BIIB067 When Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide Dismutase 1 Mutation

This study first aims to evaluate the efficacy of BIIB067 when initiated in presymptomatic adult carriers of a superoxide dismutase 1 (SOD1) mutation with elevated neurofilament (NF). It also aims are to evaluate the safety and tolerability of BIIB067 and to evaluate the effect of BIIB067 on pharmacodynamics (PD)/treatment response biomarkers when initiated prior to versus at the time of emergence of clinically manifest amyotrophic lateral sclerosis (ALS).

 

Key inclusion criteria

  • Aged 18 years or older at the time of enrollment

  • Participants should have a protocol-defined rapidly progressive SOD1 mutation

  • Participants with plasma NfL level less than the protocol-defined threshold

  • Participants who are clinically presymptomatic for ALS

Key exclusion criteria

  • History or positive test result at screening for human immunodeficiency virus (HIV).

  • Current hepatitis C infection. Participants with positive HCV antibody and undetectable HCV Ribonucleic Acid (RNA) are eligible to participate in the study.

  • Current hepatitis B infection. Participants with immunity to hepatitis B from previous natural infection or vaccination are eligible to participate in the study.

  • History of systemic hypersensitivity reaction to tofersen, the excipients contained in the formulation, and if appropriate, any diagnostic agents to be administered during the study.

  • History of confounding neuromuscular or neurological disorder that is expected to have a progressive course during the study, and/or is expected to be associated with elevations in NF.

  • Presence of risk for increased or uncontrolled bleeding and/or risk of bleeding that if not managed optimally could place a participant at an increased risk for intraoperative or postoperative bleeding.

  • Significant cognitive impairment, clinical dementia, or unstable psychiatric illness, including psychosis, suicidal ideation, suicide attempt, or untreated major depression ≤ 90 days of screening

  • Treatment with riluzole and/or edaravone. If the participant has been on riluzole and/or edaravone, the medication(s) must be discontinued for at least 5 half-lives prior to screening.

  • Use of off-label treatments for ALS

  • Treatment with another investigational drug, biological agent, or device within 1 month or 5 half-lives of study agent, whichever is longer. Specifically, no prior treatment with small interfering RNA, stem cell therapy, or gene therapy is allowed

  • Anticipated need, in the opinion of the Investigator, for administration of any antiplatelet or anticoagulant medication that cannot be safely continued or held for an LP procedure, if necessary, according to local or institutional guidelines and/or Investigator determination

  • Current enrollment or a plan to enroll in any interventional clinical study in which an investigational treatment, biological agent, device, or approved therapy for investigational use. Participation in a noninterventional study focused on ALS natural history may be allowed

“If we’re not in the beginning for rare disease, we may simply be too late for our patients to get the benefit of these new therapies.”

-Angela Genge

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