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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. Identifier: NCT01622088
Recruitment Status : Terminated (The Initial Phase 3 Study (NCTO1281189) did not meet its primary efficacy endpoint.)
First Posted : June 18, 2012
Last Update Posted : December 15, 2017
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.

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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 Drug: Dexpramipexole
Oral tablet 150 mg given twice daily up to 36 months

Primary Outcome Measures :
  1. Incidence of Adverse Events and Serious Adverse Events [ Time Frame: 36 months ]
  2. Discontinuation of study treatment due to an Adverse Events [ Time Frame: 36 months ]
  3. Changes in vital signs, clinical laboratory assessments (hematology, blood chemistry, and urinalysis), 12-lead electrocardiogram (ECGs), and body weight [ Time Frame: 36 months ]
  4. Incidence of laboratory abnormalities [ Time Frame: 36 months ]

Secondary Outcome Measures :
  1. Change in Amyotrophic Lateral Sclerosis Functional Rating Scale (revised) (ALSFRS-R) score [ Time Frame: 36 months ]
  2. Decline in sniff nasal inspiratory pressure (SNIP) [ Time Frame: 36 months ]
  3. Time to death [ Time Frame: 36 months ]
  4. Time to death or death equivalent (tracheostomy or permanent assisted ventilation (PAV), defined as use of noninvasive ventilation (NIV) for ≥ 22 hours per day for ≥ 10 days) [ Time Frame: 36 months ]

Information from the National Library of Medicine

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

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 identifier (NCT number): NCT01622088

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Sponsors and Collaborators
Knopp Biosciences
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Responsible Party: Knopp Biosciences Identifier: NCT01622088    
Other Study ID Numbers: 223AS304
EUDRA CT #: 2011-006119-70
First Posted: June 18, 2012    Key Record Dates
Last Update Posted: December 15, 2017
Last Verified: December 2017
Keywords provided by Knopp Biosciences:
ALS, Motor Neuron Disease, Amyotrophic Lateral Sclerosis
Additional relevant MeSH terms:
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Amyotrophic Lateral Sclerosis
Motor Neuron Disease
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases
Molecular Mechanisms of Pharmacological Action
Protective Agents
Physiological Effects of Drugs
Antiparkinson Agents
Anti-Dyskinesia Agents
Dopamine Agonists
Dopamine Agents
Neurotransmitter Agents