Actively Recruiting
An Early Clinical Trial to Evaluate VGN-R09b for Treatment of 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
316 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
This early Phase trial is to prove the safety and efficacy of VGN-R09b to treat patients with AADC deficiency.
CONDITIONS
Official 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 younger than 8 years, or with a head size suitable for surgery
- Confirmed AADC deficiency diagnosis consistent with clinical symptoms and lab tests (CSF neurotransmitter profile or plasma AADC activity plus genetic confirmation)
- Motor development less than or equal to 3 months at baseline
- Failed to benefit from standard medical therapies for AADC deficiency
- Stable medication regimen for AADC deficiency with no new medications for at least 6 months and no dose changes for 3 months before baseline
- Parent or legal guardian consents to study participation
- Parent or legal guardian agrees to support study requirements including symptom assessments
You will not qualify if you...
- Presence of brain tumors, structural brain abnormalities, or lesions posing excessive risk
- Significant medical or neurological conditions posing high operative or anesthetic risk (including congenital heart disease, respiratory disease requiring oxygen, history of serious anesthesia complications, cardiorespiratory arrest, liver or kidney failure, cancer, or HIV)
- Severe bleeding disorders or ongoing anticoagulant use
- Active or recent severe infection within 12 weeks before screening
- Previous stereotactic brain surgery or gene/cell therapy
- Live vaccination received within 4 weeks prior to enrollment
- Anti-AAV9 neutralizing antibody titer above 1,200 folds
- Contraindications to sedation for surgery or imaging studies such as PET or MRI
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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