Summary

for people ages 18 years and up (full criteria)
at La Jolla, California and other locations
study started
estimated completion:

Description

Summary

The primary objective of the study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of BIIB067 in adults with ALS. The secondary objective is to evaluate the effects of BIIB067 on levels of superoxide dismutase 1 (SOD1) protein in the cerebrospinal fluid (CSF).

Official Title

A Phase 1, Placebo-Controlled, Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB067 Administered to Adult Subjects With Amyotrophic Lateral Sclerosis

Keywords

Amyotrophic Lateral Sclerosis IONIS-SOD1Rx SOD1 ALS Sclerosis Motor Neuron Disease BIIB067 Single Ascending Dose Multiple Ascending Dose

Eligibility

For people ages 18 years and up

Key Inclusion Criteria:

  • Weakness attributable to ALS and documented SOD1 mutation at Screening Visit 2.
  • A forced vital capacity (FVC) ≥50% of predicted value as adjusted for sex, age, and height (from the sitting position). Participants with stable FVC <50% but ≥45%, whose FVC has not declined by more than 5% in the last 6 months may be considered for inclusion, at the discretion of the Investigator.
  • If taking riluzole, Participant must be on a stable dose for ≥30 days prior to Day 1 and expected to remain at that dose until the final study visit.
  • Medically able to undergo the study procedures, and to adhere to the visit schedule at the time of study entry, as determined by the Investigator.

Key Exclusion Criteria:

  • History of or positive test result for human immunodeficiency virus.
  • History of, or positive test result at Screening, for hepatitis C virus antibody.
  • Current hepatitis B infection (defined as positive for hepatitis B surface antigen[HBsAg] and/or hepatitis B core antibody [HBcAb]). Participants with immunity to hepatitis B from previous natural infection (defined as negative HBsAg, positive hepatitis B surface antibody immunoglobulin G, and positive HBcAb) or vaccination(defined as positive anti-HBs) are eligible to participate in the study.
  • 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 ribonucleic acid, stem cell therapy, or gene therapy is allowed.
  • Current enrollment in any other interventional study.
  • Current or recent (within 1 month) use, or anticipated need, in the opinion of the Investigator, of copper (II) (diacetyl-bis (N4-methylthiosemicarbazone)) or pyrimethamine.
  • Current or anticipated need, in the opinion of the Investigator, of a diaphragm pacing system (DPS) during the study period.
  • Tracheostomy

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

Locations

  • University of California San Diego Medical Center accepting new patients
    La Jolla California 92093-0949 United States
  • Barrow Neurological Institute accepting new patients
    Phoenix Arizona 85013 United States
  • California Pacific Medical Center accepting new patients
    San Francisco California 94115 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Biogen
ID
NCT02623699
Phase
Phase 1
Study Type
Interventional
Last Updated
May 31, 2018