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:

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:

09/06/2018

Date Study Added to alsconsortium.org:

04/10/2018
  • 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

    University of Miami
    Miami, Florida 33136
    United States

    University of Virginia Health System
    Charlottesville, Virginia 22908
    United States