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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/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
Scientific title
A Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study of the Safety and Efficacy of Dexpramipexole in Subjects With Amyotrophic Lateral Sclerosis
Secondary ID [1] 0 0
EUDRA CT NO: 2010-022818-19
Secondary ID [2] 0 0
223AS302
Universal Trial Number (UTN)
Trial acronym
EMPOWER
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Amyotrophic Lateral Sclerosis 0 0
Condition category
Condition code
Neurological 0 0 0 0
Neurodegenerative diseases
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
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.

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Composite Assessment of Function and Survival (CAFS) at 12 Months - 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.
Timepoint [1] 0 0
12 months
Primary outcome [2] 0 0
Death up to 12 Months (CAFs Individual Component) - 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.
Timepoint [2] 0 0
12 months
Primary outcome [3] 0 0
Change From Baseline in ALSFRS-R at 12 Months (CAFs Individual Component) - 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.
Timepoint [3] 0 0
12 months
Secondary outcome [1] 0 0
Death or Respiratory Insufficiency (DRI) up to Month 18 - 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.
Timepoint [1] 0 0
18 months
Secondary outcome [2] 0 0
Death up to 18 Months - 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.
Timepoint [2] 0 0
18 months
Secondary outcome [3] 0 0
=50% Predicted Upright Slow Vital Capacity (SVC) or Died up to 18 Months - 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.
Timepoint [3] 0 0
18 months

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.
Minimum age
18 Years
Maximum age
80 Years
Gender
Both males and females
Can healthy volunteers participate?
No
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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled 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
Blinded (masking used)
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
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC
Recruitment hospital [1] 0 0
Prince of Wales Hospital - Randwick
Recruitment hospital [2] 0 0
Westmead Hospital - Westmead
Recruitment hospital [3] 0 0
Royal Brisbane and Women's Hospital - Herston
Recruitment hospital [4] 0 0
Calvary Health Care Bethlehem - Melbourne
Recruitment postcode(s) [1] 0 0
- Randwick
Recruitment postcode(s) [2] 0 0
2145 - Westmead
Recruitment postcode(s) [3] 0 0
4029 - Herston
Recruitment postcode(s) [4] 0 0
3121 - Melbourne
Recruitment outside Australia
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Knopp Biosciences
Address
Country

Ethics approval
Ethics application status

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).
Trial website
https://clinicaltrials.gov/show/NCT01281189
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Merit Cudkowicz, MD, MSc
Address 0 0
Professor of Neurology of the Harvard Medical School
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

Summary results
Other publications