臨床研究等提出・公開システム

Scientific Title	Long-term observational study of Gene Therapy for Sporadic ALS
Public Title	Long-term observational study of Gene Therapy for Sporadic ALS

Contact for Scientific Queries	Name	Morita Mitsuya
	Affiliation	Jichi Medical University Hospital
	Address	3311-1,Yakushiji, Shimotsuke-shi, Tochigi
	Telephone	+81-285-44-2111
	E-mail	morita@jichi.ac.jp
Contact for Public Queries	Name	Sekiguchi Shinya
	Affiliation	SRD, Inc.
	Address	3-4-8 Hachobori, Chuo-ku, Tokyo
	Telephone	+81-3-5543-0306
	E-mail	als_gt0001x-01@cro-srd.co.jp

Recruitment status	Recruiting

Anticipated date of first enrollment		Aug. 09, 2023
Actual date of first enrollment
Target sample size		6
Study Type		Observational
Study Design	allocation
	masking
	control
	assignment
	purpose
Key inclusion & exclusion 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	-Patients who are judged by the investigator or subinvestigator to be inappropriate to participate in this study.
	Age Minimum	No limit
	Age Maximum	No limit
	Gender	Both
Health Condition(s) or Problem(s) Studied		Sporadic amyotrophic lateral sclerosis (Sporadic ALS)
Intervention(s)
Health Condition(s) Keyword
Intervention(s) Keyword
Health Condition(s) Code
Intervention(s) Code
Primary Outcome(s)		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(s)

Primary Sponsor

Source of Monetary Support / Secondary Sponsor
Secondary Sponsor

Source of Monetary Support	Gene Therapy Research Institution Co., Ltd.
Secondary Sponsor	Applicable

Name of Certified Review Board	Jichi Medical University IRB
Address	3311-1, Yakushiji, Shimotsuke-shi, Tochigi
Telephone	+81-285-58-7195
E-Mail	chiken@jichi.ac.jp
Approval Status	Approval
Date of approval

Plan to share IPD	No
Plan description

Secondary ID(s)
Issuing Authority

Countries of Recruitment（Except Japan）	none