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Trial registered on ANZCTR


Registration number
ACTRN12623000300673
Ethics application status
Approved
Date submitted
7/03/2023
Date registered
17/03/2023
Date last updated
7/09/2023
Date data sharing statement initially provided
17/03/2023
Type of registration
Prospectively registered

Titles & IDs
Public title
First in human clinical study of a novel drug PTC607 to assess effects of food intake and to compare PTC607 levels, when taken as an oral suspension and tablet formulation.
Scientific title
A phase 1, first in human study to assess the pharmacokinetics of single oral doses of PTC607 in the fasted and fed states and to evaluate the comparative bioavailability of 2 formulations of PTC607 in healthy volunteers.
Secondary ID [1] 308932 0
None
Universal Trial Number (UTN)
Trial acronym
Linked study record
This record is a sub-study of ACTRN12622001534774

Health condition
Health condition(s) or problem(s) studied:
Huntington's Disease 328939 0
Condition category
Condition code
Neurological 325926 325926 0 0
Neurodegenerative diseases
Human Genetics and Inherited Disorders 325927 325927 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
This study in an open-label, fixed sequence, 3 period crossover relative bioavailability study of 2 different formulations followed by a food effect assessment of PTC607 in 8 healthy participants. The dose of PTC607 will be determined based on at least the data from Cohort 1.1 and Cohort 1.2 from ACTRN12622001534774 study. The periods will be separated by a minimum of 5 half-lives (approximately 110 hours).
In Period 1, the suspension formulation of PTC607 will be orally administered after an overnight fast of at least 10 hours. Participants will remain fasted until 4 hours post dose.
In Period 2, the tablet dose of PTC607 will be administered after an overnight fast of at least 10 hours. Participants will remain fasted until 4 hours post dose.
In Period 3, the final tablet dose of PTC607 will be administered after participants have consumed a high fat meal. PTC607 will be administered 30 minutes after the start of the assigned breakfast. At least 90% of the meal must be consumed within 30 minutes of beginning.
The standardised meal will be the standard US Food and Drug Administration high-fat, high calorie (800 to 1000 calories) breakfast. Approximately 50% of total caloric content of the meal will be from fat. The meal will derive approximately 150, 250, and 500-600 calories from protein, carbohydrate, and fat, respectively.
Healthy volunteers will stay in the clinical research unit for the entire duration of dosing and will take study drug under direct supervision of the clinical research unit staff .
Intervention code [1] 325378 0
Treatment: Drugs
Comparator / control treatment
Oral suspension in fasted state (Period 1) will serve as the control treatment.
Control group
Active

