Essential TremorPhase 2Essential1NCT05021991
A Clinical Trial of 2 Doses of PRAX-944 in Participants With Essential Tremor
A Phase 2 study testing 100 mg PRAX-944, 60 mg PRAX-944 and Flexibly dosed 20 mg to 100 mg PRAX-944 in people with Essential Tremor, run by Praxis Precision Medicines at 39 sites in 2 countries.
Trial summary
This multi-center, randomized, double-blinded, placebo-controlled, dose-range-finding clinical trial (with an optional Extension comprised of an Extension Double-blind (DB) Lead in Period followed by an Extension Open-label (OL) Period) that will assess the efficacy, safety, and tolerability of PRAX 944 in… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Essential Tremor
- What is being tested?
- 100 mg PRAX-944, 60 mg PRAX-944 and Flexibly dosed 20 mg to 100 mg PRAX-944
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 133 participants
- Where
- 39 sitesin 2 countries
- Study dates
- 14 Oct 2021to 29 Feb 2024
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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 be due to the diagnosis of ET, it is acceptable for them to also have one or more of the following ET plus signs: (i) mild dystonic posturing, (ii) mild rest tremor in the setting of advanced ET and in the absence of other features of Parkinsonism, (iii) intention tremor, (iv) mild increase in tandem gait difficulty.
- Participant has moderate to severe functional impairment due to tremor as determined by the TETRAS and CGI-S.
- If currently receiving any medication for ET, is on a stable dose of any of these medications for ET for 1 month prior to Screening and is willing to maintain stable doses throughout the trial. If receiving primidone for ET, is willing and able to discontinue 14 days prior to Day 1.
- Body mass index (BMI) between 18 and 40 kg/m² (inclusive).
Who cannot join
- Sporadically using a benzodiazepine, sleep medication, or anxiolytic that would confound the assessment of tremor.
- Trauma to the nervous system within 3 months preceding the onset of tremor.
- History or clinical evidence of other medical, neurological, or psychiatric condition that may explain or cause tremor, including but not limited to Parkinson's disease, Huntington's disease, Alzheimer's disease, cerebellar disease (including spinocerebellar ataxias), primary dystonia, Fragile X Tremor/Ataxia syndrome or family history of Fragile X syndrome, traumatic brain injury, psychogenic tremor, alcohol or benzodiazepine abuse or withdrawal, multiple sclerosis, polyneuropathy, and endocrine states such as hyperthyroidism or unstable treatment of hypothyroidism or medication, food, or supplement induced movement disorders (eg, tremor related to beta agonists or caffeine), or other medical, neurological, or psychiatric conditions that may explain or cause tremor
- Prior magnetic resonance-guided focused ultrasound or surgical intervention for ET such as deep brain stimulation or thalamotomy.
- Botulinum toxin injection for ET in the 6 months prior to Baseline.
- Cala trio health device for ET in the 14 days prior to Baseline and throughout the study.
- History of substance use disorder consistent with Diagnostic and Statistical Manual of Mental Disorders (DSM)-5 criteria. Participants with a previous diagnosis of substance use disorder who have been in remission for at least 2 years can participate in the trial.
Trial locations and contacts
39 sites in 2 countries.
