Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12625000595415
Ethics application status
Approved
Date submitted
20/05/2025
Date registered
10/06/2025
Date last updated
10/06/2025
Date data sharing statement initially provided
10/06/2025
Type of registration
Prospectively registered

Titles & IDs
Public title
A first in human clinical study to check the effect of food on the safety, tolerability and levels of PTC844 in blood, when taken in fasted and fed state by a healthy person.
Scientific title
Phase 1 Adaptive Design Food Effect Study to Characterize the Pharmacokinetics, Safety, Tolerability, and Pharmacodynamics of PTC844 Administered Fasted or Fed state in healthy participants.
Secondary ID [1] 314402 0
PTC844-SMAD-102-HV
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Immunological Disorders 337421 0
Condition category
Condition code
Inflammatory and Immune System 333799 333799 0 0
Autoimmune diseases
Inflammatory and Immune System 333803 333803 0 0
Other inflammatory or immune system disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
This is a Phase 1 study evaluating effect of food on the PK of PTC844 tablets (20mg) single administration under fasted and 2 kinds of fed conditions (standard meal and high-fat meal).
• Arm 1: Fasted condition followed by standard meal (fed) condition
• Arm 2: Fasted condition followed by high-fat, high-calorie (fed) condition
Participants will be dosed after a fast of at least 10 hours followed by which they will not consume any food for another 4 hours. The drug will be administered in clinic and date and time of each dose will be recorded. After 21 days of washout, they will be dosed along with either
Arm 1: Standard meal (fed) condition where they will be dosed after an overnight fast of 10 hours and 30 min post start of a standard meal (600-800 kcal). OR
Arm 2. High-fat, high-calorie (fed) condition where they will be dosed after an overnight fast of at least 10 hours and after 30 minutes from the start of consuming a high-fat, high-calorie meal (800-1000 kcal).
The study is adaptive design because it provides internal study data to make decisions and modifications within the study. Dosing in Period 2 Arm 2 with high fat meal will depend on food effect increase in exposure after standard meal. We do have an option to reduce the dose if we expect higher than safe levels of exposure with high fat meal.
As per US Food and Drug Administration, a standard meal is 600-800 kcal, where approximately 50% is carbohydrates and other 50% are similar amount of fat and protein. Whereas a high-fat, high-calorie meal includes 800-1000 kcal of which roughly 60% fat, 25% carbohydrate and 15% protein.
Intervention code [1] 331018 0
Treatment: Drugs
Comparator / control treatment
Drug in fasted state will be considered as control.
Control group
Active

Outcomes
Primary outcome [1] 341397 0
To characterize the effect of food on the pharmacokinetics (PK) of single doses of PTC844 tablets by calculating PK parameters (Cmax, Tmax, AUC).
Timepoint [1] 341397 0
Blood for PK analysis of PTC844 in plasma will be drawn at pre dose and at 0.5, 1, 2, 3, 4, 6, 7, 8, 9, 10, 11, 12, 16, 24, 36, 48, 60, 72, 84, and 96 hours post dose on Days 1 and 23 (ie, while the participant is at their inpatient stay). Blood for PK analysis of PTC844 will also be taken once at 168, 264, 336, 432, and 504 hours post dose in both Treatment Periods (ie, at outpatient visits). The 504-hour post dose sample for Treatment Period 1 will be taken during admission on Day 22 for Treatment Period 2.
Secondary outcome [1] 447386 0
To characterize safety and tolerability of single doses of PTC844 tablets in fasted and fed states on Days 1 and 23 by observing 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 PTC844 is administered in fasted and fed states in a composite manner.
Timepoint [1] 447386 0
ECGs will be obtained at Screening (Days -28 to -2), Check-In (Days -1 and 22), at pre dose and at 0.5, 1, 2, 3, 4, 6, 7, 8, 9, 10, 11, 12, 16, and 24 hours post dose on Day 1 and 23, and at Discharge. Vital signs will be assessed at Screening (Days -28 to -2), at Check-in (Days -1 and Day 22), at pre dose and at 0.5, 2, 5, 8, 12, and 24 hours post dose on Day 1 and Day 23, Full physical examination will be conducted at Screening, at Check-In (Days -1 and 22), and on Days 8, 15, 30, 37, and 44. Vital signs will include systolic and diastolic blood pressure, oxygen saturation, pulse rate, respiratory rate, and body temperature. Adverse Events will be assessed from screening (Day-28) to follow up (4 weeks post dose). Clinical laboratory tests will include clinical chemistry, hematology, and urinalysis and will be performed at screening (Days -28 to -2), Check-In (Days -1 and 22), Days 1 (12 hours post dose), 2, 3, 4, 8, 12, 15, 19, 23, 24, 25, 26, 30, 34, 37, 41 and 44.

