Long-term observational study of Gene Therapy for Sporadic ALS

JRCT ID: jRCT2033230284

Registered date:09/08/2023

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Sporadic amyotrophic lateral sclerosis (Sporadic ALS)
• Date of first enrollment: 09/08/2023
• Target sample size: 6
• Study type: Observational

Outcome(s)

• Primary Outcome: To investigate the long-term tolerability, safety, and efficacy of the study product in patients with Sporadic ALS, after medullary cavity administrations of AAV.GTX-ADAR2.

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 Sporadic ALS, and who completed or discontinued the study.
• Exclude criteria: -Patients who are judged by the investigator or subinvestigator to be inappropriate to participate in this study.

Related Information

• Primary Sponsor: Morita Mitsuya
• Secondary Sponsor: Gene Therapy Research Institution Co., Ltd.