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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03070119




Registration number
NCT03070119
Ethics application status
Date submitted
28/02/2017
Date registered
3/03/2017
Date last updated
14/06/2024

Titles & IDs
Public title
Long-Term Evaluation of BIIB067 (Tofersen)
Scientific 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
Secondary ID [1] 0 0
2016-003225-41
Secondary ID [2] 0 0
233AS102
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
ALS Caused by Superoxide Dismutase 1 (SOD1) Mutation 0 0
Condition category
Condition code

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Tofersen

Experimental: BIIB067 - Participants who have completed Parts A, B, or C of study 233AS101 will be placed in this arm.


Treatment: Drugs: Tofersen
Participants will receive a loading dose regimen followed by maintenance dosing.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
Timepoint [1] 0 0
Up to Week 364
Secondary outcome [1] 0 0
Levels of BIIB067 in Plasma
Timepoint [1] 0 0
Up to Week 364
Secondary outcome [2] 0 0
Levels of BIIB067 in Cerebrospinal Fluid (CSF)
Timepoint [2] 0 0
Up to Week 360
Secondary outcome [3] 0 0
Change from Baseline in Total SOD1 Protein in CSF
Timepoint [3] 0 0
Baseline to Week 360
Secondary outcome [4] 0 0
Change from Baseline in Neurofilament Light Chain (NfL) Concentration in Plasma
Timepoint [4] 0 0
Baseline to Week 364
Secondary outcome [5] 0 0
Change from Baseline in Total ALS Functional Rating Scale - Revised (ALSFRS-R) Score
Timepoint [5] 0 0
Baseline to Week 364
Secondary outcome [6] 0 0
Change from Baseline in Slow Vital Capacity (SVC)
Timepoint [6] 0 0
Baseline to Week 364
Secondary outcome [7] 0 0
Change from Baseline in Handheld Dynamometry (HHD) Megascore and Individual Muscle Strength
Timepoint [7] 0 0
Baseline to Week 364
Secondary outcome [8] 0 0
Time to Death or Permanent Ventilation
Timepoint [8] 0 0
Up to Week 364
Secondary outcome [9] 0 0
Time to Death
Timepoint [9] 0 0
Up to Week 364

Eligibility
Key inclusion criteria
Key

* 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
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
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

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Other
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
8/03/2017
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
12/08/2024
Actual
Sample size
Target
Accrual to date
Final
139
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
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United States of America
State/province [2] 0 0
California
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United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Georgia
Country [5] 0 0
United States of America
State/province [5] 0 0
Illinois
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United States of America
State/province [6] 0 0
Maryland
Country [7] 0 0
United States of America
State/province [7] 0 0
Massachusetts
Country [8] 0 0
United States of America
State/province [8] 0 0
Minnesota
Country [9] 0 0
United States of America
State/province [9] 0 0
Missouri
Country [10] 0 0
United States of America
State/province [10] 0 0
Nebraska
Country [11] 0 0
United States of America
State/province [11] 0 0
Ohio
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United States of America
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Oregon
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United States of America
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Tennessee
Country [14] 0 0
Belgium
State/province [14] 0 0
Leuven
Country [15] 0 0
Canada
State/province [15] 0 0
Alberta
Country [16] 0 0
Canada
State/province [16] 0 0
Ontario
Country [17] 0 0
Canada
State/province [17] 0 0
Quebec
Country [18] 0 0
France
State/province [18] 0 0
Puy De Dome
Country [19] 0 0
Germany
State/province [19] 0 0
Baden Wuerttemberg
Country [20] 0 0
Italy
State/province [20] 0 0
Torino
Country [21] 0 0
Japan
State/province [21] 0 0
Bunkyo-ku
Country [22] 0 0
Japan
State/province [22] 0 0
Kagoshima-shi
Country [23] 0 0
Japan
State/province [23] 0 0
Shinjuku-ku
Country [24] 0 0
Japan
State/province [24] 0 0
Suita-shi
Country [25] 0 0
New Zealand
State/province [25] 0 0
Christchurch
Country [26] 0 0
United Kingdom
State/province [26] 0 0
South Yorkshire

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Biogen
Address
Country
Other collaborator category [1] 0 0
Commercial sector/industry
Name [1] 0 0
Ionis Pharmaceuticals, Inc.
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/study/NCT03070119
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Medical Director
Address 0 0
Biogen
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries