Please note that the copy function is not enabled for this field.
If you wish to
modify
existing outcomes, please copy and paste the current outcome text into the Update field.
LOGIN
CREATE ACCOUNT
LOGIN
CREATE ACCOUNT
MY TRIALS
REGISTER TRIAL
FAQs
HINTS AND TIPS
DEFINITIONS
Trial Review
The ANZCTR website will be unavailable from 1pm until 3pm (AEDT) on Wednesday the 30th of October for website maintenance. Please be sure to log out of the system in order to avoid any loss of data.
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 endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this
information for consumers
Download to PDF
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
19/08/2024
Titles & IDs
Public title
Long-Term Evaluation of BIIB067 (Tofersen)
Query!
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
Query!
Secondary ID [1]
0
0
2016-003225-41
Query!
Secondary ID [2]
0
0
233AS102
Query!
Universal Trial Number (UTN)
Query!
Trial acronym
Query!
Linked study record
Query!
Health condition
Health condition(s) or problem(s) studied:
ALS Caused by Superoxide Dismutase 1 (SOD1) Mutation
0
0
Query!
Condition category
Condition code
Intervention/exposure
Study type
Interventional
Query!
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.
Query!
Intervention code [1]
0
0
Treatment: Drugs
Query!
Comparator / control treatment
Query!
Control group
Query!
Outcomes
Primary outcome [1]
0
0
Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
Query!
Assessment method [1]
0
0
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.
Query!
Timepoint [1]
0
0
Up to Week 364
Query!
Secondary outcome [1]
0
0
Levels of BIIB067 in Plasma
Query!
Assessment method [1]
0
0
Query!
Timepoint [1]
0
0
Up to Week 364
Query!
Secondary outcome [2]
0
0
Levels of BIIB067 in Cerebrospinal Fluid (CSF)
Query!
Assessment method [2]
0
0
Query!
Timepoint [2]
0
0
Up to Week 360
Query!
Secondary outcome [3]
0
0
Change from Baseline in Total SOD1 Protein in CSF
Query!
Assessment method [3]
0
0
Query!
Timepoint [3]
0
0
Baseline to Week 360
Query!
Secondary outcome [4]
0
0
Change from Baseline in Neurofilament Light Chain (NfL) Concentration in Plasma
Query!
Assessment method [4]
0
0
Query!
Timepoint [4]
0
0
Baseline to Week 364
Query!
Secondary outcome [5]
0
0
Change from Baseline in Total ALS Functional Rating Scale - Revised (ALSFRS-R) Score
Query!
Assessment method [5]
0
0
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.
Query!
Timepoint [5]
0
0
Baseline to Week 364
Query!
Secondary outcome [6]
0
0
Change from Baseline in Slow Vital Capacity (SVC)
Query!
Assessment method [6]
0
0
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.
Query!
Timepoint [6]
0
0
Baseline to Week 364
Query!
Secondary outcome [7]
0
0
Change from Baseline in Handheld Dynamometry (HHD) Megascore and Individual Muscle Strength
Query!
Assessment method [7]
0
0
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.
Query!
Timepoint [7]
0
0
Baseline to Week 364
Query!
Secondary outcome [8]
0
0
Time to Death or Permanent Ventilation
Query!
Assessment method [8]
0
0
Time to death or permanent ventilation is defined as the time to the earliest occurrence of one of the following events: Death; Permanent ventilation \[=22 hours of mechanical ventilation (invasive or noninvasive) per day for =21 consecutive days\].
Query!
Timepoint [8]
0
0
Up to Week 364
Query!
Secondary outcome [9]
0
0
Time to Death
Query!
Assessment method [9]
0
0
Query!
Timepoint [9]
0
0
Up to Week 364
Query!
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
Query!
Minimum age
18
Years
Query!
Query!
Maximum age
No limit
Query!
Query!
Sex
Both males and females
Query!
Can healthy volunteers participate?
No
Query!
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
Query!
Study design
Purpose of the study
Treatment
Query!
Allocation to intervention
Non-randomised trial
Query!
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Query!
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Query!
Masking / blinding
Open (masking not used)
Query!
Who is / are masked / blinded?
Query!
Query!
Query!
Query!
Intervention assignment
Other
Query!
Other design features
Query!
Phase
Phase 3
Query!
Type of endpoint/s
Query!
Statistical methods / analysis
Query!
Recruitment
Recruitment status
Completed
Query!
Data analysis
Query!
Reason for early stopping/withdrawal
Query!
Other reasons
Query!
Date of first participant enrolment
Anticipated
Query!
Actual
8/03/2017
Query!
Date of last participant enrolment
Anticipated
Query!
Actual
Query!
Date of last data collection
Anticipated
Query!
Actual
12/08/2024
Query!
Sample size
Target
Query!
Accrual to date
Query!
Final
139
Query!
Recruitment in Australia
Recruitment state(s)
Query!
Recruitment outside Australia
Country [1]
0
0
United States of America
Query!
State/province [1]
0
0
Arizona
Query!
Country [2]
0
0
United States of America
Query!
State/province [2]
0
0
California
Query!
Country [3]
0
0
United States of America
Query!
State/province [3]
0
0
Florida
Query!
Country [4]
0
0
United States of America
Query!
State/province [4]
0
0
Georgia
Query!
Country [5]
0
0
United States of America
Query!
State/province [5]
0
0
Illinois
Query!
Country [6]
0
0
United States of America
Query!
State/province [6]
0
0
Maryland
Query!
Country [7]
0
0
United States of America
Query!
State/province [7]
0
0
Massachusetts
Query!
Country [8]
0
0
United States of America
Query!
State/province [8]
0
0
Minnesota
Query!
Country [9]
0
0
United States of America
Query!
State/province [9]
0
0
Missouri
Query!
Country [10]
0
0
United States of America
Query!
State/province [10]
0
0
Nebraska
Query!
Country [11]
0
0
United States of America
Query!
State/province [11]
0
0
Ohio
Query!
Country [12]
0
0
United States of America
Query!
State/province [12]
0
0
Oregon
Query!
Country [13]
0
0
United States of America
Query!
State/province [13]
0
0
Tennessee
Query!
Country [14]
0
0
Belgium
Query!
State/province [14]
0
0
Leuven
Query!
Country [15]
0
0
Canada
Query!
State/province [15]
0
0
Alberta
Query!
Country [16]
0
0
Canada
Query!
State/province [16]
0
0
Ontario
Query!
Country [17]
0
0
Canada
Query!
State/province [17]
0
0
Quebec
Query!
Country [18]
0
0
France
Query!
State/province [18]
0
0
Puy De Dome
Query!
Country [19]
0
0
Germany
Query!
State/province [19]
0
0
Baden Wuerttemberg
Query!
Country [20]
0
0
Italy
Query!
State/province [20]
0
0
Torino
Query!
Country [21]
0
0
Japan
Query!
State/province [21]
0
0
Bunkyo-ku
Query!
Country [22]
0
0
Japan
Query!
State/province [22]
0
0
Kagoshima-shi
Query!
Country [23]
0
0
Japan
Query!
State/province [23]
0
0
Shinjuku-ku
Query!
Country [24]
0
0
Japan
Query!
State/province [24]
0
0
Suita-shi
Query!
Country [25]
0
0
New Zealand
Query!
State/province [25]
0
0
Christchurch
Query!
Country [26]
0
0
United Kingdom
Query!
State/province [26]
0
0
South Yorkshire
Query!
Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Query!
Name
Biogen
Query!
Address
Query!
Country
Query!
Other collaborator category [1]
0
0
Commercial sector/industry
Query!
Name [1]
0
0
Ionis Pharmaceuticals, Inc.
Query!
Address [1]
0
0
Query!
Country [1]
0
0
Query!
Ethics approval
Ethics application status
Query!
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.
Query!
Trial website
https://clinicaltrials.gov/study/NCT03070119
Query!
Trial related presentations / publications
Miller TM, Cudkowicz ME, Genge A, Shaw PJ, Sobue G, Bucelli RC, Chio A, Van Damme P, Ludolph AC, Glass JD, Andrews JA, Babu S, Benatar M, McDermott CJ, Cochrane T, Chary S, Chew S, Zhu H, Wu F, Nestorov I, Graham D, Sun P, McNeill M, Fanning L, Ferguson TA, Fradette S; VALOR and OLE Working Group. Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS. N Engl J Med. 2022 Sep 22;387(12):1099-1110. doi: 10.1056/NEJMoa2204705.
Query!
Public notes
Query!
Contacts
Principal investigator
Name
0
0
Medical Director
Query!
Address
0
0
Biogen
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for public queries
Name
0
0
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for scientific queries
No information has been provided regarding IPD availability
What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT03070119
Download to PDF