Arimoclomol in Amyotropic Lateral Sclerosis - Open Label Extension Trial

• Register: ClinicalTrials.gov
• Last refreshed on: 4 September 2023
• Main ID: NCT03836716
• Date of registration: 07/02/2019
• Prospective Registration: Yes
• Primary sponsor: ZevraDenmark
• Public title: Arimoclomol in Amyotropic Lateral Sclerosis - Open Label Extension Trial
• Scientific 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
• Date of first enrolment: September 19, 2019
• Target sample size: 120
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/ct2/show/NCT03836716
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Belgium, Canada, France, Germany, Italy, Netherlands, Poland, Spain, Sweden, Switzerland, United Kingdom, United States

Contacts

• Name: Michael Benatar, MD PhD
• Affiliation: University of Miami

Key inclusion & exclusion 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



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Amyotrophic Lateral Sclerosis

Intervention(s)

Drug: Arimoclomol

Primary Outcome(s)

Mean and Change From Baseline in Clinical Safety Laboratory Tests - Cystatin C [Time Frame: Week 76]

Mean and Change From Baseline in Clinical Safety Laboratory Tests - Hematology (2) [Time Frame: Week 76]

Mean and Change From Baseline in Clinical Safety Laboratory Tests - Hemoglobin [Time Frame: Week 76]

Mean and Change From Baseline in Vital Signs - Blood Pressure [Time Frame: Week 76]

Mean and Change From Baseline in Vital Signs - Respiratory Rate [Time Frame: Week 76]

Mean and Change From Baseline in Vital Signs - Pulse Rate [Time Frame: Week 76]

Mean and Change From Baseline in Clinical Safety Laboratory Tests - Clinical Chemistry (1) [Time Frame: Week 76]

Columbia-Suicide Severity Rating Scale (C-SSRS) Over the Open-label Treatment Period [Time Frame: From Day 1 in ORARIALS-02 to Early Termination, an average of approximately 28 weeks]

Mean and Change From Baseline in Clinical Safety Laboratory Tests - Clinical Chemistry (2) [Time Frame: Week 76]

Mean and Change From Baseline in Clinical Safety Laboratory Tests - Erythrocytes [Time Frame: Week 76]

Number of Participants With Treatment-emergent Adverse Events (TEAEs) Over the Open-label Treatment Period [Time Frame: From Day 1 in ORARIALS-02 to Early Termination, an average of approximately 28 weeks]

Mean and Change From Baseline in Vital Signs - Temperature [Time Frame: Week 76]

Mean and Change From Baseline in Clinical Safety Laboratory Tests - Albumin and Protein [Time Frame: Week 76]

Mean and Change From Baseline in Clinical Safety Laboratory Tests - Hematocrit [Time Frame: Week 76]

Number of Participants With Potentially Clinically Significant Abnormalities in Clinical Safety Laboratory Tests and Vital Signs Over the Open-label Treatment Period [Time Frame: From Day 1 in ORARIALS-02 to Early Termination, an average of approximately 28 weeks]

Mean and Change From Baseline in Clinical Safety Laboratory Tests - Clinical Chemistry (3) [Time Frame: Week 76]

Mean and Change From Baseline in Clinical Safety Laboratory Tests - Hematology (1) [Time Frame: Week 76]

Secondary Outcome(s)

Change in Percentage (%) Predicted Slow Vital Capacity (SVC) From Baseline to Week 76 (for Subjects Who Did Not Meet the Survival Endpoint in the ORARIALS-01 Trial) [Time Frame: 76 weeks]

Change in ALS Functional Rating Scale - Revised (ALSFRS-R) From Baseline to Week 76 [Time Frame: Week 76]

Secondary ID(s)

ORARIALS-02

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 24/08/2023
• URL: https://clinicaltrials.gov/ct2/show/results/NCT03836716