A Phase 3, Randomised, Placebo-Controlled Trial of Arimoclomol in Amyotropic Lateral Sclerosis

Study Purpose:

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

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Not enrolling

Phase:

Phase III

Study Chair(s)/Principal Investigator(s):

Michael Benatar, MD PhD University of Miami

Clinicaltrials.gov ID (11 digit #):

NCT03491462

Neals Affiliated?

No

Coordinating Center Contact Information


Requests at Orphazyme A/S / .(JavaScript must be enabled to view this email address) / +45 51 71 68 37
.(JavaScript must be enabled to view this email address)

Full Study Summary:

Study Sponsor:

Orphazyme

Participant Duration:

Screening of up to 4 weeks Treatment of up to 76 weeks

Estimated Enrollment:

231

Estimated Study Start Date:

07/31/2018

Estimated Study Completion Date:

12/31/2020

Posting Last Modified Date:

05/02/2019

Date Study Added to alsconsortium.org:

04/10/2018
  • More Information
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Min Vital Capacity (% predicted normal):

    80

    Time since Symptom Onset:

    <18 months

    Time since Diagnosis:

    N/A

    Can participants use Riluzole?

    Yes


    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
    • ALSFRS-R equal to or above 35 and erect 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

  • Site Contact Information

    Barrow Neurological Institute
    Nicole Turcotte / .(JavaScript must be enabled to view this email address) / 602-406-4775
    Phoenix , Arizona 85013
    United States

    HonorHealth Neurology
    Mackenzie Steinbach / .(JavaScript must be enabled to view this email address) / 480-882-4916
    Phoenix, Arizona 85018
    United States

    UC Irvine Health ALS and Neuromuscular Center
    Ivonne Turner / .(JavaScript must be enabled to view this email address) / 714-456-7760
    Veronica Martin / .(JavaScript must be enabled to view this email address) / 714-456-7760
    Orange, California 92868
    United States

    University of Miami
    Maria Elena Paredes / .(JavaScript must be enabled to view this email address) / 305-243-7336
    Miami, Florida 33136
    United States

    The University of Iowa
    Jeri Sieren / .(JavaScript must be enabled to view this email address) / 319-356-8744
    Iowa City , Iowa 52242
    United States

    University of Kansas Medical Center
    Alyssa Lackey / .(JavaScript must be enabled to view this email address) / 913-945-9942
    Kansas City, Kansas 66160
    United States

    Hospital for Special Surgery
    William Mark Richardson / .(JavaScript must be enabled to view this email address) / 646-797-8657
    New York, New York 10021
    United States

    Providence Brain & Spine Institute
    Arlena Cummings / .(JavaScript must be enabled to view this email address) / 503-962-1171
    Portland , Oregon 97213
    United States

    University of Pensylvania
    Kelly Almasy / .(JavaScript must be enabled to view this email address) / 215-829-5041
    Philadelphia, Pennsylvania 19107
    United States

    University of Texas Southwestern Medical Center
    Amruta Joshi / .(JavaScript must be enabled to view this email address) / 213-648-9380
    Dallas, Texas 75390
    United States

    University of Virginia Health System
    Mary Wagoner / .(JavaScript must be enabled to view this email address) / 434-924-5541
    Charlottesville, Virginia 22908
    United States

    Catholic University Leuven
    Leuven, 3000
    Belgium

    London Health Sciences Centre
    Christine Piechowicz / .(JavaScript must be enabled to view this email address) / 519-685-8500 ext 34858
    London, Ontario N6A 5A5
    Canada

    Sunnybrook Health Sciences Centre
    Liane Phung / .(JavaScript must be enabled to view this email address) / 16-480-6100 ext 87561
    Toronto, Ontario M4N 3M5
    Canada

    Montreal Neurological Institute and Hospital
    Montréal, Quebec H3A 2B4
    Canada

    Neuroligisk afdeling Aarhus Universitets Hospital
    Charlotte Odgaard / .(JavaScript must be enabled to view this email address) / Aarhus, 8000
    Denmark

    Neurologisk afdeling Bispebjerg Hospital
    Copenhagen, 2400
    Denmark

    Centre Hospitalier Regional Universitaire (CHRU) Montpellier - Hopital Gui De Chauliac
    Montpellier, 34295
    France

    Groupe Hospitalier Pitie-Salpetriere
    Paris, 75013
    France

    Charite - Universitaetsmedizin Berlin
    Birgit Koch / .(JavaScript must be enabled to view this email address) / +49 30 450 560028
    Berlin, 13353
    Germany

    Medizinische Hochschule Hannover (MHH)
    Chantal Fischer / .(JavaScript must be enabled to view this email address) / +49 (0) 511 532-8333
    Hannover, 30625
    Germany

    Universitaetsklinikum Ulm
    Johannes Dorst / .(JavaScript must be enabled to view this email address) / +49- 731 177 5285
    Ulrike Weiland / .(JavaScript must be enabled to view this email address) / +49- 731 177 5285
    Ulm, 89081
    Germany

    Instituti Clinica Scientifici Maugeri
    Milano, 20138
    Italy

    Azienda Ospedaliero Universitaria (AUO) di Torino
    Torino, 10126
    Italy

    University Medical Center Utrecht
    Tommy Bunte / .(JavaScript must be enabled to view this email address) / 31887573110
    Utrecht , 3584CX
    Netherlands

    Centrum Medyczne NeuroProtect
    Marta Biel / .(JavaScript must be enabled to view this email address) / +48 22 468 15 48
    Warsaw, 01-684
    Poland

    Citi Clinic
    Adam R Ronert / .(JavaScript must be enabled to view this email address) / 48607616559
    Warsaw, 02-473
    Poland

    Hospital Universitario Vall d'Hebron
    Ana Canovas / .(JavaScript must be enabled to view this email address) / 0034 932746000 ext 2780
    Barcelona, 08035
    Spain

    Hospital Carlos III - Hospital Universitario La Paz
    Saul Marin / .(JavaScript must be enabled to view this email address) / +34 686061101
    Madrid, 28046
    Spain

    Umeå University Hospital
    Erica Stenberg / .(JavaScript must be enabled to view this email address) / +46 72548 74 10
    Umeå , 90737
    Sweden

    Kantonsspital St.Gallen
    Christoph Neuwirth / .(JavaScript must be enabled to view this email address) / +41 714943581
    Saint Gallen, 9007
    Switzerland

    Leonard Wolfson Experimental Neurology Centre
    Anna Bellin / .(JavaScript must be enabled to view this email address) / London , WC1N 3BG
    United Kingdom