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Quick Info
Status
This study has been completed
Phase
3
Trial Type
Randomized, parallel assignment, quadruple masking, placebo controlled
Treatment Type
Drug: Arimoclomol
Randomization
2:1
Enrollment
245
Start Date
7/31/2018
Sponsor
Orphazyme
Contact Information
Locations
Belgium, Other
Catholic University Leuven, Leuven, 3000, Belgium
Canada, Ontario
London Health Sciences Centre, London, ON, N6A 5A5, Canada
Sunnybrook Health Sciences Centre, Toronto, ON, M4N 3M5, Canada
Canada, Quebec
Montreal Neurological Institute and Hospital, Montréal, QC, H3A 2B4, Canada
France, Other
Centre Hospitalier Regional Universitaire (CHRU) Montpellier - Hopital Gui De Chauliac, Montpellier, 34295, France
Groupe Hospitalier Pitie-Salpetriere - Centre d'Investigation Clinique Neurosciences 1422, Paris, 75013, France
Germany, Other
Charite - Universitaetsmedizin Berlin - Campus Virchow-Klinikum (CVK) - Ambulanz fuer ALS und andere, Berlin, 13353, Germany
Medizinische Hochschule Hannover (MHH) - Klinik fuer Neurologie, Hannover, 30625, Germany
Universitaetsklinikum Ulm - Klinik fuer Neurologie, Ulm, 89081, Germany
Italy, Other
Instituti Clinica Scientifici Maugeri - IRCCS, Milano, 20138, Italy
Azienda Ospedaliero Universitaria (AUO) di Torino - Citta'della Salute e della Scienza di Torino, Torino, 10126, Italy
Netherlands, Other
University Medical Center Utrecht, Utrecht, 3584CX, Netherlands
Poland, Other
Centrum Medyczne NeuroProtect, Warsaw, 01-684, Poland
Citi Clinic, Warsaw, 02-473, Poland
Spain, Other
Hospital Universitario Vall d'Hebron ALS Unit. Consultas Externas; Office: 9-10-11, Barcelona, 08035, Spain
Hospital Carlos III - Hospital Universitario La Paz, ALS Unit, Madrid, 28046, Spain
Sweden, Other
Umeå University Hospital, Umeå, 90737, Sweden
Switzerland, Other
Kantonsspital St.Gallen, Muskelzentrum/ALS Clinic, Saint Gallen, 9007, Switzerland
United Kingdom, Other
Leonard Wolfson Experimental Neurology Centre, London, WC1N 3BG, United Kingdom
United States, Arizona
St. Joseph's Hospital and Medical Center (SJHMC) - Barrow Neurological Institute (BNI) - The Gregory, Phoenix, AZ, 85013, United States
HonorHealth Neurology, Phoenix, AZ, 85018, United States
United States, California
UC Irvine Health ALS and Neuromuscular Center, Orange, CA, 92868, United States
United States, Florida
University of Miami, Miami, FL, 33136, United States
United States, Kansas
University of Kansas Medical Center (KUMC) - Landon Center on Aging, Kansas City, KS, 66160, United States
United States, New York
Hospital for Special Surgery, New York, NY, 10021, United States
United States, Oregon
Providence Brain & Spine Institute, Portland, OR, 97213, United States
United States, Pennsylvania
University of Pensylvania, Perelman Center for Advanced Medicine - Penn Neuroscience Center, Philadelphia, PA, 19107, United States
United States, Texas
University of Texas Southwestern Medical Center, Dallas, TX, 75390, United States
United States, Virginia
University of Virginia Health System, Charlottesville, VA, 22908, United States
Enrollment Criteria
Breathing Ability
Percent lung function (FVC) or (SVC)
≥80% (SVC)
Months Since Onset
Number of months since first symptoms of ALS.
<18 months
Non-Invasive Ventilation (NIV)
Can PALS use a BiPAP in the trial?
No more than 2 hours/day
Diaphragm Pacer (DPS)
Can PALS use a DPS in the trial?
No
Edaravone Usage
Can a PALS use edaravone (Radicut/Radicava) while enrolled in the trial?
No
Open Label
Unknown
Update Notes
Study completed
1/20/2021
No significant updates
9/16/2020
No significant update
2/18/2020
Recruitment status updated
7/1/2019
Locations updated
6/5/2019
Recruitment status at locations updated
4/8/2019
Locations updated
3/4/2019
Locations updated
2/19/2019
Locations updated
2/5/2019
Locations updated
1/30/2019
Locations added
1/15/2019
Locations updated
1/2/2019
Locations updated
12/14/2018
Locations updated
11/23/2018
Locations updated
10/5/2018
Locations updated
8/24/2018
First patient dosed in trial.
8/14/2018
Trial added
4/9/2018

Other Information

Purpose
A multicenter, randomized, double-blind, placebo-controlled, parallel group trial to evaluate the efficacy and safety of arimoclomol in amyotropic lateral sclerosis (ALS)
Eligibility
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 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 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
Details
Screening of up to 4 weeks Treatment of up to 76 weeks
Collaborator(s)
  • Orphazyme
Trial Protocol as Published on Clinicaltrials.gov
NCT03491462 (First Published: 3/27/2018)
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