An Early Clinical Trial to Evaluate VGN-R09b for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency.
Recruiting
Early Phase 1
Interventional Study
Aromatic L-amino Acid Decarboxylase (AADC) Deficiency
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
At a Glance
- Age
- 24 Months – 7
- Sex
- Any
- Trial phase
- Early Phase 1
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 6 (estimated)
- Sponsor
- Shanghai Jiao Tong University School of Medicine · Other
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About This Trial
This early Phase trial is to prove the safety and efficacy of VGN-R09b to treat patients with AADC deficiency.
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
1. The child patient has to be ≥2 years old and \< 8 years old, or a head circumference big enough for surgery.
2. Historical diagnosis of AADC deficiency with clinical symptoms consistency, AND confirmed by one of the lab tests: (1) CSF neurotransmitter profile demonstrating re…
Contacts