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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT01281189
Registration number
NCT01281189
Ethics application status
Date submitted
20/01/2011
Date registered
21/01/2011
Date last updated
7/06/2021
Titles & IDs
Public title
Phase 3 Study of Dexpramipexole in ALS
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Scientific title
A Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study of the Safety and Efficacy of Dexpramipexole in Subjects With Amyotrophic Lateral Sclerosis
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Secondary ID [1]
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EUDRA CT NO: 2010-022818-19
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Secondary ID [2]
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223AS302
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Universal Trial Number (UTN)
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Trial acronym
EMPOWER
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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
Treatment: Drugs - Placebo
Experimental: Dexpramipexole -
Placebo comparator: Placebo -
Treatment: Drugs: Dexpramipexole
Oral tablet 150mg twice daily for up to 18 months.
Treatment: Drugs: Placebo
Oral tablet twice daily for up to 18 months.
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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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Composite Assessment of Function and Survival (CAFS) at 12 Months
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Assessment method [1]
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The Composite Assessment of Function and Survival (CAFS) is a between-group comparison of a single ranked clinical outcome based on (1) the change from baseline in ALS Functional Rating Scale-Revised (ALSFRS-R) score and (2) time to death. Each subject is ranked according to time-to-death (earlier deaths ranked lower than later deaths). Subjects who survive are ranked more favorably than subjects who died. Among the survivors, subjects are ranked according to change in ALSFRS-R (greater worsening of ALSFRS-R is ranked lower than less worsening or an improvement in ALSFRS-R). The ranked scores range from 001 to 941 (the number of subjects in the Efficacy Population) with larger rank score numbers associated with a better outcome. The ranks were analyzed using an ANCOVA model, which includes treatment as a fixed effect and adjusts for baseline ALSFRS-R score, duration of symptoms, site of onset, and use of riluzole. The least square mean rank score is presented for each treatment group.
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Timepoint [1]
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12 months
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Primary outcome [2]
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Death up to 12 Months (CAFs Individual Component)
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Assessment method [2]
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The longest duration of follow-up for this time to the death analysis was 12 months. In the study, subjects were followed for 12-18 months.
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Timepoint [2]
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12 months
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Primary outcome [3]
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Change From Baseline in ALSFRS-R at 12 Months (CAFs Individual Component)
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Assessment method [3]
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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 of 48, with higher scores representing better function.
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Timepoint [3]
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12 months
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Secondary outcome [1]
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Death or Respiratory Insufficiency (DRI) up to Month 18
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Assessment method [1]
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Time to Death or Respiratory Insufficiency (DRI) is defined as receipt of a tracheostomy or the use of non-invasive ventilation (NIV) for =22 hours per day for at least 10 consecutive days. If NIV is used to meet the criteria for respiratory insufficiency, no measured slow vital capacity (SVC) at any subsequent assessment may be \>50%. Time to DRI is calculated from the date of the first dose to the first date of one of the following events: death, tracheostomy, or the 10th day of consecutive NIV with no measured SVC \>50% at any subsequent assessment.
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Timepoint [1]
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18 months
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Secondary outcome [2]
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Death up to 18 Months
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Assessment method [2]
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Estimated time to death up to 18 months. This includes deaths reported greater than 30 days following discontinuation from the study (the time period for reporting all-cause mortality), regardless of subject disposition, up to 18 months from first dose.
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Timepoint [2]
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18 months
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Secondary outcome [3]
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=50% Predicted Upright Slow Vital Capacity (SVC) or Died up to 18 Months
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Assessment method [3]
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The date of reaching =50% of predicted upright slow vital capacity (SVC) is defined as the date of the first visit at which a predicted upright SVC is =50% and continues to remain =50% at the subsequent visit except for the last available observation. The time to reach =50% of predicted upright SVC is defined as the duration between the date of reaching =50% of predicted upright SVC and the date of the first dose of study medication. If the subject is alive and does not reach =50% of predicted upright SVC, the time to reach =50% of predicted upright SVC will be censored and equal to the number of days from the first dose of study medication until the visit date when the subject's last available SVC assessment is performed. The earliest time (Reaching =50% Predicted Upright SVC or death) is used in analysis.
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Timepoint [3]
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18 months
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Eligibility
Key inclusion criteria
* Aged 18 to 80 years old, inclusive, on Day 1.
* Diagnosis of sporadic or familial ALS.
* Onset of first ALS symptoms within 24 months prior to Day 1.
* World Federation of Neurology El Escorial criteria are met for a possible, laboratory-supported probable, probable, or definite ALS diagnosis.
* Upright slow vital capacity (SVC) of 65% or more at screening.
* Patients taking or not taking Riluzole are eligible for this study: if a patient has never taken Riluzole, he or she is eligible; if a patient is currently taking Riluzole, he or she must have been on a stable dose for at least 60 days; if a patient has discontinued Riluzole, he or she must have stopped taking it for at least 30 days.
* Must be able to swallow tablets at the time of study entry.
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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
* Other medically significant illness.
* Clinically significant abnormal laboratory values.
* Pregnant women or women breastfeeding.
* Prior exposure to dexpramipexole.
* Currently taking pramipexole or other dopamine agonists.
Other protocol-defined inclusion/exclusion criteria may apply.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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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
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
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Intervention assignment
Parallel
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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
Completed
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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
1/03/2011
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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
1/11/2012
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Sample size
Target
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Accrual to date
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Final
942
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Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC
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Prince of Wales Hospital - Randwick
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Westmead Hospital - Westmead
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Royal Brisbane and Women's Hospital - Herston
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Calvary Health Care Bethlehem - Melbourne
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- Randwick
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2145 - Westmead
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4029 - Herston
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Recruitment postcode(s) [4]
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3121 - Melbourne
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Knopp Biosciences
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Ethics approval
Ethics application status
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Summary
Brief summary
The purpose of this study is to determine whether dexpramipexole (150 mg twice daily) is safe and effective in the treatment of Amyotrophic Lateral Sclerosis (ALS).
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Trial website
https://clinicaltrials.gov/study/NCT01281189
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Trial related presentations / publications
Cudkowicz ME, van den Berg LH, Shefner JM, Mitsumoto H, Mora JS, Ludolph A, Hardiman O, Bozik ME, Ingersoll EW, Archibald D, Meyers AL, Dong Y, Farwell WR, Kerr DA; EMPOWER investigators. Dexpramipexole versus placebo for patients with amyotrophic lateral sclerosis (EMPOWER): a randomised, double-blind, phase 3 trial. Lancet Neurol. 2013 Nov;12(11):1059-67. doi: 10.1016/S1474-4422(13)70221-7. Epub 2013 Sep 23. Erratum In: Lancet Neurol. 2013 Nov;12(11):1042. Carbonell, J G [corrected to Gamez, J].
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Public notes
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Contacts
Principal investigator
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Merit Cudkowicz, MD, MSc
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Professor of Neurology of the Harvard Medical School
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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 are available at
https://clinicaltrials.gov/study/NCT01281189
Download to PDF