Trial Review

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Trial registered on ANZCTR


Registration number
Help: Registration number
ACTRN12626000933358
Ethics application status
Help: Ethics application status
Approved
Date submitted
Help: Date submitted
22/04/2026
Date registered
Help: Date registered
29/07/2026
Date last updated
Help: Date last updated
29/07/2026
Date data sharing statement initially provided
Help: Data sharing statement date
29/07/2026
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 1 (Single Ascending Dose) and Part 3 (Multiple Ascending Dose)
Scientific title
Phase 1 Dose Escalation Study Assessing the Safety, Pharmacokinetics, and Pharmacodynamics of PTC612 in Adult Volunteers - Part 1 (Single Ascending Dose) and Part 3 (Multiple Ascending Dose)
Additional trial identifiers [1] 316968 0
Nil
Universal Trial Number (UTN)
Trial acronym
Related trial records

Health condition
Health condition(s) studied:
inflammatory diseases 341427 0
Condition category
Condition code
Inflammatory and Immune System 337485 337485 0 0
Other inflammatory or immune system disorders

Intervention/exposure
Study type
Interventional
Description of the intervention or exposure
Part 1 Single Ascending Dose (SAD):

Part 1 of the study will evaluate single ascending oral doses of PTC612 in healthy participants. PTC612 will be administered as a powder formulation for oral administration (powder is mixed with a liquid
vehicle prior to administration). This part is designed to assess the safety, tolerability, and pharmacokinetics of PTC612 following single-dose exposure across increasing dose levels.

The following dose levels may be explored and administered orally (note these are estimated
doses and subject to change):
• Group 1: 3 milligram doses of PTC612 or matching placebo
• Group 2: 10 milligram doses of PTC612 or matching placebo
• Group 3: 30 milligram doses of PTC612 or matching placebo
• Group 4: 90 milligram doses of PTC612 or matching placebo

In Part 1, the safety and pharmacokinetics (PK) of single doses of PTC612 will be evaluated in a randomized, double-blind, placebo-controlled design across approximately four dose groups in eligible participants.
In each Single Ascending Dose (SAD) cohort, a sentinel dosing approach will be implemented. For the initial two participants, randomization will assign one participant to receive PTC612 and the other to receive placebo.
For the remaining six participants within the same cohort, randomization will allocate five participants to receive PTC612 and one participant to receive placebo.

Each Single Ascending Dose (SAD) group will be dosed according to a sentinel dosing design to ensure optimal safety.
Unique Subject will be enrolled in each cohort

After each group completes dosing and PK blood sample collection, the Safety Review Committee (SRC) will review the available safety data, tolerability findings, and pharmacokinetic results through at least Day 3 (and up to Day 8). Based on this review, the Safety Review Committee (SRC) will determine the appropriate dose escalation for the subsequent group, including any required dose adjustments.

Each subsequent group, with unique subjects, will commence dosing approximately two weeks following final dose administered in the previous group.

Participants will receive study drug directly from the Investigator (or designee), under medical
supervision.

Part 3 Multiple Ascending Dose (MAD):

Unique participants will be enrolled in each part and each cohort. In Part 3, the safety and PK of multiple PTC612 doses will be assessed in a randomized, double-blind, placebo-controlled design with 3 dose groups in eligible participants. In each group, 8 participants will be randomized to treatments in a ratio of 6:2 (active: placebo). PTC612 is planned to be dosed as oral tablets in Part 3, although an oral powder may be used for dosing if needed (this will be determined based on the doses selected and agreed upon by the Safety Review Committee (SRC).

The first dose group in Part 3 may be initiated upon completion of Cohort 2 in Part 1 (i.e., 10 milligrams in Single Ascending Dose), if supported by the Safety Review Committee (SRC). The second dose group in Part 3 may be initiated upon completion of Cohort 3 in Part 1. In Part 3, PTC612 will be administered in the fasted condition.

Oral doses of PTC612 or matching placebo will be administered once daily in the morning from Day 1 through Day 14.

In each MAD cohort, participants will be randomized in a 3:1 ratio, with six participants receiving PTC612 and two receiving placebo. Sentinel dosing will not be implemented in Part 3 (Multiple Ascending Dose) of the study.

The starting dose for this part of the study will be chosen based on safety and drug-level results from earlier participants. Any increase in dose will only happen after careful review of safety data by the Study Review Committee to ensure the dose remains safe for participants. Maximum dose to be tested will be no more than 90mg.

Oral Powder will be dissolved in a liquid

Participants will receive study drug directly from the Investigator (or designee), under medical
supervision.
Intervention code [1] 333704 0
Treatment: Drugs
Description of the comparator or control
Placebo: The placebo/comparator treatment is microcrystalline cellulose, a white to off-white powder, which will be constituted into an oral suspension of 0.5 mg/mL.
The placebo/comparator treatment will be provided as an oral suspension to the site (CRU).
Route of administration- Oral
Dosage levels-
Part 1: 3, 10, 30, or 90 mg
Part 3: To be determined (In Part 3, a dose can only be used if the same dose, or a higher dose, has already been tested as a single dose in an earlier part of the study and was found to be acceptable.). The minimum dose level to be tested is 3mg and the maximum dose level to be tested is 90mg.
Control group
Placebo

