Long-Term Evaluation of BIIB067 (Tofersen)

• Register: ClinicalTrials.gov
• Last refreshed on: 29 May 2023
• Main ID: NCT03070119
• Date of registration: 28/02/2017
• Prospective Registration: Yes
• Primary sponsor: Biogen
• Public title: Long-Term Evaluation of BIIB067 (Tofersen)
• Scientific title: An Extension Study to Assess the Long-Term Safety, Tolerability, Pharmacokinetics, and Effect on Disease Progression of BIIB067 Administered to Previously Treated Adults With Amyotrophic Lateral Sclerosis Caused by Superoxide Dismutase 1 Mutation
• Date of first enrolment: March 8, 2017
• Target sample size: 138
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/show/NCT03070119
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Sequential Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Belgium, Canada, France, Germany, Italy, Japan, New Zealand, United Kingdom, United States

Contacts

• Name: Medical Director
• Affiliation: Biogen

Key inclusion & exclusion criteria

Key Inclusion Criteria:

- Must have diagnosis of superoxide dismutase 1-amyotrophic lateral sclerosis
(SOD1-ALS), and must have completed the End of Study Visit for either Parts A, B, or C
of Study 233AS101 (NCT02623699) (i.e., were not withdrawn).

- If taking riluzole, participant must be receiving a stable dose for =30 days prior to
Day 1.

- If taking edaravone, participant must have initiated edaravone =60 days (2 treatment
cycles) prior to Day 1. Edaravone may not be administered on dosing days during this
study.

- Medically able to undergo the study procedures, and to adhere to the visit schedule at
the time of study entry, as determined by the Investigator.

- For female participants of childbearing potential must agree to practice effective
contraception during the study and be willing and able to continue contraception for 5
months after their last dose of study treatment.

- Participants from Study 233AS101 Parts A and B must have a washout =16 weeks between
the last dose of study treatment received in Study 233AS101 and the first dose of
BIIB067 received in the current Study 233AS102.

Key Exclusion Criteria:

- History of allergies to a broad range of anesthetics.

- Presence of risk for increased or uncontrolled bleeding and/or risk of bleeding that
is not managed optimally and could place a participant at an increased risk for
bleeding during or after a Lumbar Puncture (LP) procedure. These risks could include,
but are not limited to, anatomical factors at or near the LP site (e.g., vascular
abnormalities, neoplasms, or other abnormalities) and underlying disorders of the
coagulation cascade, platelet function, or platelet count (e.g., hemophilia, Von
Willebrand's disease, liver disease).

- Presence of an implanted shunt for the drainage of CSF or an implanted central nervous
system (CNS) catheter.

- Prior or current treatment with small interfering ribonucleic acid (RNA), stem cell
therapy, or gene therapy.

- Treatment with another investigational drug, biological agent (excluding BIIB067), or
device within 1 month or 5 half-lives of study agent, whichever is longer.

- Current or anticipated need, in the opinion of the Investigator, of a diaphragm pacing
system (DPS) during the study period.

- Current or recent (within 1 month) use, or anticipated need, in the opinion of the
Investigator, of copper (II) (diacetyl-bis(N4-methylthiosemicarbazone)) or
pyrimethamine.

- Female participants who are pregnant or currently breastfeeding.

- Current enrollment in any other interventional study.

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

ALS Caused by Superoxide Dismutase 1 (SOD1) Mutation

Intervention(s)

Drug: Tofersen

Primary Outcome(s)

Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) [Time Frame: Up to Week 364]

Secondary Outcome(s)

Change from Baseline in Handheld Dynamometry (HHD) Megascore and Individual Muscle Strength [Time Frame: Baseline to Week 364]

Time to Death [Time Frame: Up to Week 364]

Change from Baseline in Total SOD1 Protein in CSF [Time Frame: Baseline to Week 360]

Time to Death or Permanent Ventilation [Time Frame: Up to Week 364]

Change from Baseline in Neurofilament Light Chain (NfL) Concentration in Plasma [Time Frame: Baseline to Week 364]

Change from Baseline in Total ALS Functional Rating Scale - Revised (ALSFRS-R) Score [Time Frame: Baseline to Week 364]

Change from Baseline in Slow Vital Capacity (SVC) [Time Frame: Baseline to Week 364]

Levels of BIIB067 in Plasma [Time Frame: Up to Week 364]

Levels of BIIB067 in Cerebrospinal Fluid (CSF) [Time Frame: Up to Week 360]

Secondary ID(s)

2016-003225-41

233AS102

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Ionis Pharmaceuticals, Inc.