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

Study Purpose:

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.

Study Status:

Not recruiting


Amyotrophic Lateral Sclerosis

Study Type:


Type of Intervention:


Intervention Name:





Phase 3

Study Chair(s)/Principal Investigator(s):

Michael Benatar, MD PhD, University of Miami ID:


Neals Affiliated?


Coordinating Center Contact Information

Study Sponsor:


Estimated Enrollment:


Estimated Study Start Date:

09 / 19 / 2019

Estimated Study Completion Date:

07 / 02 / 2021

Posting Last Modified Date:

07 / 12 / 2021

Date Study Added to

02 / 11 / 2019

Minimum Age:

18 Years

Maximum Age:


Can participants use Riluzole?


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, or in the circumstance that the subject is incompetent, informed consent/assent is provided in accordance with local regulation and/or procedures

- Subject has completed the ORARIALS-01 trial (i.e., met one of the surrogate survival endpoints of tracheostomy or PAV or has completed the 76 weeks randomized treatment period)

- Subject completed ORARIALS-01 while on treatment, where on treatment is defined as having taken the last dose of IMP within 2 weeks of the End of Trial visit (whether at week 76 or prior)

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

- Women who are lactating or pregnant, or men or women unwilling to use a highly effective method of birth control if not surgically sterile (defined as bilateral tubal ligation, bilateral oophorectomy, or hysterectomy for women; vasectomy for men) for female participants until 4 weeks after last dose and for male participants until 3 months after last dose. Pre-menopausal women must have a negative pregnancy test prior to dosing with trial medication.

- Any of the following medically significant conditions:

1. Clinically significant renal or hepatic disease OR clinical laboratory assessment (results ≥ 3 times the upper limit of normal [ULN] for aspartate aminotransferase and/or alanine aminotransferase, bilirubin ≥ 2 times the ULN, or creatinine ≥ 1.5 times the ULN).

2. Any new condition or worsening of existing condition which, in the opinion of the investigator, would put the subject at undue risk.

- Any serious adverse event or moderate/severe adverse event from the ORARIALS-01 trial which is ongoing at the time of transitioning to ORARIALS-02 and assessed as probably related to IMP

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

UC Irvine Health ALS and Neuromuscular Center

Orange, California 92868
United States

University of Miami

Miami, Florida 33136
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 Virginia Health System

Charlottesville, Virginia 22908
United States

Catholic University Leuven


London Health Sciences Centre

London, Ontario N6A 5A5

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

Montpellier 34295

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

Paris 75013

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

Berlin 13353

Medizinische Hochschule Hannover (MHH) - Klinik fuer Neurologie

Hannover 30625

Universitaetsklinikum Ulm - Klinik fuer Neurologie

Ulm 89081

Instituti Clinica Scientifici Maugeri

Milano 20138

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

Torino 10126

University Medical Center Utrecht

Utrecht 3584CX

Centrum Medyczne NeuroProtect

Warsaw 01-684

Citi Clinic

Warsaw 02-473

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

Barcelona 08035

Hospital Carlos III - Hospital Universitario La Paz, ALS Unit

Madrid 28046

Umeå University Hospital

Umeå 90737

Leonard Wolfson Experimental Neurology Centre

London WC1N 3BG
United Kingdom