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ong-term observational study of Gene Therapy for Sporadic ALS

Not Applicable
Recruiting
Conditions
Sporadic amyotrophic lateral sclerosis (Sporadic ALS)
Registration Number
JPRN-jRCT2033230284
Lead Sponsor
Morita Mitsuya
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
Recruiting
Sex
All
Target Recruitment
6
Inclusion Criteria

Patients who enrolled in the Phase I/II Study of Gene Therapy for Sporadic ALS, and who completed or discontinued the study.

Exclusion Criteria

-Patients who are judged by the investigator or subinvestigator to be inappropriate to participate in this study.

Study & Design

Study Type
Observational
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
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.
Secondary Outcome Measures
NameTimeMethod
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