Arimoclomol in Amyotropic Lateral Sclerosis - Open Label Extension Trial
Condition: Amyotrophic Lateral Sclerosis
MeSH terms: Motor Neuron Disease; Amyotrophic Lateral Sclerosis; Sclerosis
Intervention: Arimoclomol
Intervention description: 2 capsules (2 x 124 mg arimoclomol base; equivalent to 2 x 200 mg arimoclomol citrate) taken 3 times daily
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Intervention model description: Non-randomized open label
Masking: None (Open Label)
Enrollment: 120.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: Arimoclomol; ALS

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.
    

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