NCT06272149

An Exploratory Clinical Trial of VGN-R08b in Patients With Type II Gaucher Disease

An Exploratory Clinical Trial to Evaluate the Tolerability and Safety of VGN-R08b Via Intracerebroventricular Injection in Patients With Type II Gaucher Disease

• Status: Recruiting
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 6 (estimated)
• Sponsor: Xinhua Hospital, Shanghai Jiao Tong University School of Medicine · Academic / Other
• Sex: All
• Age: 0 Months – 24 Months
• Healthy volunteers: Not accepted

Summary

This exploratory trial is to prove the tolerability and safety of VGN-R08b to treat infants with type II Gaucher disease.

Detailed description

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.

Conditions

• Type II Gaucher Disease

Interventions

Type	Name	Description
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.

Timeline

• Start date: 2023-03-01
• Primary completion: 2025-01-15
• Completion: 2029-02-28
• First posted: 2024-02-22
• Last updated: 2024-02-22

Locations

1 site across 1 country: China