None listed
Conditions
Brief summary
The safety, efficacy and Pharmacokinetics of PTC844 tablets will be assessed with multiple doses over a period of 14 days followed by a single dose. This will be a randomised, double-blind study in healthy volunteers taking standard meal..
Interventions
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
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.
Sponsors
CTI Clinical Trial and Consulting Services Australia Pty Limited
Study design
Allocation
Randomised controlled trial
Intervention model
Parallel
Primary purpose
Treatment
Masking
Blinded (masking used) (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
All
Age
18 Years to 60 Years
Healthy volunteers
Yes
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.
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.
Outcome results
None listed