Amyotrophic Lateral SclerosisPhase 3ENVISIONNCT01622088
Phase 3 Extension Study of Dexpramipexole in ALS
A Phase 3 study testing Dexpramipexole in people with Amyotrophic Lateral Sclerosis, run by Knopp Biosciences at 80 sites in 11 countries.
Trial summary
The purpose of the study is to collect long-term safety data from subjects with Amyotrophic Lateral Sclerosis (ALS) exposed to dexpramipexole. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- Dexpramipexole
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 616 participants
- Where
- 80 sitesin 11 countries
- Study dates
- Jun 2012to Feb 2013
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
80 sites in 11 countries.
80 sites in 11 countries
United States42 sites
Emory University
Atlanta, Georgia, 30322
Johns Hopkins University School of Medicine
Baltimore, Maryland, 21287
Massachusetts General Hospital
Charlestown, Massachusetts, 02129
Carolinas Medical Center
Charlotte, North Carolina, 28207
University of Virginia Health System
Charlottesville, Virginia, 22908
Northwestern University
Chicago, Illinois, 60611
The Cleveland Clinic Foundation
Cleveland, Ohio, 44195
Ohio State University
Columbus, Ohio, 43210
Texas Neurology
Dallas, Texas, 75214
Duke University Medical Center
Durham, North Carolina, 27705
University of California at San Francisco - Fresno
Fresno, California, 03721
St. Mary's Health Care
Grand Rapids, Michigan, 49503
Penn State Hershey Medical Center
Hershey, Pennsylvania, 17033
Methodist Neurological Institute
Houston, Texas, 77030
Indiana University
Indianapolis, Indiana, 46202
University of Iowa
Iowa City, Iowa, 52242
Mayo Clinic - Jacksonville
Jacksonville, Florida, 32224
University of Kansas Medical Center
Kansas City, Kansas, 66160
University of Nevada School of Medicine
Las Vegas, Nevada, 89102
Dartmouth-Hitchcock Medical Center
Lebanon, New Hampshire, 03756
Neurology Associates, P.C.
Lincoln, Nebraska, 68506
University of Miami Miller School of Medicine
Miami, Florida, 33136
Hennepin County Medical Center
Minneapolis, Minnesota, 55404
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
Hospital for Special Care
New Britain, Connecticut, 06053
Columbia University
New York, New York, 10032
University of California, Irvine
Orange, California, 92868
Drexel University College of Medicine
Philadelphia, Pennsylvania, 19102
ALS Center at Penn
Philadelphia, Pennsylvania, 19107
Barrow Neurological Institute - St. Joseph's Hospital
Phoenix, Arizona, 85013
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15213
Providence ALS Center
Portland, Oregon, 97213
Mayo Clinic - Rochester
Rochester, Minnesota, 55905
University of California, Davis
Sacramento, California, 95817
University of Utah
Salt Lake City, Utah, 84132
University of Texas Health Sciences Center
San Antonio, Texas, 78229
California Pacific Medical Center
San Francisco, California, 94115
University of Washington
Seattle, Washington, 98195
Washington University School of Medicine
St Louis, Missouri, 63110
Research Foundation of the State University of New York
Syracuse, New York, 12207
University of South Florida Medical Center
Tampa, Florida, 33612
Wake Forest University
Winston-Salem, North Carolina, 27157
Canada6 sites
Univ of Calgary / Foothills MC
Calgary, Alberta, T2V 1P9
London Health Sciences Centre
London
CHUM - Hopital Notre Dame
Montreal, Quebec, H2L 4M1
Mcgill University
Montreal, Quebec, H3A 2B4
Sunnybrook and Women's College and Health Sciences Centre
Toronto, M4N 3M5
University of British Columbia
Vancouver
United Kingdom6 sites
Queen Elizabeth Hospital
Birmingham, B15 2TH
Walton Centre for Neurology & Neurosurgery
Liverpool, L9 7LJ
Kings College Hospital NHS Foundation Trust
London, SE5 8AF
Newcastle University Hospital - Clinical Ageing Research Unit
Newcastle, NE4 5PL
John Radcliffe Hospital
Oxford
Sheffield Institute for Transnational Neuroscience
Sheffield, S10 2HQ
France5 sites
CHRU de Lille - Hôpital Roger Salengro
Lille, 59037
Centre Hospitalier La Timone
Marseille
CHU Gui de Chauliac
Montpellier, 34295
CHU de Nice - Hôpital de l'Archet 1
Nice
