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Single and Multiple Dose Study of BIIB067 in Adults With Amyotrophic Lateral Sclerosis (ALS)

This study is currently recruiting participants. (see Contacts and Locations)
Verified March 2017 by Biogen
Sponsor:
Collaborator:
Ionis Pharmaceuticals, Inc.
Information provided by (Responsible Party):
Biogen
ClinicalTrials.gov Identifier:
NCT02623699
First received: November 24, 2015
Last updated: March 27, 2017
Last verified: March 2017
  Purpose
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).

Condition Intervention Phase
Amyotrophic Lateral Sclerosis
Drug: BIIB067
Other: Placebo
Phase 1

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Participant, Care Provider, Investigator, Outcomes Assessor
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Biogen:

Primary Outcome Measures:
  • Number of participants experiencing Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: up to day 169 ]
    Part A: up to day 57; Part B: up to day 169

  • Number of participants with clinically significant laboratory assessment abnormalities [ Time Frame: up to day 169 ]
    Part A: up to day 57; Part B: up to day 169

  • Number of participants with clinically significant vital sign abnormalities [ Time Frame: up to day 169 ]
    Part A: up to day 57; Part B: up to day 169

  • Number of participants with clinically significant physical examination abnormalities [ Time Frame: up to day 169 ]
    Part A: up to day 57; Part B: up to day 169

  • Number of participants with clinically significant neurological examination abnormalities [ Time Frame: up to day 169 ]
    Part A: up to day 57; Part B: up to day 169

  • Number of participants with clinically significant 12-lead electrocardiograms (ECGs) abnormalities [ Time Frame: up to day 169 ]
    Part A: up to day 57; Part B: up to day 169

  • PK parameter of BIIB067 in plasma: Maximum observed concentration (Cmax) [ Time Frame: up to day 169 ]
    Part A: up to day 57; Part B: up to day 169

  • PK parameter of BIIB067 in plasma: Time to reach maximum observed concentration (Tmax) [ Time Frame: up to day 169 ]
    Part A: up to day 57; Part B: up to day 169

  • PK parameter of BIIB067 in plasma: Area under the concentration-time curve from time zero to infinity (AUCinf) [ Time Frame: up to day 169 ]
    Part A: up to day 57; Part B: up to day 169

  • PK parameter of BIIB067 in plasma: Area under the concentration-time curve from time zero to the time of the last measurable concentration (AUClast) [ Time Frame: up to day 169 ]
    Part A: up to day 57; Part B: up to day 169

  • PK parameter of BIIB067 in plasma: Apparent terminal elimination half-life (t1/2) [ Time Frame: up to day 169 ]
    Part A: up to day 57; Part B: up to day 169

  • PK parameters of BIIB067 in CSF levels: Terminal elimination half-life (t1/2) [ Time Frame: up to day 169 ]
    Part A: up to day 57; Part B: up to day 169


Secondary Outcome Measures:
  • Change from baseline in CSF levels of SOD1 protein. [ Time Frame: up to day 169 ]
    Part A: up to day 57; Part B: up to day 169


Estimated Enrollment: 72
Study Start Date: January 2016
Estimated Study Completion Date: July 2018
Estimated Primary Completion Date: July 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Single Ascending Dose
Part A: Randomized single ascending dose
Drug: BIIB067
Part A and B
Other: Placebo
Part A and B
Experimental: Multiple Ascending Dose
Part B: Randomized multiple ascending dose
Drug: BIIB067
Part A and B
Other: Placebo
Part A and B

  Eligibility

Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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 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

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

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02623699

Contacts
Contact: Biogen clinicaltrials@biogen.com

Locations
United States, Arizona
Barrow Neurological Institute Recruiting
Phoenix, Arizona, United States, 85013
United States, California
University of California San Diego Medical Center Recruiting
La Jolla, California, United States, 92093-0949
California Pacific Medical Center Recruiting
San Francisco, California, United States, 94115
United States, Florida
Compass Research, LLC Recruiting
Orlando, Florida, United States, 32806
United States, Georgia
The Emory Clinic Recruiting
Atlanta, Georgia, United States, 30322
United States, Maryland
Johns Hopkins Hospital Recruiting
Baltimore, Maryland, United States, 21287
United States, Massachusetts
Massachusetts General Hospital, MA Recruiting
Boston, Massachusetts, United States, 02114
United States, Missouri
Washington University School of Medicine Recruiting
St. Louis, Missouri, United States, 63110
United States, Tennessee
Volunteer Research Group, LLC Recruiting
Knoxville, Tennessee, United States, 37920
Belgium
UZ Leuven Recruiting
Leuven, Belgium, 3000
Canada, Ontario
Sunnybrook Health Sciences Centre Not yet recruiting
Toronto, Ontario, Canada, M4N 3M5
Canada, Quebec
Montreal Neurological Institute Clinical Research Unit Recruiting
Montréal, Quebec, Canada, H3A 2B4
France
Groupe Hospitalier Pitie-Salpetriere Not yet recruiting
Paris, France, 75651
Germany
Universitaetsklinikum Ulm Recruiting
Ulm, Baden Wuerttemberg, Germany, 89081
Italy
Università degli Studi di Torino Not yet recruiting
Torino, Italy, 10126
Sweden
Norrlands universitetssjukhus Not yet recruiting
Umeå, Sweden, 90185
United Kingdom
Sheffield Institute for Translational Neuroscience Recruiting
Sheffield, South Yorkshire, United Kingdom, S10 2HQ
Sponsors and Collaborators
Biogen
Ionis Pharmaceuticals, Inc.
Investigators
Study Director: Medical Director Biogen
  More Information

Responsible Party: Biogen
ClinicalTrials.gov Identifier: NCT02623699     History of Changes
Other Study ID Numbers: 233AS101
2015-004098-33 ( EudraCT Number )
Study First Received: November 24, 2015
Last Updated: March 27, 2017

Keywords provided by Biogen:
Isis-SOD1Rx
SOD1
ALS

Additional relevant MeSH terms:
Sclerosis
Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases

ClinicalTrials.gov processed this record on March 28, 2017