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Trial registered on ANZCTR


Registration number
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ACTRN12626000934347
Ethics application status
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Approved
Date submitted
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29/06/2026
Date registered
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29/07/2026
Date last updated
Help: Date last updated
29/07/2026
Date data sharing statement initially provided
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29/07/2026
Type of registration
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Prospectively registered

Titles & IDs
Public title
A Study in Adult Volunteers to Understand the Safety and Effects of a New Drug, PTC612 with Food Effect (Part2)
Scientific title
Phase 1 Dose Escalation Study Assessing the Safety, Pharmacokinetics, and Pharmacodynamics of PTC612in Adult Volunteers with effect of Food (Part2)
Additional trial identifiers [1] 317560 0
Nil
Universal Trial Number (UTN)
Trial acronym
Related trial records

Health condition
Health condition(s) studied:
inflammatory diseases 342309 0
Condition category
Condition code
Inflammatory and Immune System 338425 338425 0 0
Other inflammatory or immune system disorders

Intervention/exposure
Study type
Interventional
Description of the intervention or exposure
Unique participants will be enrolled in each part and each cohort, part 2 of the study consists of two cohorts of eligible participants to evaluate food effects on the PTC612 tablet:

• Cohort 2A: tablet under fasted condition vs tablet under fed condition – standard meal
• Cohort 2B: tablet under fasted condition vs tablet under fed condition – high-fat high-calorie (HFHC) meal

Each cohort will use an open-label, randomized, 2-sequence, 2-period, 2-treatment, 2-way cross over design. Approximately 28 participants will be enrolled in Part 2.

Cohorts 2A and 2B may begin in parallel once the next higher dose level in Part 1 (Single Ascending Dose) is well tolerated (ie, 30 mg). Within each cohort, participants will be randomized 1:1 to one of two treatment sequences (N=7 per sequence), as follows:
Cohort 2A:
• Sequence 1: Fasted (Period 1) and Fed with standard meal (Period 2)
• Sequence 2: Fed with standard meal (Period 1) and Fasted (Period 2)
Cohort 2B:
• Sequence 1: Fasted (Period 1) and Fed with HFHC meal (Period 2)
• Sequence 2: Fed with HFHC meal (Period 1) and Fasted (Period 2)
Washout period between Sequences will be 2 weeks.

Tablet fed (standard meal): Participants will receive a single 10 mg dose of PTC612 following an overnight fast of at least 10 hours, administered approximately 30 minutes after the start of a standard meal.

Tablet fed (High Fat, High-Calorie meal): Participants will receive a single 10 mg dose of PTC612 following an overnight fast of at least 10 hours, administered approximately 30 minutes after the start of a high-fat,high-calorie (HFHC) meal. The meal must be completely consumed within 30 minutes, prior to dosing. No food is permitted for 4 hours post-dose. Water intake restrictions will be the same as those under the fasted condition.
Composition of the Standard Meal -
Total Calories: - 600-800kcal
Calories from Protein: - 60-280kcal
Calories From Carbohydrates:- 270-520kcal
Calories From Fat: 210-280kcal
High Fat, High Calorie Meal
Total Calories: - 800-1000kcal
Calories from Protein: - 150kcal
Calories From Carbohydrates :- 250kcal
Calories From Fat : 500-600kcal

Participants will receive study drug directly from the Investigator (or designee), under medical supervision.
Intervention code [1] 334357 0
Treatment: Drugs
Description of the comparator or control
Tablet, fasted: Participants will receive a single 10 mg dose of PTC612 following an overnight fast of at least 10 hours. Food intake is not permitted for 4 hours after dosing. Water may be consumed up to 1 hour before dosing and from 1 hour after dosing ad libitum, except for the water taken with the dose.
Control group
Active

Outcomes
Primary outcome [1] 345741 0
Safety and tolerability of PTC612 following administration of single oral dose in fed and fasting condition of Part 2
Timepoint [1] 345741 0
Clinical Laboratory: Period 1: Screening (28 days pre-first dose), Check-in (1 day pre-first dose) Period 2: Day 14, Day 21, & End of Treatment/Day28 AE: Period 1: Screening (28 days pre-first dose), Check-In (1 day pre-first dose), then daily from first dose to day 13 post-first dose, Follow up (4 Weeks ±4 Days After Last Dose) Period2: Day14, Day15, Day16, Day17, Day18, Day 19, Day 20, Day 21, Day 22, Day 23, Day24-27, Day 28 (End of Treatment), Follow up ( 4 Weeks ±4 Days After Last Dose) Vital Signs: Screening (28 days pre-first dose), Check-In (1 day pre-first dose), Day1, Day2, Day3, Day5, Day7, Day 8, Day 9 & Day 28 (End of Treatment).
Secondary outcome [1] 463122 0
Pharmacokinetics of PTC612 following a single oral dose under fed and fasted conditions in healthy participants of Part 2
Timepoint [1] 463122 0
Period 1: 0.5, 1, 2, 3, 4, 5, 6, 8, 10, 12, 24, 36, and 48 h, 96, 144 & 192 hours post dose in both treatment period Period 2: Checkin, 320hour or Day 14 post dose

