Phase 3 Study of Dexpramipexole in ALS (EMPOWER)

• ClinicalTrials.gov Identifier: NCT01281189
• Recruitment Status: Completed
• First Posted: January 21, 2011
• Last Update Posted: December 15, 2017
• Sponsor: Knopp Biosciences
• Information provided by (Responsible Party): Knopp Biosciences

Study Description

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).

Condition or disease	Intervention/treatment	Phase
Amyotrophic Lateral Sclerosis	Drug: Dexpramipexole; Drug: Placebo	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 determine whether dexpramipexole (150 mg twice daily) is safe and effective in the treatment of ALS.

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 943 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• Official Title: A Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study of the Safety and Efficacy of Dexpramipexole in Subjects With Amyotrophic Lateral Sclerosis
• Study Start Date: March 2011
• Actual Primary Completion Date: November 2012
• Actual Study Completion Date: November 2012

Arms and Interventions

Arm	Intervention/treatment
Experimental: Dexpramipexole	Drug: Dexpramipexole; Oral tablet 150mg twice daily for up to 18 months.; Other Name: BIIB050
Placebo Comparator: Placebo	Drug: Placebo; Oral tablet twice daily for up to 18 months.

Outcome Measures

Primary Outcome Measures :

1. A joint rank of functional outcomes adjusted for mortality. [ Time Frame: 12 months ]

Secondary Outcome Measures :

1. Time to death or respiratory insufficiency [ Time Frame: 18 months ]
2. Time to death [ Time Frame: 18 months ]
3. Respiratory decline: time to reach less than or equal to 50% of predicted upright slow vital capacity (SVC) or death [ Time Frame: 18 months ]
4. Change in muscle strength measurements (MSM), as determined by the overall megascore for hand-held dynamometry (HHD) [ Time Frame: 12 months ]
5. Change in ALS-related health quality, as measured by change in the total score on the Amyotrophic Lateral Sclerosis Assessment Questionnaire-5-Item Form (ALSAQ-5) [ Time Frame: 12 months ]
6. Population pharmacokinetics. [ Time Frame: 18 months ]
7. Incidence of adverse events, serious adverse events. [ Time Frame: 18 Months ]

Eligibility Criteria

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

Criteria

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.

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.

Contacts and Locations

  Show 82 Study Locations

Sponsors and Collaborators

Knopp Biosciences

More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Cudkowicz ME, van den Berg LH, Shefner JM, Mitsumoto H, Mora JS, Ludolph A, Hardiman O, Bozik ME, Ingersoll EW, Archibald D, Meyers AL, Dong Y, Farwell WR, Kerr DA; EMPOWER investigators. Dexpramipexole versus placebo for patients with amyotrophic lateral sclerosis (EMPOWER): a randomised, double-blind, phase 3 trial. Lancet Neurol. 2013 Nov;12(11):1059-67. doi: 10.1016/S1474-4422(13)70221-7. Epub 2013 Sep 23. Erratum in: Lancet Neurol. 2013 Nov;12(11):1042. Carbonell, J G [corrected to Gamez, J].

• Responsible Party: Knopp Biosciences
• ClinicalTrials.gov Identifier: NCT01281189 History of Changes
• Other Study ID Numbers: 223AS302; EUDRA CT NO: 2010-022818-19
• First Posted: January 21, 2011
• Last Update Posted: December 15, 2017
• Last Verified: December 2017

Keywords provided by Knopp Biosciences:

Amyotrophic Lateral Sclerosis; ALS; Motor Neurone Disease

Additional relevant 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