Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
20 and PRAX-944 · 1 trial · 1 indication
The TETRAS upper limb score is calculated as the sum of the 6 separate upper-limb maneuvers assessed by the clinician: forward-outstretched postural tremor \[right\], forward outstretched postural tremor \[left\], lateral wing-beating postural tremor \[right\], lateral wing-beating postural tremor \[left\], kinetic tremor \[right\], kinetic tremor \[left\], each on a 0 (no tremor) to 4 (maximum tremor) severity scale in 0.5-point increments. TETRAS Part 4 score for both upper limbs ranged from 0 to 24, higher scores indicated more severe tremor. A negative change from baseline indicated less tremor. Baseline is defined as the last non-missing value collected prior to receiving first dose of study drug. Change from baseline will be defined as assessment value minus baseline value.
An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of a study intervention, whether or not considered related to the study intervention. Incidence and Severity of Adverse Events have been reported.
Vital signs parameters included body temperature, pulse rate, respiratory rate, blood pressure (systolic and diastolic), clinical laboratory measures (chemistry, hematology, urinalysis, and coagulation), electrocardiogram parameters (heart rate, PR, QRS, QT, and corrected QT intervals). Any change in vital sign abnormalities that was clinically significant in the medical and scientific judgment of the investigator and not related to an underlying disease was reported as an unsolicited adverse event (AE). Any clinically significant abnormality associated with underlying disease does not require reporting as an (S)AE unless judged by the investigator to be more severe than expected for the participant's condition.
The C-SSRS is described as a scale developed at Columbia University that has 2-6 questions each in categories of Suicidal Ideation, Intensity of Ideation, Suicidal Behavior, and Actual Attempts. Four constructs were measured. Severity of Suicidal ideation is rated on a 5-point ordinal scale. Intensity of ideation is comprised of 5 items (frequency, duration, controllability, deterrents, and reason for ideation), each rated on a 5-point ordinal scale. Suicidal behavior is rated on a nominal scale that includes actual, aborted, and interrupted attempts; preparatory behavior; and non-suicidal self-injurious behavior. Lethality, assesses actual attempts; actual lethality is rated on a 6-point ordinal scale, and if actual lethality is 0, potential lethality of attempts is rated on a 3-point ordinal scale.The higher the C-SSRS score, the higher the suicide risk (ie. worse outcome).
| Arm | Type | Description |
|---|---|---|
| Part A: Open-label 20 and 40 mg PRAX-944 | EXPERIMENTAL | Once daily, oral dosing with 7 days of 20 mg and 7 days of 40 mg |
| Part B: Open-label titration PRAX-944 (120 mg) followed by blinded PRAX-944 | EXPERIMENTAL | Once daily, oral dosing with titration to 120 mg: 3 days of 20 mg, 4 days of 40 mg, 7 days of 60 mg, 7 days of 80 mg, 7 days of 100 mg, 28 days of 120 mg |
| Part B: Open-label titration PRAX-944 (120 mg) followed by blinded placebo | ACTIVE_COMPARATOR | Once daily, oral dosing with titration to 120 mg: 3 days of 20 mg, 4 days of 40 mg, 7 days of 60 mg, 7 days of 80 mg, 7 days of 100 mg, 14 days of 120 mg, 14 days placebo |
| Name | Type | Description |
|---|---|---|
| Part A: 20 and 40 mg PRAX-944 | DRUG | Once daily oral treatment |
| Part B: 120 mg PRAX-944 | DRUG | Once daily oral treatment with titration |
| Part B: 120 mg PRAX-944 and Placebo | DRUG | Once daily oral treatment with titration followed by placebo |
Inclusion Criteria: 1. Clinical diagnosis of ET consistent with Movement Disorders Society Criteria, a duration of ET of at least 3 years and with onset before the age of 65 2. TETRAS upper limb score (ie, sum of bilateral upper limb items 4a, 4b, and 4c) of ≥10 as rated by the Investigator at Scre...
PRAX-944 is being developed for the treatment of essential tremor, a neurological movement disorder. It is an investigational small molecule therapy from Praxis Precision Medicines. The clinical program has focused on evaluating PRAX-944 in adults with essential tremor, with a Phase 2 trial completed.
PRAX-944 is being developed by Praxis Precision Medicines, Inc., a biopharmaceutical company that trades on the Nasdaq under the ticker symbol PRAX. The company focuses on therapies for neurological and psychiatric disorders.
PRAX-944 has completed a Phase 2 clinical trial in essential tremor. It is an investigational drug and has not been approved by the FDA. The Phase 2 study, NCT05021978, was completed and enrolled 24 participants.
PRAX-944 has been studied in a Phase 2 clinical trial, NCT05021978, titled A Clinical Trial of PRAX-944 in Participants With Essential Tremor. The trial was completed, enrolled 24 participants, and was conducted in the United States, Australia, and New Zealand.