Help us determine the safety and tolerability of an ALS gene therapy

Closed for enrollment

Study Name
Biogen-C9 Phase I Clinical Trial
Michael Benatar, MD, PhD
Study Type
Interventional (Clinical Trial), Phase I, Randomized, Placebo-controlled
Eligible Population

Individuals affected with ALS, and positive for the C9ORF72 gene mutation

Study Purpose/Goals

To investigate the safety and tolerability of the experimental drug BIIB078 in patients with C9orf72-ALS

A Phase I Multiple-Ascending-Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of BIIB078 Administered Intrathecally to Adults with C9ORF72-Associated Amyotrophic Lateral Sclerosis.


40 weeks of participation:

  • Screening: 6 weeks
  • Treatment: 12 weeks (3 doses, once every 2 weeks + 2 doses, once every 4 weeks)
  • Follow-up: 22 weeks


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

Join our family - together, we can change the course of this disease.

Join our family

Together, we can change the course of this disease.

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