Open Label, Non-randomized Extension Trial to Assess Long Term Safety and Efficacy of Arimoclomol in Subjects With Amyotropic Lateral Sclerosis Who Have Completed the ORARIALS-01 Trial

Study Purpose:

A multicenter, non-randomized, open label trial, to assess long term safety and efficacy of Arimoclomol in subjects with Amyotrophic Lateral Sclerosis (ALS)who have completed the ORARIALS-01 trial.

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 #):

NCT03836716

Neals Affiliated?

No

Coordinating Center Contact Information


Requests at Orphazyme A/S / .(JavaScript must be enabled to view this email address) / +45 39 17 82 72
.(JavaScript must be enabled to view this email address)

Full Study Summary:

Study Sponsor:

Orphazyme

Participant Duration:

Estimated Enrollment:

231

Estimated Study Start Date:

08/15/2019

Estimated Study Completion Date:

08/01/2022

Posting Last Modified Date:

11/04/2019

Date Study Added to alsconsortium.org:

08/29/2019
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    • Subject is able to comprehend and is willing to provide written informed consent and is capable and willing to comply with trial procedures, or in the circumstance that the subject is incompetent, informed consent/assent is provided in accordance with local regulation and/or procedures
    • Subject has completed the ORARIALS-01 trial (i.e., met one of the surrogate survival endpoints of tracheostomy or PAV or has completed the 76 weeks randomized treatment period)
    • On treatment with IMP defined as last dose within 2 weeks of the last visit of the blinded ORARIALS-01 trial.
    • Able and willing to travel to the site for the Baseline visit and in the investigator's opinion is expected to be able to attend the clinic for the visit at Week 4.

    Exclusion Criteria:

    • Known or suspected allergy or intolerance to the IMP (Arimoclomol or constituents)
    • Exposure to any other investigational treatment, advanced therapy medicinal product or use of any other prohibited concomitant medications
    • Significant protocol deviation in the blinded ORARIALS-01 trial based on the Investigator's judgement
    • Women who are lactating or pregnant, or men or women unwilling to use a highly effective method of birth control if not surgically sterile (defined as bilateral tubal ligation, bilateral oophorectomy, or hysterectomy for women; vasectomy for men) for female participants until 4 weeks after last dose and for male participants until 3 months after last dose. Pre-menopausal women must have a negative pregnancy test prior to dosing with trial medication.
    • Any of the following medically significant conditions:
      Clinically significant renal or hepatic disease, as indicated by clinical laboratory assessment (results ≥ 3 times the upper limit of normal [ULN] for aspartate aminotransferase, alanine aminotransferase, and lactate dehydrogenase, bilirubin ≥ 2 times the ULN, or creatinine ≥ 1.5 times the ULN).
      Any new condition or worsening of existing condition which, in the opinion of the investigator, would make the subject unsuitable for enrolment or could interfere with the subject participating in or completing the trial
    • Any serious adverse event or moderate/severe adverse event from the ORARIALS-01 trial which is ongoing at the time of transitioning to ORARIALS-02 and assessed as probably related to IMP

  • 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
    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

    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,
    Belgium

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

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

    Montreal Neurological Institute and Hospital
    Natalie Saunders / .(JavaScript must be enabled to view this email address) / +15143981779
    Montréal, Quebec
    Canada

    Centre Hospitalier Regional Universitaire (CHRU) Montpellier
    Laura Labar / .(JavaScript must be enabled to view this email address) / +330467330281
    Montpellier,
    France

    Groupe Hospitalier Pitie-Salpetriere
    Sylvie Coudoin / .(JavaScript must be enabled to view this email address) / +330142165762
    Paris,
    France

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

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

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

    Instituti Clinica Scientifici Maugeri
    Riccardo Sideri / .(JavaScript must be enabled to view this email address) / +390250725260
    Milano ,
    Italy

    Azienda Ospedaliero Universitaria (AUO) di Torino
    Gio Fuda / .(JavaScript must be enabled to view this email address) / +390116335439
    Torino,
    Italy

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

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

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

    Hospital Universitario Vall d'Hebron
    Ana Canovas / .(JavaScript must be enabled to view this email address) / 0034 93274600 ext 2780
    Barcelona,
    Spain

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

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

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

    Leonard Wolfson Experimental Neurology Centre
    Anna Bellin / .(JavaScript must be enabled to view this email address) / 2034488014
    London,
    United Kingdom