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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/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
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
Secondary ID [1] 0 0
EUDRA CT #: 2011-006119-70
Secondary ID [2] 0 0
223AS304
Universal Trial Number (UTN)
Trial acronym
ENVISION
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

Experimental: Dexpramipexole - Dexpramipexole open-label


Treatment: Drugs: Dexpramipexole
Oral tablet 150 mg given twice daily (BID)
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Subjects Who Reported an Adverse Event
Timepoint [1] 0 0
Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
Primary outcome [2] 0 0
Number of Subjects Who Experienced a Serious Adverse Event
Timepoint [2] 0 0
Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
Primary outcome [3] 0 0
Number of Subjects Who Discontinued the Study Treatment Due to an Adverse Event
Timepoint [3] 0 0
Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
Primary outcome [4] 0 0
Number of Participants With Potentially Clinically Significant Vital Sign Results
Timepoint [4] 0 0
Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
Primary outcome [5] 0 0
Number of Participants With Potentially Clinically Significant Hematology Results
Timepoint [5] 0 0
Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
Primary outcome [6] 0 0
Number of Participants With Potentially Clinically Significant Blood Chemistry Results
Timepoint [6] 0 0
Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
Primary outcome [7] 0 0
Number of Participants With Potentially Clinically Significant ECG Results
Timepoint [7] 0 0
Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
Secondary outcome [1] 0 0
Slope of ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) From Baseline to End of Study
Timepoint [1] 0 0
Up to maximum 226 days: approximately 32.2 weeks
Secondary outcome [2] 0 0
Slope of Sniff Nasal Inspiratory Pressure (SNIP) From Baseline to End of Study
Timepoint [2] 0 0
Up to maximum 226 days: approximately 32.2 weeks
Secondary outcome [3] 0 0
Death up to 6 Months
Timepoint [3] 0 0
6 Months
Secondary outcome [4] 0 0
Percentage of Participants With Death or Death Equivalent up to 6 Months
Timepoint [4] 0 0
6 months

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

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
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
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/06/2012
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/02/2013
Sample size
Target
Accrual to date
Final
616
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
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
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State/province [3] 0 0
Connecticut
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Florida
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Georgia
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Illinois
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Indiana
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Iowa
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Kansas
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Maryland
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Massachusetts
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Michigan
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Nebraska
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Utah
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Washington
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Belgium
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Gent
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Belgium
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Leuven
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London
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Canada
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Toronto
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Canada
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Vancouver
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France
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Lille
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France
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Marseille
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Montpellier
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Bochum
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Hannover
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Jena
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Ulm
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Dublin
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Amsterdam
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Nijmegen
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Utrecht
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Barcelona
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Stockholm
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Birmingham
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Liverpool
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London
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Newcastle
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Oxford
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United Kingdom
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Sheffield

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Knopp Biosciences
Address
Country
Other collaborator category [1] 0 0
Commercial sector/industry
Name [1] 0 0
Biogen
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

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.
Trial website
https://clinicaltrials.gov/study/NCT01622088
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Michael Bozik, MD
Address 0 0
Knopp Biosciences
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

For IPD and results data, please see https://clinicaltrials.gov/study/NCT01622088