Lentiviral Gene Therapy for CGD
ConditionChronic Granulomatous Disease
Trial statusRecruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexAll
AgeNot listed
SponsorShenzhen Geno-Immune Medical Institute
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.
Eligibility criteria
Qualifiers
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
Disqualifiers
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
Trial design
Treatments tested in this trial
- Infusion of lentiviral TYF-CGD-modified autologous stem cells
Treatment groups
10 Participants
are divided into 1 treatment groupSponsors and collaborators
Source ClinicalTrials.gov