Arimoclomol in Amyotropic Lateral Sclerosis - Open Label Extension Trial

NCT ID: NCT03836716

Last Updated: 2023-08-24

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE3
• Total Enrollment: 120 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2019-09-19
• Study Completion Date: 2021-07-02

Brief Summary

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.

Detailed Description

The planned duration of the open-label trial was 152 weeks, but the trial was terminated early as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints. Therefore, the actual mean duration of open-label treatment was approximately 28 weeks (range approximately 2 to 71 weeks).

Conditions

Amyotrophic Lateral Sclerosis

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

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

Interventions

Arimoclomol

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

Intervention Type: DRUG

Eligibility Criteria

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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

ZevraDenmark

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Michael Benatar, MD PhD

Role: PRINCIPAL_INVESTIGATOR

University of Miami

Locations

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

UC Irvine Health ALS and Neuromuscular Center

Orange, California, United States

University of Miami

Miami, Florida, United States

Hospital for Special Surgery

New York, New York, United States

Providence Brain & Spine Institute

Portland, Oregon, United States

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

Philadelphia, Pennsylvania, United States

University of Virginia Health System

Charlottesville, Virginia, United States

Catholic University Leuven

Leuven, , Belgium

London Health Sciences Centre

London, Ontario, Canada

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

Montpellier, , France

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

Paris, , France

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

Berlin, , Germany

Medizinische Hochschule Hannover (MHH) - Klinik fuer Neurologie

Hanover, , Germany

Universitaetsklinikum Ulm - Klinik fuer Neurologie

Ulm, , Germany

Instituti Clinica Scientifici Maugeri

Milan, , Italy

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

Torino, , Italy

University Medical Center Utrecht

Utrecht, , Netherlands

Centrum Medyczne NeuroProtect

Warsaw, , Poland

Citi Clinic

Warsaw, , Poland

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

Barcelona, , Spain

Hospital Carlos III - Hospital Universitario La Paz, ALS Unit

Madrid, , Spain

Umeå University Hospital

Umeå, , Sweden

Leonard Wolfson Experimental Neurology Centre

London, , United Kingdom

Countries

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

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

ORARIALS-02

Identifier Type: -

Identifier Source: org_study_id