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Trial registered on ANZCTR


Registration number
ACTRN12620001292965
Ethics application status
Approved
Date submitted
8/06/2020
Date registered
30/11/2020
Date last updated
12/10/2021
Date data sharing statement initially provided
30/11/2020
Date results information initially provided
12/10/2021
Type of registration
Retrospectively registered

Titles & IDs
Public title
A Trial in Healthy Participants to Evaluate the Pharmacokinetics, Safety, Tolerability and Food Effects of PRAX-562
Scientific title
A Phase 1, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Safety, Tolerability and Pharmacokinetics of Single and Multiple Ascending Doses and Food Effect of PRAX-562 in Healthy Volunteers
Secondary ID [1] 301309 0
PRAX-562-101
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Epilepsy 317494 0
Condition category
Condition code
Neurological 315595 315595 0 0
Epilepsy

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Participants will be confined at the site during the dosing period where dosing will be done via direct supervision and they can only participate in one of the following parts:
- Part A (oral capsule; screening/ baseline period of up to 28 days with total duration up to 39 days): single ascending doses of PRAX-562 between 2.5 mg and 90 mg (increases of no more than 3 times the previous dose will be based on safety and tolerability at each dose level) or placebo (Day 1) administered under fasting conditions
- Part B (oral capsule; screening/ baseline period of up to 28 days with total duration up to 47 days): single daily doses of up to 90 mg PRAX-562* or placebo (Day 1-14) administered under fasting conditions
- Part C (oral capsule; screening/ baseline period of up to 28 days with total duration up to 50 days): up to 90 mg PRAX-562* (Day 1 and 8) both fasted (no food or drink, except water, for at least 10 hours prior to dosing) and fed (30 minutes after a high-fat, high-calorie meal where approximately 50% of the calories will be from fat) in a randomized order
*Exact dose of PRAX-562 for Parts B and C will be based on safety, tolerability and PK data from Part A
Intervention code [1] 317609 0
Treatment: Drugs
Comparator / control treatment
Placebo capsules (microcrystalline cellulose capsule that is the same as PRAX-562 without the active ingredient) in Parts A and B only
Control group
Placebo

Outcomes
Primary outcome [1] 323829 0
Part A and B: Safety and tolerability of PRAX-562 will be assessed as a composite endpoint through an integrated analysis of the following: patient and clinician-reported adverse events, vital signs, clinical laboratory results (chemistry, haematology, urinalysis), electrocardiogram (ECG), and Columbia-Suicide Severity Rating Scale (C-SSRS)
Timepoint [1] 323829 0
Part A: Baseline, Day -1 to Day 6 (daily), Day 9 Part B: Baseline, Day -1 to Day 19 (daily), Day 24. This change was made after enrolment of 88 participants.
Primary outcome [2] 323830 0
Part C: The effects of food on pharmacokinetics will be performed by LC/MS quantification of PRAX-562 levels in plasma. Parameters include plasma concentrations and accumulation ratios (Cmax, tmax, AUC)
Timepoint [2] 323830 0
Part C: predose and 0.25, 0.5, 0.75, 1, 1.5, 2, 2.5, 3, 4, 6, 8, 12, 16, 24, 36, 48, 72, 96 and 120 hours post dose.
Secondary outcome [1] 383003 0
Pharmacokinetics will be performed by LC/MS quantification of PRAX-562 levels in plasma. Parameters include plasma concentrations and accumulation ratios (Cmax, tmax, AUC, t1/2).
Timepoint [1] 383003 0
Part A: predose and 0.25, 0.5, 0.75, 1, 1.5, 2, 2.5, 3, 4, 6, 8, 12, 16, 24, 36, 48, 72, 96 and 120 hours post dose.
Part B: 1: predose and 0.5, 0.75, 1, 1.5, 2, 2.5, 3, 4, 6, 8, 12, 16, 24, 36, 48, 72, 96 and 120 hours post dose.
Secondary outcome [2] 383004 0
Part C: Safety and tolerability of PRAX-562 will be assessed as a composite endpoint through an integrated analysis of the following: patient and clinician-reported adverse events, vital signs, clinical laboratory results (chemistry, haematology, urinalysis), electrocardiogram (ECG), and Columbia-Suicide Severity Rating Scale (C-SSRS)
Timepoint [2] 383004 0
Part C: Baseline, Day -1 to Day 13 (daily), 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).
- Medically healthy (without significant medical issues, e.g. high blood pressure)
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 findings, laboratory value or physical examination findings
- Positive ethanol, drug screen or 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 400 mL or more of 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
19/06/2020
Date of last participant enrolment
Anticipated
Actual
8/07/2021
Date of last data collection
Anticipated
Actual
28/07/2021
Sample size
Target
120
Accrual to date
Final
112
Recruitment in Australia
Recruitment state(s)
SA

Funding & Sponsors
Funding source category [1] 305746 0
Commercial sector/Industry
Name [1] 305746 0
Praxis Precision Medicines
Country [1] 305746 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] 306176 0
None
Name [1] 306176 0
Address [1] 306176 0
Country [1] 306176 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 306021 0
Bellberry Human Research Ethics Committees
Ethics committee address [1] 306021 0
123 Glen Osmond Rd
Eastwood SA 5063
Ethics committee country [1] 306021 0
Australia
Date submitted for ethics approval [1] 306021 0
08/04/2020
Approval date [1] 306021 0
08/06/2020
Ethics approval number [1] 306021 0

Summary
Brief summary
This clinical trial is to evaluate the PK, safety, tolerability and food effects of PRAX-562 in healthy participants aged 18 to 55 years inclusive.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 102446 0
Prof Sepehr Shakib
Address 102446 0
Department of Clinical Pharmacology
8F411
Royal Adelaide Hospital
North Terrace, ADELAIDE, South Australia, 5000
Country 102446 0
Australia
Phone 102446 0
+61 0411100278
Fax 102446 0
Email 102446 0
[email protected]
Contact person for public queries
Name 102447 0
Dr Bernard Ravina
Address 102447 0
Praxis Precision Medicines
One Broadway, 16th Floor
Cambridge, MA 02142
Country 102447 0
United States of America
Phone 102447 0
+1 617 300 8460
Fax 102447 0
Email 102447 0
[email protected]
Contact person for scientific queries
Name 102448 0
Dr Bernard Ravina
Address 102448 0
Praxis Precision Medicines
One Broadway, 16th Floor
Cambridge, MA 02142
Country 102448 0
United States of America
Phone 102448 0
+1 617 300 8460
Fax 102448 0
Email 102448 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.