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A Trial to Evaluate Safety and Efficacy of a Product Named VGN-R09b in Severe AADC Deficiency

NCT06432140 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL · Enrollment: 13

Last updated 2026-04-21

No results posted yet for this study

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

Conditions

  • Aromatic L-amino Acid Decarboxylase Deficiency

Interventions

GENETIC

VGN-R09b injection

Two levels of VGN-R09b will be injected into bilateral putamen in dose-escalating phase, and one dose level will be injected in dose confirming phase

Sponsors & Collaborators

  • Shanghai Vitalgen BioPharma Co., Ltd.

    lead INDUSTRY

Principal Investigators

  • yi Wang, MD · Children's Hospital of Fudan University

  • jiwen Wang, MD · Shanghai Children's Medical Center

  • yunhai song, MD · Shanghai Children's Medical Center

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Months
Max Age
8 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-07-02
Primary Completion
2026-07-23
Completion
2030-09-20

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 NCT06432140 on ClinicalTrials.gov