Long-term observational study of Gene Therapy for Sporadic ALS

Status

Active, not recruiting

Phases

Unknown

Study type

Observational

Source

JPRN

Registry ID

JPRN-jRCT2033230284

Enrollment

Registered

2023-08-09

Start date

2023-08-21

Completion date

Unknown

Last updated

2026-09-14

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Sporadic amyotrophic lateral sclerosis (Sporadic ALS)

Interventions

None listed

Eligibility

Sex/Gender

All

Inclusion criteria

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

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

Design outcomes

Primary

Measure	Time frame
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

Measure	Time frame
To evaluate the long-term efficacy of an intrathecal administration of an ADAR2-expressing AAV vector in patients with sporadic ALS, the following measures will be assessed: -ALSFRS-R -Manual Muscle Testing -%FVC -Time to initiation of enteral nutrition	—

Contacts

Public ContactMitsuya Morita

Jichi Medical University Hospital

morita@jichi.ac.jp+81-285-44-2111

Outcome results