39 sites in 2 countries
United States35 sites
Praxis Research Site
Alexandria, Virginia, 22311
Praxis Research Site
Aurora, Colorado, 80045
Praxis Research Site
Birmingham, Alabama, 35294
Praxis Research Site
Boca Raton, Florida, 33486
Praxis Research Site
Boston, Massachusetts, 02118
Praxis Research Site
Burlington, Massachusetts, 01805
Praxis Research Site
Burlington, Vermont, 05401
Praxis Research Site
Chicago, Illinois, 60612
Praxis Research Site
Cincinnati, Ohio, 45212
Praxis Research Site
Farmington Hills, Michigan, 48334
Praxis Research Site
Gainesville, Florida, 32608
Praxis Research Site
Georgetown, Texas, 78628
Praxis Research Site
Golden Valley, Minnesota, 55427
Praxis Research Site
Houston, Texas, 77030
Praxis Research Site
Jacksonville, Florida, 32209
Praxis Research Site
Kansas City, Kansas, 66160
Praxis Research Site
Kirkland, Washington, 98034
Praxis Research Site
Las Vegas, Nevada, 89106
Praxis Research Site
Little Rock, Arkansas, 72205
Praxis Research Site
Louisville, Kentucky, 40202
Praxis Research Site
Milwaukee, Wisconsin, 53226
Praxis Research Site
New York, New York, 10029
Praxis Research Site
New York, New York, 10032
Praxis Research Site
Philadelphia, Pennsylvania, 19107
Praxis Research Site
Port Charlotte, Florida, 33980
Praxis Research Site
Rockville, Maryland, 20852
Praxis Research Site
Round Rock, Texas, 78681
Praxis Research Site
San Diego, California, 92103
Praxis Research Site
Santa Monica, California, 90404
Praxis Research Site
Spokane, Washington, 99202
Praxis Research Site
St. Petersburg, Florida, 33713
Praxis Research Site
Tampa, Florida, 33612
Praxis Research Site
Torrance, California, 90503
Praxis Research Site
Virginia Beach, Virginia, 23456
Praxis Research Site
West Palm Beach, Florida, 33407
Canada4 sites
Praxis Research Site
Halifax, Nova Scotia, B3S 1N2
Praxis Research Site
Montreal, Quebec, H3A 2B4
Praxis Research Site
Toronto, Ontario, M5T 2S8
Praxis Research Site
Vancouver, British Columbia, V6T 2B5
About this study
Official title: A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Dose Range Finding Clinical Trial to Evaluate the Efficacy, Tolerability, and Safety of PRAX-944 in the Treatment of Adults With Essential Tremor
This multi-center, randomized, double-blinded, placebo-controlled, dose-range-finding clinical trial (with an optional Extension comprised of an Extension Double-blind (DB) Lead in Period followed by an Extension Open-label (OL) Period) that will assess the efficacy, safety, and tolerability of PRAX 944 in participants aged 18 years or older who have a diagnosis of Essential Tremor (ET) and have had symptoms for at least 3 years.
- Conditions
- Essential Tremor
- Being tested
- 100 mg PRAX-944, 60 mg PRAX-944, Placebo, Flexibly dosed 20 mg to 100 mg PRAX-944
- Keywords
- Movement Disorder, Benign Essential Tremor, Familial Tremor, Hereditary Essential Tremor, Movement Disorder Agents, Calcium Channels, T-type
- Medical subject headings
- Essential Tremor, Movement Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
100 mg PRAX-944Drug
Once daily oral treatment with titration
60 mg PRAX-944Drug
Once daily oral treatment with titration
PlaceboDrug
Once daily oral treatment
Flexibly dosed 20 mg to 100 mg PRAX-944Drug
Once daily oral treatment
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: 100 mg PRAX-944; Drug: 60 mg PRAX-944; Drug: Placebo; Drug: Flexibly dosed 20 mg to 100 mg PRAX-944 |
| 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. Receives: Drug: 100 mg PRAX-944; Drug: 60 mg PRAX-944; Drug: Placebo; Drug: Flexibly dosed 20 mg to 100 mg PRAX-944 |
| 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. Receives: Drug: 100 mg PRAX-944; Drug: 60 mg PRAX-944; Drug: Placebo; Drug: Flexibly dosed 20 mg to 100 mg PRAX-944 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline to Day 56 on the modified ADL
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.
Measured: 56 days
Secondary outcomes (16)
Change from baseline to Day 56 on the Clinical Global Impression-Severity (CGI-S)
The 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).
Measured: 56 days
Clinical Global Impression-Improvement (CGI-I) score at Day 56
The CGI-I assesses the participant's improvement (or worsening). The clinician is required to assess the participant's condition relative to pre-treatment 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 rater believes the improvement/worsening is drug-related or not.
