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A Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC - An Expansion

NCT02926066 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2023-02-10

No results posted yet for this study

Summary

This clinical trial expansion is to offer patients, who are not enrolled into the Phase I/II trial, a chance of treatment, to provide the experience in this gene therapy, and to increase the dose slightly.

Conditions

  • Aromatic Amino Acid Decarboxylase Deficiency

Interventions

DRUG

AAV2-hAADC

Dosage form: Aqueous solution Dose(s): 2.37x10\^11 vg/case(High dose) Dosing schedule: Intracerebral infusion, single dose Mechanism of action (if known): supplement a gene defect Dosage form: Aqueous solution Dose(s): 1.81x10\^11 vg/case(Standard dose) Dosing schedule: Intracerebral infusion, single dose Mechanism of action (if known): supplement a gene defect

Sponsors & Collaborators

  • PTC Therapeutics

    collaborator INDUSTRY

  • National Taiwan University Hospital

    lead OTHER

Principal Investigators

  • Yin-Hsiu Chien, MD.,PhD · National Taiwan University Hospital

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
6 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2016-11-09
Primary Completion
2022-01-24
Completion
2022-05-16

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 NCT02926066 on ClinicalTrials.gov