Trial Review

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Trial registered on ANZCTR


Registration number
Help: Registration number
ACTRN12626001204336
Ethics application status
Help: Ethics application status
Approved
Date submitted
Help: Date submitted
9/09/2026
Date registered
Help: Date registered
29/09/2026
Date last updated
Help: Date last updated
29/09/2026
Date data sharing statement initially provided
Help: Data sharing statement date
29/09/2026
Type of registration
Help: Type of registration
Retrospectively registered

Titles & IDs
Public title
A Study in Adult Volunteers to Understand the Safety and Effects of a New Drug, PTC612 - Part 4 (Biomarker Evaluation in Participants With Elevated Baseline Inflammation)
Scientific title
Phase 1 Dose Escalation Study Assessing the Safety, Pharmacokinetics, and Pharmacodynamics of PTC612in Adult Volunteers. Part 4 (Biomarker Evaluation in Participants With Elevated Baseline Inflammation)
Additional trial identifiers [1] 317990 0
PTC612
Universal Trial Number (UTN)
Trial acronym
Related trial records
Part1&3: ACTRN12626000933358
Part2: ACTRN12626000934347

Health condition
Health condition(s) studied:
inflammatory diseases 343202 0
Condition category
Condition code
Inflammatory and Immune System 339293 339293 0 0
Other inflammatory or immune system disorders

Intervention/exposure
Study type
Interventional
Description of the intervention or exposure
Part 4 of the study involves a multiple-dose evaluation of PTC612 compared with placebo in eligible participants in up to 3 cohorts . Following a screening period of up to 28 days, participants who meet eligibility criteria will be admitted to the study site on Day -1. Participants will receive once-daily oral doses of PTC612 or matching placebo from Day 1 through Day 14 with a starting dose of 10mg PTC612. The maximum dose to be tested in this Part is expected to be no more than 50mg.

Pharmacokinetic (PK) and pharmacodynamic (PD) blood samples will be collected according to the Schedule of Activities (SoA), with the possibility of adjusting collection timepoints based on data generated from Parts 1, 2,(ACTRN12626000933358) and 3(ACTRN12626000934347). PD assessments will include inflammatory biomarkers such as hs-CRP, ESR, IL-1ß, IL-6, IL-18, fibrinogen, and lipoprotein(a) [Lp(a)], along with other relevant inflammation markers if required.

Participants will remain at the study site until Day 21 and will be discharged after completing all study procedures. A follow-up safety phone call will be conducted approximately 4 weeks (±4 days) after the last study drug dose.

After each cohort completes the required dosing and PK assessments, the Safety Review Committee (SRC) will review available safety, tolerability, and PK data through at least Day 8 (up to Day 21). Based on these findings, the SRC will determine whether dose escalation should proceed and whether any dose modifications are necessary. Dose escalation will continue unless predefined stopping criteria are met or the Sponsor decides otherwise. Active is a white to off-white, round tablet in strengths of 5mg and 20 mg.
Intervention code [1] 335063 0
Treatment: Drugs
Description of the comparator or control
Placebo is white to off-white, round tablet matched to the 5mg and 20 mg strengths.
Route of administration- Oral
Control group
Placebo

Outcomes
Primary outcome [1] 346541 0
Type, frequency, severity, timing, and relationship to study intervention of any AEs and abnormalities in clinical laboratory, ECGs, vital signs, and/or physical examinations when PTC612 is administered.
Timepoint [1] 346541 0
Clinical Laboratory: Screening (28 days pre-first dose), Check-in (1 day pre-first dose), Day 2, Day 4, Day 7, Day 11, Day 14 and Day 21 post-first dose (Discharge/End of Treatment). 12-Lead ECG: Screening (28 days pre-first dose), Check-in (1 day pre-first dose), Day 1 (45, 30 &15 minutes pre-dose and 0.5, 1, 2, 3, 4, 5, 6, 8, 10, & 12 hrs post-dose), Days 1-6 (pre-dose), Day 7 (pre-dose and 0.5, 1, 2, 3, 4, 5, 6, 8, 10, & 12 hrs post-dose), Days 8-13 (pre-dose), Day 14 (pre-dose and 0.5, 1, 2, 3, 4, 5, 6, 8, 10, & 12 hrs), Day 15 (24 & 26hrs post last dose), Day 16 (48hrs post last dose), Day 17 (72hrs post last dose), Day 18 (96 hrs post last dose), Day 18 (96hrs post last dose), Day 19 (120hrs post last dose), Day 20 (144hrs post last dose), and Day 21 (168hrs post last dose) (Discharge/End of Treatment). Vital Signs: Screening (28 days pre-first dose), Check-in (1 day pre-first dose), Day 1 (pre-dose and 05, 2, 5, 8 and 12 hrs post-dose), Days 2-6 (pre-dose), Day 7 (pre-dose and 05, 2, 5, 8 and 12 hrs post-dose), Days 8-13 (pre-dose), Day 14 (pre-dose and 05, 2, 5, 8 and 12 hrs post-dose), Day 15 (24 & 26hrs post last dose), Day 16 (48hrs post last dose), Day 17 (72hrs post last dose), Day 18 (96 hrs post last dose), Day 18 (96hrs post last dose), Day 19 (120hrs post last dose), Day 20 (144hrs post last dose), and Day 21 (168hrs post last dose) (Discharge/End of Treatment). Physical Examination: On Screening only (- 28 days pre-first dose)
Secondary outcome [1] 466315 0
Change from baseline in endogenous blood concentration of high-sensitivity C-reactive protein (hs-CRP).
Timepoint [1] 466315 0
Day 1 pre-dose, 4hr & 24hrs postdose, Day 4 predose, Day 5 predose, Day 6 predose, Day 7 predose, 4hr & 24hrs postdose, Day 11 predose, Day 12 predose, Day 13 predose, Day 14 predose, 4hr, 24hr, 48hr, 96hr, & 168hr postdose.
Secondary outcome [2] 466316 0
PTC612 plasma Pharmacokinetic (PK) parameters (including, but not limited to, Cmax, AUC0-t, AUC0-inf, Tmax, and T1/2).
Timepoint [2] 466316 0
Day 1, Day 7, and Day 14 Predose (between waking up and dosing), 0.5hour postdose, 1hour postdose, 2hours postdose, 3hours postdose, 4hours postdose, 5hours postdose, 6hours postdose, 8hours postdose, 10hours postdose, 12hours postdose, 24hours postdose (corresponds to Day 2 predose for Day 1 dosing and Day 8 predose for Day 7 dosing), and at 36hr, 48hr, 72hr, 96hr, 120hr, 144hr, and 168hrs post Day 14 dose. Single predose sample collected on: Day 3, Day 4, Day 5, Day 6, Day 9, Day 10, Day 11, Day 12, Day 13,

