Previous gene therapy trials for CGD using lentiviral vectors have produced promising immune reconstitution in early studies, so this approach builds on prior positive but still evolving clinical experience.
Eligibility criteria
Show full inclusion / exclusion 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 \<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
Where this trial is running
Shenzhen, Guangdong
- Shenzhen Geno-immune Medical Institute — Shenzhen, Guangdong, China (Recruiting)
Study contacts
- Study coordinator: Lung-Ji Chang, Ph.D
- Email: c@szgimi.org
- Phone: 86-0755-86725195
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.