Try the modernized ClinicalTrials.gov beta website. Learn more about the modernization effort.
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

Phase 3 Extension Study of Dexpramipexole in ALS (ENVISION)

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 evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01622088
Recruitment Status : Terminated (The Initial Phase 3 Study (NCTO1281189) did not meet its primary efficacy endpoint.)
First Posted : June 18, 2012
Results First Posted : April 7, 2022
Last Update Posted : May 3, 2022
Sponsor:
Collaborator:
Biogen
Information provided by (Responsible Party):
Knopp Biosciences

Brief Summary:
The purpose of the study is to collect long-term safety data from subjects with Amyotrophic Lateral Sclerosis (ALS) exposed to dexpramipexole.

Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis Drug: Dexpramipexole Phase 3

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.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 616 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Study Start Date : June 2012
Actual Primary Completion Date : February 2013
Actual Study Completion Date : February 2013


Arm Intervention/treatment
Experimental: Dexpramipexole
Dexpramipexole open-label
Drug: Dexpramipexole
Oral tablet 150 mg given twice daily (BID)
Other Name: BIIB050




Primary Outcome Measures :
  1. 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

  2. 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

  3. 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

  4. 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.

  5. 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.

  6. 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.

  7. 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.


Secondary Outcome Measures :
  1. 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 ]
    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.

  2. Slope of Sniff Nasal Inspiratory Pressure (SNIP) From Baseline to End of Study [ Time Frame: Up to maximum 226 days: approximately 32.2 weeks ]
    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.

  3. Death up to 6 Months [ Time Frame: 6 Months ]
    Kaplan-Meier estimate of percentage of subjects who died up to 6 months

  4. Percentage of Participants With Death or Death Equivalent up to 6 Months [ Time Frame: 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



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   18 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01622088


Locations
Show Show 80 study locations
Sponsors and Collaborators
Knopp Biosciences
Biogen
Investigators
Layout table for investigator information
Study Chair: Michael Bozik, MD Knopp Biosciences
Layout table for additonal information
Responsible Party: Knopp Biosciences
ClinicalTrials.gov Identifier: NCT01622088    
Other Study ID Numbers: 223AS304
EUDRA CT #: 2011-006119-70
First Posted: June 18, 2012    Key Record Dates
Results First Posted: April 7, 2022
Last Update Posted: May 3, 2022
Last Verified: April 2022
Keywords provided by Knopp Biosciences:
ALS, Motor Neuron Disease, Amyotrophic Lateral Sclerosis
Additional relevant MeSH terms:
Layout table for MeSH terms
Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases
Pramipexole
Antioxidants
Molecular Mechanisms of Pharmacological Action
Protective Agents
Physiological Effects of Drugs
Antiparkinson Agents
Anti-Dyskinesia Agents
Dopamine Agonists
Dopamine Agents
Neurotransmitter Agents