Ignite Creation Date:
2024-05-06 @ 8:09 PM
Last Modification Date:
2024-10-26 @ 3:21 PM
Study NCT ID:
NCT06272149
Status:
RECRUITING
Last Update Posted:
2024-02-22
First Post:
2023-07-17
Brief Title:
An Exploratory Clinical Trial of VGN-R08b in Patients With Type II Gaucher Disease
Sponsor:
Xinhua Hospital Shanghai Jiao Tong University School of Medicine
Organization:
Xinhua Hospital Shanghai Jiao Tong University School of Medicine
Study Overview
Official Title:
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
Status Verified Date:
2023-02
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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 24 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
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 24 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
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None