RECRUITING

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

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

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

Quick Facts

Study Start:2016-07-01
Study Completion:2031-07
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT02852213

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:24 Months
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. Age 18 years or older
  2. Willing and able to provide informed consent
  3. Able to understand and follow study procedures
  4. Stable medical condition
  1. Pregnancy or breastfeeding
  2. Severe psychiatric disorders
  3. Active substance abuse
  4. Unstable medical conditions
  5. Inability to comply with study requirements

Contacts and Locations

Study Contact

Andrea Davis, MS
CONTACT
614-688-6412
andrea.hesse@osumc.edu

Principal Investigator

Krystof Bankiewicz, MD, PhD
STUDY_DIRECTOR
OSU Professor of Neurological Surgery

Study Locations (Sites)

University of California San Francisco, Benioff Children's Hospital
San Francisco, California, 94143
United States
Nationwide Children's Hospital
Columbus, Ohio, 43205
United States
The Ohio State University Medical Center
Columbus, Ohio, 43221
United States

Collaborators and Investigators

Sponsor: Krzysztof Bankiewicz

  • Krystof Bankiewicz, MD, PhD, STUDY_DIRECTOR, OSU Professor of Neurological Surgery

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2016-07-01
Study Completion Date2031-07

Study Record Updates

Study Start Date2016-07-01
Study Completion Date2031-07

Terms related to this study

Keywords Provided by Researchers

  • AADC
  • gene therapy

Additional Relevant MeSH Terms

  • AADC Deficiency