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Trial registered on ANZCTR


Registration number
ACTRN12625000846426
Ethics application status
Approved
Date submitted
31/07/2025
Date registered
6/08/2025
Date last updated
6/08/2025
Date data sharing statement initially provided
6/08/2025
Type of registration
Prospectively registered

Titles & IDs
Public title
A healthy volunteer study to check the effect and safety of PTC844 tablets with standard meal in multiple ascending doses across the period of 14 days followed by a study of single ascending dose in the participants.
Scientific title
Phase 1 Study to Characterize the Pharmacokinetics, Safety, Tolerability, and Pharmacodynamics of PTC844 Administered as Single and Multiple Ascending Doses with a Standard Meal in Healthy Participants.
Secondary ID [1] 314956 0
PTC844-SMAD-102-HV
Universal Trial Number (UTN)
Trial acronym
Linked study record
ACTRN12625000595415 is Part A (1st part) of the study and linked to the current application (Part B and C) as they are sequential and were to be carried out only if Part A had favourable outcomes. They are separate applications as Part B and C include a placebo arm and it was not in Part A study design.

Health condition
Health condition(s) or problem(s) studied:
Immunological Disorders 338286 0
Condition category
Condition code
Inflammatory and Immune System 334588 334588 0 0
Autoimmune diseases
Inflammatory and Immune System 334589 334589 0 0
Other inflammatory or immune system disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
This is a Phase 1 study in 2 parts - Multiple Ascending Doses followed by Single Ascending Dose.
Multiple Ascending Dose (MAD): The safety and Pharmacokinetics (PK) of multiple PTC844 tablet doses, administered within 30 minutes with a standard meal and approximately 240 mL of water, will be assessed in a randomized, double-blind, placebo-controlled, parallel-treatment design with up to 4 groups. Each group will have 8 participants with 6 administered study drug and 2 given placebo. Participant will be dosed on site and the study coordinator will record it. Screening will go up to 28 days after which the participant will be admitted on Day -1. Drug will be administered from Day 1 to Day 14, and the below doses will be explored (once daily).
• Group 1: 10 mg PTC844 tablets or matching placebo
• Group 2: 20 mg PTC844 tablets or matching placebo
• Group 3: 30 mg PTC844 tablets or matching placebo
• Group 4: 40 mg PTC844 tablets or matching placebo
Participants will be discharged on Day 21 after completing all of their procedures and complete subsequent visits as out-patients at Nucleus Network.
Single Ascending Dose: SAD part will only be initiated if required based on the results from the MAD part of the study. The safety and PK of multiple PTC844 tablet doses administered with a standard meal and approximately 240 mL of water will be assessed in a randomized, double-blind, placebo-controlled, parallel-treatment design with up to 3 groups. Each group will have 8 participants with 6 administered study drug and 2 given placebo. Screening will go up to 28 days after which the participant will be admitted on Day -1. A single dose will be administered on Day 1 and the below doses will be explored
• Group 1: 40 mg PTC844 tablet or matching placebo
• Group 2: 60 mg PTC844 tablet or matching placebo
• Group 3: 80 mg PTC844 tablet or matching placebo
The patient will be discharged on Day 5 after carrying out all planned procedures. They will complete all subsequent tests during their outpatient visits at Nucleus Network.
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. Participants may enroll in different parts of the study after completing their first part participation, completing adequate investigational drug washout (4 weeks), and rescreening.
Intervention code [1] 331563 0
Treatment: Drugs
Comparator / control treatment
Matching Placebo Tablets. The ingredients of the placebo tablet are:
• Lactose monohydrate
• Microcrystalline cellulose
• Croscarmellose sodium
• Sodium lauryl sulfate
• Povidone K30
• Colloidal silicon dioxide
• Magnesium stearate
• Opadry II 85F18422 White
Control group
Placebo

