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

JRCT ID: jRCT2071240022

Registered date:14/06/2024

Basic Information

• Recruitment status: Pending
• Health condition(s) or Problem(s) studied: Amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1)
• Date of first enrollment: 25/06/2024
• Target sample size: 20
• Study type: Interventional

Intervention(s)

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.

Outcome(s)

• Primary Outcome: Adverse event assessment

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: Not applicable
• Gender: Both
• Include criteria: Weakness attributable to ALS and associated with a mutation in the SOD1 gene (SOD1-ALS).

Exclude criteria

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.

Related Information

• Primary Sponsor: Cho Kyo