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Long-Term Evaluation of BIIB067

This study is enrolling participants by invitation only.
Sponsor:
ClinicalTrials.gov Identifier:
NCT03070119
First Posted: March 3, 2017
Last Update Posted: October 11, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Collaborator:
Ionis Pharmaceuticals, Inc.
Information provided by (Responsible Party):
Biogen
  Purpose
The primary objective of the study is to evaluate the long-term safety and tolerability of BIIB067 in participants with Amyotrophic Lateral Sclerosis Caused by Superoxide Dismutase 1 Mutation (SOD1-ALS). The secondary objective is to evaluate the pharmacokinetic (PK) profile of BIIB067 in participants with SOD1-ALS.

Condition Intervention Phase
ALS Caused by Superoxide Dismutase 1 (SOD1) Mutation Drug: BIIB067 Phase 1

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Extension Study to Assess the Long-Term Safety, Tolerability, Pharmacokinetics, and Effect on Disease Progression of BIIB067 Administered to Previously Treated Adults With Amyotrophic Lateral Sclerosis Caused by Superoxide Dismutase 1 Mutation

Resource links provided by NLM:


Further study details as provided by Biogen:

Primary Outcome Measures:
  • Number of participants experiencing AEs and serious adverse events (SAEs) [ Time Frame: Up to 15 months ]
    Safety Surveillance

  • Number of participants with clinically significant laboratory assessment abnormalities [ Time Frame: Up to 15 months ]
    Safety Surveillance

  • Number of participants with clinically significant vital sign abnormalities [ Time Frame: Up to 15 months ]
    Safety Surveillance

  • Number of participants with clinically significant physical examination abnormalities [ Time Frame: Up to 15 months ]
    Safety Surveillance

  • Number of participants with clinically significant neurological examination abnormalities [ Time Frame: Up to 15 months ]
    Safety Surveillance

  • Number of participants with clinically significant 12-lead electrocardiograms (ECGs) abnormalities [ Time Frame: Up to 15 months ]
    Safety Surveillance


Secondary Outcome Measures:
  • PK parameter of BIIB067 in plasma: Maximum observed concentration (Cmax) [ Time Frame: Up to 15 months ]
    Venipuncture for PK data collection

  • PK parameter of BIIB067 in cerebrospinal fluid (CSF): Cmax [ Time Frame: Up to 15 months ]
    Venipuncture for PK data collection

  • PK parameter of BIIB067 in plasma: Time to reach the maximum observed concentration (Tmax) [ Time Frame: Up to 15 months ]
    Venipuncture for PK data collection

  • PK parameter of BIIB067 in CSF: Tmax [ Time Frame: Up to 15 months ]
    Venipuncture for PK data collection

  • PK parameter of BIIB067 in plasma: Area under the concentration-time curve from time 0 to infinity (AUCinf) [ Time Frame: Up to 15 months ]
    Venipuncture for PK data collection

  • PK parameter of BIIB067 in CSF: AUCinf [ Time Frame: Up to 15 months ]
    Venipuncture for PK data collection

  • PK parameter of BIIB067 in plasma: Area under the concentration-time curve from time 0 to time of the last measurable (AUClast) [ Time Frame: Up to 15 months ]
    Venipuncture for PK data collection

  • PK parameter of BIIB067 in CSF: AUClast [ Time Frame: Up to 15 months ]
    Venipuncture for PK data collection

  • PK parameter of BIIB067 in plasma: Apparent terminal elimination half-life (t½) [ Time Frame: Up to 15 months ]
    Venipuncture for PK data collection

  • PK parameter of BIIB067 in CSF: t½ [ Time Frame: Up to 15 months ]
    Venipuncture for PK data collection


Estimated Enrollment: 48
Actual Study Start Date: March 8, 2017
Estimated Study Completion Date: January 1, 2020
Estimated Primary Completion Date: January 1, 2020 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: BIIB067 Low dose Drug: BIIB067
Participants will receive a loading dose regimen followed by maintenance dosing.
Experimental: BIIB067 Mid dose Drug: BIIB067
Participants will receive a loading dose regimen followed by maintenance dosing.
Experimental: BIIB067 High dose Drug: BIIB067
Participants will receive a loading dose regimen followed by maintenance dosing.