Eligibility
Key inclusion criteria
1. Evidence of signed and dated informed consent document(s) indicating that the study candidate has been informed of all pertinent aspects of the study.
2. Age between 18 and 60 years, inclusive, at Screening.
3. Body mass index (BMI) greater than 18 and less than or equal to 32.0 kg/m2 with a body weight more than or equal to 50.0 kg at Screening.
4. Healthy, as determined by the investigator on the basis of medical history, physical examination, laboratory results, ECG (QT-interval corrected using Fridericia’s formula [QTcF] more than or equal to 450 msec), and vital signs. The clinical laboratory tests, ECG, and/or vital sign measurements may be repeated as necessary if, in the judgment of the investigator, there is a reason to believe the initial result is inaccurate or due to transient issues (eg, white coat effect, clumped platelets).
5. Male participants, sexually active with women of childbearing potential (WOCBP), must agree to use a barrier method of birth control during the study and up to 98 days after the (last) dose of study intervention.
6. Male participants must agree to not donate sperm during the study and up to 98 days
after the (last) dose of study intervention.
7. Female participants must be of non-childbearing potential. Female participants are considered WOCBP unless they are postmenopausal (at least 12 months consecutive amenorrhea without other known or suspected cause, and with a follicle-stimulating hormone (FSH) and estradiol level in the postmenopausal range at Screening) or have
been sterilized surgically (eg, hysterectomy, bilateral salpingectomy, or bilateral oophorectomy).
8. Female participants must have a negative serum pregnancy test at Screening and a negative urine pregnancy test at each Check-In.
9. No use of nicotine-containing products within 1 month prior to Check-In and willingness to abstain from nicotine-containing products until the last Discharge.
10. All prescribed medications must have been stopped at least 14 days or 5 half-lives (whichever is longer) prior to Check-In. Also, all over-the-counter medications, vitamin preparations or other food supplements, and herbal medications (eg, St. John’s wort) must have been stopped at least 7 days or 5 half-lives (whichever is longer) prior to Check-In. An exception is made for paracetamol (up to 2 g per day), which is allowed up to Check-In.
11. Ability and willingness to abstain from alcohol from 48 hours prior to each Check-In until Discharge.
12. Ability and willingness to abstain from grapefruit (juice) from 7 days prior to each Check-In until Discharge.
13. Negative screen for alcohol, drugs-of-abuse, and cotinine at Screening and each Check-In.
14. Participants must indicate willingness and ability to comply with scheduled visits, treatment plan, laboratory tests, other study procedures, and study restrictions.
Minimum age
18 Years
Maximum age
60 Years
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
1. Participation in any drug or device clinical investigation, other than this study, within 60 days or 5 half-lives (whichever is longer) prior to Check-In or are anticipating participating in any other drug or device clinical investigation within the duration of this study.
2. Prior or ongoing medical condition (eg, concomitant illness or psychiatric condition), medical history, 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. Presence of any clinically significant abnormality (including abnormal liver function tests; eg, aspartate aminotransferase (AST), alanine aminotransferase (ALT), or bilirubin [total, conjugated, and unconjugated) more than or equal to 1.5 times the upper limit of normal).
4. History of tuberculosis or positive Quantiferon Gold test at Screening.
5. Any psychological, psychiatric, or emotional issues, or 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. Being a WOCBP (see definition in Inclusion Criterion 7).
7. Donation of plasma within 7 days prior to the (first) dose of study intervention. Donation or loss of more than 450 mL of blood (excluding volume drawn at Screening) within 30 days prior to the (first) dose of study intervention.
8. History of alcohol abuse (regular alcohol intake more than or equal to 21 units per week for male participants and more than or equal to 14 units per week for female participants) within 2 years prior to Screening. One unit (8 g) is equivalent to a ½ pint (280 mL) of beer, 1 measure (25 mL) of spirits, or 1 small glass (125 mL) of wine.
9. Positive hepatitis B surface antigen (HBsAg), positive hepatitis C virus (HCV) antibody, or HIV antibody result at Screening.
10. Any vaccination planned between 2 weeks prior to Check-In and Discharge and throughout the study. Live vaccines are not allowed between 4 weeks prior to Check-In and Discharge and throughout the study.
11. Known hypersensitivity or intolerance to the drug substance or any excipient of the drug product.

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
Not yet recruiting
Date of first participant enrolment
Anticipated
7/07/2025
Actual
Date of last participant enrolment
Anticipated
14/10/2025
Actual
Date of last data collection
Anticipated
2/03/2026
Actual
Sample size
Target
28
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
QLD

Funding & Sponsors
Funding source category [1] 318920 0
Commercial sector/Industry
Name [1] 318920 0
PTC Therapeutics, Inc.
Country [1] 318920 0
United States of America
Primary sponsor type
Commercial sector/Industry
Name
CTI Clinical Trial and Consulting Services Australia Pty Limited
Address
Country
Australia
Secondary sponsor category [1] 321389 0
None
Name [1] 321389 0
Address [1] 321389 0
Country [1] 321389 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 317537 0
Alfred Hospital Ethics Committee
Ethics committee address [1] 317537 0
Ethics committee country [1] 317537 0
Australia
Date submitted for ethics approval [1] 317537 0
07/05/2025
Approval date [1] 317537 0
30/05/2025
Ethics approval number [1] 317537 0

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

Contacts
Principal investigator
Name 141362 0
Dr Michael Wong
Address 141362 0
Nucleus Network, Level 5, 300C Herston Road, Herston, QLD 4006
Country 141362 0
Australia
Phone 141362 0
+61 07 3707 2793
Fax 141362 0
Email 141362 0
[email protected]
Contact person for public queries
Name 141363 0
PTC844 Study Coordinator
Address 141363 0
Nucleus Network, Level 5, 300C Herston Road, Herston, QLD 4006
Country 141363 0
Australia
Phone 141363 0
+61 1800 243 733
Fax 141363 0
Email 141363 0
[email protected]
Contact person for scientific queries
Name 141364 0
Aaron Tansy
Address 141364 0
PTC Therapeutics, 500 Warren Corporate Center Drive, Warren, NJ 07059
Country 141364 0
United States of America
Phone 141364 0
+1 908 222 7000
Fax 141364 0
Email 141364 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.