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Trial record 2 of 11 for:    Arimoclomol

Arimoclomol in Amyotropic Lateral Sclerosis

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ClinicalTrials.gov Identifier: NCT03491462
Recruitment Status : Active, not recruiting
First Posted : April 9, 2018
Last Update Posted : June 21, 2019
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

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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 Slow Vital Capacity [ 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 (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 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


  Show 29 Study Locations
Sponsors and Collaborators
Orphazyme
Investigators
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Principal Investigator: Michael Benatar, MD PhD University of Miami

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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: June 21, 2019
Last Verified: June 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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