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

Study Purpose:

The primary objective of the study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of BIIB067 (Isis-SOD1Rx) in adults with ALS.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Active, currently recruiting

Phase:

Phase I

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

Clinicaltrials.gov ID (11 digit #):

NCT02623699

Neals Affiliated?

No

Coordinating Center Contact Information

Biogen
.(JavaScript must be enabled to view this email address)
.(JavaScript must be enabled to view this email address) United States

Full Study Summary:

The primary objective of the study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of BIIB067 (Isis-SOD1Rx) in adults with ALS. The secondary objective is to evaluate the effects of BIIB067 (Isis-SOD1Rx) on levels of superoxide dismutase 1 (SOD1) protein in the cerebrospinal fluid (CSF).

Study Sponsor:

Biogen

Participant Duration:

Estimated Enrollment:

72

Estimated Study Start Date:

12/31/2015

Estimated Study Completion Date:

03/31/2018

Posting Last Modified Date:

01/31/2016

Date Study Added to alsconsortium.org:

12/13/2015
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Min Vital Capacity (% predicted normal):

    45

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:
    -Subjects with sporadic ALS, eligible for Part A only, must have weakness attributable to sporadic ALS and a diagnosis of possible, laboratory supported probable, probable, or definite ALS according to the World Federation of Neurology El Escorial criteria (revised according to the Airlie House Conference 1998 [Brooks 2000]). Presence of SOD1 mutation is not required for inclusion in Part A.
    -Subjects with SOD1-ALS, eligible for Part A and Part B, must have weakness attributable to ALS and documented SOD1 mutation.
    -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, subject 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.

    Exclusion Criteria:
    -History of or positive test result for human immunodeficiency virus.
    -History of or positive test result for hepatitis C virus antibody or hepatitis B virus (defined as positive for both hepatitis B surface antigen and hepatitis B core antibody).
    -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

  • Site Contact Information

    University of California San Diego Medical Center
    La Jolla, California 92093
    United States

    Barrow Neurological Institute
    Phoenix, Arizona 85013
    United States

    Volunteer Research Group, LLC
    Knoxville, Tennessee 37920
    United States

    Washington University School of Medicine
    St. Louis, Missouri 63110
    United States

    Massachusetts General Hospital
    Boston, Massachusetts 02114
    United States

    Johns Hopkins Hospital
    Baltimore, Maryland 21287
    United States

    The Emory Clinic
    Atlanta, Georgia 30322
    United States

    Compass Research, LLC
    Orlando, Florida 32806
    United States

    California Pacific Medical Center
    San Francisco, California 94115
    United States

    Sheffield Institute for Translational Neuroscience
    Sheffield, South Yorkshire S10 2HQ
    United Kingdom

    Norrlands universitetssjukhus
    Umea, 90185
    Sweden

    Universita degli Studi di Torino
    Torino, 10126
    Italy

    Universitaetsklinikum Ulm
    Ulm, Baden Wuerttemberg 89081
    Germany

    Groupe Hospitalier Pitie-Salpetriere
    Paris, 75013
    France

    Montreal Neurological Institute Clinical Research Unit
    Montreal, Quebec H3A 2B4
    Canada

    Sunnybrook Health Sciences Centre
    Toronto, Ontario M4N 3M5
    Canada

    UZ Leuven
    Leuven, 3000
    Belgium