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A Phase I/II Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC

NCT01395641 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2023-02-10

No results posted yet for this study

Summary

This Phase I/II trial is to prove the efficacy and safety of AAV2-hAADC to treat patients with AADC deficiency.

Conditions

  • Aromatic L-amino Acid Decarboxylase (AADC) Deficiency

Interventions

DRUG

gene therapy

AAV2-hAADC viral vector will be injected into bilateral putamen by stereotactic surgery.

Sponsors & Collaborators

  • PTC Therapeutics

    collaborator INDUSTRY

  • National Taiwan University Hospital

    lead OTHER

Principal Investigators

  • Wuh-Liang Hwu, M.D., Ph.D. · National Taiwan University Hospital

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
24 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-10-22
Primary Completion
2020-12-18
Completion
2022-03-07

Countries

  • Taiwan

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01395641 on ClinicalTrials.gov