Research Studies

Study Title

Biogen C9 ASO Trial (A Phase I Multiple-Ascending-Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of BIIB078 Administered Intrathecally to Adults with C9ORF72-Associated ALS)

Enrollment Status


Principal Investigators

Site-PI: Michael Benatar, MD, PhD

Study Type

Drug Trial


  • Investigate the safety and tolerability of the experimental antisense oligonucleotide (ASO) BIIB078 in participants with C9ORF72-ALS


C9ORF72 ALS patients


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Individuals affected with ALS who also have a C9ORF72 mutation

Study Involvement

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Multiple study visits with spinal tap and intrathecal administration of experimental agent. “Loading” comprises 3 administrations, with each dose separated by 2 weeks. “Maintenance” comprises 2-5 administrations, depending on dosing cohort.
Study procedures include:

  • Medical history and medication review
  • Drug dosing
  • Blood and urine collection
  • Neurological exam
  • Muscle testing
  • Electrocardiogram
  • Physical Exam
  • Neuropsychiatric testing
  • Surveys
  • Breathing testing

Monthly intrathecal administration (following a blinded induction phase) of BIIB078 during the open label extension phase.

Primary Funding Source


Related Publications

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Related Presentations & Lectures

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