Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
PRAX-944 · 1 trial · 1 indication
The modified ADL is a composite sum of items 1 to 11 of the TETRAS-ADL subscale and items 6 and 7 on the TETRAS-PS. 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 ADL score is calculated as the sum of all 13 items (with scoring adjustments) and ranges from 0 to 42 where larger values represent increased direct tremor impact to activities of daily living.
| Arm | Type | Description |
|---|---|---|
| Regimen 1 | EXPERIMENTAL | Double-blind Part: Oral dosing, once daily in the morning with titration over 56 days to 100 mg PRAX-944: 7 days of 5 mg, 7 days of 10 mg, 7 days of 20 mg, 7 days of 40 mg, 7 days of 60 mg, 7 days of 80 mg, 14 days of 100 mg Extension Part: Double-blind Lead-in: Oral dosing, once daily in the morning over 43 days: 100 mg PRAX-944 Extension Part: Open-label Flexible PRAX-944 Dosing: Oral dosing, once daily in the morning of 20 mg to 100 mg PRAX-944 for 469 days Crossover Part: Following double-blind lead-in/open-label: 1:1 randomization to placebo or stable dose of PRAX-944 for 21 days followed by cross-over to either placebo or PRAX-944 oral dosing, once daily in the morning with titration over 7 days (3 days at 20 mg, 4 days at 40 mg) to 60 mg (14 days) before returning to open-label part. |
| Regimen 2 | EXPERIMENTAL | Double-blind Part: Oral dosing, once daily in the morning with titration over 56 days to 60 mg PRAX-944: 7 days of 5 mg, 7 days of 10 mg, 7 days of 20 mg, 7 days of 40 mg, 28 days of 60 mg Extension Part: Double-blind Lead-in: Oral dosing, once daily in the morning with titration over 43 days to 100 mg PRAX-944: 7 days of 80 mg, 36 days of 100 mg Extension Part: Open-label Flexible PRAX-944 Dosing: Oral dosing, once daily in the morning of 20 mg to 100 mg PRAX-944 for 469 days Crossover Part: Following double-blind lead-in/open-label: 1:1 randomization to placebo or stable dose of PRAX-944 for 21 days followed by cross-over to either placebo or PRAX-944 oral dosing, once daily in the morning with titration over 7 days (3 days at 20 mg, 4 days at 40 mg) to 60 mg (14 days) before returning to open-label part. |
| Regimen 3 | PLACEBO_COMPARATOR | Double-blind Part: Oral dosing, once daily in the morning: 56 days of placebo Extension Part: Double-blind Lead-in: Oral dosing, once daily in the morning with titration over 43 days to 100 mg PRAX-944: 7 days of 5 mg, 7 days of 10 mg, 7 days of 20 mg, 7 days of 40 mg, 7 days of 60 mg, 7 days of 80 mg, 14 days of 100 mg Extension Part: Open-label Flexible PRAX-944 Dosing: Oral dosing, once daily in the morning of 20 mg to 100 mg PRAX-944 for 469 days Crossover Part: Following double-blind lead-in/open-label: 1:1 randomization to placebo or stable dose of PRAX-944 for 21 days followed by cross-over to either placebo or PRAX-944 oral dosing, once daily in the morning with titration over 7 days (3 days at 20 mg, 4 days at 40 mg) to 60 mg (14 days) before returning to open-label part. |
| Name | Type | Description |
|---|---|---|
| 100 mg PRAX-944 | DRUG | Once daily oral treatment with titration |
| 60 mg PRAX-944 | DRUG | Once daily oral treatment with titration |
| Placebo | DRUG | Once daily oral treatment |
| Flexibly dosed 20 mg to 100 mg PRAX-944 | DRUG | Once daily oral treatment |
Inclusion Criteria: 1. Clinical diagnosis of ET, including: (a) tremor syndrome of bilateral upper limb action tremor, (b) at least 3 years in duration, (c) with or without tremor in other locations (eg, head, voice, or lower limbs), (d) If the symptoms and signs are judged by the investigator to b...
PRAX-944 is an investigational small molecule being developed by Praxis Precision Medicines, Inc. for the treatment of essential tremor, a neurological movement disorder. It has completed a Phase 2 clinical trial and is in the NDA/BLA regulatory stage. PRAX-944 is not yet approved for commercial use.
PRAX-944 is being developed as a treatment for essential tremor, a neurological condition characterized by involuntary rhythmic shaking. It is a small molecule therapeutic candidate. The drug has completed a Phase 2 clinical trial in this indication and is currently in the NDA/BLA stage of development.
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 movement disorders.
PRAX-944 is in the NDA/BLA stage of development. It has completed one Phase 2 clinical trial in essential tremor. The drug is investigational and has not been approved by the FDA. The completed trial was a randomized, double-blind, placebo-controlled study.
PRAX-944 has been studied in one completed clinical trial, NCT05021991, titled "A Clinical Trial of 2 Doses of PRAX-944 in Participants With Essential Tremor." This Phase 2 trial enrolled 133 participants across the United States and Canada. It was randomized, double-blind, and placebo-controlled, with participants aged 18 years and older.