A Phase 1 Multiple-Ascending-Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of BIIB078 Administered Intrathecally to Adults With C9ORF72-Associated Amyotrophic Lateral Sclerosis

Study Purpose:

The primary objective of this study is to evaluate the safety and tolerability of BIIB078 in adults with C9ORF72-Amyotrophic Lateral Sclerosis (ALS). The secondary objectives of this study are to evaluate the pharmacokinetic profile of BIIB078 and to evaluate the effects of BIIB078 on clinical function. As the first-in-human study, the study enrolls a small number of participants in each cohort. Every participant in a cohort is treated with the same dose or placebo. The study is designed to evaluate and confirm the safety of each dose before enrolling and exposing new participants to a higher dose in the next cohort. Therefore, proceeding from cohort to cohort, the recruitment status of "Recruiting" may change. Please contact clinicaltrials@biogen.com for further information.


Amyotrophic Lateral Sclerosis (ALS),  Familial ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:



Phase I

Study Chair(s)/Principal Investigator(s):

Biogen Medical Director

Clinicaltrials.gov ID (11 digit #):


Neals Affiliated?


Coordinating Center Contact Information

US Biogen Clinical Trial Center / .(JavaScript must be enabled to view this email address) / 866-633-4636
.(JavaScript must be enabled to view this email address)

Full Study Summary:

Study Sponsor:


Participant Duration:

Estimated Enrollment:


Estimated Study Start Date:


Estimated Study Completion Date:


Posting Last Modified Date:


Date Study Added to alsconsortium.org:

  • Eligibility Criteria


    Female, Male

    Minimum Age:


    Maximum Age:


    Min Vital Capacity (% predicted normal):


    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?


    Key Inclusion Criteria:

    • Ability of the participant to understand the purpose and risks of the study, to provide signed and dated informed consent, and to authorize the use of confidential health information in accordance with national and local participant privacy regulations; or, in the event of the participant's physical incapacity to sign, to confirm that understanding and consent orally to a legally authorized representative (LAR) for the express purpose of having said informed consent and authorization signed on his/her behalf.
    • All participants of childbearing potential must agree to practice highly effective contraception during the study and be willing and able to continue contraception for 5 months after their last dose of study treatment.
    • Must meet the possible, laboratory-supported probable, probable, or definite criteria for diagnosing ALS according to the World Federation of Neurology El Escorial criteria and have documentation of a clinical genetic test demonstrating the presence of a pathogenic mutation in C9ORF72.
    • Slow vital capacity (SVC) ≥ 50% of predicted value as adjusted for sex, age, and height (from the sitting position).
    • Participants taking concomitant riluzole at study entry must be on a stable dose for ≥ 30 days prior to the first dose of study treatment (Day 1).
    • Participants taking concomitant edaravone at study entry must be on a stable dose for ≥ 60 days prior to the first dose of study treatment (Day 1).
    • ALS Cognitive Behavioral Screen (ALS-CBS) score ≥ 11 for the cognitive portion; ≥ 33 for the behavioral portion.
    • 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.
    • Screening values of coagulation parameters including platelet count, international normalized ratio (INR), prothrombin time (PT), and activated partial thromboplastin time (APTT) should be within normal ranges.
    • Has an informant/caregiver who, in the Investigator's judgment, has frequent and sufficient contact with the participant as to be able to provide accurate information about the participant's cognitive and functional abilities at Screening.

    Key Exclusion Criteria:

    • History of drug abuse or alcoholism ≤ 6 months of Screening that would limit participation in the study, as determined by the Investigator.
    • Tracheostomy.
    • Prescreening ALSFRS-R slope less than 0.4 points/month, where prescreening ALSFRS-R slope is defined as follows: (48 - ALSFRS-R score at Screening) / (months from date of symptom onset to date of Screening).
    • History of or positive test result at Screening for human immunodeficiency virus. .
    • History of, or positive test result at Screening for, hepatitis C virus antibody.
    • Treatment with another investigational drug or biological agent within 1 month of Screening or 5 half-lives of study agent, whichever is longer.
    • Treatment with an antiplatelet or anticoagulant therapy that cannot safely be interrupted for lumbar puncture (LP) according to local standard of care and/or institutional guidelines, in the opinion of the Investigator or Prescriber.
    • Current or anticipated need, in the opinion of the Investigator, of a diaphragm pacing system during the study period.
    • Female participants who are pregnant or currently breastfeeding.

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

  • Site Contact Information

    Research Site
    La Jolla, California 92093
    United States

    Research Site
    Los Angeles, California 90048
    United States

    Research Site
    Palo Alto, California 94303
    United States

    Research Site
    Jacksonville, Florida 32224
    United States

    Research Site
    Miami, Florida 33136
    United States

    Research Site
    Atlanta, Georgia 30322
    United States

    Research Site
    Baltimore, Maryland 21205
    United States

    Research Site
    Boston, Massachusetts 02114
    United States

    Research Site
    Saint Louis, Missouri 63110
    United States

    Research Site
    Lincoln, Nebraska 68506
    United States

    Research Site
    New York, New York 10032
    United States

    Research Site
    Knoxville, Tennessee 37920
    United States

    Research Site
    Calgary, Alberta T2N 1N4

    Research Site
    Toronto, Ontario M4N 3M5

    Research Site
    Montreal, Quebec H2X 0A9

    Research Site

    Research Site

    Research Site
    St. Gallen,

    Research Site
    United Kingdom

    Research Site
    United Kingdom