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.
The planned duration of the open-label trial was 152 weeks, but the trial was terminated early as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints. Therefore, the actual mean duration of open-label treatment was approximately 28 weeks (range approximately 2 to 71 weeks).
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