Outcomes
Primary outcome [1] 333772 0
Bioavailability of single doses of PTC607 suspension and tablet in healthy participants following a single dose under fasted condition through the comparison of PK parameters (Cmax, Tmax, AUC, t1/2 measured in plasma).
Timepoint [1] 333772 0
Pre-dose, 0.25 hr, 0.5 hr, 1 hr, 2 hr, 3 hr, 4 hr, 6 hr, 8 hr and 12 hr post dose on Day 1 of each treatment period; 24 hr and 36 hr post dose on Day 2 of each treatment period, and 48 hr post dose on Day 3 of each treatment period.
Primary outcome [2] 333773 0
Pharmacokinetics (Cmax, Tmax, AUC, t1/2 measured in plasma) of single doses of PTC607 administered in the fed state through comparison with fasted states in healthy participants
Timepoint [2] 333773 0
Pre-dose, 0.25 hr, 0.5 hr, 1 hr, 2 hr, 3 hr, 4 hr, 6 hr, 8 hr and 12 hr post dose on Day 1 of each treatment period; 24 hr and 36 hr post dose on Day 2 of each treatment period, and 48 hr post dose on Day 3 of each treatment period.
Secondary outcome [1] 418247 0
Safety and tolerability of single oral dose of PTC607 as suspension and tablet formulation through review of:
- vital signs (blood pressure and heart rate assessed by sphygmomanometer, body temperature by thermometer)
- 12-lead electrocardiograms
- clinical safety laboratory tests: serum chemistry (including liver function tests, electrolytes, kidney function tests, cholesterol and triglycerides), full blood count and coagulation tests will be assessed using whole blood sample, urinalysis will be assessed using urine sample.
- treatment emergent adverse events (including events leading to premature discontinuation of study drug)
Timepoint [1] 418247 0
Vital signs: Screening (Day -28 to -2); Day -1, post dose on Day 1, Day 2 and Day 3 of each treatment period and at early discontinuation visit.
12-lead electrocardiograms: Screening (Day -28 to -2); Day -1, post dose on Day 1 and Day 3 of each treatment period and at early discontinuation visit
- Clinical safety laboratory tests: Screening (Day -28 to -2); Day -1 and post dose on Day 3 of each treatment period and at early discontinuation visit.
- Adverse events: continuously from admission (Day -1 of Treatment Period 1) until discharge on post dose Day 3 of Treatment Period 3.
Secondary outcome [2] 418249 0
Safety and tolerability of PTC607 administered in the fed and fasted states through review of:
- vital signs (blood pressure and heart rate assessed by sphygmomanometer, body temperature by thermometer)
- 12-lead electrocardiograms
- clinical safety laboratory tests: serum chemistry (including liver function tests, electrolytes, kidney function tests, cholesterol and triglycerides), full blood count and coagulation tests will be assessed using whole blood sample, urinalysis will be assessed using urine sample.
- treatment emergent adverse events (including events leading to premature discontinuation of study drug)
Timepoint [2] 418249 0
Vital signs: Screening (Day -28 to -2); Day -1, post dose on Day 1, Day 2 and Day 3 of each treatment period and at early discontinuation visit.
12-lead electrocardiograms: Screening (Day -28 to -2); Day -1, post dose on Day 1 and Day 3 of each treatment period and at early discontinuation visit
- Clinical safety laboratory tests: Screening (Day -28 to -2); Day -1 and post dose on Day 3 of each treatment period and at early discontinuation visit.
- Adverse events: continuously from admission (Day -1 of Treatment Period 1) until discharge on post dose on Day 3 of Treatment Period 3.

Eligibility
Key inclusion criteria
1. Males and females aged 18 to 65 years inclusive at Screening
2. Participants able to provide informed consent
3. Body mass index of greater than or equal to 18.5 and less than or equal to 30.0 kg/m2 with a body weight of greater than or equal to 50.0 kg for male
participants and a body weight of greater than or equal to 45.0 kg for female participants at Screening
4. Generally healthy as determined by the investigator based on medical evaluation, including medical history, physical examination, laboratory test results, ECG, and vital signs.
5. Male participants must be willing to use 2 acceptable contraceptive methods for the duration of the study and for a minimum of 6 months after the last dose, and female participants of childbearing potential must be willing to use 2 acceptable contraceptive methods for the duration of the study and for a minimum of 30 days after the last dose.
6. All female participants of childbearing potential must have a negative serum pregnancy
test result at Screening and a negative urine pregnancy test on Day -1.
7. Male participants must agree to not donate sperm for the duration of the study and for at
least 6 months after the last dose.
8. Participants must be willing and able to consume the entire high-fat breakfast in the designated timeframe. Participants with allergies or with vegetarian or vegan lifestyle choices that would prevent them from eating these breakfasts will be excluded.
Minimum age
18 Years
Maximum age
65 Years
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
1. Participants that participated in any drug or device clinical investigation within 60 days
prior to Screening or who anticipate participating in any drug or device clinical
investigation within the duration of this study.
2. Prior or ongoing medical condition (eg, concomitant illness, psychiatric condition),
medical history, and/or physical findings that, in the investigator’s opinion, could
adversely affect the safety of the participant or could impair the assessment of study
results.
3. An abnormal general neurological examination.
4. Presence of any clinically significant abnormality during Screening.
5. Any psychological or emotional problems, any disorders, or resultant therapy that is
likely to invalidate informed consent or limit the ability of the participant to comply with
the protocol requirements.
6. A positive hepatitis B surface antigen, positive hepatitis C antibody, or HIV antibody
result at Screening.
7. Donation of plasma within 7 days prior to dosing. Donation or loss of blood (excluding
volume drawn at Screening or menses) of 50 to 499 mL within 30 days or more than
499 mL within 56 days prior to dosing.
8. Excessive alcohol consumption (regular alcohol intake greater than or equal to 21 units per week for male
participants and greater than or equal to 14 units per week for female participants) within 6 months prior to
Screening. One unit (8 g) is equivalent to a half pint (280 mL) of beer, 1 measure
(25 mL) of spirits, or 1 small glass (125 mL) of wine.
9. The participant is a smoker or uses other nicotine-containing products. Ex-smokers must
have ceased smoking >3 months prior to Screening. Smokers who consume <4 tobacco
products per week are allowed.
10. The participant has consumed grapefruit (or its juice), star fruit, pomegranate, pomelo,
tangelo, or Seville orange-containing products in the 1 week before Screening.
11. A positive urine drug screen, cotinine screen, or alcohol breath test at Screening or on
Day -1 of each treatment period.
12. Females who are pregnant or nursing.
13. Participant has previously received PTC607.