Measured: 56 days
Change from baseline to Day 56 on the TETRAS-ADL score
The TETRAS-ADL subscale is a 12-item assessment of typical daily activities that are impacted by tremor. Activities are assessed in the following functional domains: speaking, feeding, drinking, personal hygiene, dressing, writing, and social activity. The impact to each function is rated on a 5-point Likert scale from 0 to 4. The ADL subscale score is calculated as the sum of all 12 items and ranges from 0 to 48 where larger values represent increased direct tremor impact to activities of daily living.
Measured: 56 days
Change from baseline to Day 56 on the TETRAS-Performance Subscale (PS) total score
There are 9 items covering different body regions in the Performance Subscale. Each Performance Subscale item is rated on a scale of 0 to 4, with higher scores indicating higher tremor severity. Item 4 of the Performance Subscale is the upper limb item. It is comprised of 6 sub-items (4a, 4b, and 4c assessed for both the right and left upper limbs). The Performance subscale score is calculated as the sum of all 9 items and ranges from 0 to 64 where larger values represent higher tremor severity.
Measured: 56 days
Change from baseline to Day 56 on the TETRAS-upper limb (UL) score (TETRAS-PS item 4)
Item 4 of the Performance subscale is the upper limb item. It is comprised of 6 sub-items (4a, 4b, and 4c assessed for both the right and left upper limbs). Each sub-item is rated on a scale from 0 to 4, with higher scores indicating higher tremor amplitude of the upper limb. The upper limb total score is the sum of these 6 sub-items and ranges from 0 to 24 where larger values represent higher tremor severity.
Measured: 56 days
Change from baseline to Day 56 on the TETRAS-combined upper limb (CUL) score (TETRAS-PS sum of items 4, 6, 7, and 8)
The combined upper limb score is the sum of the 6 sub-item scores of the upper limb item and the handwriting and spirals scores. The combined upper limb score ranges from 0 to 32 where larger values represent higher tremor severity.
Measured: 56 days
Patient Global Impression-Change (PGI-C) score at Day 56
The PGI-C assesses the participant's change in condition. 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.
Measured: 56 days
Change from baseline to Days 14, 28, and 42 on the modified ADL
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.
Measured: Up to 42 days
Change from baseline to Days 14, 28, and 42 on the CGI-S
The 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).
Measured: Up to 42 days
Change from baseline to Days 14, 28, and 42 on the TETRAS-ADL total score
The TETRAS-ADL subscale is a 12-item assessment of typical daily activities that are impacted by tremor. Activities are assessed in the following functional domains: speaking, feeding, drinking, personal hygiene, dressing, writing, and social activity. The impact to each function is rated on a 5-point Likert scale from 0 to 4. The ADL subscale score is calculated as the sum of all 12 items and ranges from 0 to 48 where larger values represent increased direct tremor impact to activities of daily living.
Measured: Up to 42 days
Change from baseline to Days 14, 28, and 42 on the TETRAS-UL score
Item 4 of the Performance subscale is the upper limb item. It is comprised of 6 sub-items (4a, 4b, and 4c assessed for both the right and left upper limbs). Each sub-item is rated on a scale from 0 to 4, with higher scores indicating higher tremor amplitude of the upper limb. The upper limb total score is the sum of these 6 sub-items and ranges from 0 to 24 where larger values represent higher tremor severity.
Measured: Up to 42 days
Change from baseline to Days 14, 28, and 42 on the TETRAS-CUL score
The combined upper limb score is the sum of the 6 sub-item scores of the upper limb item and the handwriting and spirals scores. The combined upper limb score ranges from 0 to 32 where larger values represent higher tremor severity.
Measured: Up to 42 days
CGI-I scores at Days 14, 28, and 42
The CGI-I assesses the participant's improvement (or worsening). The clinician is required to assess the participant's condition relative to pre-treatment 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 rater believes the improvement/worsening is drug-related or not.
