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
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
Study Chair(s)/Principal Investigator(s):
Clinicaltrials.gov ID (11 digit #):NCT02623699
Coordinating Center Contact InformationBiogen
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).
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
Time since Symptom Onset:
Time since Diagnosis:
Can participants use Riluzole?Yes
-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.
-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.
Site Contact Information
University of California San Diego Medical Center
La Jolla, California 92093
Barrow Neurological Institute
Phoenix, Arizona 85013
Volunteer Research Group, LLC
Knoxville, Tennessee 37920
Washington University School of Medicine
St. Louis, Missouri 63110
Massachusetts General Hospital
Boston, Massachusetts 02114
Johns Hopkins Hospital
Baltimore, Maryland 21287
The Emory Clinic
Atlanta, Georgia 30322
Compass Research, LLC
Orlando, Florida 32806
California Pacific Medical Center
San Francisco, California 94115
Sheffield Institute for Translational Neuroscience
Sheffield, South Yorkshire S10 2HQ
Universita degli Studi di Torino
Ulm, Baden Wuerttemberg 89081
Groupe Hospitalier Pitie-Salpetriere
Montreal Neurological Institute Clinical Research Unit
Montreal, Quebec H3A 2B4
Sunnybrook Health Sciences Centre
Toronto, Ontario M4N 3M5