Clinical trial NCT03491462: Arimoclomol in Amyotropic Lateral Sclerosis
Official title: A Phase 3, Randomised, Placebo-Controlled Trial of Arimoclomol in Amyotropic Lateral Sclerosis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Amyotrophic Lateral Sclerosis
Keywords: Arimoclomol
ALS
Lead sponsor: ZevraDenmark
Sponsor class: INDUSTRY
First posted: 2018-04-09
Enrollment: 245
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Placebo-controlled, parallel group
Primary purpose: TREATMENT

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 up to 4 weeks Treatment up to 76 weeks

Eligibility criteria: Inclusion Criteria:

* Subject meets revised El Escorial criteria for clinically possible, clinically probable / clinically probable ALS laboratory-supported, clinically definite ALS or clinically definite familial ALS laboratory-supported
* 18 months or less since first appearance of weakness (e.g. limb weakness, dysarthria, dysphagia, shortness of breath)
* ALS Functional Rating Scale-Revised (ALSFRS-R) equal to or above 35 and erect (seated) Slow Vital Capacity (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 and 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

Primary outcomes:
measure: Combined Assessment of Function and Survival (CAFS)
timeFrame: Over 76 Weeks
Secondary outcomes:
measure: Time to Permanent Assisted Ventilation (PAV) / Tracheostomy / Death
timeFrame: Over 76 weeks
measure: Change From Baseline to Week 76 (or End-of-trial) in the Revised ALS Functional Rating Scale (ALSFRS-R)
timeFrame: Week 76 (or end of trial)
measure: Change From Baseline to Week 76 (or End-of-trial) in Percent (%) Predicted Slow Vital Capacity (SVC)
timeFrame: Week 76 (or end of trial)
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: St. Joseph's Hospital and Medical Center (SJHMC) - Barrow Neurological Institute (BNI) - The Gregory W. Fulton ALS and Neuromuscular Disease Center
state: Arizona
city: Phoenix
country: United States
facility: HonorHealth Neurology
state: Arizona
city: Orange
country: United States
facility: UC Irvine Health ALS and Neuromuscular Center
state: California
city: Miami
country: United States
facility: University of Miami
state: Florida
city: Kansas City
country: United States
facility: University of Kansas Medical Center (KUMC) - Landon Center on Aging
state: Kansas
city: New York
country: United States
facility: Hospital for Special Surgery
state: New York
city: Portland
country: United States
facility: Providence Brain & Spine Institute
state: Oregon
city: Philadelphia
country: United States
facility: University of Pensylvania, Perelman Center for Advanced Medicine - Penn Neuroscience Center
state: Pennsylvania
city: Dallas
country: United States
facility: University of Texas Southwestern Medical Center
state: Texas
city: Charlottesville
country: United States
facility: University of Virginia Health System
state: Virginia
city: Leuven
country: Belgium
facility: Catholic University Leuven
city: London
country: Canada
facility: London Health Sciences Centre
state: Ontario
city: Toronto
country: Canada
facility: Sunnybrook Health Sciences Centre
state: Ontario
city: Montréal
country: Canada
facility: Montreal Neurological Institute and Hospital
state: Quebec
city: Montpellier
country: France
facility: Centre Hospitalier Regional Universitaire (CHRU) Montpellier - Hopital Gui De Chauliac
city: Paris
country: France
facility: Groupe Hospitalier Pitie-Salpetriere - Centre d'Investigation Clinique Neurosciences 1422
city: Berlin
country: Germany
facility: Charite - Universitaetsmedizin Berlin - Campus Virchow-Klinikum (CVK) - Ambulanz fuer ALS und andere Motoneuronenerkrankungen
city: Hannover
country: Germany
facility: Medizinische Hochschule Hannover (MHH) - Klinik fuer Neurologie
city: Ulm
country: Germany
facility: Universitaetsklinikum Ulm - Klinik fuer Neurologie
city: Milano
country: Italy
facility: Instituti Clinica Scientifici Maugeri - IRCCS
city: Torino
country: Italy
facility: Azienda Ospedaliero Universitaria (AUO) di Torino - Citta'della Salute e della Scienza di Torino
city: Utrecht
country: Netherlands
facility: University Medical Center Utrecht
city: Warsaw
country: Poland
facility: Centrum Medyczne NeuroProtect
city: Warsaw
country: Poland
facility: Citi Clinic
city: Barcelona
country: Spain
facility: Hospital Universitario Vall d'Hebron ALS Unit. Consultas Externas; Office: 9-10-11
city: Madrid
country: Spain
facility: Hospital Carlos III - Hospital Universitario La Paz, ALS Unit
city: Umeå
country: Sweden
facility: Umeå University Hospital
city: Saint Gallen
country: Switzerland
facility: Kantonsspital St.Gallen, Muskelzentrum/ALS Clinic
city: London
country: United Kingdom
facility: Leonard Wolfson Experimental Neurology Centre
Study officials:
affiliation: University of Miami
name: Michael Benatar, MD PhD
role: PRINCIPAL_INVESTIGATOR
References: