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Long-Term Evaluation of BIIB067 (Tofersen)

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.
 
ClinicalTrials.gov Identifier: NCT03070119
Recruitment Status : Active, not recruiting
First Posted : March 3, 2017
Last Update Posted : May 22, 2023
Sponsor:
Collaborator:
Ionis Pharmaceuticals, Inc.
Information provided by (Responsible Party):
Biogen

Study Description
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Brief Summary:
The primary objective of the study is to evaluate the long-term safety and tolerability of BIIB067 (tofersen) in participants with amyotrophic lateral sclerosis (ALS) and confirmed superoxide dismutase 1 (SOD1) mutation. The secondary objectives are to evaluate the pharmacokinetic (PK), pharmacodynamic (PD), biomarker effects, and efficacy of BIIB067 administered to participants with ALS and a confirmed SOD1 mutation.

Condition or disease Intervention/treatment Phase
ALS Caused by Superoxide Dismutase 1 (SOD1) Mutation Drug: Tofersen Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 138 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 : June 13, 2024
Estimated Study Completion Date : June 13, 2024

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: BIIB067
Participants who have completed Parts A, B, or C of study 233AS101 will be placed in this arm.
 Drug: Tofersen
Participants will receive a loading dose regimen followed by maintenance dosing.
Other Names:
  • BIIB067
  • QALSODY


Outcome Measures
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Primary Outcome Measures :
  1. Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: Up to Week 364 ]
    An AE is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. An SAE is any untoward medical occurrence that at any dose results in death, life-threatening event, requires inpatient hospitalization, significant disability/incapacity or congenital anomaly.


Secondary Outcome Measures :
  1. Levels of BIIB067 in Plasma [ Time Frame: Up to Week 364 ]
  2. Levels of BIIB067 in Cerebrospinal Fluid (CSF) [ Time Frame: Up to Week 360 ]
  3. Change from Baseline in Total SOD1 Protein in CSF [ Time Frame: Baseline to Week 360 ]
  4. Change from Baseline in Neurofilament Light Chain (NfL) Concentration in Plasma [ Time Frame: Baseline to Week 364 ]
  5. Change from Baseline in Total ALS Functional Rating Scale - Revised (ALSFRS-R) Score [ Time Frame: Baseline to Week 364 ]
    The ALSFRS-R measures 4 functional domains, including respiratory, bulbar function, gross motor skills, and fine motor skills. There are 12 questions, each scored from 0 to 4, for a total possible score of 48, with higher scores representing better function.

  6. Change from Baseline in Slow Vital Capacity (SVC) [ Time Frame: Baseline to Week 364 ]
    Vital capacity will be measured by means of an SVC test, administered in the upright position. Upright SVC will be determined by performing 3 to 5 measures, in accordance with criteria established by the American Thoracic Society and the European Respiratory Society.

  7. Change from Baseline in Handheld Dynamometry (HHD) Megascore and Individual Muscle Strength [ Time Frame: Baseline to Week 364 ]
    Quantitative muscle strength will be evaluated using HHD, which tests isometric strength of multiple muscles using standard participant positioning. Approximately 8 muscle groups will be examined (per each side) in both upper and lower extremities.

  8. Time to Death or Permanent Ventilation [ Time Frame: Up to Week 364 ]
    Time to death or permanent ventilation is defined as the time to the earliest occurrence of one of the following events: Death; Permanent ventilation [≥22 hours of mechanical ventilation (invasive or noninvasive) per day for ≥21 consecutive days].

  9. Time to Death [ Time Frame: Up to Week 364 ]

Eligibility Criteria
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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.


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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 superoxide dismutase 1-amyotrophic lateral sclerosis (SOD1-ALS), and must have completed the End of Study Visit for either Parts A, B, or C of Study 233AS101 (NCT02623699) (i.e., were not withdrawn).
  • If taking riluzole, participant must be receiving a stable dose for ≥30 days prior to Day 1.
  • If taking edaravone, participant must have initiated edaravone ≥60 days (2 treatment cycles) prior to Day 1. Edaravone may not be administered on dosing days during this study.
  • 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.
  • For female participants of childbearing potential must agree to practice effective contraception during the study and be willing and able to continue contraception for 5 months after their last dose of study treatment.
  • Participants from Study 233AS101 Parts A and B 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.

Key Exclusion Criteria:

  • 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.
  • 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.
  • Female participants who are pregnant or currently breastfeeding.
  • Current enrollment in any other interventional study.

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply

Contacts and Locations
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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
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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, 94118
United States, Florida
Research Site
Jacksonville, Florida, United States, 32224
Research Site
Miami, Florida, United States, 33136
Research Site
Orlando, Florida, United States, 32806
United States, Georgia
Research Site
Atlanta, Georgia, United States, 30322
United States, Illinois
Research Site
Chicago, Illinois, United States, 60611
United States, Maryland
Research Site
Baltimore, Maryland, United States, 21287
United States, Massachusetts
Research Site
Boston, Massachusetts, United States, 02114
United States, Minnesota
Research Site
Rochester, Minnesota, United States, 55905
United States, Missouri
Research Site
Saint Louis, Missouri, United States, 63110
United States, Nebraska
Research Site
Lincoln, Nebraska, United States, 68510
United States, Ohio
Research Site
Cleveland, Ohio, United States, 44106
United States, Oregon
Research Site
Portland, Oregon, United States, 97213
United States, Tennessee
Research Site
Knoxville, Tennessee, United States, 37920
Belgium
Research Site
Leuven, Belgium, 3000
Canada, Alberta
Research Site
Calgary, Alberta, Canada, T2N 4Z6
Research Site
Edmonton, Alberta, Canada, T6G 2B7
Canada, Ontario
Research Site
Toronto, Ontario, Canada, M4N 3M5
Canada, Quebec
Research Site
Montreal, Quebec, Canada, H3A2B4
France
Research Site
Clermont-Ferrand cedex, Puy De Dome, France, 63003
Germany
Research Site
Ulm, Baden Wuerttemberg, Germany, 89081
Italy
Research Site
Torino, Italy, 10126
Japan
Research Site
Bunkyo-ku, Japan
Research Site
Kagoshima-shi, Japan
Research Site
Shinjuku-ku, Japan
Research Site
Suita-shi, Japan
New Zealand
Research Site
Christchurch, New Zealand, 8011
United Kingdom
Research Site
Sheffield, South Yorkshire, United Kingdom, S102HQ
Sponsors and Collaborators
Biogen
Ionis Pharmaceuticals, Inc.
Investigators
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Study Director: Medical Director Biogen
More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Biogen
ClinicalTrials.gov Identifier: NCT03070119    
Other Study ID Numbers: 233AS102
2016-003225-41 ( EudraCT Number )
First Posted: March 3, 2017    Key Record Dates
Last Update Posted: May 22, 2023
Last Verified: May 2023

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No