Ignite Creation Date:
2024-06-16 @ 11:49 AM
Last Modification Date:
2024-10-26 @ 3:30 PM
Study NCT ID:
NCT06432140
Status:
NOT_YET_RECRUITING
Last Update Posted:
2024-06-03
First Post:
2024-05-17
Brief Title:
A Trial to Evaluate Safety and Efficacy of a Product Named VGN-R09b in Severe AADC Deficiency
Sponsor:
Shanghai Vitalgen BioPharma Co Ltd
Organization:
Shanghai Vitalgen BioPharma Co Ltd
Study Overview
Official Title:
An Open Dose-escalating and Dose Confirmation Trial to Evaluate the Safety and Efficacy of VGN-R09b by Intra Putamen Injection in Patients With Severe Aromatic L-amino Acid Decarboxylase AADC Deficiency
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-05
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 trial includes dose-escalating part phase 1 and dose confirming part to prove the safety and efficacy of VGN-R09b to treat patients with severe AADC deficiency
Detailed Description:
Aromatic L-amino acid decarboxylase AADC is an enzyme responsible for the final step in the synthesis of neurotransmitters dopamine and serotonin AADC deficiency is a rare genetic disorder VGN-R09b is a kind of Gene therapy with adeno-associated virus AAV serotype 9 AAV9 driven human AADC hAADC being injected directly into putamen
This is an open dose-escalating and dose confirming study The sponsor plans to explore two dose levels 601011vg and 1281012vg in dose-escalating phase three subjects each cohort then plans to have 10 subjects enrolled for dose confirmation phase
This study is to give evidence for the safety and efficacy of VGN-R09b treatment for patients with severe Aromatic L-amino acid decarboxylase AADC deficiency
This is an open dose-escalating and dose confirming study The sponsor plans to explore two dose levels 601011vg and 1281012vg in dose-escalating phase three subjects each cohort then plans to have 10 subjects enrolled for dose confirmation phase
This study is to give evidence for the safety and efficacy of VGN-R09b treatment for patients with severe Aromatic L-amino acid decarboxylase AADC deficiency
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