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Arimoclomol in Amyotropic Lateral Sclerosis - Open Label Extension Trial

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ClinicalTrials.gov Identifier: NCT03836716
Recruitment Status : Not yet recruiting
First Posted : February 11, 2019
Last Update Posted : February 11, 2019
Sponsor:
Information provided by (Responsible Party):
Orphazyme

Brief Summary:
A multicenter, non-randomized, open label trial, to assess long term safety and efficacy of Arimoclomol in subjects with Amyotrophic Lateral Sclerosis (ALS)who have completed the ORARIALS-01 trial.

Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis Drug: Arimoclomol Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 121 participants
Intervention Model: Single Group Assignment
Intervention Model Description: Non-randomized open label
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Open Label, Non-randomized Extension Trial to Assess Long Term Safety and Efficacy of Arimoclomol in Subjects With Amyotropic Lateral Sclerosis Who Have Completed the ORARIALS-01 Trial
Estimated Study Start Date : April 30, 2019
Estimated Primary Completion Date : July 30, 2022
Estimated Study Completion Date : July 30, 2022


Arm Intervention/treatment
Experimental: Arimoclomol
Arimoclomol, capsule
Drug: Arimoclomol
Active treatment
Other Name: Arimoclomol citrate




Primary Outcome Measures :
  1. Assess long term safety of Arimoclomol in treatment of ALS [ Time Frame: Over 76 weeks ]
    Incidence and severity of Treatment-emergent adverse events (TEAEs)

  2. Assess long term safety of Arimoclomol in treatment of ALS [ Time Frame: Over 76 weeks ]
    Mean and change from Baseline to Week 76 (or end of trial) in clinical safety laboratory tests and vital signs

  3. Assess long term safety of Arimoclomol in treatment of ALS [ Time Frame: Over 76 weeks ]
    Incidence of potentially clinically significant abnormalities in clinical trial safety laboratory tests and vital signs over a treatment period of 76 weeks

  4. Assess long term safety of Arimoclomol in treatment of ALS [ Time Frame: Over 76 weeks ]
    Columbia-Suicide Severity Rating Scale (C-SSRS) over a treatment period of 76 weeks


Secondary Outcome Measures :
  1. Evaluate long term efficacy of Arimoclomol treatment of ALS [ Time Frame: Over 76 weeks ]
    Time to Permanent Assisted Ventilation (PAV)/tracheostomy/death /for subjects entering this trial having completed 76 weeks of randomized treatment in ORARIALS-01

  2. Evaluate long term efficacy of Arimoclomol treatment of ALS [ Time Frame: Over 76 weeks ]
    Time to death (for subjects entering this trial having met the survival endpoint of PAV or tracheostomy in ORARIALS-01

  3. Evaluate long term efficacy of Arimoclomol treatment of ALS [ Time Frame: Over 76 weeks ]
    Change in ALS Functional Rating Scale Revised (ALSFRS-R) from Baseline to the end of the trial

  4. Evaluate long term efficacy of Arimoclomol treatment of ALS [ Time Frame: Over 76 weeks ]
    Change in Slow Vital Capacity (SVC) from Baseline to the end of the trial (for subjects who did not meet the survival endpoint in the ORARIALS-01 trial



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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 is able to comprehend and is willing to provide written informed consent and is capable and willing to comply with trial procedures.
  • Subject has completed the ORARIALS-01 trial
  • On treatment with IMP defined as last dose within 2 weeks of the last visit of the blinded ORARIALS-01 trial.

Exclusion Criteria:

  • Known or suspected allergy or intolerance to the IMP (Arimoclomol or constituents)
  • Exposure to any other investigational treatment, advanced therapy medicinal product or use of any other prohibited concomitant medications
  • Significant protocol deviation in the blinded ORARIALS-01 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): NCT03836716


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

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: NCT03836716     History of Changes
Other Study ID Numbers: ORARIALS-02
First Posted: February 11, 2019    Key Record Dates
Last Update Posted: February 11, 2019
Last Verified: February 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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Sclerosis
Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases