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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT01622088
Registration number
NCT01622088
Ethics application status
Date submitted
3/05/2012
Date registered
18/06/2012
Date last updated
3/05/2022
Titles & IDs
Public title
Phase 3 Extension Study of Dexpramipexole in ALS
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Scientific title
An Open-Label, Multicenter, Extension Study to Evaluate the Long-Term Safety and Efficacy of Dexpramipexole (BIIB050) in Subjects With Amyotrophic Lateral Sclerosis
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Secondary ID [1]
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EUDRA CT #: 2011-006119-70
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Secondary ID [2]
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223AS304
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Universal Trial Number (UTN)
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Trial acronym
ENVISION
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Amyotrophic Lateral Sclerosis
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Condition category
Condition code
Neurological
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Neurodegenerative diseases
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Dexpramipexole
Experimental: Dexpramipexole - Dexpramipexole open-label
Treatment: Drugs: Dexpramipexole
Oral tablet 150 mg given twice daily (BID)
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Number of Subjects Who Reported an Adverse Event
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Assessment method [1]
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The number of subjects who reported an adverse event during the study
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Timepoint [1]
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Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
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Primary outcome [2]
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Number of Subjects Who Experienced a Serious Adverse Event
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Assessment method [2]
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The number of subjects enrolled who reported a serious adverse event during the study
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Timepoint [2]
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Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
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Primary outcome [3]
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Number of Subjects Who Discontinued the Study Treatment Due to an Adverse Event
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Assessment method [3]
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The number of subjects enrolled who discontinued the study treatment due to an adverse event during the study
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Timepoint [3]
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Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
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Primary outcome [4]
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Number of Participants With Potentially Clinically Significant Vital Sign Results
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Assessment method [4]
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Number of Participants with Potentially Clinically Significant Vital Sign Abnormalities. Percentages based on number of patients with at least one non-missing post-baseline value in each treatment group. Patients are only counted once per criterion per laboratory test.
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Timepoint [4]
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Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
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Primary outcome [5]
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Number of Participants With Potentially Clinically Significant Hematology Results
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Assessment method [5]
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Number of Participants with Potentially Clinically Significant Hematology Results. Percentages based on number of patients with at least one non-missing post-baseline value in each treatment group. Patients are only counted once per criterion per laboratory test.
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Timepoint [5]
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Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
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Primary outcome [6]
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Number of Participants With Potentially Clinically Significant Blood Chemistry Results
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Assessment method [6]
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Number of Participants with Potentially Clinically Significant Blood Chemistry Results. Percentages based on number of patients with at least one non-missing post-baseline value in each treatment group. Patients are only counted once per criterion per laboratory test.
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Timepoint [6]
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Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
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Primary outcome [7]
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Number of Participants With Potentially Clinically Significant ECG Results
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Assessment method [7]
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Number of Participants with Potentially Clinically Significant ECG Results. Percentages based on number of patients with at least one non-missing post-baseline value in each treatment group. Patients are only counted once per criterion per laboratory test.
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Timepoint [7]
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Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
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Secondary outcome [1]
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Slope of ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) From Baseline to End of Study
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Assessment method [1]
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The ALSFRS-R (ALS functional rating scale with respiratory component) is a validated scale which measures 4 functional domains, comprising respiratory function, bulbar function, gross motor skills, and fine motor skills. There are a total of 12 questions, each scored from 0 to 4 for a total possible score between 0 to 48, with higher scores representing better function. Slope is calculated using a linear mixed effects model with x-axis time in months and y-axis ALSFRS-R score. Units for slope are change per month in units on the ALSFRS-R scale.
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Timepoint [1]
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Up to maximum 226 days: approximately 32.2 weeks
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Secondary outcome [2]
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Slope of Sniff Nasal Inspiratory Pressure (SNIP) From Baseline to End of Study
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Assessment method [2]
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SNIP is a test of inspiratory force (sternocleidomastoid and diaphragm) measured via a nasal cannula and is used to assess respiratory muscle weakness and to monitor changes in respiratory muscle strength over time. During the SNIP maneuver, the patient is asked to perform a strong, sharp, maximal sniff, whereby nasal pressure is measured via nasal cannula. The maximum recorded value after several attempts, with rest in between attempts, was use in the analysis.
