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)

Nov 19, 2021

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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

Completed

Principal Investigators

Site-PI: Michael Benatar, MD, PhD

Study Type

Drug Trial

Goals

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

Enrolling

C9ORF72 ALS patients

Eligibility

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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

Biogen

Related Publications

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van den Berg LH, Rothstein JD, Shaw PJ, Babu S, Benatar M, Bucelli RC, Genge A, Glass JD, Hardiman O, Libri V, Mobach T, Oskarsson B, Pattee GL, Ravits J, Shaw CE, Weber M, Zinman L, Jafar-Nejad P, Rigo F, Lin L, Ferguson TA, Gotter AL, Graham D, Monine M, Inra J, Sinks S, Eraly S, Garafalo S, Fradette S. Safety, tolerability, and pharmacokinetics of antisense oligonucleotide BIIB078 in adults with C9orf72-associated amyotrophic lateral sclerosis: a phase 1, randomised, double blinded, placebo-controlled, multiple ascending dose study. Lancet Neurol. 2024 Sep;23(9):901-912. [PubMed link: 39059407]

https://pubmed.ncbi.nlm.nih.gov/39059407

Related Presentations & Lectures

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