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?



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

Recruitment
Recruitment status
Completed
Date of first participant enrolment
Anticipated
11/04/2023
Actual
17/04/2023
Date of last participant enrolment
Anticipated
11/04/2023
Actual
7/07/2023
Date of last data collection
Anticipated
19/05/2023
Actual
18/08/2023
Sample size
Target
8
Accrual to date
Final
8
Recruitment in Australia
Recruitment state(s)
SA
Recruitment hospital [1] 23930 0
CMAX Clinical Research Pty Ltd - Adelaide
Recruitment postcode(s) [1] 39423 0
5000 - Adelaide

Funding & Sponsors
Funding source category [1] 313142 0
Commercial sector/Industry
Name [1] 313142 0
PTC Therapeutics, Inc.
Country [1] 313142 0
United States of America
Primary sponsor type
Commercial sector/Industry
Name
CTI Clinical Trial and Consulting Services Australia Pty Ltd.
Address
Level 21, 207 Kent Street,
Sydney NSW 2000
Country
Australia
Secondary sponsor category [1] 314844 0
None
Name [1] 314844 0
Address [1] 314844 0
Country [1] 314844 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 312385 0
Bellberry Human Research Ethics Committee
Ethics committee address [1] 312385 0
123 Glen Osmond Road, Eastwood SA 5063
Ethics committee country [1] 312385 0
Australia
Date submitted for ethics approval [1] 312385 0
03/03/2023
Approval date [1] 312385 0
12/04/2023
Ethics approval number [1] 312385 0

Summary
Brief summary
This will be a phase 1, first-in-human, open-label, 3-period, 3-treatment crossover design study to evaluate the food effects and relative bioavailability of 2 formulations of PTC607 in 8 healthy participants.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 124474 0
Prof Sepehr Shakib
Address 124474 0
CMAX Clinical Research,
Level 5, 21 North Terrace,
Adelaide SA 5000
Country 124474 0
Australia
Phone 124474 0
+61 411100278
Fax 124474 0
Email 124474 0
[email protected]
Contact person for public queries
Name 124475 0
Sepehr Shakib
Address 124475 0
CMAX Clinical Research,
Level 5, 21 North Terrace,
Adelaide SA 5000
Country 124475 0
Australia
Phone 124475 0
+61 411100278
Fax 124475 0
Email 124475 0
[email protected]
Contact person for scientific queries
Name 124476 0
Amy-Lee Bredlau
Address 124476 0
PTC Therapeutics, Inc.
100 Corporate Court
South Plainfield, NJ 07080
Country 124476 0
United States of America
Phone 124476 0
+1 914 707 2785
Fax 124476 0
Email 124476 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


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.