Arimoclomol in Amyotropic Lateral Sclerosis

Study Description

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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)

Condition or disease	Intervention/treatment	Phase
Amyotrophic Lateral Sclerosis	Drug: Arimoclomol; Drug: Placebo oral capsule	Phase 3

Detailed Description:

Screening of up to 4 weeks Treatment of up to 76 weeks

Study Design

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Study Type :	Interventional (Clinical Trial)
Estimated Enrollment :	231 participants
Allocation:	Randomized
Intervention Model:	Parallel Assignment
Intervention Model Description:	placebo-controlled, parallel group
Masking:	Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description:	Double-blind
Primary Purpose:	Treatment
Official Title:	A Phase 3, Randomised, Placebo-Controlled Trial of Arimoclomol in Amyotropic Lateral Sclerosis
Actual Study Start Date :	July 31, 2018
Estimated Primary Completion Date :	December 2020
Estimated Study Completion Date :	January 2021

Arms and Interventions

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Arm	Intervention/treatment
Experimental: Arimoclomol; Arimoclomol, capsule	Drug: Arimoclomol; Active treatment; Other Name: Arimoclomol citrate
Placebo Comparator: Placebo; Placebo oral capsule (matching to experimental Arm)	Drug: Placebo oral capsule; Matching placebo capsule; Other Name: Placebo

Outcome Measures

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Primary Outcome Measures :

1. Combined Assessment of Function and Survival (CAFS) [ Time Frame: over 76 Weeks ]
   A nonparametric rank analysis of covariance considering Time to Permanent assisted ventilation, tracheostomy or death or ALSFRS-R score (12 functions on a 5-point ordinal rating scale with maximum score of 48)

Secondary Outcome Measures :

1. Time to permanent assisted ventilation (PAV)/tracheostomy/death [ Time Frame: over 76 weeks ]
   time from baseline to event where PAV is defined as the first of 7 consecutive days on which PAV was used for >22 hours/day.
2. Change from Baseline to Week 76 (or end-of-trial) in the revised ALS Functional Rating Scale (ALSFRS-R) [ Time Frame: Week 76 (or end of trial) ]
   Change in functional rating scale over time as recorded by the revised ALS functional rating scale (ALSFRS-R), a scale where 12 functions are rated on a 4-point scale giving a maximum score of 48
3. Change from Baseline to Week 76 (or end-of-trial) in SVC [ Time Frame: Week 76 (or end of trial) ]
   Change in Slow Vital Capacity (a measure of breathing function) over time

Eligibility Criteria

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Ages Eligible for Study:	18 Years and older (Adult, Older Adult)
Sexes Eligible for Study:	All
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

• Subject meets revised El Escorial criteria for clinically possible, clinically probable / Clinically probable ALS laboratory-supported or clinically definite ALS, or familial ALS
• 18 months or less since first appearance of weakness
• ALSFRS-R equal to or above 35 and erect SVC% predicted equal to or above 80% at screening

Exclusion Criteria:

• Tracheostomy or use of non-invasive ventilation for more than 2 hours during waking hours at the time of screening or 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

Contacts and Locations

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Contacts

Contact: Requests at Orphazyme A/S	+45 39 17 82 72	Request@orphazyme.com

Locations

United States, Arizona
St. Joseph's Hospital and Medical Center (SJHMC) - Barrow Neurological Institute (BNI) - The Gregory W. Fulton ALS and Neuromuscular Disease Center	Recruiting
Phoenix, Arizona, United States, 85013
Contact: Nicole Turcotte 602-406-4775 Nicole.Turcotte@DignityHealth.org
Principal Investigator: Shafeeq Ladha, MD
HonorHealth Neurology	Recruiting
Phoenix, Arizona, United States, 85018
Contact: Mackenzie Steinbach 480-882-4916 Mackenzie.Steinbach@honorhealth.com
Principal Investigator: Todd Levine, MD
United States, California
UC Irvine Health ALS and Neuromuscular Center	Recruiting
Orange, California, United States, 92868
Contact: Ivonne Turner 714-456-7760 imturner@uci.edu
Contact: Veronica Martin +1 714-456-7760 vero@uci.edu
Principal Investigator: Namita Goyal, MD
United States, Florida
University of Miami	Recruiting
Miami, Florida, United States, 33136
Contact: Maria Elena Paredes 305-243-7336 mep132@med.miami.edu
Principal Investigator: Michael Benatar, MD, PhD
United States, Kansas
University of Kansas Medical Center (KUMC) - Landon Center on Aging	Recruiting
Kansas City, Kansas, United States, 66160
Contact: Alyssa Lackey 913-945-9942 alackey@kumc.edu
Principal Investigator: Duaa Jabari, MD
United States, New York
Hospital for Special Surgery	Recruiting
New York, New York, United States, 10021
Contact: William Mark Richardson 646-797-8657 richardsonw@HSS.EDU
Principal Investigator: Dale J. Lange, MD
United States, Oregon
Providence Brain & Spine Institute	Recruiting
Portland, Oregon, United States, 97213
Contact: Arlena Cummings 503-962-1171 arlena.cummings@providence.org
Principal Investigator: Kimberly L Goslin, MD, PhD
United States, Virginia
University of Virginia Health System	Recruiting
Charlottesville, Virginia, United States, 22908
Contact: Mary Wagoner 434-924-5541 MIW9B@hscmail.mcc.virginia.edu
Principal Investigator: Ted Burns, MD
Canada, Quebec
Montreal Neurological Institute and Hospital	Active, not recruiting
Montréal, Quebec, Canada, H3A 2B4
Spain
Hospital Universitario Vall d'Hebron ALS Unit. Consultas Externas; Office: 9-10-11	Recruiting
Barcelona, Spain, 08035
Contact: Ana Canovas 0034 932746000 ext 2780 cs.ana.belen@gmail.com
Principal Investigator: Josep Gamez, MD
Hospital Carlos III - Hospital Universitario La Paz, ALS Unit	Active, not recruiting
Madrid, Spain, 28046
Sweden
Umeå University Hospital	Recruiting
Umeå, Sweden, 90737
Contact: Erica Stenberg +46 72548 74 10 erica.stenberg@umu.se
Principal Investigator: Peter Andersen, MD

Sponsors and Collaborators

Orphazyme

Investigators

Principal Investigator:	Michael Benatar, MD PhD	University of Miami

More Information

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Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Responsible Party:	Orphazyme
ClinicalTrials.gov Identifier:	NCT03491462 History of Changes
Other Study ID Numbers:	ORARIALS-01
First Posted:	April 9, 2018
Last Update Posted:	November 26, 2018
Last Verified:	November 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD:	Undecided

Studies a U.S. FDA-regulated Drug Product:		Yes
Studies a U.S. FDA-regulated Device Product:		No

Keywords provided by Orphazyme:

arimoclomol; ALS

Additional relevant MeSH terms:

Amyotrophic Lateral Sclerosis; Sclerosis; Motor Neuron Disease; Pathologic Processes; Neurodegenerative Diseases; Nervous System Diseases	Neuromuscular Diseases; Spinal Cord Diseases; Central Nervous System Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Metabolic Diseases