ong-term observational study of Gene Therapy for AADC deficiency
Not Applicable
Recruiting
- Conditions
- Aromatic L-amino acid decarboxylase deficiency
- Registration Number
- JPRN-jRCT2033220245
- Lead Sponsor
- Yamagata Takanori
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 1
Inclusion Criteria
Patients who enrolled in the Phase I/II Study of Gene Therapy for AADC deficiency, and who completed or discontinued the study.
-Female patients who are able to become pregnant, and whose partners agree to use medically effective contraceptive methods throughout the study period.
Exclusion Criteria
-Patients who cannot undergo MRI.
Study & Design
- Study Type
- Observational
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method To investigate the long-term tolerability, safety, and efficacy of the study product in patients with AADC deficiency, after intracerebral administrations of AAV-hAADC-2 into the putamen.
- Secondary Outcome Measures
Name Time Method