Outcomes
Primary outcome [1] 344993 0
Safety and tolerability will be assessed by composite evaluation of PTC612 following single ascending dose by oral administration in healthy participants of Part 1 (single ascending doses)
Timepoint [1] 344993 0
Part 1: Clinical Laboratory: Screening (Day -28 to Day 1), Check-in (Day -1), Day 2 (Post dose), Day8/Discharge/End of Treatment. 12-Lead ECG: Screening (28 days pre-dose), Check-in (1 day pre-dose), Day 1 (pre and post dose), Day 2, Day 3, Day 4, Day 5, Day 6 & Day 7 (post-dose) and Day8/ Discharge/End of Treatment. Vital Signs: Check-In (1 day pre-dose), at Predose and at 0.5, 2, 5, 8, 12, and 24 hours post dose on Day 1, and on Day 2, Day 3, Day 4, Day 5, Day 6, & Day 7 (post-dose), Day 8/ Discharge/End of Treatment. Physical Examination: On Screening only (- 28 days pre-dose)
Primary outcome [2] 344994 0
Safety and tolerability will be assessed by composite evaluation of PTC612 following single ascending dose by oral administration in healthy participants of Part 3 (Multiple Ascending Doses (MAD))
Timepoint [2] 344994 0
Part 3: Clinical Laboratory: Screening (28 days pre-first dose), Check-in (1 day pre-first dose), Day 2, Day4, Day 7, Day 11, Day 14 and Day 21 (Discharge/End of Treatment). 12-Lead ECG: Screening (28 days pre-first dose), Check-in (1 day pre-first dose), Day 1 (pre-dose), Day 2-3, Day 4-13, Day 14, Day 15-20 & Day 21 (Discharge/End of Treatment). Vital Signs: Screening (28 days pre-first dose), Check-in (1 day pre-first dose), Day 1, Day 2-3, Day 4-13, Day 14, Day 15-20 & Day21 (Discharge/End of Treatment). Physical Examination: On Screening only (- 28 days pre-first dose)
Secondary outcome [1] 460077 0
Pharmacokinetics of PTC612 following single and multiple ascending doses of Part 1
Timepoint [1] 460077 0
0.5, 1, 2, 3, 4, 5, 6, 8, 10, 12, 24, 36, 48, 72, 96, 120, 144, and 168 hours postdose
Secondary outcome [2] 460079 0
Pharmacokinetics of PTC612 following multiple ascending doses administered orally in healthy participants of Part 3
Timepoint [2] 460079 0
0.5, 1, 2, 3, 4, 5, 6, 8, 10, 12, and 24 hours postdose on Days 1, 7, and 14 and additionally at 36, 48, 72, 96, 120, 144, and 168 hours after the last dose on Day 14.
Secondary outcome [3] 461565 0
Part 1 : Pharmacodynamics Single Ascending Doses
Timepoint [3] 461565 0
at predose and at 2, 4, 6, 24, 48, 72, 96, and 144 hours postdose
Secondary outcome [4] 461567 0
Part 3: Pharmacodynamics - Multiple Ascending Doses
Timepoint [4] 461567 0
predose and at 2, 4, 6, and 24 hours postdose on Days 1, 7, and 14 and additionally at 36, 48, 96, 120, and 168 hours after the last dose on Day 14

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 poppy seeds), 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. 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
25/05/2026
Date of last participant enrolment
Anticipated
4/08/2026
Actual
Date of last data collection
Anticipated
14/09/2026
Actual
Sample size
Target
56
Accrual to date
24
Final
Recruitment in Australia
Recruitment state(s)
SA
Recruitment hospital [1] 29370 0
CMAX Clinical Research Pty Ltd - Adelaide
Recruitment postcode(s) [1] 45688 0
5000 - Adelaide

Funding & Sponsors
Funding source category [1] 321566 0
Commercial sector/Industry
Name [1] 321566 0
PTC Therapeutics, Inc.
Country [1] 321566 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] 324352 0
None
Name [1] 324352 0
Address [1] 324352 0
Country [1] 324352 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 320096 0
Bellberry Human Research Ethics Committee A
Ethics committee address [1] 320096 0
Ethics committee country [1] 320096 0
Australia
Date submitted for ethics approval [1] 320096 0
11/03/2026
Approval date [1] 320096 0
16/04/2026
Ethics approval number [1] 320096 0

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

Contacts
Principal investigator
Name 149414 0
Dr Jonathan Newchurch
Address 149414 0
CMAX Clinical Research- Level 6, 21-24 North Terrace, Adelaide, South Australia 5000
Country 149414 0
Australia
Phone 149414 0
+61 423 223 756
Fax 149414 0
Email 149414 0
[email protected]
Contact person for public queries
Name 149415 0
Dr. Jonathan Newchurch
Address 149415 0
CMAX Clinical Research-Level 6,  21-24 North Terrace, Adelaide, South Australia 5000
Country 149415 0
Australia
Phone 149415 0
+61 423 223 756
Fax 149415 0
Email 149415 0
[email protected]
Contact person for scientific queries
Name 149416 0
Patient Advocacy
Address 149416 0
PTC Bio- 500 Warren Corporate Center Drive, Warren, New Jersey 07059
Country 149416 0
United States of America
Phone 149416 0
+1 866 562 4620
Fax 149416 0
Email 149416 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.