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Arimoclomol in Amyotropic Lateral Sclerosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03491462
Recruitment Status : Recruiting
First Posted : April 9, 2018
Last Update Posted : April 3, 2019
Information provided by (Responsible Party):

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

Information from the National Library of Medicine

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

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 identifier (NCT number): NCT03491462

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Contact: Requests at Orphazyme A/S +45 39 17 82 72

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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 Recruiting
Phoenix, Arizona, United States, 85013
Contact: Nicole Turcotte    602-406-4775   
Principal Investigator: Shafeeq Ladha, MD         
HonorHealth Neurology Recruiting
Phoenix, Arizona, United States, 85018
Contact: Mackenzie Steinbach    480-882-4916   
Principal Investigator: Todd Levine, MD         
United States, California
UC Irvine Health ALS and Neuromuscular Center Recruiting
Orange, California, United States, 92868
Contact: Ivonne Turner    714-456-7760   
Contact: Veronica Martin    +1 714-456-7760   
Principal Investigator: Namita Goyal, MD         
United States, Florida
University of Miami Recruiting
Miami, Florida, United States, 33136
Contact: Maria Elena Paredes    305-243-7336   
Principal Investigator: Michael Benatar, MD, PhD         
United States, Iowa
The University of Iowa - The Institute for Clinical and Translational Science (ICTS) Recruiting
Iowa City, Iowa, United States, 52242-1007
Contact: Jeri Sieren    319-356-8744   
Principal Investigator: Andrea Swenson, MD         
United States, Kansas
University of Kansas Medical Center (KUMC) - Landon Center on Aging Recruiting
Kansas City, Kansas, United States, 66160
Contact: Alyssa Lackey    913-945-9942   
Principal Investigator: Duaa Jabari, MD         
United States, New York
Hospital for Special Surgery Recruiting
New York, New York, United States, 10021
Contact: William Mark Richardson    646-797-8657    richardsonw@HSS.EDU   
Principal Investigator: Dale J. Lange, MD         
United States, Oregon
Providence Brain & Spine Institute Recruiting
Portland, Oregon, United States, 97213
Contact: Arlena Cummings    503-962-1171   
Principal Investigator: Kimberly L Goslin, MD, PhD         
United States, Pennsylvania
University of Pensylvania, Perelman Center for Advanced Medicine - Penn Neuroscience Center Recruiting
Philadelphia, Pennsylvania, United States, 19107
Contact: Kelly Almasy    215-829-5041   
Principal Investigator: Colin Quinn, MD         
United States, Texas
University of Texas Southwestern Medical Center Recruiting
Dallas, Texas, United States, 75390
Contact: Amruta Joshi    213-648-9380   
Principal Investigator: Sharon Nations, MD         
United States, Virginia
University of Virginia Health System Recruiting
Charlottesville, Virginia, United States, 22908
Contact: Mary Wagoner    434-924-5541   
Principal Investigator: Matthew Elliott, MD         
Catholic University Leuven Recruiting
Leuven, Belgium, 3000
Principal Investigator: Phillip Van Damme, MD         
Canada, Ontario
London Health Sciences Centre Not yet recruiting
London, Ontario, Canada, N6A 5A5
Contact: Christine Piechowicz    519-685-8500 ext 34858   
Principal Investigator: Christen Shoesmith, MD         
Sunnybrook Health Sciences Centre Recruiting
Toronto, Ontario, Canada, M4N 3M5
Contact: Liane Phung    16-480-6100 ext 87561   
Principal Investigator: Lorne Zinman, MD         
Canada, Quebec
Montreal Neurological Institute and Hospital Recruiting
Montréal, Quebec, Canada, H3A 2B4
Principal Investigator: Angela Genge, MD         
