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Trial record 2 of 11 for:    Arimoclomol

Arimoclomol in Amyotropic Lateral Sclerosis

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ClinicalTrials.gov Identifier: NCT03491462
Recruitment Status : Active, not recruiting
First Posted : April 9, 2018
Last Update Posted : June 21, 2019
Sponsor:
Information provided by (Responsible Party):
Orphazyme

Brief Summary:
A multicenter, randomized, double-blind, placebo-controlled, parallel group trial to evaluate the efficacy and safety of arimoclomol in amyotropic lateral sclerosis (ALS)

Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis Drug: Arimoclomol Drug: Placebo oral capsule Phase 3

Detailed Description:
Screening of up to 4 weeks Treatment of up to 76 weeks

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 231 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: placebo-controlled, parallel group
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description: Double-blind
Primary Purpose: Treatment
Official Title: A Phase 3, Randomised, Placebo-Controlled Trial of Arimoclomol in Amyotropic Lateral Sclerosis
Actual Study Start Date : July 31, 2018
Estimated Primary Completion Date : December 2020
Estimated Study Completion Date : January 2021


Arm Intervention/treatment
Experimental: Arimoclomol
Arimoclomol, capsule
Drug: Arimoclomol
Active treatment
Other Name: Arimoclomol citrate

Placebo Comparator: Placebo
Placebo oral capsule (matching to experimental Arm)
Drug: Placebo oral capsule
Matching placebo capsule
Other Name: Placebo




Primary Outcome Measures :
  1. Combined Assessment of Function and Survival (CAFS) [ Time Frame: over 76 Weeks ]
    A nonparametric rank analysis of covariance considering Time to Permanent assisted ventilation, tracheostomy or death or ALSFRS-R score (12 functions on a 5-point ordinal rating scale with maximum score of 48)


Secondary Outcome Measures :
  1. Time to permanent assisted ventilation (PAV)/tracheostomy/death [ Time Frame: over 76 weeks ]
    time from baseline to event where PAV is defined as the first of 7 consecutive days on which PAV was used for >22 hours/day.

  2. 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) ]
    Change in functional rating scale over time as recorded by the revised ALS functional rating scale (ALSFRS-R), a scale where 12 functions are rated on a 4-point scale giving a maximum score of 48

  3. Change from Baseline to Week 76 (or end-of-trial) in Slow Vital Capacity [ Time Frame: Week 76 (or end of trial) ]
    Change in Slow Vital Capacity (a measure of breathing function) over time



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Subject meets revised El Escorial criteria for clinically possible, clinically probable / Clinically probable ALS laboratory-supported or clinically definite ALS, or familial ALS
  • 18 months or less since first appearance of weakness (e.g. limb weakness, dysarthria, dysphagia, shortness of breath).
  • ALSFRS-R equal to or above 35 and erect (seated) SVC% predicted equal to or above 80% at screening

Exclusion Criteria:

  • Tracheostomy or use of non-invasive ventilation for more than 2 hours during waking hours at the time of screening or 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03491462


  Hide Study Locations
Locations
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United States, Arizona
St. Joseph's Hospital and Medical Center (SJHMC) - Barrow Neurological Institute (BNI) - The Gregory W. Fulton ALS and Neuromuscular Disease Center
Phoenix, Arizona, United States, 85013
HonorHealth Neurology
Phoenix, Arizona, United States, 85018
United States, California
UC Irvine Health ALS and Neuromuscular Center
Orange, California, United States, 92868
United States, Florida
University of Miami
Miami, Florida, United States, 33136
United States, Kansas
University of Kansas Medical Center (KUMC) - Landon Center on Aging
Kansas City, Kansas, United States, 66160
United States, New York
Hospital for Special Surgery
New York, New York, United States, 10021
United States, Oregon
Providence Brain & Spine Institute
Portland, Oregon, United States, 97213
United States, Pennsylvania
University of Pensylvania, Perelman Center for Advanced Medicine - Penn Neuroscience Center
Philadelphia, Pennsylvania, United States, 19107
United States, Texas
University of Texas Southwestern Medical Center
Dallas, Texas, United States, 75390
United States, Virginia
University of Virginia Health System
Charlottesville, Virginia, United States, 22908
Belgium
Catholic University Leuven
Leuven, Belgium, 3000
Canada, Ontario
London Health Sciences Centre
London, Ontario, Canada, N6A 5A5
Sunnybrook Health Sciences Centre
Toronto, Ontario, Canada, M4N 3M5
Canada, Quebec
Montreal Neurological Institute and Hospital
Montréal, Quebec, Canada, H3A 2B4
France
Centre Hospitalier Regional Universitaire (CHRU) Montpellier - Hopital Gui De Chauliac
Montpellier, France, 34295
Groupe Hospitalier Pitie-Salpetriere - Centre d'Investigation Clinique Neurosciences 1422
Paris, France, 75013
Germany
Charite - Universitaetsmedizin Berlin - Campus Virchow-Klinikum (CVK) - Ambulanz fuer ALS und andere Motoneuronenerkrankungen
Berlin, Germany, 13353
Medizinische Hochschule Hannover (MHH) - Klinik fuer Neurologie
Hannover, Germany, 30625
Universitaetsklinikum Ulm - Klinik fuer Neurologie
Ulm, Germany, 89081
Italy
Instituti Clinica Scientifici Maugeri - IRCCS
Milano, Italy, 20138
Azienda Ospedaliero Universitaria (AUO) di Torino - Citta'della Salute e della Scienza di Torino
Torino, Italy, 10126
Netherlands
University Medical Center Utrecht
Utrecht, Netherlands, 3584CX
Poland
Centrum Medyczne NeuroProtect
Warsaw, Poland, 01-684
Citi Clinic
Warsaw, Poland, 02-473
Spain
Hospital Universitario Vall d'Hebron ALS Unit. Consultas Externas; Office: 9-10-11
Barcelona, Spain, 08035
Hospital Carlos III - Hospital Universitario La Paz, ALS Unit
Madrid, Spain, 28046
Sweden
Umeå University Hospital
Umeå, Sweden, 90737
Switzerland
Kantonsspital St.Gallen, Muskelzentrum/ALS Clinic
Saint Gallen, Switzerland, 9007
United Kingdom
Leonard Wolfson Experimental Neurology Centre
London, United Kingdom, WC1N 3BG
Sponsors and Collaborators
Orphazyme
Investigators
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Principal Investigator: Michael Benatar, MD PhD University of Miami

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Responsible Party: Orphazyme
ClinicalTrials.gov Identifier: NCT03491462     History of Changes
Other Study ID Numbers: ORARIALS-01
First Posted: April 9, 2018    Key Record Dates
Last Update Posted: June 21, 2019
Last Verified: June 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Orphazyme:
arimoclomol
ALS
Additional relevant MeSH terms:
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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