Hôpital La Pitié Salpétrière
Paris, 75013
Germany5 sites
Charité - Universitätsmedizin Berlin
Berlin
Bergmannsheil Gmbh
Bochum
Medizinische Hochschule Hannover (MHH)
Hanover
Universitätsklinikum Jena
Jena
University of Ulm, RKU
Ulm
Australia4 sites
Royal Brisbane and Women's Hospital
Herston, Queensland, 4029
Calvary Health Care Bethlehem
Melbourne, Victoria, 3121
Prince of Wales Hospital
Randwick, New South Wales
Westmead Hospital
Westmead, New South Wales, 2145
Spain4 sites
Hospital Universitario de Bellvitge
Barcelona, 8907
Hospital Vall d'Hebron
Barcelona
Hospital La Paz
Madrid, 28046
Hospital Carlos III
Madrid
Netherlands3 sites
Academisch Medisch Centrum
Amsterdam, 1105 AZ
UMC St. Radboud
Nijmegen, 6525 GA
Universitair Medisch Centrum Utrecht
Utrecht, 3584 CX
Sweden2 sites
Sahlgrenska Universitetssjukhuset
Gothenburg, 41345
Karolinska Universitetssjukhuset, Solna
Stockholm, 17176
About this study
Official title: An Open-Label, Multicenter, Extension Study to Evaluate the Long-Term Safety and Efficacy of Dexpramipexole (BIIB050) in Subjects With Amyotrophic Lateral Sclerosis
The purpose of the study is to collect long-term safety data from subjects with Amyotrophic Lateral Sclerosis (ALS) exposed to dexpramipexole.
Read the detailed description
Amyotrophic Lateral Sclerosis (ALS) is a rapidly progressive, degenerative disease of motor neurons in the brain and spinal cord that leads to muscle atrophy and spasticity in limb and bulbar muscles resulting in weakness and loss of ambulation, oropharyngeal dysfunction, weight loss, and ultimately respiratory failure. The purpose of this study is to collect long-term safety data from subjects with Amyotrophic Lateral Sclerosis (ALS) exposed to dexpramipexole.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- Dexpramipexole
- Keywords
- ALS, Motor Neuron Disease, Amyotrophic Lateral Sclerosis
- Medical subject headings
- Amyotrophic Lateral Sclerosis, Motor Neuron Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DexpramipexoleDrug
Also called BIIB050
Oral tablet 150 mg given twice daily (BID)
| Study group | Role | What they receive |
|---|---|---|
| Dexpramipexole | Experimental | Dexpramipexole open-label Receives: Drug: Dexpramipexole |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Subjects Who Reported an Adverse Event
The number of subjects who reported an adverse event during the study
Measured: Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
Number of Subjects Who Experienced a Serious Adverse Event
The number of subjects enrolled who reported a serious adverse event during the study
Measured: Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
Number of Subjects Who Discontinued the Study Treatment Due to an Adverse Event
The number of subjects enrolled who discontinued the study treatment due to an adverse event during the study
Measured: Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
Number of Participants With Potentially Clinically Significant Vital Sign Results
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.
Measured: Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
Number of Participants With Potentially Clinically Significant Hematology Results
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.
Measured: Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
Number of Participants With Potentially Clinically Significant Blood Chemistry Results
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.
Measured: Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
Number of Participants With Potentially Clinically Significant ECG Results
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.
Measured: Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
Secondary outcomes (4)
Slope of ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) From Baseline to End of Study
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.
Measured: Up to maximum 226 days: approximately 32.2 weeks
Slope of Sniff Nasal Inspiratory Pressure (SNIP) From Baseline to End of Study
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.
