None listed
Conditions
Brief summary
This is Part B of a 2-part, Phase 2, Clinical Trial Evaluating the Efficacy, Safety, Tolerability, and Pharmacokinetics of PRAX-944 in Adults with Essential Tremor. Each participant will complete 3 study periods: Screening, Treatment Period (21 or 28 days) and Safety Follow-up.
Interventions
Open-label Titration Phase - Titration to 120mg PRAX-944 orally according to the following titration schedule: PRAX-944 20mg orally once a day for 3 days, 40mg orally once a day for 4 days, 60mg orally once a day for 7 days, 80mg orally once a day for 7 days, 100mg orally once a day for 7 days, and 120mg orally for 7 days.
If at any point during the open-label titration phase a participant does not tolerate escalation (based on AEs and safety assessments) and the Investigator returns the parti
Open-label Titration Phase - Titration to 120mg PRAX-944 orally according to the following titration schedule: PRAX-944 20mg orally once a day for 3 days, 40mg orally once a day for 4 days, 60mg orally once a day for 7 days, 80mg orally once a day for 7 days, 100mg orally once a day for 7 days, and 120mg orally for 7 days. If at any point during the open-label titration phase a participant does not tolerate escalation (based on AEs and safety assessments) and the Investigator returns the participant to a lower dose level, the participant will continue according to the schedule outlined above, but no further dose changes will be allowed. Once participants complete the open label phase, they will immediately start the double-blind phase. They will be randomly assigned to continue with PRAX-944 or placebo for the double-blind phase. Double-blind Phase: PRAX-944 or Placebo orally once a day for 14 days after the final dose of the open-label phase. Total duration of participation in the trial will be 70 days (56 on treatment plus a 14 day follow-up period). Study drug adherence will be monitored by a compliance application and verified by unused product return.
Sponsors
Praxis Precision Medicines
Study design
Allocation
Randomised controlled trial
Intervention model
Parallel
Primary purpose
Treatment
Masking
Blinded (masking used) (Subject, Caregiver, Outcomes Assessor)
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
- Male or females 18 years of age or older - Clinical diagnosis of essential tremor (ET) - Stable dose of 1 tremor medication throughout the clinical trial, or no other tremor medications
Exclusion criteria
- Clinical evidence of psychogenic tremor - History of other medical, neurological or psychiatric condition 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 Screening - Unwilling or unable to discontinue primidone - Unwilling or unable to refrain from alcohol 24 hours before and during the clinical trial visits. - Any other significant disease, disorder or lab abnormalities that may either put the patient at risk due to participation in the clinical trial, may influence or confound the result of the clinical trial, or affect the patient’s ability to participate in the clinical trial.
Outcome results
None listed