Phase 1 Clinical Trial to Test BIIB067 in People with ALS Currently Recruiting Participants

ionis biogenResearchers are looking for people with amyotrophic lateral sclerosis (ALS) to participate in a phase 1 clinical trial, sponsored by Biogen and Ionis Pharmaceuticals, to test the experimental drug BIIB067 (Ionis-SOD1Rx).

BIIB067 is an antisense oligonucleotide, composed of short segments of synthetic genetic material (nucleic acid) that bind to RNA. The drug is designed to block production of flawed SOD1 protein in people with ALS caused by defects in the SOD1 gene. It is administered by intrathecal injection, in which a small needle is inserted into a space in the lower back below the end of the spinal cord in a medical procedure commonly referred to as a “lumbar puncture.”

The goals of the study are to determine the safety and tolerability of the drug in people with ALS and determine how the drug distributes throughout the body.

The study, which is taking place at 17 trial sites in the United States, Canada and Western Europe, is being conducted in two distinct parts:

  • In Part A of the trial, participants with sporadic or familial SOD1-ALS will receive a single dose of BIIB067. Duration of this first part of the study is 12 weeks, to include a 4-week screening period and 8-week follow-up period.
  • In Part B of the trial, participants with familial SOD1-ALS will receive several doses of BIIB067 or placebo. Duration for each participant in this part of the study will be approximately 31 weeks, to include a 7-week screening period, a 12-week treatment period and a 12-week follow-up period.

Participants in Part A of the trial must have a diagnosis of possible, laboratory supported probable, probable or definite ALS according to El Escorial criteria. Participants in Part B of the trial must have weakness attributable to ALS and documented SOD1 mutation. In addition, all participants must:

  • have a forced vital capacity (FVC) of at least 50 percent of predicted value, or if stable for at least 6 months, 45 percent;
  • if taking riluzole, remain on a stable dose;
  • be medically able to undergo lumbar puncture; and
  • meet other eligibility criteria.

To learn more about this trial, including site locations and complete inclusion and exclusion criteria, visit and enter NCT02623699 into the search box.