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Trial record 4 of 4 for:    arimoclomol | ALS

Arimoclomol in Amyotropic Lateral Sclerosis

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ClinicalTrials.gov Identifier: NCT03491462
Recruitment Status : Recruiting
First Posted : April 9, 2018
Last Update Posted : November 26, 2018
Sponsor:
Information provided by (Responsible Party):
Orphazyme

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

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 SVC [ Time Frame: Week 76 (or end of trial) ]
    Change in Slow Vital Capacity (a measure of breathing function) over time



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

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

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 ClinicalTrials.gov identifier (NCT number): NCT03491462


Contacts
Contact: Requests at Orphazyme A/S +45 39 17 82 72 Request@orphazyme.com

Locations
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    Nicole.Turcotte@DignityHealth.org   
Principal Investigator: Shafeeq Ladha, MD         
HonorHealth Neurology Recruiting
Phoenix, Arizona, United States, 85018
Contact: Mackenzie Steinbach    480-882-4916    Mackenzie.Steinbach@honorhealth.com   
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    imturner@uci.edu   
Contact: Veronica Martin    +1 714-456-7760    vero@uci.edu   
Principal Investigator: Namita Goyal, MD         
United States, Florida
University of Miami Recruiting
Miami, Florida, United States, 33136
Contact: Maria Elena Paredes    305-243-7336    mep132@med.miami.edu   
Principal Investigator: Michael Benatar, MD, PhD         
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    alackey@kumc.edu   
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    arlena.cummings@providence.org   
Principal Investigator: Kimberly L Goslin, MD, PhD         
United States, Virginia
University of Virginia Health System Recruiting
Charlottesville, Virginia, United States, 22908
Contact: Mary Wagoner    434-924-5541    MIW9B@hscmail.mcc.virginia.edu   
Principal Investigator: Ted Burns, MD         
Canada, Quebec
Montreal Neurological Institute and Hospital Active, not recruiting
Montréal, Quebec, Canada, H3A 2B4
Spain
Hospital Universitario Vall d'Hebron ALS Unit. Consultas Externas; Office: 9-10-11 Recruiting
Barcelona, Spain, 08035
Contact: Ana Canovas    0034 932746000 ext 2780    cs.ana.belen@gmail.com   
Principal Investigator: Josep Gamez, MD         
Hospital Carlos III - Hospital Universitario La Paz, ALS Unit Active, not recruiting
Madrid, Spain, 28046
Sweden
Umeå University Hospital Recruiting
Umeå, Sweden, 90737
Contact: Erica Stenberg    +46 72548 74 10    erica.stenberg@umu.se   
Principal Investigator: Peter Andersen, MD         
Sponsors and Collaborators
Orphazyme
Investigators
Principal Investigator: Michael Benatar, MD PhD University of Miami

Responsible Party: Orphazyme
ClinicalTrials.gov Identifier: NCT03491462     History of Changes
Other Study ID Numbers: ORARIALS-01
First Posted: April 9, 2018    Key Record Dates
Last Update Posted: November 26, 2018
Last Verified: November 2018
Individual Participant Data (IPD) Sharing Statement:
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 Orphazyme:
arimoclomol
ALS

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