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Trial registered on ANZCTR


Registration number
ACTRN12620000675921
Ethics application status
Approved
Date submitted
7/05/2020
Date registered
12/06/2020
Date last updated
28/02/2022
Date data sharing statement initially provided
12/06/2020
Type of registration
Retrospectively registered

Titles & IDs
Public title
A Trial in Healthy Participants to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of PRAX-944
Scientific title
A Phase 1 Clinical Trial in Healthy Participants to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of PRAX-944 Administered in a Within-Participant Escalating Dose Paradigm
Secondary ID [1] 300627 0
PRAX-944-105
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Essential Tremor 316396 0
Condition category
Condition code
Neurological 314663 314663 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Participants will be confined at the site during the dosing period and all dosing will be witnessed by study staff. Part B will only commence once the safety of PRAX-944 low doses is established in Part A. Participants can only take part in either Part A or Part B of the study:
- Part A (PRAX-944 oral tablet): 5mg (Day 1-4); 10mg (Day 5-8); 20mg (Day 9-12)
- Part B (PRAX-944 oral tablet): 20mg (Day 1-3); 40mg (Day 4-6); 60mg (Day 7-13); 80mg (Day 14-20); 100mg (Day 21-27); 120mg (Day 28-31) or placebo (Day 1-31)
Intervention code [1] 316952 0
Treatment: Drugs
Comparator / control treatment
Matching microcrystalline cellulose placebo tablets (identical to PRAX-944 tablets without the active ingredient) in Part B only
Control group
Placebo

Outcomes
Primary outcome [1] 322988 0
Part A: Evaluate the pharmacodynamic effects of PRAX-944 as assessed by changes in EEG.
Timepoint [1] 322988 0
Part A: Baseline, Day 1, Day 4, Day 5, Day 8, Day 9 Day 12 and Day 13
Primary outcome [2] 323937 0
Part B: Safety and tolerability of PRAX-944 as assessed through an integrated analysis of the following endpoints: patient and clinician-reported adverse events, physical exams, vital signs, clinical laboratory results, electrocardiogram (ECG) and Columbia-Suicide Severity Rating Scale (C-SSRS)
Timepoint [2] 323937 0
Part B: Baseline, Day 1 to Day 33, Day 36
Secondary outcome [1] 380435 0
Part A and Part B: Pharmacokinetics will be performed by LC/MS quantification of PRAX-944 levels in plasma. Parameters include plasma concentrations (Cmax, tmax, t1/2) and accumulation ratios (AUC).
Timepoint [1] 380435 0
Part A and Part B: pre-dose and 0.25, 0.5, 0.75, 1, 1.5, 2, 2.5, 3, 4, 6, 8, 12 hours post-dose
Secondary outcome [2] 380436 0
Part A: Safety and tolerability of PRAX-944 as assessed through an integrated analysis of the following endpoints: patient and clinician-reported adverse events, physical exams, vital signs, clinical laboratory results, electrocardiogram (ECG) and Columbia-Suicide Severity Rating Scale (C-SSRS),
Timepoint [2] 380436 0
Part A: Baseline, Day 1 to Day 13, Day 17

Eligibility
Key inclusion criteria
- Female or male and between the ages of 18 and 55 years, inclusive.
- Weight of at least 50 kg with body mass index (BMI) between 18 and 32 kg/m2 (inclusive).
Minimum age
18 Years
Maximum age
55 Years
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
- Ongoing or history of any psychiatric, medical or surgical condition that might jeopardize the participant’s safety or interfere with the absorption, distribution, metabolism or excretion of the study drug.
- Any abnormal ECG, laboratory value or physical examination
- Positive breath test for ethanol, urine drug screen, urine cotinine test
- Use of systemic prescription medications or over-the-counter (OTC) medication, including multivitamins, and dietary and herbal supplement within 2 weeks
- Use of any experimental or investigational drug or device within 30 days
- Donation or loss of greater than or equal to 400 mL blood within 8 weeks and/or donation of plasma within 7 days

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
Who is / are masked / blinded?



Intervention assignment
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Date of first participant enrolment
Anticipated
Actual
29/05/2020
Date of last participant enrolment
Anticipated
Actual
22/07/2020
Date of last data collection
Anticipated
Actual
6/09/2020
Sample size
Target
24
Accrual to date
Final
24
Recruitment in Australia
Recruitment state(s)
VIC

Funding & Sponsors
Funding source category [1] 305050 0
Commercial sector/Industry
Name [1] 305050 0
Praxis Precision Medicines
Country [1] 305050 0
United States of America
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] 305414 0
None
Name [1] 305414 0
Address [1] 305414 0
Country [1] 305414 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 305438 0
Alfred Hospital Ethics Committee
Ethics committee address [1] 305438 0
55 Commercial Road
Melbourne VIC 3004
Ethics committee country [1] 305438 0
Australia
Date submitted for ethics approval [1] 305438 0
04/03/2020
Approval date [1] 305438 0
31/03/2020
Ethics approval number [1] 305438 0

Summary
Brief summary
This single-center, two-part, Phase 1 clinical trial will assess the safety, tolerability, pharmacokinetics (PK) and PD of multiple-ascending doses of PRAX-944 in healthy male and female participants aged 18 to 55 years, inclusive.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 100386 0
Dr Ben Synder
Address 100386 0
Nucleus Network Limited
Level 5 Burnet Institute
AMREP Precinct
89 Commercial Road
Melbourne, VIC 3004
Country 100386 0
Australia
Phone 100386 0
+61 3 9076 8960
Fax 100386 0
Email 100386 0
[email protected]
Contact person for public queries
Name 100387 0
Bernard Ravina
Address 100387 0
Praxis Precision Medicines
One Broadway
Cambridge, MA 02142
Country 100387 0
United States of America
Phone 100387 0
+1 617 300 8460
Fax 100387 0
Email 100387 0
[email protected]
Contact person for scientific queries
Name 100388 0
Bernard Ravina
Address 100388 0
Praxis Precision Medicines
One Broadway
Cambridge, MA 02142
Country 100388 0
United States of America
Phone 100388 0
+1 617 300 8460
Fax 100388 0
Email 100388 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
SourceTitleYear of PublicationDOI
EmbaseTranslational Pharmacology of PRAX-944, a Novel T-Type Calcium Channel Blocker in Development for the Treatment of Essential Tremor.2022https://dx.doi.org/10.1002/mds.28969
N.B. These documents automatically identified may not have been verified by the study sponsor.