Long-term observational study of Gene Therapy for AADC deficiency

JRCT ID: jRCT2033220245

Registered date:05/08/2022

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Aromatic L-amino acid decarboxylase deficiency
• Date of first enrollment: 05/08/2022
• Target sample size: 1
• Study type: Observational

Outcome(s)

• Primary Outcome: 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.

Key inclusion & exclusion criteria

• Age minimum: Not applicable
• Age maximum: Not applicable
• Gender: Both

Include 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.

• Exclude criteria: -Patients who cannot undergo MRI.

Related Information

• Primary Sponsor: Yamagata Takanori
• Secondary Sponsor: Gene Therapy Research Institution Co., Ltd.