Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
ulixacaltamide · 1 trial · 1 indication
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.
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.
The number of participants with Adverse Events (AE) will be reported by preferred term
| Arm | Type | Description |
|---|---|---|
| Parallel Design: ulixacaltamide arm | EXPERIMENTAL | Double-blind Part: Oral dosing, once daily in the morning with titration over 14 days to 60 mg ulixacaltamide: 7 days of 20 mg, 7 days of 40 mg, 70 days of 60 mg |
| Parallel Design: placebo arm | PLACEBO_COMPARATOR | Double-blind Part: Oral dosing, once daily in the morning: 84 days of placebo |
| Randomized Withdrawal | EXPERIMENTAL | Double-blind Part: Oral dosing, once daily in the morning with titration over 14 days to 60 mg ulixacaltamide: 7 days of 20 mg, 7 days of 40 mg, 42 days of 60 mg Randomized Withdrawal Part: Following double-blind part: 1:1 randomization to placebo or 60 mg ulixacaltamide for 28 days |
| Long-term Safety Study: Essential1 rollovers | EXPERIMENTAL | Open-label Part: Oral dosing, once daily in the morning up to 3 years 60 mg ulixacaltamide |
| Long-term Safety Study: Parallel Design and Randomized Withdrawal rollovers | EXPERIMENTAL | Open-label Part for patients previously on placebo: Oral dosing, once daily in the morning with titration over 14 days to 60 mg ulixacaltamide: 7 days of 20 mg, 7 days of 40 mg, up to 3 years 60 mg ulixacaltamide Open-label Part for patients previously on 60 mg ulixacaltamide: Oral dosing, once daily in the morning up to 3 years 60 mg ulixacaltamide |
| Long-term Safety Study: Direct Enrollment to LTSS Study | EXPERIMENTAL | Oral dosing, once daily in the morning with titration over 14 days to 60 mg ulixacaltamide: 7 days of 20 mg, 7 days of 40 mg, up to 3 years 60 mg ulixacaltamide |
| Name | Type | Description |
|---|---|---|
| 60 mg ulixacaltamide | DRUG | Once daily oral treatment with titration |
| Placebo | DRUG | Once daily oral treatment |
Inclusion Criteria for Study 1 PD and Study 2 RW: 1. Has a body mass index (BMI) at Screening of ≥18 kg/m2. 2. Has a clinical diagnosis of ET confirmed during screening and characterized by postural and action tremor. 3. If currently receiving medication prescribed for ET, must be on ≤1 medications...
Ulixacaltamide is an investigational small molecule being developed for the treatment of Essential Tremor, a neurological condition characterized by involuntary shaking. It is currently in Phase 3 clinical development and has not yet been approved by the FDA. The drug is being studied in a large, placebo-controlled trial to evaluate its safety and efficacy.
Ulixacaltamide is being developed by Praxis Precision Medicines, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol PRAX. The company is conducting a Phase 3 clinical trial of the drug for Essential Tremor. Praxis has received Breakthrough Therapy designation from the FDA for ulixacaltamide, which is intended to expedite its development and review.
Ulixacaltamide is currently in Phase 3 clinical development for Essential Tremor. It is an investigational drug, meaning it has not yet been approved by the FDA for commercial use. The ongoing Phase 3 trial is active but not recruiting participants, and the drug has received Breakthrough Therapy designation from the FDA.
Ulixacaltamide is being evaluated in a Phase 3 clinical trial with the identifier NCT06087276, titled 'Essential 3 - Decentralized, Phase 3 Study Evaluating the Safety and Efficacy of Ulixacaltamide in Essential Tremor (ET)'. This randomized, double-blind, placebo-controlled trial is enrolling approximately 1,000 participants aged 18 years and older in the United States.
No, ulixacaltamide is not FDA approved. It is an investigational drug currently in Phase 3 clinical development for Essential Tremor. The FDA has granted it Breakthrough Therapy designation, which is a status given to drugs that may offer substantial improvement over existing therapies for serious conditions. Approval will depend on the results of ongoing clinical trials.