  Eligibility

Information from the National Library of Medicine

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, Learn About Clinical Studies.


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

Key Inclusion Criteria:

  • Must have diagnosis of SOD1-ALS, and must have completed Part A and/or Part B of Study 233AS101 (NCT02623699) (i.e., were not withdrawn and did not miss more than 1 dose of study drug).
  • If taking riluzole, must be receiving a stable dose for ≥30 days prior to Day 1 and expected to remain at that dose until the final study visit.
  • For participants of childbearing potential must agree to practice effective contraception during the study and for 5 months after their last dose of study treatment.
  • 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).
  • History of allergies to a broad range of anesthetics.
  • Presence of risk for increased or uncontrolled bleeding and/or risk of bleeding that is not managed optimally and could place a participant at an increased risk for bleeding during or after a Lumbar Puncture (LP) procedure. These risks could include, but are not limited to, anatomical factors at or near the LP site (e.g., vascular abnormalities, neoplasms, or other abnormalities) and underlying disorders of the coagulation cascade, platelet function, or platelet count (e.g., hemophilia, Von Willebrand's disease, liver disease).
  • Presence of an implanted shunt for the drainage of CSF or an implanted central nervous system (CNS) catheter.
  • Prior or current treatment with small interfering ribonucleic acid (RNA), stem cell therapy, or gene therapy.
  • Treatment with another investigational drug, biological agent (excluding BIIB067), or device within 1 month or 5 half-lives of study agent, whichever is longer.
  • Current or anticipated need, in the opinion of the Investigator, of a diaphragm pacing system (DPS) during the study period.
  • Female participants who are pregnant or currently breastfeeding.
  • 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.

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply

  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03070119


Locations
United States, Arizona
Research Site
Phoenix, Arizona, United States, 85013
United States, California
Research Site
La Jolla, California, United States, 92093-0949
Research Site
San Francisco, California, United States, 94115
United States, Florida
Research Site
Orlando, Florida, United States, 32806
United States, Georgia
Research Site
Atlanta, Georgia, United States, 30322
United States, Maryland
Research Site
Baltimore, Maryland, United States, 21287
United States, Massachusetts
Research Site
Boston, Massachusetts, United States, 02114
United States, Missouri
Research Site
Saint Louis, Missouri, United States, 63110
United States, Tennessee
Research Site
Knoxville, Tennessee, United States, 37920
Belgium
Research Site
Leuven, Belgium, 3000
Canada, Ontario
Research Site
Toronto, Ontario, Canada, M4N 3M5
Canada, Quebec
Research Site
Montreal, Quebec, Canada, H3A2B4
Germany
Research Site
Ulm, Baden Wuerttemberg, Germany, 89081
United Kingdom
Research Site
Sheffield, South Yorkshire, United Kingdom, S102HQ
Sponsors and Collaborators
Biogen
Ionis Pharmaceuticals, Inc.
Investigators
Study Director: Medical Director Biogen
  More Information

Responsible Party: Biogen
ClinicalTrials.gov Identifier: NCT03070119     History of Changes
Other Study ID Numbers: 233AS102
2016-003225-41 ( EudraCT Number )
First Submitted: February 28, 2017
First Posted: March 3, 2017
Last Update Posted: October 11, 2017
Last Verified: October 2017

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Amyotrophic Lateral Sclerosis
Spinal Cord Diseases
Central Nervous System Diseases
Nervous System Diseases
Motor Neuron Disease
Neurodegenerative Diseases
TDP-43 Proteinopathies
Neuromuscular Diseases
Proteostasis Deficiencies
Metabolic Diseases
Superoxide Dismutase
Free Radical Scavengers
Antioxidants
Molecular Mechanisms of Pharmacological Action
Protective Agents
Physiological Effects of Drugs