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    Summary
    EudraCT Number:2010-022818-19
    Sponsor's Protocol Code Number:223AS302
    National Competent Authority:Belgium - FPS Health-DGM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2011-01-24
    Trial results View results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedBelgium - FPS Health-DGM
    A.2EudraCT number2010-022818-19
    A.3Full title of the trial
    A randomized, double-blind, placebo-controlled, multi-center study of the safety and efficacy of Dexpramipexole in subjects with amyotrophic lateral sclerosis.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Clinical Study of Dexpramipexole in Amyotrophic Lateral Sclerosis (ALS)
    A.3.2Name or abbreviated title of the trial where available
    EMPOWER
    A.4.1Sponsor's protocol code number223AS302
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01281189
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorBiogen Idec Limited
    B.1.3.4CountryUnited Kingdom
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportBiogen Idec Limited
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationBiogen Idec Ltd
    B.5.2Functional name of contact pointALS Clinical Trials Team
    B.5.3 Address:
    B.5.3.1Street Address70 Norden Road
    B.5.3.2Town/ cityMaidenhead
    B.5.3.3Post codeSL6 4AY
    B.5.3.4CountryUnited Kingdom
    B.5.6E-mailALSclinicaltrials@biogenidec.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/09/616
    D.3 Description of the IMP
    D.3.1Product nameDexpramipexole
    D.3.2Product code BIIB050 / KNS-760704
    D.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNDexpramipexole Dichydrochloride
    D.3.9.1CAS number 908244-04-2
    D.3.9.2Current sponsor codeBIIB050 / KNS-760704
    D.3.9.3Other descriptive nameDexpramipexole
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number150
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboFilm-coated tablet
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Amyotrophic lateral sclerosis (ALS)
    E.1.1.1Medical condition in easily understood language
    Lou Gehrig's disease (ALS)
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level PT
    E.1.2Classification code 10002026
    E.1.2Term Amyotrophic lateral sclerosis
    E.1.2System Organ Class 10029205 - Nervous system disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of the study is to evaluate the efficacy of oral administration of dexpramipexole 150 mg twice daily compared to placebo for 12 months in subjects with amyotrophic lateral sclerosis.
    E.2.2Secondary objectives of the trial
    The secondary objectives are to evaluate the safety and pharmacokinetic (PK) profiles of oral administration of 150 mg twice daily dexpramipexole.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Ability to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use protected health information (PHI) in accordance with national and local subject privacy regulations.
    2. Subject is between 18 and 80 years of age (inclusive) on Day 1.
    3. Subject is diagnosed with sporadic or familial ALS.
    4. Subject had onset of first ALS symptoms ≤24 months prior to Day 1.
    5. Subject meets the possible, laboratory-supported probable, probable, or definite criteria for diagnosing ALS according to the World Federation of Neurology El Escorial criteria (revised according to the Airlie House Conference 1998). Subjects meeting the definition of “possible ALS” must have upper motor neuron (UMN) and lower motor neuron (LMN) signs/symptoms in at least 1 region).
    6. Subject has an upright SVC ≥65% of predicted value for age, height, and gender at screening.
    7. Subject has not taken riluzole for at least 30 days prior to Day 1, or has been on a stable dose of riluzole for at least 60 days prior to Day 1. (Riluzole-naïve subjects are permitted in the study.)
    8. Subject is medically able to undergo the study procedures and to adhere to the visit schedule at the time of study entry.
    9. Subject is able to take oral treatment without crushing or breaking the tablets at the time of study entry as assessed by the site Investigator.
    10. 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.
    E.4Principal exclusion criteria
    1. Subject in whom causes of neuromuscular weakness other than ALS have not been excluded.
    2. Subject with significant cognitive impairment, clinical dementia, or psychiatric illness
    3. Subject with a diagnosis of other neurodegenerative diseases (e.g., Parkinson’s disease, Alzheimer’s disease, etc.)
    4. Subject with a clinically significant history of unstable or severe cardiac, oncologic, hepatic, or renal disease, or other medically significant illness
    5. Subject with a clinically significant pre-existing pulmonary disorder not attributed to ALS
    6. Subject with a history of severe drug allergy or severe allergic disease (anaphylactic shock)
    7. Subject with clinically significant ECG abnormalities at the Screening visit (e.g. QTc >500 msec (where QTc is the interval between the start of the QRS complex and the end of the T wave corrected for heart rate), or acute ST segment elevations)
    8. Subject with clinically significant abnormal clinical laboratory values, as determined by the Investigator at the Screening visit
    9. Subject with absolute neutrophil count (ANC) <1.96 x 1000/μL at the Screening visit or any documented history of neutropenia
    10. Subject with aspartate aminotransferase (AST) or alanine aminotransferase (ALT) value >3.0 times the upper limit of normal at the Screening visit
    11. Subject with creatinine clearance (CrCl, calculated according to the Modification of Diet in Renal Disease equation) ≤50 mL/minute at the Screening visit
    12. Pregnant women or women breastfeeding
    13. Subject with a history of alcohol or drug abuse within 1 year of Day 1, as determined by Investigator
    14. Subject unlikely to comply with study requirements
    15. Subject who has been exposed to any other experimental agent (off-label use or investigational) within 30 days of Day 1.
    16. Subject with prior exposure to dexpramipexole
    17. Subject taking pramipexole or other dopamine agonists
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint is a joint rank of functional outcomes adjusted for mortality. The joint rank analysis is based on change from baseline in ALSFRS-R score and time to death using follow-up data through 12 months.
    E.5.1.1Timepoint(s) of evaluation of this end point
    ALSFRS-R is measured at screening, baseline and then monthly (+/- 7 days)
    E.5.2Secondary end point(s)
    • Time to death or respiratory insufficiency (DRI)
    Respiratory insufficiency is defined as receipt of a tracheostomy or the use of non-invasive ventilation (NIV) for ≥22 hours per day for ≥10 consecutive days. If NIV is used to meet the criteria for respiratory insufficiency, no measured slow vital capacity (SVC) at any subsequent assessment may be >50%.
    • Time to death
    • Respiratory decline: time to reach ≤50% of predicted upright slow vital capacity (SVC) or death
    • Change in muscle strength measurements (MSM), as determined by the overall megascore for hand-held dynamometry (HHD), through 12 months
    • 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), through 12 months
    • Final parameters will depend on the population PK model.
    • Relationship between population PK parameters and potential
    determinant covariates (e.g., age, gender, race, co-medication, renal function)
    • Relationship between changes from baseline in efficacy endpoints
    (e.g., ALSFRS-R) and population PK parameters
    • Incidence of AEs (including SAEs), vital signs, clinical laboratory
    assessments, physical examination, electrocardiogram (ECG) tests, and body weight.
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic Yes
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA28
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Australia
    Canada
    United States
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    The end of the study is last subject's last visit.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months8
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months10
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 915
    F.1.3Elderly (>=65 years) Yes
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state30
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 407
    F.4.2.2In the whole clinical trial 915
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    Subjects who continue study treatment until study completion (between 12 and 18 months depending on their date of enrollment) will be offered the option to enter an open-label extension study at that time.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.1Name of Organisation DeNDRoN Co-ordinating Centre
    G.4.3.4Network Country United Kingdom
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2011-03-04
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2011-06-07
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2012-11-16
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