A Single-Stage, Adaptive, Open-label, Dose Escalation Safety and Efficacy Study of AADC Deficiency in Pediatric Patients

ClinicalTrials.gov ID: NCT02852213

Public ClinicalTrials.gov record NCT02852213. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

View official ClinicalTrials.gov record → Back to search

ClinicalTrials.gov public records Last synced May 8, 2026, 10:45 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Official title

SIngle-Stage, Open-Label, Safety and Efficacy Study of Adeno-Associated Virus Encoding Human Aromatic L-Amino Acid Decarboxylase by Magnetic Resonance MR-guided Infusion Into Midbrain in Pediatric Patients With AADC Deficiency

Study identification

NCT ID: NCT02852213
Recruitment status: Recruiting
Study type: Interventional
Phase: Phase 1
Lead sponsor: Krzysztof Bankiewicz; Other
Enrollment: 42 participants

Conditions and interventions

Conditions

AADC Deficiency

Interventions

AAV2-hAADC Drug

Drug

Eligibility (public fields only)

Age range: 24 Months and older
Sex: All
Healthy volunteers: Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date: Jun 30, 2016
Primary completion: Jun 30, 2027
Completion: Jun 30, 2031
Last update posted: Oct 20, 2025

2016 – 2031

United States locations

U.S. sites: 3
U.S. states: 2
U.S. cities: 2

Facility	City	State	ZIP	Site status
University of California San Francisco, Benioff Children's Hospital	San Francisco	California	94143	Recruiting
Nationwide Children's Hospital	Columbus	Ohio	43205	Recruiting
The Ohio State University Medical Center	Columbus	Ohio	43221	Recruiting

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

About this trial record page

What this page shows: Public field values for ClinicalTrials.gov record NCT02852213, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do: No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from: Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh: Last update posted Oct 20, 2025 · Synced May 8, 2026

Open the official record

The complete protocol, eligibility criteria, and contact information for NCT02852213 live on ClinicalTrials.gov.

View official ClinicalTrials.gov record →