Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12622000408785
Ethics application status
Approved
Date submitted
21/09/2021
Date registered
9/03/2022
Date last updated
9/03/2022
Date data sharing statement initially provided
9/03/2022
Type of registration
Retrospectively registered

Titles & IDs
Public title
A Clinical Trial to Evaluate the Efficacy, Safety, Tolerability, and Pharmacokinetics of PRAX-944 in Essential Tremor - Part B
Scientific title
A Phase 2 Clinical Trial Evaluating the Efficacy, Safety, Tolerability, and Pharmacokinetics of PRAX-944 in Adults with Essential Tremor - Part B
Secondary ID [1] 305266 0
PRAX-944-221
Secondary ID [2] 305267 0
PRAX-944-221, Part B
Universal Trial Number (UTN)
Trial acronym
Linked study record
This study is a follow-up study of ACTRN12619001052123

Health condition
Health condition(s) or problem(s) studied:
Essential Tremor 323554 0
Condition category
Condition code
Neurological 321110 321110 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
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.
Intervention code [1] 321663 0
Treatment: Drugs
Comparator / control treatment
Random assignment to placebo during the double-blind phase of the trial

Placebo is a cellulose fiber tablet containing microcrystalline cellulose, hydroxypropyl methylcellulose, silicon dioxide, triacetin, titanium dioxide, talc and magnesium stearate.
Control group
Placebo

Outcomes
Primary outcome [1] 328885 0
Incidence and severity of Adverse Events (AE) as a composite measure as assessed by clinical laboratory assessments (clinical chemistry, hematology, and urinalysis), ECG, self-report in response to single physician question about AEs, and physical examination
Timepoint [1] 328885 0
56 Days post-intervention commencement
Secondary outcome [1] 400787 0
Change from baseline to Day 42 in the Essential Tremor Rating Assessment Scale (TETRAS) upper limb score
Timepoint [1] 400787 0
Day 42 post-randomization
Secondary outcome [2] 400788 0
Change from baseline to Day 7 and Day 21 in the Essential Tremor Rating Assessment Scale (TETRAS) upper limb score
Timepoint [2] 400788 0
Day 7, Day 21 post-randomization
Secondary outcome [3] 400789 0
Change from baseline to Day 7, Day 21, and Day 42 on the Essential Tremor Rating Assessment Scale (TETRAS) Performance subscale
Timepoint [3] 400789 0
Day 7, Day 21, and Day 42 post-randomization
Secondary outcome [4] 400790 0
Change from baseline to Day 7, Day 21, and Day 42 in the Essential Tremor Rating Assessment Scale (TETRAS) upper limb score measured by accelerometry
Timepoint [4] 400790 0
Day 7, Day 21, Day 42 post-randomization
Secondary outcome [5] 400791 0
Change from baseline to Day 7, Day 21, and Day 42 on the TETRAS Activities of Daily Living (ADL) score
Timepoint [5] 400791 0
Day 7, Day 21, Day 42 post-randomization
Secondary outcome [6] 400792 0
Change from baseline to Day 7, Day 21, and Day 42 on the Quality of Life in Essential Tremor Questionnaire (QUEST) total and subscale scores
Timepoint [6] 400792 0
Day 7, Day 21, Day 42 post-randomization

Eligibility
Key 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
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Date of first participant enrolment
Anticipated
Actual
5/08/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
12
Accrual to date
4
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC
Recruitment outside Australia
Country [1] 24106 0
New Zealand
State/province [1] 24106 0
Christchurch
Country [2] 24124 0
New Zealand
State/province [2] 24124 0
Auckland

Funding & Sponsors
Funding source category [1] 309636 0
Commercial sector/Industry
Name [1] 309636 0
Praxis Precision Medicines Australia, Pty Ltd
Country [1] 309636 0
Australia
Primary sponsor type
Commercial sector/Industry
Name
Praxis Precision Medicines
Address
One Broadway, 16th Floor
Cambridge, MA 02142
Country
United States of America
Secondary sponsor category [1] 310655 0
None
Name [1] 310655 0
Address [1] 310655 0
Country [1] 310655 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 309408 0
Alfred Hospital Ethics Committee
Ethics committee address [1] 309408 0
Ethics committee country [1] 309408 0
Australia
Date submitted for ethics approval [1] 309408 0
Approval date [1] 309408 0
30/11/2020
Ethics approval number [1] 309408 0

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 114054 0
Prof Kelly Bertram
Address 114054 0
The Alfred Hospital
55 Commercial Road
Melbourne VIC 3004
Country 114054 0
Australia
Phone 114054 0
+61 03 90763795
Fax 114054 0
Email 114054 0
[email protected]
Contact person for public queries
Name 114055 0
Bernard Ravina
Address 114055 0
Praxis Precision Medicines
One Broadway
Cambridge, MA 02142
Country 114055 0
United States of America
Phone 114055 0
+1 617 300 8460
Fax 114055 0
Email 114055 0
[email protected]
Contact person for scientific queries
Name 114056 0
Bernard Ravina
Address 114056 0
Praxis Precision Medicines
One Broadway
Cambridge, MA 02142
Country 114056 0
United States of America
Phone 114056 0
+1 617 300 8460
Fax 114056 0
Email 114056 0
[email protected]

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment
Individual participant data will not be shared


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

Documents added manually
No documents have been uploaded by study researchers.

Documents added automatically
No additional documents have been identified.