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
Disease:
Amyotrophic Lateral Sclerosis
Study Type:
Interventional
Type of Intervention:
Drug
Intervention Name:
Arimoclomol
Placebo:
No
Phase:
Phase 3
Study Chair(s)/Principal Investigator(s):
Michael Benatar, MD PhD, University of Miami
Clinicaltrials.gov ID:
Neals Affiliated?
No
Coordinating Center Contact Information
Study Sponsor:
KemPharm Denmark A/S
Estimated Enrollment:
120
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 neals.org:
02 / 11 / 2019
Minimum Age:
18 Years
Maximum Age:
N/A
Can participants use Riluzole?
Yes
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
Leuven
Belgium
London Health Sciences Centre
London, Ontario
N6A 5A5
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
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
Leonard Wolfson Experimental Neurology Centre
London WC1N 3BG
United Kingdom