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

Abroad (travel likely)RecruitingPHASE1

Conditions

AADC Deficiency

Simple summaryAI-assisted

This summary is produced by a language model and is experimental. It may be incomplete or inaccurate and is not a substitute for professional medical advice.

Locations

University of California San Francisco, Benioff Children's Hospital, San Francisco, California, United States
Contact: Study coordinator (Catalina Pen) · catalina.pen@ucsf.edu
E
Contact: Principal Investigator (Nalin Gupta, MD)
Nationwide Children's Hospital, Columbus, Ohio, United States
Contact: Study coordinator (Hannah Lehmann) · hannah.lehmann@nationwidechildrens.org
Contact: Study coordinator (Margaret Carey) · margaret.carey@nationwidechildrens.org
Contact: Principal Investigator (Jeffrey Leonard, MD)
Toni Pearson, MD (Sub-investigator)
The Ohio State University Medical Center, Columbus, Ohio, United States
Contact: Study coordinator (Andrea Davis, MS) · andrea.davis@osumc.edu · 614-814-6871
Contact: Principal Investigator (Russell Lonser, MD)
Krystof Bankiewicz, PhD (Sub-investigator)

Study leads

Krystof Bankiewicz, MD, PhD
Study Director
OSU Professor of Neurological Surgery

View source on ClinicalTrials.gov