- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03491462
Arimoclomol in Amyotropic Lateral Sclerosis
A Phase 3, Randomised, Placebo-Controlled Trial of Arimoclomol in Amyotropic Lateral Sclerosis
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
Study Type
Enrollment (Actual)
Phase
- Phase 3
Contacts and Locations
Study Locations
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Leuven, Belgium, 3000
- Catholic University Leuven
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Ontario
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London, Ontario, Canada, N6A 5A5
- London Health Sciences Centre
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Toronto, Ontario, Canada, M4N 3M5
- Sunnybrook Health Sciences Centre
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Quebec
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Montréal, Quebec, Canada, H3A 2B4
- Montreal Neurological Institute and Hospital
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Montpellier, France, 34295
- Centre Hospitalier Regional Universitaire (CHRU) Montpellier - Hopital Gui De Chauliac
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Paris, France, 75013
- Groupe Hospitalier Pitie-Salpetriere - Centre d'Investigation Clinique Neurosciences 1422
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Berlin, Germany, 13353
- Charite - Universitaetsmedizin Berlin - Campus Virchow-Klinikum (CVK) - Ambulanz fuer ALS und andere Motoneuronenerkrankungen
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Hannover, Germany, 30625
- Medizinische Hochschule Hannover (MHH) - Klinik fuer Neurologie
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Ulm, Germany, 89081
- Universitaetsklinikum Ulm - Klinik fuer Neurologie
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Milano, Italy, 20138
- Instituti Clinica Scientifici Maugeri - IRCCS
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Torino, Italy, 10126
- Azienda Ospedaliero Universitaria (AUO) di Torino - Citta'della Salute e della Scienza di Torino
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Utrecht, Netherlands, 3584CX
- University Medical Center Utrecht
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Warsaw, Poland, 01-684
- Centrum Medyczne NeuroProtect
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Warsaw, Poland, 02-473
- Citi Clinic
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Barcelona, Spain, 08035
- Hospital Universitario Vall d'Hebron ALS Unit. Consultas Externas; Office: 9-10-11
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Madrid, Spain, 28046
- Hospital Carlos III - Hospital Universitario La Paz, ALS Unit
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Umeå, Sweden, 90737
- Umeå University Hospital
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Saint Gallen, Switzerland, 9007
- Kantonsspital St.Gallen, Muskelzentrum/ALS Clinic
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London, United Kingdom, WC1N 3BG
- Leonard Wolfson Experimental Neurology Centre
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Arizona
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Phoenix, Arizona, United States, 85013
- St. Joseph's Hospital and Medical Center (SJHMC) - Barrow Neurological Institute (BNI) - The Gregory W. Fulton ALS and Neuromuscular Disease Center
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Phoenix, Arizona, United States, 85018
- HonorHealth Neurology
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California
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Orange, California, United States, 92868
- UC Irvine Health ALS and Neuromuscular Center
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Florida
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Miami, Florida, United States, 33136
- University of Miami
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Kansas
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Kansas City, Kansas, United States, 66160
- University of Kansas Medical Center (KUMC) - Landon Center on Aging
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New York
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New York, New York, United States, 10021
- Hospital for Special Surgery
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Oregon
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Portland, Oregon, United States, 97213
- Providence Brain & Spine Institute
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Pennsylvania
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Philadelphia, Pennsylvania, United States, 19107
- University of Pensylvania, Perelman Center for Advanced Medicine - Penn Neuroscience Center
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Texas
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Dallas, Texas, United States, 75390
- University of Texas Southwestern Medical Center
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Virginia
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Charlottesville, Virginia, United States, 22908
- University of Virginia Health System
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Description
Inclusion Criteria:
- Subject meets revised El Escorial criteria for clinically possible, clinically probable / clinically probable ALS laboratory-supported, clinically definite ALS or clinically definite familial ALS laboratory-supported
- 18 months or less since first appearance of weakness (e.g. limb weakness, dysarthria, dysphagia, shortness of breath)
- ALS Functional Rating Scale-Revised (ALSFRS-R) equal to or above 35 and erect (seated) Slow Vital Capacity (SVC) % predicted equal to or above 70% at Screening
Exclusion Criteria:
- Tracheostomy or use of non-invasive ventilation for more than 2 hours during waking hours at the time of Screening and Baseline
- Pregnant or breast-feeding
- Current or anticipated use of diaphragmatic pacing
- Any other relevant medically significant condition which could present risk to the subject or interfere with the assessment of safety or has an increased risk of causing death during the trial
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Quadruple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Experimental: Arimoclomol
248 mg arimoclomol base (equivalent to 400 mg arimoclomol citrate) 3 times daily
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2 capsules (2 x 124 mg arimoclomol base; equivalent to 2 x 200 mg arimoclomol citrate) taken 3 times daily
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Placebo Comparator: Placebo
248 mg matching placebo 3 times daily
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2 matched placebo capsules taken 3 times daily
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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Combined Assessment of Function and Survival (CAFS)
Time Frame: Over 76 Weeks
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Combined Assessment of Function and Survival (CAFS) is a composite endpoint that includes 1) the change from baseline in revised ALS functional rating scale (ALSFRS-R) and 2) the survival endpoint (time to permanent assisted ventilation [PAV], tracheostomy or death). On the ALSFRS-R, 12 functions are rated on 5-point ordinal rating scales (from 0 to 4) with a total score range (minimum and maximum score) of 0-48 (sum of all 12 items). The higher the score the better functioning. For the survival endpoint, the longer time to PAV, tracheostomy, or death the better outcome. A patient's CAFS score represents a patient's rank in the study based on comparing the patient's outcome for both the change in ALSFRS-R and the time to event (PAV, tracheostomy, or death) to the outcome for all other patients in the study in a pairwise fashion. A higher rank score (range 0-1) is considered a better outcome. The reported values are the mean rank scores in each group for the composite endpoint. |
Over 76 Weeks
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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Time to Permanent Assisted Ventilation (PAV) / Tracheostomy / Death
Time Frame: Over 76 weeks
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Time from baseline to one of the events (PAV / tracheostomy / death).
PAV is defined as the first of 7 consecutive days on which PAV was used for >22 hours/day
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Over 76 weeks
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Change From Baseline to Week 76 (or End-of-trial) in the Revised ALS Functional Rating Scale (ALSFRS-R)
Time Frame: Week 76 (or end of trial)
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The ALSFRS-R score is based on a rating scale where 12 functions are rated on 5-point ordinal rating scales (from 0 to 4) with a maximum score of 48 (sum of all 12 items).
The higher the score the better functioning.
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Week 76 (or end of trial)
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Change From Baseline to Week 76 (or End-of-trial) in Percent (%) Predicted Slow Vital Capacity (SVC)
Time Frame: Week 76 (or end of trial)
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Slow Vital Capacity (SVC) is a measure of breathing function.
SVC measures the volume that can be exhaled from a full inhalation after exhaling to a maximum as slowly as possible.
The percent (%) of predicted SVC is reported.
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Week 76 (or end of trial)
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Collaborators and Investigators
Sponsor
Investigators
- Principal Investigator: Michael Benatar, MD PhD, University of Miami
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- ORARIALS-01
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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