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

> **NCT02852213** · PHASE1 · RECRUITING · sponsor: **Krzysztof Bankiewicz** · enrollment: 42 (estimated)

## Conditions studied

- AADC Deficiency

## Interventions

- **DRUG:** AAV2-hAADC

## Key facts

- **NCT ID:** NCT02852213
- **Lead sponsor:** Krzysztof Bankiewicz
- **Sponsor class:** OTHER
- **Phase:** PHASE1
- **Study type:** INTERVENTIONAL
- **Status:** RECRUITING
- **Start date:** 2016-07-01
- **Primary completion:** 2027-07
- **Final completion:** 2031-07
- **Target enrollment:** 42 (ESTIMATED)
- **Last updated:** 2025-10-21

## Collaborators

- [object Object]
- [object Object]

## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT02852213

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT02852213, "A Single-Stage, Adaptive, Open-label, Dose Escalation Safety and Efficacy Study of AADC Deficiency in Pediatric Patients". Retrieved via AI Analytics 2026-07-16 from https://api.ai-analytics.org/clinical/NCT02852213. Licensed CC0.

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*[Clinical trials dataset](/datasets/clinical-trials) · CC0 1.0*