An Early Phase 1 interventional study of VGN-R08b in Type II Gaucher Disease, sponsored by Xinhua Hospital, Shanghai Jiao Tong University School of Medicine. Recruiting at 1 site in China. Open to participants aged 0 Months to 24 Months. Per ClinicalTrials.gov, last updated 2024-02-22.
Sponsored by Xinhua Hospital, Shanghai Jiao Tong University School of Medicine · Early Phase 1, Interventional, and Treatment
This exploratory trial is to prove the tolerability and safety of VGN-R08b to treat infants with type II Gaucher disease.
Gaucher disease (GD) is an autosomal recessive genetic metabolic disorder. Due to the mutation of Glucocerebrosidase gene (GBA1), the activity of glucocerebrosidase (GCase) in the lysosome of the body is reduced, causing its substrate glucocerceramide to be accumulated in macrophage lysosomes in the liver, spleen, bone, lung, brain and eyes. Type II, acute neuropathy, with extensive and severe visceral involvement, usually develops within the first year of life, and most children die before the age of 2. VGN-R08b is a kind of Gene therapy with adeno-associated virus (AAV) serotype 9 (AAV9) driven human GBA1 being injected directly into intracerebroventricular.
This is a single-center, open, dose-climbing investigator-sponsored exploratory clinical study that included a dose-climbing phase and a dose-expanding phase. The sponsor plans to explore two dose levels in dose-climbing phase (one subject each cohort), then have additional 2\~4 subjects in dose-expanding phase.
This study is to give preliminary evidence for the safety and efficacy of VGN-R08b treatment for patients with type II Gaucher disease.
171 studies on the registry are indexed under Gaucher Disease; 37 are open to participants now.
This study's planned enrollment of 6 is below the median of 20 across 98 interventional studies indexed under Gaucher Disease.
Browse Gaucher Disease studies →Xinhua Hospital, Shanghai Jiao Tong University School of Medicine is the lead sponsor of 222 studies on the registry; 54 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
This is a single-center, open, dose-climbing investigator-sponsored exploratory clinical study that included a dose-climbing phase and a dose-expanding phase. The sponsor plans to explore two dose levels in dose-climbing phase (one subject each cohort), then have additional 2\~4 subjects in dose-expanding phase
Drug: VGN-R08b
VGN-R08b is a kind of Gene therapy with adeno-associated virus (AAV) serotype 9 (AAV9) driven human GBA1 being injected directly into intracerebroventricular.
Number of Adverse Events (AEs), Serious Adverse Events (SAEs)
Adverse Events (AEs), Serious Adverse Events (SAEs)
Time frame: Week 52
Long-term safety follow-up
Number of Adverse Events (AEs), Serious Adverse Events (SAEs)
Time frame: Up to Year 5
Survival ratio at age of 24 months
Survival ratio at age of 24 months
Time frame: Baseline until event, or reach the age of 24 months, Up to Year 5
Changes in the activity of glucose cerebroside lipase (GCase)
Pharmacodynamic indicators
Time frame: Up to Year 5
Changes in the activity of glucose cerebroside (GC) levels
Pharmacodynamic indicators
Time frame: Up to Year 5
Changes in the activity of glucose sphingosine (Lyso GL1) levels in peripheral blood and CSF after medication
Pharmacodynamic indicators
Time frame: Up to Year 5
Immunogenicity
Number of subjects producing antibodies against AAV9 and GCase
Time frame: 26 weeks
Changes in the genomic level of VGN-R08b vector in peripheral blood after medication
Pharmacokinetics
Time frame: 26 weeks
Plan to share: No
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Xinhua Hospital, Shanghai Jiao Tong University School of Medicine