Actively Recruiting
An Open, Dose-escalating Early Clinical Trial to Evaluate the Tolerability, Safety, and Efficacy of VGN-R09b by Intra Striatum Injection in Patients With Aromatic L-amino Acid Decarboxylase (AADC) Deficiency
Led by Shanghai Jiao Tong University School of Medicine · Updated on 2023-03-13
6
Participants Needed
1
Research Sites
208 weeks
Total Duration
On this page
Sponsors
S
Shanghai Jiao Tong University School of Medicine
Lead Sponsor
S
Shanghai Vitalgen BioPharma Co., Ltd.
Collaborating Sponsor
AI-Summary
What this Trial Is About
The trial focuses on patients with Aromatic L-amino acid decarboxylase (AADC) deficiency, a rare genetic disorder affecting the production of important neurotransmitters dopamine and serotonin. Researchers are evaluating the safety and effectiveness of VGN-R09b, a gene therapy delivered directly into the brain, to improve symptoms related to this condition. This early phase clinical study aims to provide preliminary evidence for this treatment's impact on patients with AADC deficiency. Participants will receive VGN-R09b through injections into the bilateral putamen area of the brain using stereotactic surgery. The study includes a dose-escalation phase followed by a dose-expansion phase to assess different dose levels. This open-label, single-center trial monitors patients after receiving the gene therapy to understand its effects and tolerability. During the study, participants will be closely monitored for adverse events and their motor development milestones, such as head control, sitting independently, standing or stepping with support, and walking with minimal assistance, over a period of 52 weeks. Researchers will conduct various assessments and follow-ups to track the treatment's safety and effectiveness, with the overall participation lasting about one year.
CONDITIONS
Brief Title
An Early Clinical Trial to Evaluate VGN-R09b for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency.
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Child aged 2 years or older and under 8 years, or with a head size suitable for surgery
- Confirmed diagnosis of AADC deficiency by clinical symptoms and specific lab tests
- Motor development delay of 3 months or more at baseline
- No benefit from standard medical therapies like dopamine agonists or Vitamin B6
- Stable medication regimen for at least 6 months with no dose changes for 3 months prior to baseline
- Parent or legal guardian consent and agreement to support study requirements
You will not qualify if you...
- Presence of intracranial tumors, severe brain abnormalities, or lesions posing high risk
- Significant medical or neurological conditions causing high operative or anesthesia risk
- Severe blood clotting problems or need for ongoing blood thinners
- Active or recent serious infections within 12 weeks before screening
- Previous stereotactic neurosurgery or gene/cell therapy
- Receipt of live vaccination within 4 weeks prior to enrollment
- Anti-AAV9 antibody levels above 1,200 folds
- Contraindications to sedation during surgery or imaging procedures
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Single treatment session with follow-up up to Week 52
Participants receive VGN-R09b gene therapy through stereotactic surgery involving injection into the bilateral putamen.
1 surgery visit and multiple follow-up visits during 52 weeks
Trial Site Locations
Total: 1 location
1
Shanghai Children's Medical Center Affiliated to Shanghai Jiao Tong University School of Medicine
Shanghai, No. 1678, Dongfang Road, Pudong New Area, Shanghai, China, 200127
Actively Recruiting
Research Team
J
Jiwen Wang, Doctor
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
Frequently Asked Questions
Have more questions? Get in touch with our team for quick support
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here