Active, not recruitingNCT06432140Updated Apr 21, 2026

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

A Phase 2/3 interventional study of VGN-R09b injection in Aromatic L-amino Acid Decarboxylase Deficiency, sponsored by Shanghai Vitalgen BioPharma Co., Ltd.. Active, not recruiting at 1 site in China. Open to participants aged 18 Months to 8 Years. Per ClinicalTrials.gov, last updated 2026-04-21.

Sponsored by Shanghai Vitalgen BioPharma Co., Ltd. · Phase 2/3, Interventional, and Treatment

Phase: Phase 2/3
Study type: Interventional
Enrollment: 13
Allocation: Not applicable
Ages: 18 Months to 8 Years
Sex: All

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

Read the 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 (6.0×1011vg and 1.28×1012vg) 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.

Conditions studied

Aromatic L-amino Acid Decarboxylase Deficiency

Keywords

AADC
AAV9
Central Nervous System (CNS) gene therapy

In context

Lead sponsor

Shanghai Vitalgen BioPharma Co., Ltd. is the lead sponsor of 10 studies on the registry; 4 are open to participants now.

Counted across the registry records on this site, refreshed daily.

Who can participate

Ages eligible: 18 Months to 8 Years
Sexes eligible: All
Accepts healthy volunteers: No

Inclusion criteria

The child patient has to be ≥18 months old and \< 8 years old, and a head circumference big enough for surgery as judged by investigator.
Historical diagnosis of AADC deficiency with clinical symptoms consistency, AND with Molecular genetic confirmation of homozygous or compound heterozygous mutation point of IVS6+4A>T in DDC gene.
With Plasma AADC activity less than or equal to 12 pmol/min/mL.
Motor development at baseline \<3 months (head fully uncontrollable at baseline）, and Failed to benefit from standard medical therapy (dopamine agonists, monoamine oxidase inhibitor or related form of Vitamin B6) at discretion of investigators.
Parent(s)/legal guardian(s) with custody of subject must give their consent for subject to enroll in the study.
Parent(s)/legal guardian(s) of the subject must agree to comply with the requirements of the study, including providing disease information and support disease assessment of symptoms.

Exclusion criteria

Exclusion Criteria:

Intracranial neoplasm or any structural brain abnormality or lesion (e.g., severe brain atrophy, white matter degenerative changes), which, in the opinion of the study investigators, would confer excessive risk and/or inadequate potential for benefit.
Presence of other significant medical or neurological conditions that would create an unacceptable operative or anesthetic risk (including congenital heart disease, respiratory disease with home oxygen requirement, history of serious anesthesia complications during previous elective procedures, history of cardiorespiratory arrest), liver or renal failure, malignancy, or HIV positive.
Severe coagulopathy, or need for ongoing anticoagulant therapy.
clinically active infection or with severe infection within 12 weeks before screening (e.g. adenovirus or herpes virus, pneumonia, sepsis, central nervous system infection).
Previous stereotactic neurosurgery, or any gene/cell therapy.
Received live vaccination within 4 weeks.
Contraindication to sedation during surgery or imaging studies (PET or MRI).

Study design

Phase: Phase 2 / Phase 3
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrollment: 13 participants (actual)

Study arms

Experimental
VGN-R09b injection
Different levels of VGN-R09b will be injected into bilateral putamen by stereotactic surgery
Genetic: VGN-R09b injection

Interventions

GeneticVGN-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

What researchers measure

Primary outcomes

Number of Adverse Events (AEs), Serious Adverse Events (SAEs)
Vital signs, physical examination, laboratory test will be monitored after drug injection
Time frame: up to Week 52
Number of subjects who achieved motor development milestones
Four milestones, including Head control, Sit independently, Stand/stepping with support, Walk with minimal assistant, would be assessed according to definition in Peabody Developmental Motor Scale 2nd edition (PDMS-2). Each milestone would be scored as 0, 1 or 2, and score 2 means achievement of the milestone.
Time frame: up to 24 months

Secondary outcomes

Change in brain AADC activity
Increase in signal in the putamen and nigra on Fluorodopa-PET imaging as brain AADC activity measure
Time frame: up to 5 Years
Change in Cerebrospinal Fluid (CSF) neurotransmitter metabolite concentrations
Neurotransmitter metabolite concentrations of Homovanillic Acid/Hydroxyindoleacetic Acid (HVA/5-HIAA) would be measured
Time frame: up to 5 Years
Change from baseline in motor function
Motor function would be assessed by Peabody Developmental Motor Scale 2nd edition (PDMS-2). The score ranges from 0 to 482, and higher score means the better in motor function.
Time frame: up to 5 Years
Change in number of Clinical symptoms
Number of disease related symptoms
Time frame: up to 5 Years
Viral shedding
Concentrations of Viral genome in serum/urine would be measured
Time frame: up to 1 week
Immunogenicity after injection
Subject number with positive antibodies of AAV9/AADC/Glial Cell Line-Derived Neurotrophic Factor (GDNF) in blood would be reported
Time frame: up to 26 weeks
Number of Adverse Events (AEs), Serious Adverse Events (SAEs) in Long-term follow-up
Drug-related AEs and SAEs would be monitored as long as 5 years after injection
Time frame: up to 5 Years

Study locations

1 site

Shanghai
Shanghai, Shanghai Municipality 200120, China

Updates

Tracking since Sep 25, 2026

No changes since tracking began. The registry record was last updated on Apr 21, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗

Registry details

Key details

Study ID: NCT06432140
Lead sponsor: Shanghai Vitalgen BioPharma Co., Ltd.
Responsible party: Sponsor
First posted: May 29, 2024
Start date: Jul 2, 2024
Primary completion: Jul 23, 2026 (estimated)
Completion: Sep 20, 2030 (estimated)
Last update: Apr 21, 2026

Study contacts

yi Wang, MD

principal investigator · Children's Hospital of Fudan University

jiwen Wang, MD

principal investigator · Shanghai Children's Medical Center

yunhai song, MD

principal investigator · Shanghai Children's Medical Center

Oversight

Data monitoring committee: No
FDA-regulated drug: No
FDA-regulated device: No

View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is active, not recruiting, as verified in Apr 2026. You cannot join it, but the record below documents what was studied.

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Discussion

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Other studies in this condition

Aromatic amino acid decarboxylase deficiency

Other studies from this sponsor

Shanghai Vitalgen BioPharma Co., Ltd.

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