Measured: Up to 42 days
PGI-C scores at Days 14, 28, and 42
The PGI-C assesses the participant's improvement (or worsening). 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 improvement/worsening is drug-related or not.
Measured: Up to 42 days
Change from baseline to Day 42 on the TETRAS-PS total score
There are 9 items covering different body regions in the Performance Subscale. Each Performance Subscale item is rated on a scale of 0 to 4, with higher scores indicating higher tremor severity. Item 4 of the Performance Subscale is the upper limb item. It is comprised of 6 sub-items (4a, 4b, and 4c assessed for both the right and left upper limbs). The Performance subscale score is calculated as the sum of all 9 items and ranges from 0 to 64.
Measured: 42 days
Number of participants with Adverse Events (AE)
The number of participants with Adverse Events (AE) will be reported by preferred term.
Measured: Up to 56 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Aug 2021
- Study start14 Oct 2021
- Primary completion9 Feb 2023
- Study completion29 Feb 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Essential TremorStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 133 participants (actual)
Sponsor and investigators
- Lead sponsor
- Praxis Precision Medicines
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Director, Clinical Development, Study director, Praxis Precision Medicines
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05021991
- Sponsor's protocol ID
- PRAX-944-222
- Acronym
- Essential1
- Status last verified
- Mar 2024
- First submitted
- 20 Aug 2021
- Last updated
- 13 Mar 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Dose Range Finding Clinical Trial to Evaluate the Efficacy, Tolerability, and Safety of PRAX-944 in the Treatment of Adults With Essential Tremor
- ClinicalTrials.gov ID
- NCT05021991
- Acronym / sponsor ID
- Essential1 / PRAX-944-222
- Conditions
- Essential Tremor
- Keywords
- Movement Disorder, Benign Essential Tremor, Familial Tremor, Hereditary Essential Tremor, Movement Disorder Agents, Calcium Channels, T-type
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 133 participants (actual)
- Lead sponsor
- Praxis Precision MedicinesIndustry (pharma, biotech, devices)
- Study start
- 14 Oct 2021 (actual)
- Primary completion
- 9 Feb 2023 (actual)
- Study completion
- 29 Feb 2024 (actual)
- First posted
- 26 Aug 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This multi-center, randomized, double-blinded, placebo-controlled, dose-range-finding clinical trial (with an optional Extension comprised of an Extension Double-blind (DB) Lead in Period followed by an Extension Open-label (OL) Period) that will assess the efficacy, safety, and tolerability of PRAX 944 in participants aged 18 years or older who have a diagnosis of Essential Tremor (ET) and have had symptoms for at least 3 years.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Regimen 1 | Experimental | Drug: 100 mg PRAX-944, Drug: 60 mg PRAX-944, Drug: Placebo, Drug: Flexibly dosed 20 mg to 100 mg PRAX-944Double-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 | Drug: 100 mg PRAX-944, Drug: 60 mg PRAX-944, Drug: Placebo, Drug: Flexibly dosed 20 mg to 100 mg PRAX-944Double-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 | Drug: 100 mg PRAX-944, Drug: 60 mg PRAX-944, Drug: Placebo, Drug: Flexibly dosed 20 mg to 100 mg PRAX-944Double-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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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 be due to the diagnosis of ET, it is acceptable for them to also have one or more of the following ET plus signs: (i) mild dystonic posturing, (ii) mild rest tremor in the setting of advanced ET and in the absence of other features of Parkinsonism, (iii) intention tremor, (iv) mild increase in tandem gait difficulty.
- Participant has moderate to severe functional impairment due to tremor as determined by the TETRAS and CGI-S.
- If currently receiving any medication for ET, is on a stable dose of any of these medications for ET for 1 month prior to Screening and is willing to maintain stable doses throughout the trial. If receiving primidone for ET, is willing and able to discontinue 14 days prior to Day 1.
