MedTrace Logo

An Early Clinical Trial to Evaluate VGN-R09b for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency.

NCT05765981 · Status: RECRUITING · Phase: EARLY_PHASE1 · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2023-03-13

No results posted yet for this study

Summary

This early Phase trial is to prove the safety and efficacy of VGN-R09b to treat patients with AADC deficiency.

Conditions

  • Aromatic L-amino Acid Decarboxylase (AADC) Deficiency

Interventions

DRUG

VGN-R09b

VGN-R09b will be injected into bilateral putamen by stereotactic surgery.（Through the SmartFlow Ventricular Cannula or the parenchymal injection tube actually used in the research center）

Sponsors & Collaborators

  • Shanghai Vitalgen BioPharma Co., Ltd.

    collaborator INDUSTRY

  • Shanghai Jiao Tong University School of Medicine

    lead OTHER

Principal Investigators

  • Jiwen Wang, Doctor · Shanghai Children's Medical Center, affiliated to Shanghai Jiao Tong University School of Medicine

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
24 Months
Max Age
7 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-01-30
Primary Completion
2025-02-20
Completion
2029-02-20

Countries

  • China

Study Locations

More Related Trials

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 NCT05765981 on ClinicalTrials.gov