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.
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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

Jiwen Wang, Doctor

18916613192

wangjiwen@scmc.com.cn

CONTACT