A Phase 3, Randomised, Placebo-Controlled Trial of Arimoclomol in Amyotropic Lateral Sclerosis
Arimoclomol in Amyotropic Lateral Sclerosis
Sponsors
Source
Orphazyme
Oversight Info
Has Dmc
Yes
Is Fda Regulated Drug
Yes
Is Fda Regulated Device
No
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
Overall Status
Active, not recruiting
Start Date
2018-07-31
Completion Date
2021-01-01
Primary Completion Date
2020-12-01
Phase
Phase 3
Study Type
Interventional
Primary Outcome
Measure |
Time Frame |
|
Combined Assessment of Function and Survival (CAFS) |
over 76 Weeks |
Secondary Outcome
Measure |
Time Frame |
|
Time to permanent assisted ventilation (PAV)/tracheostomy/death |
over 76 weeks |
|
Change from Baseline to Week 76 (or end-of-trial) in the revised ALS Functional Rating Scale (ALSFRS-R) |
Week 76 (or end of trial) |
|
Change from Baseline to Week 76 (or end-of-trial) in Slow Vital Capacity |
Week 76 (or end of trial) |
Enrollment
231
Condition
Intervention
Intervention Type
Drug
Intervention Name
Description
Active treatment
Arm Group Label
Arimoclomol
Other Name
Arimoclomol citrate
Intervention Type
Drug
Intervention Name
Description
Matching placebo capsule
Arm Group Label
Placebo
Other Name
Placebo
Eligibility
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
Gender
All
Minimum Age
18 Years
Maximum Age
N/A
Healthy Volunteers
No
Overall Official
Last Name |
Role |
Affiliation |
|
Michael Benatar, MD PhD |
Principal Investigator |
University of Miami |
Location
Facility |
|
St. Joseph's Hospital and Medical Center (SJHMC) - Barrow Neurological Institute (BNI) - The Gregory W. Fulton ALS and Neuromuscular Disease Center Phoenix Arizona 85013 United States |
|
HonorHealth Neurology Phoenix Arizona 85018 United States |
|
UC Irvine Health ALS and Neuromuscular Center Orange California 92868 United States |
|
University of Miami Miami Florida 33136 United States |
|
University of Kansas Medical Center (KUMC) - Landon Center on Aging Kansas City Kansas 66160 United States |
|
Hospital for Special Surgery New York New York 10021 United States |
|
Providence Brain & Spine Institute Portland Oregon 97213 United States |
|
University of Pensylvania, Perelman Center for Advanced Medicine - Penn Neuroscience Center Philadelphia Pennsylvania 19107 United States |
|
University of Texas Southwestern Medical Center Dallas Texas 75390 United States |
|
University of Virginia Health System Charlottesville Virginia 22908 United States |
|
Catholic University Leuven Leuven 3000 Belgium |
|
London Health Sciences Centre London Ontario N6A 5A5 Canada |
|
Sunnybrook Health Sciences Centre Toronto Ontario M4N 3M5 Canada |
|
Montreal Neurological Institute and Hospital Montréal Quebec H3A 2B4 Canada |
|
Centre Hospitalier Regional Universitaire (CHRU) Montpellier - Hopital Gui De Chauliac Montpellier 34295 France |
|
Groupe Hospitalier Pitie-Salpetriere - Centre d'Investigation Clinique Neurosciences 1422 Paris 75013 France |
|
Charite - Universitaetsmedizin Berlin - Campus Virchow-Klinikum (CVK) - Ambulanz fuer ALS und andere Motoneuronenerkrankungen Berlin 13353 Germany |
|
Medizinische Hochschule Hannover (MHH) - Klinik fuer Neurologie Hannover 30625 Germany |
|
Universitaetsklinikum Ulm - Klinik fuer Neurologie Ulm 89081 Germany |
|
Instituti Clinica Scientifici Maugeri - IRCCS Milano 20138 Italy |
|
Azienda Ospedaliero Universitaria (AUO) di Torino - Citta'della Salute e della Scienza di Torino Torino 10126 Italy |
|
University Medical Center Utrecht Utrecht 3584CX Netherlands |
|
Centrum Medyczne NeuroProtect Warsaw 01-684 Poland |
|
Citi Clinic Warsaw 02-473 Poland |
|
Hospital Universitario Vall d'Hebron ALS Unit. Consultas Externas; Office: 9-10-11 Barcelona 08035 Spain |
|
Hospital Carlos III - Hospital Universitario La Paz, ALS Unit Madrid 28046 Spain |
|
Umeå University Hospital Umeå 90737 Sweden |
|
Kantonsspital St.Gallen, Muskelzentrum/ALS Clinic Saint Gallen 9007 Switzerland |
|
Leonard Wolfson Experimental Neurology Centre London WC1N 3BG United Kingdom |
Location Countries
Country
Belgium
Canada
France
Germany
Italy
Netherlands
Poland
Spain
Sweden
Switzerland
United Kingdom
United States
Verification Date
2019-06-01
Lastchanged Date
N/A
Firstreceived Date
N/A
Responsible Party
Responsible Party Type
Sponsor
Keywords
Condition Browse
Number Of Arms
2
Arm Group
Arm Group Label
Arimoclomol
Arm Group Type
Experimental
Description
Arimoclomol, capsule
Arm Group Label
Placebo
Arm Group Type
Placebo Comparator
Description
Placebo oral capsule (matching to experimental Arm)
Firstreceived Results Date
N/A
Removed Countries
Country
Denmark
Patient Data
Sharing Ipd
Undecided
Firstreceived Results Disposition Date
N/A
Study Design Info
Allocation
Randomized
Intervention Model
Parallel Assignment
Intervention Model Description
placebo-controlled, parallel group
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description
Double-blind
Study First Submitted
March 27, 2018
Study First Submitted Qc
April 6, 2018
Study First Posted
April 9, 2018
Last Update Submitted
June 20, 2019
Last Update Submitted Qc
June 20, 2019
Last Update Posted
June 21, 2019
ClinicalTrials.gov processed this data on December 05, 2019
Conditions
Conditions usually refer to a disease, disorder, syndrome, illness, or injury. In ClinicalTrials.gov,
conditions include any health issue worth studying, such as lifespan, quality of life, health risks, etc.
Interventions
Interventions refer to the drug, vaccine, procedure, device, or other potential treatment being studied.
Interventions can also include less intrusive possibilities such as surveys, education, and interviews.
Study Phase
Most clinical trials are designated as phase 1, 2, 3, or 4, based on the type of questions
that study is seeking to answer:
In Phase 1 (Phase I) clinical trials, researchers test a new drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
In Phase 2 (Phase II) clinical trials, the study drug or treatment is given to a larger group of people (100-300) to see if it is effective and to further evaluate its safety.
In Phase 3 (Phase III) clinical trials, the study drug or treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
In Phase 4 (Phase IV) clinical trials, post marketing studies delineate additional information including the drug's risks, benefits, and optimal use.
These phases are defined by the Food and Drug Administration in the Code of Federal Regulations.
In Phase 1 (Phase I) clinical trials, researchers test a new drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
In Phase 2 (Phase II) clinical trials, the study drug or treatment is given to a larger group of people (100-300) to see if it is effective and to further evaluate its safety.
In Phase 3 (Phase III) clinical trials, the study drug or treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
In Phase 4 (Phase IV) clinical trials, post marketing studies delineate additional information including the drug's risks, benefits, and optimal use.
These phases are defined by the Food and Drug Administration in the Code of Federal Regulations.