Neuroligisk afdeling Aarhus Universitets Hospital Not yet recruiting
Aarhus, Denmark, 8000
Contact: Charlotte Odgaard    21555118   
Principal Investigator: Anette Torvin Møller, MD         
Neurologisk afdeling Bispebjerg Hospital Not yet recruiting
Copenhagen, Denmark, 2400
Principal Investigator: Merete Karlsborg, MD         
Centre Hospitalier Regional Universitaire (CHRU) Montpellier - Hopital Gui De Chauliac Recruiting
Montpellier, France, 34295
Principal Investigator: William Camu, MD         
Groupe Hospitalier Pitie-Salpetriere - Centre d'Investigation Clinique Neurosciences 1422 Recruiting
Paris, France, 75013
Principal Investigator: Pierre-Francois Pradat, MD         
Charite - Universitaetsmedizin Berlin - Campus Virchow-Klinikum (CVK) - Ambulanz fuer ALS und andere Motoneuronenerkrankungen Not yet recruiting
Berlin, Germany, 13353
Contact: Birgit Koch    +49 30 450 560028   
Principal Investigator: Thomas Meyer, MD         
Medizinische Hochschule Hannover (MHH) - Klinik fuer Neurologie Recruiting
Hannover, Germany, 30625
Contact: Chantal Fischer    +49 (0) 511 532-8333   
Principal Investigator: Susanne Petri, MD         
Universitaetsklinikum Ulm - Klinik fuer Neurologie Not yet recruiting
Ulm, Germany, 89081
Contact: Johannes Dorst, MD    +49- 731 177 5285   
Contact: Ulrike Weiland, MD    +49- 731 177 5285   
Principal Investigator: Albert Christian Ludolph, MD         
Instituti Clinica Scientifici Maugeri - IRCCS Recruiting
Milano, Italy, 20138
Principal Investigator: Gabriele Mora, MD         
Azienda Ospedaliero Universitaria (AUO) di Torino - Citta'della Salute e della Scienza di Torino Recruiting
Torino, Italy, 10126
Principal Investigator: Adriano Chio, MD         
University Medical Center Utrecht Recruiting
Utrecht, Netherlands, 3584CX
Contact: Tommy Bunte    31887573110   
Principal Investigator: Leonard Van den Berg, MD         
Centrum Medyczne NeuroProtect Recruiting
Warsaw, Poland, 01-684
Contact: Marta Biel    +48 22 468 15 48   
Principal Investigator: Mariusz Grudniak, MD         
Citi Clinic Recruiting
Warsaw, Poland, 02-473
Contact: Adam R Ronert    48607616559   
Principal Investigator: Magdalena Kuzma-Kozakiewicz, MD         
Hospital Universitario Vall d'Hebron ALS Unit. Consultas Externas; Office: 9-10-11 Recruiting
Barcelona, Spain, 08035
Contact: Ana Canovas    0034 932746000 ext 2780   
Principal Investigator: Josep Gamez, MD         
Hospital Carlos III - Hospital Universitario La Paz, ALS Unit Recruiting
Madrid, Spain, 28046
Contact: Saul Marin    +34 686061101   
Principal Investigator: Dalia Chaverri, MD         
Umeå University Hospital Recruiting
Umeå, Sweden, 90737
Contact: Erica Stenberg    +46 72548 74 10   
Principal Investigator: Peter Andersen, MD         
Kantonsspital St.Gallen, Muskelzentrum/ALS Clinic Recruiting
Saint Gallen, Switzerland, 9007
Contact: Christoph Neuwirth, MD    +41 714943581;   
Principal Investigator: Christoph Neuwirth, MD         
United Kingdom
Leonard Wolfson Experimental Neurology Centre Recruiting
London, United Kingdom, WC1N 3BG
Contact: Anna Bellin    203448811   
Principal Investigator: Michael Hanna, MD         
Sponsors and Collaborators
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Principal Investigator: Michael Benatar, MD PhD University of Miami

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Responsible Party: Orphazyme Identifier: NCT03491462     History of Changes
Other Study ID Numbers: ORARIALS-01
First Posted: April 9, 2018    Key Record Dates
Last Update Posted: April 3, 2019
Last Verified: February 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:

Additional relevant MeSH terms:
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Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases