Summary

for people ages 18 years and up (full criteria)
at Orange, California and other locations
study started
estimated completion

Description

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.

Official Title

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

Keywords

Amyotrophic Lateral Sclerosis Arimoclomol ALS Motor Neuron Disease Sclerosis

Eligibility

You can join if…

Open to people ages 18 years and up

  • 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)
  • On treatment with IMP defined as last dose within 2 weeks of the last visit of the blinded ORARIALS-01 trial.
  • Able and willing to travel to the site for the Baseline visit and in the investigator's opinion is expected to be able to attend the clinic for the visit at Week 4.

You CAN'T join if...

  • 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
  • Significant protocol deviation in the blinded ORARIALS-01 trial based on the Investigator's judgement
  • 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:
  • Clinically significant renal or hepatic disease, as indicated by clinical laboratory assessment (results ≥ 3 times the upper limit of normal [ULN] for aspartate aminotransferase, alanine aminotransferase, and lactate dehydrogenase, bilirubin ≥ 2 times the ULN, or creatinine ≥ 1.5 times the ULN).
  • Any new condition or worsening of existing condition which, in the opinion of the investigator, would make the subject unsuitable for enrolment or could interfere with the subject participating in or completing the trial
  • 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

Locations

  • UC Irvine Health ALS and Neuromuscular Center
    Orange California 92868 United States
  • 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

Details

Status
accepting new patients by invitation only
Start Date
Completion Date
(estimated)
Sponsor
Orphazyme
ID
NCT03836716
Phase
Phase 3
Study Type
Interventional
Last Updated