The goal of this clinical study is to compare ulixacaltamide and placebo treatment in essential tremor. The main question it aims to answer is:
• Is ulixacaltamide a safe and efficacious treatment for patients with essential tremor?
Participants will be asked to participate in one of two clinical studies where they will be treated with either ulixacaltamide or placebo for a period of up to 12 weeks. After the controlled study completion, they will be eligible to participate in a long-term, open-label safety study (LTSS) and be treated with ulixacaltamide. Participants are eligible to enroll directly into the LTSS if they previously participated in an essential tremor trial, received sponsor invitation after being deemed ineligible for the controlled study, or following enrollment closure of the controlled study.
Has a body mass index (BMI) at Screening of ≥18 kg/m2.
Has a clinical diagnosis of ET confirmed during screening and characterized by postural and action tremor.
If currently receiving medication prescribed for ET, must be on ≤1 medications, on stable dose(s) for at least 1 month prior to Screening, and willing to maintain stable dose(s) throughout the study. As needed (PRN) use of prescribed ET medicines is not allowed with the exception of propranolol PRN. Participants prescribed propranolol PRN are eligible but must discontinue the PRN dose after the first day of screening. Primidone use is not allowed within 2 weeks prior to screening and throughout duration of study.
Women of childbearing potential must undertake pregnancy tests, with a documented negative serum pregnancy test at Screening, negative urine pregnancy tests at Baseline (Day 1) prior to administration of study drug, throughout the intervention periods (as outlined in the SoA) and at the SFU or ED Visit, as appropriate.
Neuropathy, muscle weakness, arthropathy or other musculoskeletal diagnosis of the upper extremity that impairs dexterity or function.
Has a known hypersensitivity to any component of the formulation of ulixacaltamide.
Is unwilling or unable to refrain from episodic use of medication(s)/substance(s) that might interfere with the evaluation of tremor during the study.
Is sporadically using a benzodiazepine, sleep medication or anxiolytic (as further defined in the protocol), that in the judgement of the investigator or sponsor would confound the assessment of tremor.
Parallel
60 mg ulixacaltamide
DrugOnce daily oral treatment with titration
Placebo
DrugOnce daily oral treatment
6
Treatment groupsSee each treatment group below.
Parallel Design: Change from Baseline (CFB) to Week 8 (Day 56) on mADL11 score
Modified Activities of Daily Living 11 (mADL11) is a composite sum of items 1 to 11 of the TETRAS-ADL subscale. The impact to each function is rated on a 5-point Likert scale from 0 to 4. Before calculating the total score, a scoring adjustment is applied to each item score. The modified score is calculated as the sum of all 11 items (with scoring adjustments) and ranges from 0 to 33 where larger values represent increased direct tremor impact to activities of daily living.
Randomized Withdrawal: The proportion of participants that maintain response, as defined by change in mADL11 score, following randomized withdrawal
Modified Activities of Daily Living 11 (mADL11) is a composite sum of items 1 to 11 of the TETRAS-ADL subscale. The impact to each function is rated on a 5-point Likert scale from 0 to 4. Before calculating the total score, a scoring adjustment is applied to each item score. The modified score is calculated as the sum of all 11 items (with scoring adjustments) and ranges from 0 to 33 where larger values represent increased direct tremor impact to activities of daily living. Change in patient response will compare the proportion of patients in the ulixacaltamide and placebo arm who maintain response based on RW baseline established at Week 8 (Day 56) following randomized withdrawal.
Long-term Safety Study: Number of participants with Adverse Events (AE) and their severity.
The number of participants with Adverse Events (AE) will be reported by preferred term
Parallel Design: Rate of Disease improvement as Measured by mADL11
Compare the rate of disease improvement through Day 84 of ulixacaltamide and placebo arms, as measured by the slope of mADL11 over time.
Parallel Design: PGI-C Change at Week 8 (Day 56)
PGI-C assesses the participant's change in condition since baseline. The participant is required to assess their condition relative to Baseline (Pre-dose on Day 1) on a 7-point scale from 1 (very much improved) to 7 (very much worse). In all cases, the assessment should be made independent of whether the participant believes the change is drug-related or not.
Parallel Design: CGI-S Change from Baseline to Week 8 (Day 56)
CGI-S assesses the clinician's impression of the participant's current illness state. The clinician should use his/her total clinical experience with this patient population and rate the current severity of the participant's ET on a 7-point scale from 1 (normal, not at all ill) to 7 (among the most extremely ill patients).
Randomized Withdrawal: Rate of Disease Improvement as Measured by Slope of mADL11 from Day 56 to Day 84
Compare the rate of disease improvement from Day 56 to Day 84 of ulixacaltamide and placebo arms.
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