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-ALS. The secondary objective of this study is to evaluate the pharmacokinetic profile of BIIB078.

Disease:

Amyotrophic Lateral Sclerosis (ALS)

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Enrolling

Phase:

Phase I

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

Biogen Medical Director

Clinicaltrials.gov ID (11 digit #):

NCT03626012

Neals Affiliated?

No

Coordinating Center Contact Information


US Biogen Medical Information / .(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:

Biogen

Participant Duration:

Estimated Enrollment:

59

Estimated Study Start Date:

09/10/2018

Estimated Study Completion Date:

11/29/2020

Posting Last Modified Date:

10/19/2018

Date Study Added to alsconsortium.org:

10/19/2018
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Min Vital Capacity (% predicted normal):

    50

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Key Inclusion Criteria:

    Ability of the subject 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 subject privacy regulations; or, in the event of the subject'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 subjects 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).
    Subjects 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).
    Subjects 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 subject as to be able to provide accurate information about the subject'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.
    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 anti-platelet or anticoagulant therapy ≤ 14 days before Screening (with the exception of aspirin ≤ 325 mg/day) or anticipated use during the study.
    Current or anticipated need, in the opinion of the Investigator, of a diaphragm pacing system during the study period.
    Female subjects 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
    Miami, Florida 33136
    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
    Knoxville, Tennessee 37920
    United States

    Research Site
    Montreal, Quebec H3A 2B4
    Canada