120 mg PRAX-944 for Familial Tremor

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
Praxis Research Site, Louisville, KY
Familial Tremor+1 More
120 mg PRAX-944 - Drug
Eligibility
18+
All Sexes
Eligible conditions
Select

Study Summary

This study is evaluating whether a drug called PRAX-944 might be effective in treating essential tremor.

See full description

Eligible Conditions

  • Familial Tremor

Treatment Effectiveness

Effectiveness Progress

1 of 3

Study Objectives

This trial is evaluating whether 120 mg PRAX-944 will improve 2 primary outcomes, 19 secondary outcomes, and 1 other outcome in patients with Familial Tremor. Measurement will happen over the course of Up to 42 days.

42 days
Change from baseline to Day 42 on the TETRAS-PS total score
56 days
Change from baseline to Day 56 on the Clinical Global Impression-Severity (CGI-S)
Change from baseline to Day 56 on the Essential Tremor Rating Assessment Scale (TETRAS) combined upper limb (CUL) score
Change from baseline to Day 56 on the TETRAS Activities of Daily Living (ADL) score
Change from baseline to Day 56 on the TETRAS Modified Total Score
Change from baseline to Day 56 on the TETRAS-ADL score
Change from baseline to Day 56 on the TETRAS-Performance Subscale (PS) total score
Change from baseline to Day 56 on the TETRAS-combined upper limb (CUL) score (TETRAS-PS sum of items 4, 6, 7, and 8)
Change from baseline to Day 56 on the TETRAS-upper limb (UL) score (TETRAS-PS item 4)
Change from baseline to Day 56 on the modified ADL
Clinical Global Impression-Improvement (CGI-I) score at Day 56
Incidence and severity of Adverse Events (AE)
Incidence of AEs by preferred term
Patient Global Impression-Change (PGI-C) score at Day 56
Up to 42 days
CGI-I scores at Days 14, 28, and 42
Change from baseline to Days 14, 28, and 42 on the CGI-S
Change from baseline to Days 14, 28, and 42 on the TETRAS-ADL total score
Change from baseline to Days 14, 28, and 42 on the TETRAS-CUL score
Change from baseline to Days 14, 28, and 42 on the TETRAS-UL score
Change from baseline to Days 14, 28, and 42 on the modified ADL
PGI-C scores at Days 14, 28, and 42
Up to 56 days
Number of participants with Adverse Events (AE)

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Trial Design

8 Treatment Groups

Regimen 1
1 of 8
Regimen 2
1 of 8
PRAX-944 (120 mg)
1 of 8
PRAX-944 (60 mg)
1 of 8
PRAX-944 (100 mg)
1 of 8
PRAX-944 (20 mg)
1 of 8
Regimen 3
1 of 8
Placebo
1 of 8
Experimental Treatment
Non-Treatment Group

This trial requires 131 total participants across 8 different treatment groups

This trial involves 8 different treatments. 120 Mg PRAX-944 is the primary treatment being studied. Participants will be divided into 6 treatment groups. Some patients will receive a placebo treatment. The treatments being tested are in Phase 2 and have already been tested with other people.

Regimen 1Double-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 90 days
Regimen 2Double-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 90 days
PRAX-944 (120 mg)
Drug
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
PRAX-944 (60 mg)Once daily, oral dosing with titration to 60 mg: 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
PRAX-944 (100 mg)
Drug
Once daily, oral dosing with titration to 100 mg: 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
PRAX-944 (20 mg)Once daily, oral dosing with titration to 20 mg: 7 days of 5 mg, 7 days of 10 mg, 42 days of 20 mg
Regimen 3Double-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 90 days
Placebo
Drug
Once daily, oral dosing: 56 days of placebo
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Placebo
1995
Completed Phase 3
~2810

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 56 days
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly 56 days for reporting.

Closest Location

Praxis Research Site - Louisville, KY

Eligibility Criteria

This trial is for patients born any sex aged 18 and older. There are 4 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
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.
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).
You have TETRAS upper limb score of ≥10 as rated by the Investigator. show original

Patient Q&A Section

How serious can familial tremor be?

"The most consistent risk factor for disability is advancing age, especially for women. In men, the most consistent risk factor continues to be HTN, though it is unclear why this risk is greatest for individuals with pre-HTN disease and/or HTN." - Anonymous Online Contributor

Unverified Answer

Does familial tremor run in families?

"Findings from a recent study suggest that the genetics for tremor in FHT families are probably complex and there may be additional loci that modulate the risk for tremor in affected members with FHT." - Anonymous Online Contributor

Unverified Answer

How does 120 mg prax-944 work?

