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Japan Expanded Clinical Study (ECS) to Provide Tofersen (BIIB067) to Patients With Amyotrophic Lateral Sclerosis (ALS) Associated with a Mutation in the Superoxide Dismutase 1 (SOD1) Gene

Expanded Clinical Study of Tofersen (BIIB067) in SOD1-ALS

The participants of the 233AS002 study are adults aged 18 and older with genetically confirmed SOD1-ALS, characterized by progressive muscle weakness and high unmet medical need, including both tofersen-naive individuals and those previously treated in 233AS102 or CMAP programs, provided they are medically eligible for lumbar puncture and meet all safety-related inclusion and exclusion criteria.

This study is an expanded access program, and its primary purpose is to provide the investigational drug to patients.

This study is an expanded access program intended to provide the investigational drug to patients, and therefore no formal data collection or analysis is conducted.

This study is an expanded access program intended to provide the investigational drug to patients, and therefore no formal data collection or analysis is conducted.

This study is an expanded access program intended to provide the investigational drug to patients, and therefore no formal data collection or analysis is conducted.

Yes

In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/.

https://jrct.mhlw.go.jp/latest-detail/jRCT2071240022

Cho Kyo

Biogen Japan Ltd.

Nihonbashi 1-chome Mitsui Building 14F, 1-4-1, Nihonbashi, Chuo-ku, Tokyo

japan-medinfo@biogen.com

Biogen Japan Medical Information

Biogen Japan Ltd.

Nihonbashi 1-chome Mitsui Building 14F, 1-4-1, Nihonbashi, Chuo-ku, Tokyo

+81-120-560-086

japan-medinfo@biogen.com

Complete

June. 25, 2024

Interventional

single arm study

open(masking not used)

uncontrolled control

single assignment

treatment purpose

Weakness attributable to ALS and associated with a mutation in the SOD1 gene (SOD1-ALS).

1. Any comorbidities or conditions that, in the opinion of the Investigator, would unacceptably increase the risk of participation, including contraindications to lumbar punctures (LPs).
2. Anticipated need, in the opinion of the Investigator, for administration of any antiplatelet or anticoagulant medication that cannot be safely held before and/or after an LP procedure according to local or institutional guidelines and/or Investigator determination.
3. Treatment with another investigational drug, biological agent (excluding tofersen), or device within 1 month or 5 half-lives of study agent, whichever is longer, before the first dose of tofersen in this study.
4. Female participants who are pregnant or breastfeeding.
5. Patient's primary place of residence is outside of the country of treatment.

Both

Amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1)

Research Name: BIIB067
Generic Name: Tofersen
Trade Names: Not applicable
Loading dose regimen for tofersen-naive participants only (doses 1 to 3): Tofersen will be administered on Days 0, 14, and 28.
Maintenance dose regimen (doses 4 and after for tofersen-naive participants): A maintenance dose of tofersen will be administered approximately every 28 days (or 4 weeks).
For tofersen-experienced participants, tofersen will be administered on Day 0, Day 28, and then approximately every 28 days (or 4 weeks) thereafter for maintenance doses.

Adverse event assessment

+81-99-275-5553

May. 20, 2024

none