Phase 3 Study of Dexpramipexole in ALS - Full Text View

Sponsor:	Biogen Idec
Information provided by:	Biogen Idec
ClinicalTrials.gov Identifier:	NCT01281189

Purpose

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	Intervention	Phase
Amyotrophic Lateral Sclerosis	Drug: Dexpramipexole Drug: Placebo	Phase 3

Study Type:	Interventional
Study Design:	Allocation: Randomized Endpoint Classification: Efficacy Study Intervention Model: Parallel Assignment Masking: Double Blind (Subject, Caregiver, 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

Resource links provided by NLM:

MedlinePlus related topics: Amyotrophic Lateral Sclerosis

U.S. FDA Resources

Further study details as provided by Biogen Idec:

Primary Outcome Measures:

A joint rank of functional outcomes adjusted for mortality. [ Time Frame: 12 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

Time to death using all available follow-up data. [ Time Frame: 18 months ] [ Designated as safety issue: No ]
Respiratory decline: time to reach 50% of predicted upright SVC or death. [ Time Frame: 18 months ] [ Designated as safety issue: No ]
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: 18 months ] [ Designated as safety issue: No ]
Change in muscle strength measurements (MSM), as determined by the overall megascore for hand-held dynamometry (HHD) [ Time Frame: 12 months ] [ Designated as safety issue: No ]
Incidence of adverse events, serious adverse events, vital signs, clinical laboratory assessments, physical examination, electrocardiogram tests, and body weight. [ Time Frame: 18 months ] [ Designated as safety issue: Yes ]
Population pharmacokinetics. [ Time Frame: 6 months ] [ Designated as safety issue: No ]

Estimated Enrollment:	804
Study Start Date:	March 2011
Estimated Study Completion Date:	February 2013
Estimated Primary Completion Date:	January 2013 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
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.

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.

Eligibility

Ages Eligible for Study:	18 Years to 80 Years
Genders Eligible for Study:	Both
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

Please refer to this study by its ClinicalTrials.gov identifier: NCT01281189

Show 82 Study Locations

Sponsors and Collaborators

Biogen Idec

More Information

No publications provided

Responsible Party:	Medical Director EMPOWER Study, Biogen Idec
ClinicalTrials.gov Identifier:	NCT01281189 History of Changes
Other Study ID Numbers:	223AS302, EUDRA CT NO: 2010-022818-19
Study First Received:	January 20, 2011
Last Updated:	December 22, 2011
Health Authority:	United States: Food and Drug Administration

Keywords provided by Biogen Idec:

Amyotrophic Lateral Sclerosis
ALS
Motor Neurone Disease

Additional relevant MeSH terms:

Amyotrophic Lateral Sclerosis
Sclerosis
Motor Neuron Disease
Spinal Cord Diseases
Central Nervous System Diseases
Nervous System Diseases

Neurodegenerative Diseases
TDP-43 Proteinopathies
Neuromuscular Diseases
Proteostasis Deficiencies
Metabolic Diseases
Pathologic Processes

ClinicalTrials.gov processed this record on May 23, 2012