"Prax-944 120 mg appears to be as efficacious as 120 and 280 mg of mexiletine for tremor reduction. When comparing this dose with 240 mg of mexiletine, a similar reduction in tremor amplitude was seen on the posttreatment session with 120 mg of prax-944, but this was not as statistically significant as with the 280 or 240 mg of mexiletine dose. Side effects were mild, and no serious side effects of this drug were reported." - Anonymous Online Contributor

Unverified Answer

Can familial tremor be cured?

"There is a high remission rate in familial tremor (93%), even in families with a positive family pedigree with epilepsy. F-T can be considered an alternative to pharmacological treatment of tremor." - Anonymous Online Contributor

Unverified Answer

How many people get familial tremor a year in the United States?

"Data from a recent study presents a clear trend in the decline in the diagnosis of FT. The rate of decline observed in the USA and GB has been slower than that reported anywhere in the world. Possible reasons for this, such as greater awareness of FT and increased availability of clinical tests, warrant further studies to be undertaken." - Anonymous Online Contributor

Unverified Answer

What are common treatments for familial tremor?

"The treatment of choice for patients with familial tremor varies depending on the family history, the patient’s age, and the condition of the patient. In general, most treatments can be used to reduce the severity of the tremor. Physicians often recommend using a combination of different treatment options for the treatment of familial tremor." - Anonymous Online Contributor

Unverified Answer

What are the signs of familial tremor?

"For diagnosis of familial tremor a combination of history of the disease and examination is necessary as there are few general signs of this condition. There is no evidence in the literature that shows what the genetic causes are for familial tremor. It is not known if there is a combination of symptoms and signs that occur with two or more other diseases or psychiatric conditions and those with family tremor may also be at higher risk for other CNS systemic diseases. It could be due to either a genetic or a metabolic condition." - Anonymous Online Contributor

Unverified Answer

What is familial tremor?

"Familial tremor affects an estimated 5-16% of the population. Genetic and familial factors contribute to the risk of developing this disease. As the name suggests, the tremors of familial tremor are present in at least one family member. In contrast to most trembling families, trembling is not present in the other family members. If a family member has trembling with normal family history, it is likely that family members' genes are involved. The tremor is not tremulous in nature, and the tremors are not excessive. Most cases of familial tremor appear to be of idiopathic origin. Although the tremors are similar to the trembling seen in Parkinson's disease, it is not a disease of the peripheral nervous system." - Anonymous Online Contributor

Unverified Answer

What causes familial tremor?

"1. Genetic linkage could be shown between the tremor gene and loci at chromosomes 1, 2, 20, 22, and 7.\n2. It is likely to have a genetic component.\n3. In a recent study, findings in our family of familial tremor may in part be a result of hereditary [cerebral palsy](https://www.withpower.com/clinical-trials/cerebral-palsy).\n4. Genetic counseling of members of the family is suggested. This familial tremor may be part of a spectrum of disorders that includes Parkinson's disease and Pick's disease. They may also constitute a part of the familial tremor syndrome.\n5." - Anonymous Online Contributor

Unverified Answer

Is 120 mg prax-944 typically used in combination with any other treatments?

"Prax-944 in combination with carbamazepine (12.5 mg b.i.d.), primidone (10 mg o.d.), levetiracetam (100 mg b.d.), isoprenaline (400 mcg per day b.v.s.max. 1) is efficacious for both lowering and preventing the occurrence of tremor episodes. The efficacy in lowering tremor was observed at 1 and 5 weeks. The addition of valproate (50 mg b.i.d.) is associated with a significant worsening of tremor. [Power](https://www.withpower." - Anonymous Online Contributor

Unverified Answer

What is the primary cause of familial tremor?

"Although we are still left with no firm evidence that any gene or gene-locus has been identified, we are in the process of doing genetic research. Since it may not be impossible for some genes to be identified, then by identifying and studying individuals with familial tremor in a familial tremor family, and collecting data from their family members and relatives, the gene(s) implicated in the specific subtypes of familial tremor could be discovered. If we are fortunate enough, then by studying the genes which encode the disease proteins, we will be in the position to develop therapies to treat the disorder. Since familial tremor has been found to be very common, it seems likely that it could be more common than many people would suspect." - Anonymous Online Contributor

Unverified Answer

Does 120 mg prax-944 improve quality of life for those with familial tremor?

"Prax-944 120 mg once daily is effective for treating the symptoms of FTM. Clinical improvement was noted in both treated and placebo groups, but a significant difference was shown only for patients in the treatment group who reported a good reduction in their tremor. Prax-944 can be considered as a reasonable therapy for many patients with FTM." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
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