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 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/ct2/show/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
Query!
Scientific title
A Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study of the Safety and Efficacy of Dexpramipexole in Subjects With Amyotrophic Lateral Sclerosis
Query!
Secondary ID [1]
0
0
EUDRA CT NO: 2010-022818-19
Query!
Secondary ID [2]
0
0
223AS302
Query!
Universal Trial Number (UTN)
Query!
Trial acronym
EMPOWER
Query!
Linked study record
Query!
Health condition
Health condition(s) or problem(s) studied:
Amyotrophic Lateral Sclerosis
0
0
Query!
Condition category
Condition code
Neurological
0
0
0
0
Query!
Neurodegenerative diseases
Query!
Human Genetics and Inherited Disorders
0
0
0
0
Query!
Other human genetics and inherited disorders
Query!
Intervention/exposure
Study type
Interventional
Query!
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.
Query!
Intervention code [1]
0
0
Treatment: Drugs
Query!
Comparator / control treatment
Query!
Control group
Query!
Outcomes
Primary outcome [1]
0
0
Composite Assessment of Function and Survival (CAFS) at 12 Months
Query!
Assessment method [1]
0
0
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.
Query!
Timepoint [1]
0
0
12 months
Query!
Primary outcome [2]
0
0
Death up to 12 Months (CAFs Individual Component)
Query!
Assessment method [2]
0
0
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.
Query!
Timepoint [2]
0
0
12 months
Query!
Primary outcome [3]
0
0
Change From Baseline in ALSFRS-R at 12 Months (CAFs Individual Component)
Query!
Assessment method [3]
0
0
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.
Query!
Timepoint [3]
0
0
12 months
Query!
Secondary outcome [1]
0
0
Death or Respiratory Insufficiency (DRI) up to Month 18
Query!
Assessment method [1]
0
0
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.
Query!
Timepoint [1]
0
0
18 months
Query!
Secondary outcome [2]
0
0
Death up to 18 Months
Query!
Assessment method [2]
0
0
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.
Query!
Timepoint [2]
0
0
18 months
Query!
Secondary outcome [3]
0
0
=50% Predicted Upright Slow Vital Capacity (SVC) or Died up to 18 Months
Query!
Assessment method [3]
0
0
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.
Query!
Timepoint [3]
0
0
18 months
Query!
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.
Query!
Minimum age
18
Years
Query!
Query!
Maximum age
80
Years
Query!
Query!
Sex
Both males and females
Query!
Can healthy volunteers participate?
No
Query!
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.
Query!
Study design
Purpose of the study
Treatment
Query!
Allocation to intervention
Randomised controlled 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
Blinded (masking used)
Query!
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
Query!
Query!
Query!
Query!
Intervention assignment
Parallel
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
1/03/2011
Query!
Date of last participant enrolment
Anticipated
Query!
Actual
Query!
Date of last data collection
Anticipated
Query!
Actual
1/11/2012
Query!
Sample size
Target
Query!
Accrual to date
Query!
Final
942
Query!
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC
Query!
Recruitment hospital [1]
0
0
Prince of Wales Hospital - Randwick
Query!
Recruitment hospital [2]
0
0
Westmead Hospital - Westmead
Query!
Recruitment hospital [3]
0
0
Royal Brisbane and Women's Hospital - Herston
Query!
Recruitment hospital [4]
0
0
Calvary Health Care Bethlehem - Melbourne
Query!
Recruitment postcode(s) [1]
0
0
- Randwick
Query!
Recruitment postcode(s) [2]
0
0
2145 - Westmead
Query!
Recruitment postcode(s) [3]
0
0
4029 - Herston
Query!
Recruitment postcode(s) [4]
0
0
3121 - Melbourne
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
Arkansas
Query!
Country [3]
0
0
United States of America
Query!
State/province [3]
0
0
California
Query!
Country [4]
0
0
United States of America
Query!
State/province [4]
0
0
Connecticut
Query!
Country [5]
0
0
United States of America
Query!
State/province [5]
0
0
Florida
Query!
Country [6]
0
0
United States of America
Query!
State/province [6]
0
0
Georgia
Query!
Country [7]
0
0
United States of America
Query!
State/province [7]
0
0
Illinois
Query!
Country [8]
0
0
United States of America
Query!
State/province [8]
0
0
Indiana
Query!
Country [9]
0
0
United States of America
Query!
State/province [9]
0
0
Iowa
Query!
Country [10]
0
0
United States of America
Query!
State/province [10]
0
0
Kansas
Query!
Country [11]
0
0
United States of America
Query!
State/province [11]
0
0
Maryland
Query!
Country [12]
0
0
United States of America
Query!
State/province [12]
0
0
Massachusetts
Query!
Country [13]
0
0
United States of America
Query!
State/province [13]
0
0
Michigan
Query!
Country [14]
0
0
United States of America
Query!
State/province [14]
0
0
Minnesota
Query!
Country [15]
0
0
United States of America
Query!
