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Arimoclomol in Amyotropic Lateral Sclerosis

Sponsor
KemPharm Denmark A/S (Industry)
Overall Status
Completed
CT.gov ID
NCT03491462
Collaborator
(none)
245
Enrollment
29
Locations
2
Arms
28.6
Actual Duration (Months)
8.4
Patients Per Site
0.3
Patients Per Site Per Month

Study Details

Study Description

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
Phase 3

Detailed Description

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

Study Design

Study Type:
Interventional
Actual Enrollment :
245 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Intervention Model Description:
placebo-controlled, parallel groupplacebo-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 :
Jul 31, 2018
Actual Primary Completion Date :
Dec 18, 2020
Actual Study Completion Date :
Dec 18, 2020

Arms and Interventions

Arm Intervention/Treatment
Experimental: Arimoclomol

Arimoclomol, capsule

Drug: Arimoclomol
Active treatment
Other Names:
  • Arimoclomol citrate

    		•
    Placebo Comparator: Placebo

    Placebo oral capsule (matching to experimental Arm)

    Drug: Placebo oral capsule
    Matching placebo capsule
    Other Names:
  • Placebo

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Combined Assessment of Function and Survival (CAFS) [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 [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) [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 [Week 76 (or end of trial)]

      Change in Slow Vital Capacity (a measure of breathing function) over time

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years and Older
    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 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

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 St. Joseph's Hospital and Medical Center (SJHMC) - Barrow Neurological Institute (BNI) - The Gregory W. Fulton ALS and Neuromuscular Disease Center Phoenix Arizona United States 85013
    2 HonorHealth Neurology Phoenix Arizona United States 85018
    3 UC Irvine Health ALS and Neuromuscular Center Orange California United States 92868
    4 University of Miami Miami Florida United States 33136
    5 University of Kansas Medical Center (KUMC) - Landon Center on Aging Kansas City Kansas United States 66160
    6 Hospital for Special Surgery New York New York United States 10021
    7 Providence Brain & Spine Institute Portland Oregon United States 97213
    8 University of Pensylvania, Perelman Center for Advanced Medicine - Penn Neuroscience Center Philadelphia Pennsylvania United States 19107
    9 University of Texas Southwestern Medical Center Dallas Texas United States 75390
    10 University of Virginia Health System Charlottesville Virginia United States 22908
    11 Catholic University Leuven Leuven Belgium 3000
    12 London Health Sciences Centre London Ontario Canada N6A 5A5
    13 Sunnybrook Health Sciences Centre Toronto Ontario Canada M4N 3M5
    14 Montreal Neurological Institute and Hospital Montréal Quebec Canada H3A 2B4
    15 Centre Hospitalier Regional Universitaire (CHRU) Montpellier - Hopital Gui De Chauliac Montpellier France 34295
    16 Groupe Hospitalier Pitie-Salpetriere - Centre d'Investigation Clinique Neurosciences 1422 Paris France 75013
    17 Charite - Universitaetsmedizin Berlin - Campus Virchow-Klinikum (CVK) - Ambulanz fuer ALS und andere Motoneuronenerkrankungen Berlin Germany 13353
    18 Medizinische Hochschule Hannover (MHH) - Klinik fuer Neurologie Hannover Germany 30625
    19 Universitaetsklinikum Ulm - Klinik fuer Neurologie Ulm Germany 89081
    20 Instituti Clinica Scientifici Maugeri - IRCCS Milano Italy 20138
    21 Azienda Ospedaliero Universitaria (AUO) di Torino - Citta'della Salute e della Scienza di Torino Torino Italy 10126
    22 University Medical Center Utrecht Utrecht Netherlands 3584CX
    23 Centrum Medyczne NeuroProtect Warsaw Poland 01-684
    24 Citi Clinic Warsaw Poland 02-473
    25 Hospital Universitario Vall d'Hebron ALS Unit. Consultas Externas; Office: 9-10-11 Barcelona Spain 08035
    26 Hospital Carlos III - Hospital Universitario La Paz, ALS Unit Madrid Spain 28046
    27 Umeå University Hospital Umeå Sweden 90737
    28 Kantonsspital St.Gallen, Muskelzentrum/ALS Clinic Saint Gallen Switzerland 9007
    29 Leonard Wolfson Experimental Neurology Centre London United Kingdom WC1N 3BG

    Sponsors and Collaborators

    • KemPharm Denmark A/S

    Investigators

    • Principal Investigator: Michael Benatar, MD PhD, University of Miami

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    KemPharm Denmark A/S
    ClinicalTrials.gov Identifier:
    NCT03491462
    Other Study ID Numbers:
    • ORARIALS-01
    First Posted:
    Apr 9, 2018
    Last Update Posted:
    Jan 20, 2021
    Last Verified:
    Jan 1, 2021
    Individual Participant Data (IPD) Sharing Statement:
    Undecided
    Plan to Share IPD:
    Undecided
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Keywords provided by KemPharm Denmark A/S
    arimoclomol
    ALS
    Additional relevant MeSH terms:
    Motor Neuron Disease
    Amyotrophic Lateral Sclerosis
    Sclerosis

    Study Results

    No Results Posted as of Jan 20, 2021