Ignite Creation Date:
2024-05-06 @ 8:00 PM
Last Modification Date:
2024-10-26 @ 3:18 PM
Study NCT ID:
NCT06217861
Status:
RECRUITING
Last Update Posted:
2024-05-17
First Post:
2023-12-12
Brief Title:
A Study to Evaluate the Tolerability Safety and Efficacy of VGM-R02b
Sponsor:
Shanghai Vitalgen BioPharma Co Ltd
Organization:
Shanghai Vitalgen BioPharma Co Ltd
Study Overview
Official Title:
An Open-Label Dose-Escalation and Dose-Expansion Phase I Clinical Study to Evaluate the Tolerability Safety and Efficacy of VGM-R02b in Patients With Glutaric Acidemia Type I
Status:
RECRUITING
Status Verified Date:
2024-01
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:
Phase I open-label single-arm single-dose trial of VGM-R02b gene replacement therapy in patients with Glutaric Acidemia Type I GA-I who meet enrollment criteria and are genetically confirmed by GCDH gene mutation 1 to 3 patients aged 6 years at the time of screening will be enrolled in each dose group in the dose escalation part In the dose expansion part the sample size will be statistically calculated and adjusted according to the efficacy and safety data in the dose escalation part
Detailed Description:
This study consists of screening period treatment period and postoperative monitoring period and follow-up period During the screening period Days -28 to -1 patients whose parentslegal guardians provide informed consent will complete screening procedures to determine eligibility for trial enrollment Eligible subjects will be admitted to the clinical research center before surgical administration to complete the preoperative examination and determine the surgical plan Ommaya fluid reservoir capsule implantation will be used in this study if there were problems with Ommaya implantation intra-cerebroventricular injection could also be used for drug administration The day of administration set to be D1 Prophylactic immunosuppressive therapy including Methylprednisolone Prednisolone and Rapamycin was initiated on D1 Then all the examinations during the 7-day postoperative observation period will be completed based on the evaluation time point specified in the Schedule of Assessments table Subjects may be discharged 7 days after the infusion based on Investigator judgment During the outpatient follow-up period up to 52 weeks after administration subjects will return at regularly scheduled intervals for efficacy and safety assessments until the End of Trial After the End of Trial visit eligible patients will be asked to participate into the long-term follow up trial
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
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 2023LP01348 | OTHER | CDE | None |