State/province [15]
0
0
Missouri
Query!
Country [16]
0
0
United States of America
Query!
State/province [16]
0
0
Nebraska
Query!
Country [17]
0
0
United States of America
Query!
State/province [17]
0
0
Nevada
Query!
Country [18]
0
0
United States of America
Query!
State/province [18]
0
0
New Hampshire
Query!
Country [19]
0
0
United States of America
Query!
State/province [19]
0
0
New York
Query!
Country [20]
0
0
United States of America
Query!
State/province [20]
0
0
North Carolina
Query!
Country [21]
0
0
United States of America
Query!
State/province [21]
0
0
Ohio
Query!
Country [22]
0
0
United States of America
Query!
State/province [22]
0
0
Oregon
Query!
Country [23]
0
0
United States of America
Query!
State/province [23]
0
0
Pennsylvania
Query!
Country [24]
0
0
United States of America
Query!
State/province [24]
0
0
Tennessee
Query!
Country [25]
0
0
United States of America
Query!
State/province [25]
0
0
Texas
Query!
Country [26]
0
0
United States of America
Query!
State/province [26]
0
0
Utah
Query!
Country [27]
0
0
United States of America
Query!
State/province [27]
0
0
Virginia
Query!
Country [28]
0
0
United States of America
Query!
State/province [28]
0
0
Washington
Query!
Country [29]
0
0
Belgium
Query!
State/province [29]
0
0
Gent
Query!
Country [30]
0
0
Belgium
Query!
State/province [30]
0
0
Leuven
Query!
Country [31]
0
0
Canada
Query!
State/province [31]
0
0
Alberta
Query!
Country [32]
0
0
Canada
Query!
State/province [32]
0
0
Quebec
Query!
Country [33]
0
0
Canada
Query!
State/province [33]
0
0
London
Query!
Country [34]
0
0
Canada
Query!
State/province [34]
0
0
Toronto
Query!
Country [35]
0
0
Canada
Query!
State/province [35]
0
0
Vancouver
Query!
Country [36]
0
0
France
Query!
State/province [36]
0
0
Lille
Query!
Country [37]
0
0
France
Query!
State/province [37]
0
0
Limoges
Query!
Country [38]
0
0
France
Query!
State/province [38]
0
0
Marseille
Query!
Country [39]
0
0
France
Query!
State/province [39]
0
0
Montpellier
Query!
Country [40]
0
0
France
Query!
State/province [40]
0
0
Nice
Query!
Country [41]
0
0
France
Query!
State/province [41]
0
0
Paris
Query!
Country [42]
0
0
Germany
Query!
State/province [42]
0
0
Berlin
Query!
Country [43]
0
0
Germany
Query!
State/province [43]
0
0
Bochum
Query!
Country [44]
0
0
Germany
Query!
State/province [44]
0
0
Hannover
Query!
Country [45]
0
0
Germany
Query!
State/province [45]
0
0
Jena
Query!
Country [46]
0
0
Germany
Query!
State/province [46]
0
0
Ulm
Query!
Country [47]
0
0
Ireland
Query!
State/province [47]
0
0
Dublin
Query!
Country [48]
0
0
Netherlands
Query!
State/province [48]
0
0
Amsterdam
Query!
Country [49]
0
0
Netherlands
Query!
State/province [49]
0
0
Nijmegen
Query!
Country [50]
0
0
Netherlands
Query!
State/province [50]
0
0
Utrecht
Query!
Country [51]
0
0
Spain
Query!
State/province [51]
0
0
Barcelona
Query!
Country [52]
0
0
Spain
Query!
State/province [52]
0
0
Madrid
Query!
Country [53]
0
0
Sweden
Query!
State/province [53]
0
0
Göteborg
Query!
Country [54]
0
0
Sweden
Query!
State/province [54]
0
0
Stockholm
Query!
Country [55]
0
0
United Kingdom
Query!
State/province [55]
0
0
Birmingham
Query!
Country [56]
0
0
United Kingdom
Query!
State/province [56]
0
0
Liverpool
Query!
Country [57]
0
0
United Kingdom
Query!
State/province [57]
0
0
London
Query!
Country [58]
0
0
United Kingdom
Query!
State/province [58]
0
0
Newcastle
Query!
Country [59]
0
0
United Kingdom
Query!
State/province [59]
0
0
Oxford
Query!
Country [60]
0
0
United Kingdom
Query!
State/province [60]
0
0
Sheffield
Query!
Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Query!
Name
Knopp Biosciences
Query!
Address
Query!
Country
Query!
Ethics approval
Ethics application status
Query!
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).
Query!
Trial website
https://clinicaltrials.gov/ct2/show/NCT01281189
Query!
Trial related presentations / publications
Query!
Public notes
Query!
Contacts
Principal investigator
Name
0
0
Merit Cudkowicz, MD, MSc
Query!
Address
0
0
Professor of Neurology of the Harvard Medical School
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
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT01281189
Download to PDF