Measured: Up to maximum 226 days: approximately 32.2 weeks
Death up to 6 Months
Kaplan-Meier estimate of percentage of subjects who died up to 6 months
Measured: 6 Months
Percentage of Participants With Death or Death Equivalent up to 6 Months
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
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jun 2012 (estimated)
- Study startJun 2012
- Primary completionFeb 2013
- Study completionFeb 2013
Why it stopped: The Initial Phase 3 Study (NCTO1281189) did not meet its primary efficacy endpoint.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 616 participants (actual)
Sponsor and investigators
- Lead sponsor
- Knopp Biosciences
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Biogen
- Responsible party
- Sponsor
- Investigators
- Michael Bozik, MD, Study chair, Knopp Biosciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01622088
- Sponsor's protocol ID
- 223AS304
- Other IDs
- EUDRA CT #: 2011-006119-70
- Acronym
- ENVISION
- Status last verified
- Apr 2022
- First submitted
- 3 May 2012
- Last updated
- 3 May 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 3 May 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label, Multicenter, Extension Study to Evaluate the Long-Term Safety and Efficacy of Dexpramipexole (BIIB050) in Subjects With Amyotrophic Lateral Sclerosis
- ClinicalTrials.gov ID
- NCT01622088
- Acronym / sponsor ID
- ENVISION / 223AS304
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- ALS, Motor Neuron Disease, Amyotrophic Lateral Sclerosis
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The Initial Phase 3 Study (NCTO1281189) did not meet its primary efficacy endpoint.
- Enrolment
- 616 participants (actual)
- Lead sponsor
- Knopp BiosciencesIndustry (pharma, biotech, devices)
- Collaborators
- Biogen
- Study start
- Jun 2012
- Primary completion
- Feb 2013 (actual)
- Study completion
- Feb 2013 (actual)
- First posted
- 18 Jun 2012 (estimated)
- Results
- Posted 7 Apr 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of the study is to collect long-term safety data from subjects with Amyotrophic Lateral Sclerosis (ALS) exposed to dexpramipexole.
Detailed description
Amyotrophic Lateral Sclerosis (ALS) is a rapidly progressive, degenerative disease of motor neurons in the brain and spinal cord that leads to muscle atrophy and spasticity in limb and bulbar muscles resulting in weakness and loss of ambulation, oropharyngeal dysfunction, weight loss, and ultimately respiratory failure. The purpose of this study is to collect long-term safety data from subjects with Amyotrophic Lateral Sclerosis (ALS) exposed to dexpramipexole.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Dexpramipexole | Experimental | Drug: DexpramipexoleDexpramipexole open-label |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (7 listed)
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| DexpramipexoleAlso: BIIB050Oral tablet 150 mg given twice daily (BID) | Drug | Dexpramipexole |
Primary outcome
Number of Subjects Who Reported an Adverse Event
Time frame: Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
The number of subjects who reported an adverse event during the study
Number of Subjects Who Experienced a Serious Adverse Event
Time frame: Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
The number of subjects enrolled who reported a serious adverse event during the study
Number of Subjects Who Discontinued the Study Treatment Due to an Adverse Event
Time frame: Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
The number of subjects enrolled who discontinued the study treatment due to an adverse event during the study
Number of Participants With Potentially Clinically Significant Vital Sign Results
Time frame: Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
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.
Number of Participants With Potentially Clinically Significant Hematology Results
Time frame: Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
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.
Number of Participants With Potentially Clinically Significant Blood Chemistry Results
Time frame: Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
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.
Number of Participants With Potentially Clinically Significant ECG Results
Time frame: Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
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.
4 secondary outcomes
Slope of ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) From Baseline to End of Study
Time frame: Up to maximum 226 days: approximately 32.2 weeks
Slope of Sniff Nasal Inspiratory Pressure (SNIP) From Baseline to End of Study
Time frame: Up to maximum 226 days: approximately 32.2 weeks
Death up to 6 Months
Time frame: 6 Months
Percentage of Participants With Death or Death Equivalent up to 6 Months
Time frame: 6 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 May 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Michael Bozik, MD study chair
Knopp Biosciences
80 sites in 11 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Dexpramipexole in people with Amyotrophic Lateral Sclerosis, run by Knopp Biosciences at 80 sites in 11 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 3 May 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 80 sites in United States, Canada, United Kingdom, France, Germany and 6 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 7 Apr 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Subjects Who Reported an Adverse Event (time frame: Baseline through end of study (maximum 226 days: approximately 32.2 weeks)). The study lists 7 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 May 2022.