- Body mass index (BMI) between 18 and 40 kg/m² (inclusive).
Key exclusion criteria (7 listed)
- Sporadically using a benzodiazepine, sleep medication, or anxiolytic that would confound the assessment of tremor.
- Trauma to the nervous system within 3 months preceding the onset of tremor.
- History or clinical evidence of other medical, neurological, or psychiatric condition that may explain or cause tremor, including but not limited to Parkinson's disease, Huntington's disease, Alzheimer's disease, cerebellar disease (including spinocerebellar ataxias), primary dystonia, Fragile X Tremor/Ataxia syndrome or family history of Fragile X syndrome, traumatic brain injury, psychogenic tremor, alcohol or benzodiazepine abuse or withdrawal, multiple sclerosis, polyneuropathy, and endocrine states such as hyperthyroidism or unstable treatment of hypothyroidism or medication, food, or supplement induced movement disorders (eg, tremor related to beta agonists or caffeine), or other medical, neurological, or psychiatric conditions that may explain or cause tremor
- Prior magnetic resonance-guided focused ultrasound or surgical intervention for ET such as deep brain stimulation or thalamotomy.
- Botulinum toxin injection for ET in the 6 months prior to Baseline.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 100 mg PRAX-944Once daily oral treatment with titration | Drug | Regimen 1, Regimen 2, Regimen 3 |
| 60 mg PRAX-944Once daily oral treatment with titration | Drug | Regimen 1, Regimen 2, Regimen 3 |
| PlaceboOnce daily oral treatment | Drug | Regimen 1, Regimen 2, Regimen 3 |
| Flexibly dosed 20 mg to 100 mg PRAX-944Once daily oral treatment | Drug | Regimen 1, Regimen 2, Regimen 3 |
Primary outcome
Change from baseline to Day 56 on the modified ADL
Time frame: 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.
16 secondary outcomes
Change from baseline to Day 56 on the Clinical Global Impression-Severity (CGI-S)
Time frame: 56 days
Clinical Global Impression-Improvement (CGI-I) score at Day 56
Time frame: 56 days
Change from baseline to Day 56 on the TETRAS-ADL score
Time frame: 56 days
Change from baseline to Day 56 on the TETRAS-Performance Subscale (PS) total score
Time frame: 56 days
Change from baseline to Day 56 on the TETRAS-upper limb (UL) score (TETRAS-PS item 4)
Time frame: 56 days
Change from baseline to Day 56 on the TETRAS-combined upper limb (CUL) score (TETRAS-PS sum of items 4, 6, 7, and 8)
Time frame: 56 days
Patient Global Impression-Change (PGI-C) score at Day 56
Time frame: 56 days
Change from baseline to Days 14, 28, and 42 on the modified ADL
Time frame: Up to 42 days
Change from baseline to Days 14, 28, and 42 on the CGI-S
Time frame: Up to 42 days
Change from baseline to Days 14, 28, and 42 on the TETRAS-ADL total score
Time frame: Up to 42 days
Change from baseline to Days 14, 28, and 42 on the TETRAS-UL score
Time frame: Up to 42 days
Change from baseline to Days 14, 28, and 42 on the TETRAS-CUL score
Time frame: Up to 42 days
CGI-I scores at Days 14, 28, and 42
Time frame: Up to 42 days
PGI-C scores at Days 14, 28, and 42
Time frame: Up to 42 days
Change from baseline to Day 42 on the TETRAS-PS total score
Time frame: 42 days
Number of participants with Adverse Events (AE)
Time frame: Up to 56 days
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Mar 2024.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Director, Clinical Development study director
Praxis Precision Medicines
39 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing 100 mg PRAX-944, 60 mg PRAX-944 and Flexibly dosed 20 mg to 100 mg PRAX-944 in people with Essential Tremor, run by Praxis Precision Medicines at 39 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 13 Mar 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 39 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from baseline to Day 56 on the modified ADL (time frame: 56 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Mar 2024.