Eligibility
Key inclusion criteria
1. Participant must be between 18 to 65 years of age.
2. Be generally healthy, as determined by medical history, physical examination, blood and urine tests, heart test (ECG), and vital signs.
3. Body mass index BMI greater than 18.5 and less than or equal to 32.0 kilograms per square meter with a body weight of at least 50.0 kilograms for males or at least 45.0 kilograms for females at screening.
4. Have read, understood, and signed the informed consent form.
5. Be willing and able to attend all study visits and follow all study instructions and restrictions.
6. Be willing to stop most medications, supplements, and herbal products before the study, as required by the study doctor.
7. Be willing to avoid alcohol, nicotine products, caffeine, certain foods (including grapefruit and poppyseeds), and strenuous exercise for specified periods before and during the study.
8. Have negative screening tests for alcohol, drugs of abuse, and nicotine.
9. Women:
•Must have negative pregnancy tests if able to become pregnant.
•Must agree to use reliable contraception or abstain from sexual activity during the study and for a short time after the last dose.
•Must not donate eggs during the study and shortly after.
10. Men:
•Must agree to use barrier contraception if sexually active with women who could become pregnant.
•Must not donate sperm during the study and for a period after the last dose.
11. For study parts requiring food before dosing, Participants must be willing and able to consume the entire high-fat high-calorie or standard meal in the designated timeframe.
Minimum age
18 Years
Maximum age
65 Years
Sex
Both males and females
Healthy volunteers
Yes
Key exclusion criteria
1. Have taken part in another clinical trial involving a drug or medical device within the last 30 days or plan to take part in another trial during this study.
2. Have any current or past medical or mental health condition that the study doctor believes could affect safety or interfere with study results.
3. Had a fever or symptoms of a viral or bacterial infection within 7 days before screening.
4. Have had cancer within the last 5 years, unless it was surgically removed and considered cured.
5. Have a history of severe allergic reactions or serious drug allergies.
6. Have any clinically significant abnormal findings at screening, including abnormal liver test results.
7. Have abnormal liver blood test results above accepted limits at screening.
8. Have a history of tuberculosis or a positive tuberculosis test at screening.
9. Have psychological or emotional conditions that may affect informed consent or ability to follow study requirements.
10. Are pregnant, breastfeeding, or planning to become pregnant or breastfeed during the study.
11. Have donated whole blood within the last 3 months, plasma within the last 2 weeks, or platelets within the last 6 weeks.
12. Have consumed alcohol within 48 hours before screening or study check in visits.
13. Have a history of alcohol abuse within the last 2 years.
14. Have evidence of active hepatitis B, hepatitis C, or human immunodeficiency virus infection at screening.

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)
The randomization is to be performed by an external vendor and will adhere to the vendor’s
processes.
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Simple randomisation using a randomisation table created by computer software (i.e. computerisedsequence 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
Not yet recruiting
Date of first participant enrolment
Anticipated
1/01/2027
Actual
Date of last participant enrolment
Anticipated
1/02/2027
Actual
Date of last data collection
Anticipated
15/03/2027
Actual
Sample size
Target
28
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
SA

Funding & Sponsors
Funding source category [1] 322116 0
Commercial sector/Industry
Name [1] 322116 0
PTC Therapeutics, Inc.
Country [1] 322116 0
United States of America
Primary sponsor type
Commercial sector/Industry
Name
PTC Therapeutics, Inc.
Address
Country
United States of America
Secondary sponsor category [1] 325008 0
None
Name [1] 325008 0
Address [1] 325008 0
Country [1] 325008 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 320645 0
Bellberry Human Research Ethics Committee A
Ethics committee address [1] 320645 0
Ethics committee country [1] 320645 0
Australia
Date submitted for ethics approval [1] 320645 0
11/03/2026
Approval date [1] 320645 0
16/04/2026
Ethics approval number [1] 320645 0

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 151158 0
Dr Jonathan Newchurch
Address 151158 0
CMAX Clinical Research- Level 6, 21-24 North Terrace, Adelaide, South Australia 5000
Country 151158 0
Australia
Phone 151158 0
+61 423 223 756
Fax 151158 0
Email 151158 0
[email protected]
Contact person for public queries
Name 151159 0
Jonathan Newchurch
Address 151159 0
CMAX Clinical Research- Level 6, 21-24 North Terrace, Adelaide, South Australia 5000
Country 151159 0
Australia
Phone 151159 0
+61 423 223 756
Fax 151159 0
Email 151159 0
[email protected]
Contact person for scientific queries
Name 151160 0
Patient Advocacy
Address 151160 0
PTC Bio- 500 Warren Corporate Center Drive, Warren, New Jersey 07059
Country 151160 0
United States of America
Phone 151160 0
+1 866 562 4620
Fax 151160 0
Email 151160 0
[email protected]

Data sharing statement
Will the study consider sharing individual participant data?
No


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.