Outcomes
Primary outcome [1] 342262 0
For multiple ascending dose: To characterize the safety and tolerability of multiple ascending doses of PTC844 tablets.
Timepoint [1] 342262 0
Full physical examination will be conducted at Screening, at Check-In (Day -1), on Day 3, Day 14, Day 21, Day 28, and Day 35 post first dose and at PI discretion. Clinical laboratory tests will include clinical chemistry, hematology, and urinalysis performed on days -1, 1, 2, 3, 5, 7, 10, 14, 15, 17, 21, 25, 28 and 32 post first dose. Triplicate 12-lead ECGs using Electrocardiograms (with each reading taken 1 to 2 minutes apart) will be obtained after at least 10 minutes of rest in a supine position at Screening, at Predose and at 0.5, 1, 2, 3, 4, 6, 7, 8, 9, 10, 11, 12, and 24 hours post first dose on Day 1 (24 hours post first dose is Day 2) and Day 14 (24 hours post first dose is Day 15), and at Discharge. Vital signs including systolic and diastolic blood pressure using a sphygmomanometer, oxygen saturation, pulse rate and respiratory rate using pulse oximeter, and body temperature using thermometer will be performed after at least 5 minutes of rest in a supine position at Screening, at Check-In (Day -1), at predose and at 0.5, 2, 5, 8, 12, and 24 hours post first dose on Day 1, and once on each day indicated on the table above. On Days 2 to 14, vital signs will be performed predose. Adverse events will be assessed from screening to 4 weeks post first dose.
Primary outcome [2] 342263 0
For Single ascending dose: To characterize the safety and tolerability of single ascending doses of PTC844 tablets.
Timepoint [2] 342263 0
Full physical examination will be conducted at Screening, at Check-In (Day -1), and on Days 2, 8, 15, and 22 post first dose. Clinical laboratory tests will include clinical chemistry, hematology, and urinalysis and will be performed on Days -1, 1, 2,3,5,8,12,15,19 post first dose. Triplicate 12-lead ECGs using Electrocardiograms (with each reading taken 1 to 2 minutes apart) will be obtained after at least 10 minutes of rest in a supine position at Screening, at predose and at 0.5, 1, 2, 3, 4, 6, 7, 8, 9, 10, 11, 12, 16, and 24 hours post first dose on Day 1 (24 hours post first dose is Day 2), and at Discharge. Vital signs including systolic and diastolic blood pressure using a sphygmomanometer, oxygen saturation, pulse rate and respiratory rate using pulse oximeter, and body temperature using thermometer will be performed after at least 5 minutes of rest in a supine position at Screening, at Check-In (Day -1), at predose and at 0.5, 2, 5, 8, 12, and 24 hours post first dose on Day 1, followed by Days 2,3,4,5,8,12,15,19. Adverse events will be assessed from screening to 4 weeks post first dose.
Secondary outcome [1] 450194 0
Multiple Ascending Dose: To characterize the PK of PTC844 tablets.
Timepoint [1] 450194 0
Blood for PK analysis of PTC844 in plasma will be drawn at predose and at 0.5, 1, 2, 3, 4, 6, 7, 8, 9, 10, 11, 12, and 24 hours postdose on Days 1 and 7, Predose on Days 2, 3 to 6, and Days 8 to 13, and at Predose and at 0.5, 1, 2, 3, 4, 6, 7, 8, 9, 10, 11, 12, 24, 36, 48, 72, 96, 120, 144, and 168 hours postdose on Day 14 (ie, while the participant is at their inpatient stay). Blood for PK analysis of PTC844 and Dihydroorotate (DHO) in plasma will also be taken once at 264, 336, 432, and 504 hours postdose of the Day 14 dose (ie, at outpatient visits).
Secondary outcome [2] 450195 0
Single Ascending Dose: To characterize the PK of PTC844 tablets.
Timepoint [2] 450195 0
Blood for PK analysis of PTC844 and DHO in plasma will be drawn at predose 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 postdose on Day 1 (ie, while the participant is at their inpatient stay). Blood for PK analysis of PTC844 in plasma will also be taken once at 168, 264, 336, 432, and 504 hours postdose in both Treatment Periods (ie, at outpatient visits).

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 450msec), 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
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Concealed and involves contacting the holder of the allocation schedule (randomisation list), who is an off-site unblinded pharmacist.
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Simple randomisation using a software
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 1
Type of endpoint/s
Safety/efficacy
Statistical methods / analysis
A formal statistical analysis plan will be developed and finalised before database lock will be developed and finalised before database lock.
Analysis populations:
Safety Analysis set: includes participants who received at least 1 dose of study drug. The safety set will be employed in the analysis of tolerability and safety variables.
PK Full Analysis set: this set is defined as all participants who received at least 1 dose of study drug and have at least 1 reported concentration.
PK Analysis set: this analysis set is defined as all participants in the PK full analysis set who have at least 1 measurable drug concentration without a major protocol deviation or any other events that may affect the evaluation of the effect of the study drug on the key PK parameters. The PK set will be employed in summary and/or analysis of PK concentration and parameters. Participants dosed with placebo will not be included in the PK set.

Recruitment
Recruitment status
Not yet recruiting
Date of first participant enrolment
Anticipated
11/08/2025
Actual
Date of last participant enrolment
Anticipated
23/03/2026
Actual
Date of last data collection
Anticipated
27/04/2026
Actual
Sample size
Target
56
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
QLD

Funding & Sponsors
Funding source category [1] 319514 0
Commercial sector/Industry
Name [1] 319514 0
PTC Therapeutics, Inc.
Country [1] 319514 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] 322010 0
None
Name [1] 322010 0
Address [1] 322010 0
Country [1] 322010 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 318089 0
Alfred Hospital Ethics Committee
Ethics committee address [1] 318089 0
Ethics committee country [1] 318089 0
Australia
Date submitted for ethics approval [1] 318089 0
07/05/2025
Approval date [1] 318089 0
30/05/2025
Ethics approval number [1] 318089 0

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

Contacts
Principal investigator
Name 143078 0
Dr Michael Wong
Address 143078 0
Nucleus Network, Level 5, 300C Herston Road, Herston, QLD 4006
Country 143078 0
Australia
Phone 143078 0
+61 07 3707 2793
Fax 143078 0
Email 143078 0
[email protected]
Contact person for public queries
Name 143079 0
Study Coordinator
Address 143079 0
Nucleus Network, Level 5, 300C Herston Road, Herston, QLD 4006
Country 143079 0
Australia
Phone 143079 0
+61 1800 243 733
Fax 143079 0
Email 143079 0
[email protected]
Contact person for scientific queries
Name 143080 0
Aaron Tansy
Address 143080 0
PTC Therapeutics, 500 Warren Corporate Center Drive, Warren, NJ 07059
Country 143080 0
United States of America
Phone 143080 0
+1 9082227000
Fax 143080 0
Email 143080 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.