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A Study to Evaluate the Tolerability, Safety and Efficacy of VGM-R02b

NCT06217861 · Status: RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2026-05-13

No results posted yet for this study

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.

Conditions

  • Glutaric Acidemia Type I
  • Glutaric Aciduria Type I

Interventions

BIOLOGICAL

VGM-R02b

Administered as specified in the treatment arm.

Sponsors & Collaborators

  • Shanghai Vitalgen BioPharma Co., Ltd.

    lead INDUSTRY

Principal Investigators

  • Qiang Shu · The Children's Hospital of Zhejiang University School of Medicine

  • Rulai Yang · The Children's Hospital of Zhejiang University School of Medicine

  • Guanping Dong · The Children's Hospital of Zhejiang University School of Medicine

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
6 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-04-29
Primary Completion
2027-08-31
Completion
2027-08-31

Countries

  • China

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06217861 on ClinicalTrials.gov