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PRAX-944

Phase 2

Essential Tremor | Small molecule | Neurology |Praxis Precision Medicines, Inc.|Last Updated: Mar 13, 2024

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials1
Total Enrollment133

FDA Designations

No designations recorded

Clinical trial landscape

PRAX-944 · 1 trial · 1 indication

Phase 2 1
NCT05021991A Clinical Trial of 2 Doses of PRAX-944 in Participants With Essential TremorEssential Tremor
COMPLETED133 Analytics
PHASE2COMPLETED
A Clinical Trial of 2 Doses of PRAX-944 in Participants With Essential Tremor
Essential TremorUnlock trial analytics

Study Endpoints

Primary Endpoints

Change from baseline to Day 56 on the modified ADL
56 days

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.

Secondary Endpoints

Change from baseline to Day 56 on the Clinical Global Impression-Severity (CGI-S)
56 days
Clinical Global Impression-Improvement (CGI-I) score at Day 56
56 days
Change from baseline to Day 56 on the TETRAS-ADL score
56 days
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Regimen 1EXPERIMENTALDouble-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 2EXPERIMENTALDouble-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 3PLACEBO_COMPARATORDouble-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.

Interventions

NameTypeDescription
100 mg PRAX-944DRUGOnce daily oral treatment with titration
60 mg PRAX-944DRUGOnce daily oral treatment with titration
PlaceboDRUGOnce daily oral treatment
Flexibly dosed 20 mg to 100 mg PRAX-944DRUGOnce daily oral treatment
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites39

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...

Countries:United StatesCanada
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Frequently asked questions about PRAX-944

What is PRAX-944 used for?

PRAX-944 is an investigational small molecule being developed for the treatment of Essential Tremor, a neurological condition characterized by involuntary shaking. It is currently in clinical development and has not been approved by regulatory authorities.

Who makes PRAX-944?

PRAX-944 is being developed by Praxis Precision Medicines, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol PRAX. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with Essential Tremor.

What phase is PRAX-944 in?

PRAX-944 is in Phase 2 clinical development for Essential Tremor. A Phase 2 trial has been completed, and the drug remains investigational, meaning it has not yet received regulatory approval for commercial use.

What clinical trials is PRAX-944 in?

PRAX-944 has one completed clinical trial, NCT05021991, titled "A Clinical Trial of 2 Doses of PRAX-944 in Participants With Essential Tremor." This Phase 2, randomized, double-blind, placebo-controlled study enrolled 133 participants in the United States and Canada.

Is PRAX-944 the same as any other drug?

No alternative names for PRAX-944 have been disclosed. The drug is identified solely by its investigational code name PRAX-944 in clinical trial registries and public information from its developer, Praxis Precision Medicines.