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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
12/06/2020
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 30 kg/m2 (inclusive).
Minimum age
18 Years
Maximum age
55 Years
Gender
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
Recruiting
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
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
Address [1] 305050 0
One Broadway, 16th Floor
Cambridge, MA 02142
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
b.snyder@nucleusnetwork.com.au
Contact person for public queries
Name 100387 0
Dr 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
clinicaltrials@praxismedicines.com
Contact person for scientific queries
Name 100388 0
Dr 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
clinicaltrials@praxismedicines.com

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 other documents available
Summary results
No Results