A Phase 3, Randomised, Placebo-Controlled Trial of Arimoclomol in Amyotrophic Lateral Sclerosis - ORARIALS-01

Orphazyme A/S0 sites19 target enrollmentTBD

Overview

Trial is onging in other countries

Registry

who.int

Start Date

TBD

End Date

December 17, 2020

Last Updated

2 years ago

Study Type

Interventional

Sponsor

•1\. Capable of\- and willing to\- provide written informed consent and comply with
•trial procedures.
•2\. Subject is male or female \*18 years of age.
•3\. 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.
•4\. 18 months or less since first appearance of weakness (e.g. limb weakness,
•dysarthria, dysphagia, shortness of breath).
•5\. ALSFRS\-R \*35 and erect (seated) SVC % predicted \* 70% at Screening.
•6\. Able and willing to travel to the site, and in the investigator\*s opinion is

•1\. Tracheostomy or use of non\-invasive ventilation for more than 2 hours during
•waking hours at the time of Screening and Baseline visits.
•2\. Pregnant or breast\-feeding.
•3\. Current or anticipated use of diaphragmatic pacing during the trial.
•4\. Exposure to any investigational treatment within 4 weeks or \<5 half\-lives of
•the Screening visit, whichever is longest and/or advanced therapy medicinal
•product (ATMP), i.e. treatments based on genes, cells or tissues and/or
•participated in any prior ALS clinical trial receiving active drug treatment
•(with the exception described in exclusion criterion 5\).
•5\. Treatment with edaravone within 4 weeks of the Baseline visit. However, up