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Timepoint [2]
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Up to maximum 226 days: approximately 32.2 weeks
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Secondary outcome [3]
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Death up to 6 Months
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Assessment method [3]
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Kaplan-Meier estimate of percentage of subjects who died up to 6 months
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Timepoint [3]
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6 Months
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Secondary outcome [4]
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Percentage of Participants With Death or Death Equivalent up to 6 Months
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Assessment method [4]
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Kaplan-Meier estimate of percentage of subjects who died or had a death equivalent event (tracheostomy or permanent assisted ventilation [PAV], defined as use of noninvasive ventilation [NIV] for =22 hours per day for =10 days) up to 6 months
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Timepoint [4]
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6 months
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Eligibility
Key inclusion criteria
- Subject has the ability to understand the purpose and risks of the study and provide
signed and dated informed consent (or have the consent confirmed by a witness if
unable to write) and authorization to use protected health information (PHI) in
accordance with national and local subject privacy regulations.
- Subject was enrolled in either CL211 (NCT00931944) or Study 223AS302 (NCTO1281189).
- Subject has completed their last visit in Study CL211 (NCT00931944) or Study 223AS302
(NCTO1281189).
- Subjects of childbearing potential must practice effective contraception during the
study and be willing and able to continue contraception for 1 month (females) or 3
months (males) after their last dose of study treatment.
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Minimum age
18
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Maximum age
80
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
- Subject withdrew prematurely from Study CL211 (NCT00931944) or Study 223AS302
(NCTO1281189).
- Subject permanently discontinued study treatment in Study CL211 (NCT00931944) or Study
223AS302 (NCTO1281189) for any reason other than enrollment into this study.
- Subject from Study CL211 (NCT00931944) or Study 223AS302 (NCTO1281189) has a
significant change in medical history (including laboratory tests or a clinically
significant condition) that in the opinion of the Investigator would impair the
subject's medical fitness for participation and preclude treatment.
- Female subject who is pregnant or breastfeeding.
- Subject is currently enrolled in any investigational drug study other than Study CL211
(NCT00931944) or Study 223AS302 (NCTO1281189).
- Subject is taking pramipexole, other dopamine agonists, any other agent with
dopaminergic activity, or any other disallowed concomitant medication.
- Subject is unwilling or unable to comply with the requirements of the protocol
including the presence of any condition (physical, mental, or social) that is likely
to affect the subject's ability to comply with the protocol. At a minimum, subjects
who are not able to travel to the study site must be willing to agree to remote blood
draws for clinical laboratory evaluations and telephone visits to report Adverse
Events, concomitant medications, and Amyotrophic Lateral Sclerosis Functional Rating
Scale (revised) (ALSFRS-R) scores.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
N/A
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Terminated
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
1/06/2012
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
1/02/2013
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Sample size
Target
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Accrual to date
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Final
616
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Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC
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Recruitment hospital [1]
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Prince of Wales Hospital - Randwick
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Recruitment hospital [2]
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Westmead Hospital - Westmead
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Recruitment hospital [3]
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Royal Brisbane and Women's Hospital - Herston
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Recruitment hospital [4]
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Calvary Health Care Bethlehem - Melbourne
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Recruitment postcode(s) [1]
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- Randwick
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Recruitment postcode(s) [2]
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2145 - Westmead
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Recruitment postcode(s) [3]
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4029 - Herston
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Recruitment postcode(s) [4]
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3121 - Melbourne
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Recruitment outside Australia
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Arizona
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California
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Sheffield
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Knopp Biosciences
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Address
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Biogen
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Ethics approval
Ethics application status
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Summary
Brief summary
The purpose of the study is to collect long-term safety data from subjects with Amyotrophic
Lateral Sclerosis (ALS) exposed to dexpramipexole.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT01622088
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Michael Bozik, MD
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Address
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Knopp Biosciences
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT01622088
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