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

NCT06432140 | Active Not Recruiting Phase 2

• 1 other identifier: VGN-R09b-102
• Study Type: interventional
• Target: 13
• Locations: 1 country
• Sites: 1

Brief 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

Keywords

AADC; AAV9; Central Nervous System (CNS) gene therapy

Outcome Measures

Primary Outcomes (2)

• Number of Adverse Events (AEs), Serious Adverse Events (SAEs)

  Vital signs, physical examination, laboratory test will be monitored after drug injection

  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.

  up to 24 months

Secondary Outcomes (7)

• Change in brain AADC activity

  up to 5 Years

• Change in Cerebrospinal Fluid (CSF) neurotransmitter metabolite concentrations

  up to 5 Years

• Change from baseline in motor function

  up to 5 Years

• Change in number of Clinical symptoms

  up to 5 Years

• Viral shedding

  up to 1 week

Study Arms (1)

VGN-R09b injection

EXPERIMENTAL

Different levels of VGN-R09b will be injected into bilateral putamen by stereotactic surgery

Genetic: VGN-R09b injection

Interventions

VGN-R09b injection GENETIC

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

VGN-R09b injection

Eligibility Criteria

• Age: 18 Months - 8 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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).

Sponsors & Collaborators

• Shanghai Vitalgen BioPharma Co., Ltd.: lead

Study Sites (1)

Shanghai

Shanghai, Shanghai Municipality, 200120, China

Location

MeSH Terms

Conditions

Aromatic amino acid decarboxylase deficiency

Study Officials

• yi Wang, MD

  Children's Hospital of Fudan University

  PRINCIPAL INVESTIGATOR

• jiwen Wang, MD

  Shanghai Children's Medical Center

  PRINCIPAL INVESTIGATOR

• yunhai song, MD

  Shanghai Children's Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: VGN-R09b will be injected into bilateral putamen by stereotactic surgery

Study Record Dates

• First Submitted: May 17, 2024
• First Posted: May 29, 2024
• Study Start: July 2, 2024
• Primary Completion (Estimated): July 23, 2026
• Study Completion (Estimated): September 20, 2030
• Last Updated: April 21, 2026

Record last verified: 2026-04

Locations

CN (1)