amyotrophic lateral sclerosis (ALS) MedDRA version: 14.1 Level: PT Classification code 10002026 Term: Amyotrophic lateral sclerosis System Organ Class: 10029205 - Nervous system disorders
Conditions
Interventions
Product Name: Dexpramipexole
Product Code: BIIB050 / KNS-760704
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: Dexpramipexole Dichydrochloride
CAS Number: 908244-04-2
Current Sponsor cod
Sponsors
Biogen Idec Limited
Eligibility
Sex/Gender
All
Inclusion criteria
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. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 915 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 0
Exclusion criteria
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) 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: 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.;Secondary Objective: The secondary objectives are to evaluate the safety and pharmacokinetic (PK) profiles of oral administration of 150 mg twice daily dexpramipexole.;Primary 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.;Timepoint(s) of evaluation of this end point: ALSFRS-R is measured at screening, baseline and then monthly (+/- 7 days) | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary 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 SVC or death - Change in muscle strength measurements (MSM), as determined by the overall megascore for HHD, through 12 months - - Change in ALS-related health quality, as measured by change in the total score on the ALSAQ-5, through 12 months;Timepoint(s) of evaluation of this end point: Secondary end points are variable. | — |
Countries
Australia, Canada, Germany, Ireland, Netherlands, Spain, Sweden, United Kingdom, United States
Contacts
Public ContactALS Clinical Trials Team
Biogen Idec Ltd
Outcome results
None listed