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NCT02852213 · ClinicalTrials.gov registry record · Phase 1
A Single-Stage, Adaptive, Open-label, Dose Escalation Safety and Efficacy Study of AADC Deficiency in Pediatric Patients
A Phase 1 study of AADC Deficiency, sponsored by Krzysztof Bankiewicz.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 42
- Enrollment target
- 3
- Study locations
NCT02852213: Recruiting Phase 1 study of AADC Deficiency, sponsored by Krzysztof Bankiewicz.
NCT02852213 is a Phase 1 study of AADC Deficiency that is actively recruiting participants, run by Krzysztof Bankiewicz. The registered enrollment target is 42 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (30% lower). The trial reports 3 study locations across 2 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT02852213, a Phase 1 study of AADC Deficiency, is actively recruiting participants, sponsored by Krzysztof Bankiewicz.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 42 participants
- Enrollment target
- 3
- Study locations
Study Summary
The overall objective of this study is to determine the safety and efficacy of AAV2-hAADC delivered to the substantia nigra pars compacta (SNc) and ventral tegmental area (VTA) in children with aromatic L-amino acid decarboxylase (AADC) deficiency.
Primary Outcome
Assessment of adverse events related to surgery (including intracerebral hemorrhage or stroke, CNS infection) and gene transfer (including severity of post-operative dyskinesia)
Conditions Studied
Interventions
- DRUG AAV2-hAADC
Study Locations (3)
Ohio
- Nationwide Children's Hospital - Columbus
- The Ohio State University Medical Center - Columbus
California
- University of California San Francisco, Benioff Children's Hospital - San Francisco
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 42 participants |
| Start Date | 2016-07-01 |
| Est. Completion | 2031-07 |
| Phase | Phase 1 |
What NCT02852213 shows while recruiting
NCT02852213 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 42 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (30% lower).
The record links to 1 condition, with AADC Deficiency appearing as the primary indexed condition, and to 1 intervention - of which AAV2-hAADC is the first listed.
NCT02852213 reports a single indexed study location in Ohio, California.
Frequently Asked Questions
What is clinical trial NCT02852213 about?
NCT02852213 is a clinical study titled "A Single-Stage, Adaptive, Open-label, Dose Escalation Safety and Efficacy Study of AADC Deficiency in Pediatric Patients". The overall objective of this study is to determine the safety and efficacy of AAV2-hAADC delivered to the substantia nigra pars compacta (SNc) and ventral tegmental area (VTA) in children with aromatic L-amino acid decarboxylase (AADC) deficiency.
What is the current status of trial NCT02852213?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 42 participants. The study started on 2016-07-01. Estimated completion is 2031-07.
What conditions does trial NCT02852213 study?
This clinical trial studies the following conditions: AADC Deficiency.
What interventions are being tested in trial NCT02852213?
The interventions under investigation include: AAV2-hAADC (DRUG).
Who is sponsoring clinical trial NCT02852213?
This trial is sponsored by Krzysztof Bankiewicz, which has 2 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT02852213 being conducted?
This trial has 3 study locations across California, Ohio. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT02852213's enrollment target sits among peer trials
42 1105th of 2000 higher than 876 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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