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Arimoclomol in Amyotropic Lateral Sclerosis

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ClinicalTrials.gov Identifier: NCT03491462
Recruitment Status : Recruiting
First Posted : April 9, 2018
Last Update Posted : April 3, 2019
Sponsor:
Information provided by (Responsible Party):
Orphazyme

Tracking Information
First Submitted Date  ICMJE March 27, 2018
First Posted Date  ICMJE April 9, 2018
Last Update Posted Date April 3, 2019
Actual Study Start Date  ICMJE July 31, 2018
Estimated Primary Completion Date December 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 16, 2018)
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)
Original Primary Outcome Measures  ICMJE
 (submitted: April 6, 2018)
Combined Assessment of Function and Survival (CAFS) [ Time Frame: over 76 Weeks ]
Time to event (Permanent assisted ventilation, tracheostomy or death) is combined with ALSFRS-R score (12 functions on a 5-point ordinal rating scale with maximum score of 48) to create a nonparametric rank analysis of covariance
Change History Complete list of historical versions of study NCT03491462 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: April 1, 2019)
  • 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.
  • 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
  • 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
Original Secondary Outcome Measures  ICMJE
 (submitted: April 6, 2018)
  • Time to permanent assisted ventilation (PAV)/tracheostomy/death [ Time Frame: over 76 weeks ]
    time from baseline to event
  • ALS Function Rating Scale, Revised (ALSFRS-R) [ Time Frame: over 76 weeks ]
    measure of patient function
  • Slow Vital Capacity (SVC) [ Time Frame: over 76 weeks ]
    measurement for the quality of breathing
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Arimoclomol in Amyotropic Lateral Sclerosis
Official Title  ICMJE A Phase 3, Randomised, Placebo-Controlled Trial of Arimoclomol in Amyotropic Lateral Sclerosis
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)
Detailed Description Screening of up to 4 weeks Treatment of up to 76 weeks
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE 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
Condition  ICMJE Amyotrophic Lateral Sclerosis
Intervention  ICMJE
  • Drug: Arimoclomol
    Active treatment
    Other Name: Arimoclomol citrate
  • Drug: Placebo oral capsule
    Matching placebo capsule
    Other Name: Placebo
Study Arms  ICMJE
  • Experimental: Arimoclomol
    Arimoclomol, capsule
    Intervention: Drug: Arimoclomol
  • Placebo Comparator: Placebo
    Placebo oral capsule (matching to experimental Arm)
    Intervention: Drug: Placebo oral capsule
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: April 6, 2018)
231
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE January 2021
Estimated Primary Completion Date December 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Requests at Orphazyme A/S +45 39 17 82 72 Request@orphazyme.com
Listed Location Countries  ICMJE Belgium,   Canada,   Denmark,   France,   Germany,   Italy,   Netherlands,   Poland,   Spain,   Sweden,   Switzerland,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03491462
Other Study ID Numbers  ICMJE ORARIALS-01
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: Undecided
Responsible Party Orphazyme
Study Sponsor  ICMJE Orphazyme
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Michael Benatar, MD PhD University of Miami
PRS Account Orphazyme
Verification Date February 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP