Arimoclomol in Amyotropic Lateral Sclerosis
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| ClinicalTrials.gov Identifier: NCT03491462 |
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Recruitment Status :
Completed
First Posted : April 9, 2018
Last Update Posted : January 20, 2021
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Sponsor:
KemPharm Denmark A/S
Information provided by (Responsible Party):
KemPharm Denmark A/S
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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 |
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 245 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 |
| Actual Primary Completion Date : | December 18, 2020 |
| Actual Study Completion Date : | December 18, 2020 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Amyotrophic lateral sclerosis
Juvenile primary lateral sclerosis
| Arm | Intervention/treatment |
|---|---|
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Experimental: Arimoclomol
Arimoclomol, capsule
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Drug: Arimoclomol
Active treatment
Other Name: Arimoclomol citrate |
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Placebo Comparator: Placebo
Placebo oral capsule (matching to experimental Arm)
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Drug: Placebo oral capsule
Matching placebo capsule
Other Name: Placebo |
Primary Outcome Measures :
- 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 :
- 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
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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 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
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
Locations
Show 29 study locations
Sponsors and Collaborators
KemPharm Denmark A/S
Investigators
| Principal Investigator: | Michael Benatar, MD PhD | University of Miami |
| Responsible Party: | KemPharm Denmark A/S |
| ClinicalTrials.gov Identifier: | NCT03491462 |
| Other Study ID Numbers: |
ORARIALS-01 |
| First Posted: | April 9, 2018 Key Record Dates |
| Last Update Posted: | January 20, 2021 |
| Last Verified: | January 2021 |
| 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 KemPharm Denmark A/S:
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arimoclomol ALS |
Additional relevant MeSH terms:
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Amyotrophic Lateral Sclerosis Motor Neuron Disease Sclerosis Pathologic Processes Neurodegenerative Diseases Nervous System Diseases |
Neuromuscular Diseases Spinal Cord Diseases Central Nervous System Diseases TDP-43 Proteinopathies Proteostasis Deficiencies Metabolic Diseases |