Arimoclomol in Amyotropic Lateral Sclerosis

NCT ID: NCT03491462

Last Updated: 2023-08-24

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

245 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-07-31

Study Completion Date

2020-12-18

Brief Summary

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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

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Screening up to 4 weeks Treatment up to 76 weeks

Conditions

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Amyotrophic Lateral Sclerosis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Placebo-controlled, parallel group
Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors
Double-blind

Study Groups

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Arimoclomol

248 mg arimoclomol base (equivalent to 400 mg arimoclomol citrate) 3 times daily

Group Type EXPERIMENTAL

Arimoclomol

Intervention Type DRUG

2 capsules (2 x 124 mg arimoclomol base; equivalent to 2 x 200 mg arimoclomol citrate) taken 3 times daily

Placebo

248 mg matching placebo 3 times daily

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

2 matched placebo capsules taken 3 times daily

Interventions

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Arimoclomol

2 capsules (2 x 124 mg arimoclomol base; equivalent to 2 x 200 mg arimoclomol citrate) taken 3 times daily

Intervention Type DRUG

Placebo

2 matched placebo capsules taken 3 times daily

Intervention Type DRUG

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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ZevraDenmark

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Michael Benatar, MD PhD

Role: PRINCIPAL_INVESTIGATOR

University of Miami

Locations

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St. Joseph's Hospital and Medical Center (SJHMC) - Barrow Neurological Institute (BNI) - The Gregory W. Fulton ALS and Neuromuscular Disease Center

Phoenix, Arizona, United States

Site Status

HonorHealth Neurology

Phoenix, Arizona, United States

Site Status

UC Irvine Health ALS and Neuromuscular Center

Orange, California, United States

Site Status

University of Miami

Miami, Florida, United States

Site Status

University of Kansas Medical Center (KUMC) - Landon Center on Aging

Kansas City, Kansas, United States

Site Status

Hospital for Special Surgery

New York, New York, United States

Site Status

Providence Brain & Spine Institute

Portland, Oregon, United States

Site Status

University of Pensylvania, Perelman Center for Advanced Medicine - Penn Neuroscience Center

Philadelphia, Pennsylvania, United States

Site Status

University of Texas Southwestern Medical Center

Dallas, Texas, United States

Site Status

University of Virginia Health System

Charlottesville, Virginia, United States

Site Status

Catholic University Leuven

Leuven, , Belgium

Site Status

London Health Sciences Centre

London, Ontario, Canada

Site Status

Sunnybrook Health Sciences Centre

Toronto, Ontario, Canada

Site Status

Montreal Neurological Institute and Hospital

Montreal, Quebec, Canada

Site Status

Centre Hospitalier Regional Universitaire (CHRU) Montpellier - Hopital Gui De Chauliac

Montpellier, , France

Site Status

Groupe Hospitalier Pitie-Salpetriere - Centre d'Investigation Clinique Neurosciences 1422

Paris, , France

Site Status

Charite - Universitaetsmedizin Berlin - Campus Virchow-Klinikum (CVK) - Ambulanz fuer ALS und andere Motoneuronenerkrankungen

Berlin, , Germany

Site Status

Medizinische Hochschule Hannover (MHH) - Klinik fuer Neurologie

Hanover, , Germany

Site Status

Universitaetsklinikum Ulm - Klinik fuer Neurologie

Ulm, , Germany

Site Status

Instituti Clinica Scientifici Maugeri - IRCCS

Milan, , Italy

Site Status

Azienda Ospedaliero Universitaria (AUO) di Torino - Citta'della Salute e della Scienza di Torino

Torino, , Italy

Site Status

University Medical Center Utrecht

Utrecht, , Netherlands

Site Status

Centrum Medyczne NeuroProtect

Warsaw, , Poland

Site Status

Citi Clinic

Warsaw, , Poland

Site Status

Hospital Universitario Vall d'Hebron ALS Unit. Consultas Externas; Office: 9-10-11

Barcelona, , Spain

Site Status

Hospital Carlos III - Hospital Universitario La Paz, ALS Unit

Madrid, , Spain

Site Status

Umeå University Hospital

Umeå, , Sweden

Site Status

Kantonsspital St.Gallen, Muskelzentrum/ALS Clinic

Sankt Gallen, , Switzerland

Site Status

Leonard Wolfson Experimental Neurology Centre

London, , United Kingdom

Site Status

Countries

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United States Belgium Canada France Germany Italy Netherlands Poland Spain Sweden Switzerland United Kingdom

References

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Benatar M, Hansen T, Rom D, Geist MA, Blaettler T, Camu W, Kuzma-Kozakiewicz M, van den Berg LH, Morales RJ, Chio A, Andersen PM, Pradat PF, Lange D, Van Damme P, Mora G, Grudniak M, Elliott M, Petri S, Olney N, Ladha S, Goyal NA, Meyer T, Hanna MG, Quinn C, Genge A, Zinman L, Jabari D, Shoesmith C, Ludolph AC, Neuwirth C, Nations S, Shefner JM, Turner MR, Wuu J, Bennett R, Dang H, Sundgreen C; ORARIALS-01 trial team. Safety and efficacy of arimoclomol in patients with early amyotrophic lateral sclerosis (ORARIALS-01): a randomised, double-blind, placebo-controlled, multicentre, phase 3 trial. Lancet Neurol. 2024 Jul;23(7):687-699. doi: 10.1016/S1474-4422(24)00134-0. Epub 2024 May 20.

Reference Type DERIVED
PMID: 38782015 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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ORARIALS-01

Identifier Type: -

Identifier Source: org_study_id

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