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Long-Term Evaluation of BIIB067

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ClinicalTrials.gov Identifier: NCT03070119
Recruitment Status : Enrolling by invitation
First Posted : March 3, 2017
Last Update Posted : August 15, 2018
Sponsor:
Collaborator:
Ionis Pharmaceuticals, Inc.
Information provided by (Responsible Party):
Biogen

Brief Summary:
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 or disease Intervention/treatment Phase
ALS Caused by Superoxide Dismutase 1 (SOD1) Mutation Drug: BIIB067 Phase 1

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 48 participants
Allocation: Non-Randomized
Intervention Model: Sequential 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
Actual Study Start Date : March 8, 2017
Estimated Primary Completion Date : January 31, 2020
Estimated Study Completion Date : January 31, 2020


Arm Intervention/treatment
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 A Drug: BIIB067
Participants will receive a loading dose regimen followed by maintenance dosing.

Experimental: BIIB067 High dose B Drug: BIIB067
Participants will receive a loading dose regimen followed by maintenance dosing.




Primary Outcome Measures :
  1. Number of participants experiencing AEs and serious adverse events (SAEs) [ Time Frame: Up to Month 27 ]
    Safety Surveillance


Secondary Outcome Measures :
  1. PK parameter of BIIB067 in plasma: Maximum observed concentration (Cmax) [ Time Frame: Up to Month 27 ]
    Venipuncture for PK data collection

  2. PK parameter of BIIB067 in cerebrospinal fluid (CSF): Cmax [ Time Frame: Up to Month 27 ]
    Venipuncture for PK data collection

  3. PK parameter of BIIB067 in plasma: Time to reach the maximum observed concentration (Tmax) [ Time Frame: Up to Month 27 ]
    Venipuncture for PK data collection

  4. PK parameter of BIIB067 in CSF: Tmax [ Time Frame: Up to Month 27 ]
    Venipuncture for PK data collection

  5. PK parameter of BIIB067 in plasma: Area under the concentration-time curve from time 0 to infinity (AUCinf) [ Time Frame: Up to Month 27 ]
    Venipuncture for PK data collection

  6. PK parameter of BIIB067 in CSF: AUCinf [ Time Frame: Up to Month 27 ]
    Venipuncture for PK data collection

  7. 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 Month 27 ]
    Venipuncture for PK data collection

  8. PK parameter of BIIB067 in CSF: AUClast [ Time Frame: Up to Month 27 ]
    Venipuncture for PK data collection

  9. PK parameter of BIIB067 in plasma: Apparent terminal elimination half-life (t½) [ Time Frame: Up to Month 27 ]
    Venipuncture for PK data collection

  10. PK parameter of BIIB067 in CSF: t½ [ Time Frame: Up to Month 27 ]
    Venipuncture for PK data collection



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
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.
  • Must have a washout ≥16 weeks between the last dose of study treatment received in Study 233AS101 and the first dose of BIIB067 received in the current Study 233AS102.
  • Participants taking concomitant edaravone at study entry must be on a stable dose for ≥ 30 days prior to the first dose of study treatment (Day 1) and must continue with the same dose regimen throughout the study, unless the Investigator determines that edaravone should be discontinued for medical reasons, in which case it may not be restarted during the study. Edaravone may not be administered on dosing days during this study.

Key Exclusion Criteria:

  • History of or positive test result for human immunodeficiency virus.
  • History of, or positive test result at Screening for hepatitis C virus 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.
  • Current hepatitis B infection (defined as positive for hepatitis B surface antigen [HBsAg] and/or hepatitis B core antibody [HBcAb]). Participants with immunity to hepatitis B from previous natural infection (defined as negative HBsAg, positive hepatitis B surface antibody immunoglobulin G, and positive HBcAb) or vaccination (defined as positive anti-HBs) are eligible to participate in the study.
  • Presence of an implanted intravenous port/catheter.

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply


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

Responsible Party: Biogen
ClinicalTrials.gov Identifier: NCT03070119     History of Changes
Other Study ID Numbers: 233AS102
2016-003225-41 ( EudraCT Number )
First Posted: March 3, 2017    Key Record Dates
Last Update Posted: August 15, 2018
Last Verified: August 2018

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