Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12619001052123
Ethics application status
Approved
Date submitted
13/07/2019
Date registered
26/07/2019
Date last updated
11/02/2022
Date data sharing statement initially provided
26/07/2019
Type of registration
Prospectively registered

Titles & IDs
Public title
A Clinical Trial to Evaluate the Efficacy, Safety, Tolerability, and Pharmacokinetics of PRAX-944 in Essential Tremor
Scientific title
A Phase 2 Clinical Trial Evaluating the Efficacy, Safety, Tolerability, and Pharmacokinetics of PRAX-944 in Adults with Essential Tremor
Secondary ID [1] 298668 0
PRAX-944-221
Secondary ID [2] 306402 0
PRAX-944-221, Part A
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Essential Tremor 313565 0
Condition category
Condition code
Neurological 311995 311995 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
PRAX-944 20mg orally once a day for 7 days and 40mg orally once a day for 7 days (total of 14 days consecutively)

Study drug adherence will be monitored by a compliance application and verified by unused product return.
Intervention code [1] 314937 0
Treatment: Drugs
Comparator / control treatment
None
Control group
Uncontrolled

Outcomes
Primary outcome [1] 320642 0
Efficacy of PRAX-944 on upper limb tremor assessed by The Essential Tremor Rating Assessment Scale (TETRAS)
Timepoint [1] 320642 0
Baseline, Day 7 and Day 14
Secondary outcome [1] 372303 0
Efficacy of PRAX-944 on other measures of tremor severity assessed by TETRAS and accelerometer
Timepoint [1] 372303 0
Baseline, Day 7 and Day 14
Secondary outcome [2] 372304 0
Safety and tolerability of PRAX-944 will be assessed through an integrated analysis of the following endpoints: patient and clinician-reported adverse events (e.g. dizziness or headache), vital signs, clinical laboratory results, electrocardiogram (ECG), and Columbia-Suicide Severity Rating Scale (C-SSRS)
Timepoint [2] 372304 0
Baseline, Day 1, Day 7, Day 14, Day 21

Eligibility
Key inclusion criteria
- Male or females between the ages of 18 and 75 years
- 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
75 Years
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
Non-randomised 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
Open (masking not used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Date of first participant enrolment
Anticipated
3/02/2020
Actual
3/03/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
12
Accrual to date
7
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,VIC
Recruitment outside Australia
Country [1] 21674 0
New Zealand
State/province [1] 21674 0
Christchurch

Funding & Sponsors
Funding source category [1] 303210 0
Commercial sector/Industry
Name [1] 303210 0
Praxis Precision Medicines Australia, Pty Ltd
Country [1] 303210 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] 303221 0
None
Name [1] 303221 0
Address [1] 303221 0
Country [1] 303221 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 303767 0
Alfred Hospital Ethics Committee
Ethics committee address [1] 303767 0
55 Commercial Road
Melbourne VIC 3004
Ethics committee country [1] 303767 0
Australia
Date submitted for ethics approval [1] 303767 0
Approval date [1] 303767 0
12/04/2019
Ethics approval number [1] 303767 0

Summary
Brief summary
This is Part A 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.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 94726 0
Prof Dominic Thyagarajan
Address 94726 0
the Alfred Hospital
55 Commercial Road
Melbourne VIC 3004
Country 94726 0
Australia
Phone 94726 0
+61 03 90762000
Fax 94726 0
Email 94726 0
[email protected]
Contact person for public queries
Name 94727 0
Dr Bernard Ravina
Address 94727 0
Praxis Precision Medicines
One Broadway
Cambridge, MA 02142
Country 94727 0
United States of America
Phone 94727 0
+1 617 300 8460
Fax 94727 0
Email 94727 0
[email protected]
Contact person for scientific queries
Name 94728 0
Dr Bernard Ravina
Address 94728 0
Praxis Precision Medicines
One Broadway
Cambridge, MA 02142
Country 94728 0
United States of America
Phone 94728 0
+1 617 300 8460
Fax 94728 0
Email 94728 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.