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. Participants must have a BMI greater than 30 kg/m² and less than or equal to 42 kg/m² at Screening. Additionally, participants must demonstrate relative weight stability during the 6 weeks prior to Screening, as determined by the Investigator or their delegate.
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.
12. Participants must be generally healthy, with the exception of the following required conditions: obesity (as specified in Inclusion Criterion #3), at least one cardiovascular risk factor (diagnosed hypertension or hypercholesterolemia), and elevated baseline inflammation (hs-CRP greater than or equal to 2 mg/L). Participants with type 2 diabetes mellitus, obstructive sleep apnea, gastroesophageal reflux disease (GERD), hypothyroidism, need for hormonal replacement therapy (HRT) (eg.menopause or perimenopause) and/or osteoarthritis are eligible to participate in the study.
13. The use of metformin, proton pump inhibitors (PPIs), levothyroxine, HRT, low-dose aspirin and cardiovascular medications prescribed for hypertension or hypercholesterolemia is permitted, provided the participant has been on a stable regimen for at least 28 days prior to study participation.
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. computerised sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s

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
7/08/2026
Date of last participant enrolment
Anticipated
28/01/2027
Actual
Date of last data collection
Anticipated
15/03/2027
Actual
Sample size
Target
54
Accrual to date
1
Final
Recruitment in Australia
Recruitment state(s)
SA
Recruitment outside Australia
Country [1] 28508 0
New Zealand
State/province [1] 28508 0
Auckland and Christchurch

Funding & Sponsors
Funding source category [1] 322539 0
Commercial sector/Industry
Name [1] 322539 0
PTC Therapeutics, Inc.
Country [1] 322539 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] 325461 0
None
Name [1] 325461 0
Address [1] 325461 0
Country [1] 325461 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 321021 0
Bellberry Human Research Ethics Committee A
Ethics committee address [1] 321021 0
Ethics committee country [1] 321021 0
Australia
Date submitted for ethics approval [1] 321021 0
11/03/2026
Approval date [1] 321021 0
16/04/2026
Ethics approval number [1] 321021 0
Ethics committee name [2] 321374 0
Central Health and Disability Ethics Committee
Ethics committee address [2] 321374 0
Ethics committee country [2] 321374 0
New Zealand
Date submitted for ethics approval [2] 321374 0
30/07/2026
Approval date [2] 321374 0
20/08/2026
Ethics approval number [2] 321374 0
2026 FULL 26454

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

Contacts
Principal investigator
Name 152394 0
Dr Jonathan Newchurch
Address 152394 0
CMAX Clinical Research- Level 6, 21-24 North Terrace, Adelaide, South Australia 5000
Country 152394 0
Australia
Phone 152394 0
+61 423 223 756
Fax 152394 0
Email 152394 0
[email protected]
Contact person for public queries
Name 152395 0
Jonathan Newchurch
Address 152395 0
CMAX Clinical Research- Level 6, 21-24 North Terrace, Adelaide, South Australia 5000
Country 152395 0
Australia
Phone 152395 0
+61 423 223 756
Fax 152395 0
Email 152395 0
[email protected]
Contact person for scientific queries
Name 152396 0
Patient Advocacy
Address 152396 0
PTC Bio- 500 Warren Corporate Center Drive, Warren, New Jersey 07059
Country 152396 0
United States of America
Phone 152396 0
+1 866 562 4620
Fax 152396 0
Email 152396 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.