None listed
Conditions
Brief summary
This will be a phase 1 first-in-human study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of PTC607 at various incremental single doses in healthy volunteers.
Interventions
Part 1 is a single ascending dose part of the study in healthy volunteers. It will consist with up to 5 sequential cohorts of 8 participants each. In each cohort, participants will be randomised to receive a single dose of either PTC607 or placebo in a 3:1 ratio (6 participants will receive PTC607 and 2 will receive placebo) in a fasted state and in a double-blinded fashion. PTC607 and placebo will be administered as a suspension orally. In each cohort, sentinel dosing will be performed in 2 par
Part 1 is a single ascending dose part of the study in healthy volunteers. It will consist with up to 5 sequential cohorts of 8 participants each. In each cohort, participants will be randomised to receive a single dose of either PTC607 or placebo in a 3:1 ratio (6 participants will receive PTC607 and 2 will receive placebo) in a fasted state and in a double-blinded fashion. PTC607 and placebo will be administered as a suspension orally. In each cohort, sentinel dosing will be performed in 2 participants (1 participant with PTC607 and 1 participant with placebo). The remaining 6 participants (5 of whom will be randomised to PTC607 and 1 of whom to placebo) will be dosed after at least 24 hours, if no clinically significant safety issues are observed in sentinels. The starting dose will be 5 mg of PTC607. The maximum dose escalation between cohorts will be no more than 3-fold to a maximum of 120 mg. The Safety Review Committee will meet to determine the dose-level for the next cohort based on PK and safety results from the previous cohorts. Healthy volunteers will stay in the clinical research unit for 3 nights and will take study drug after at least 10 hours fasting under direct supervision of the clinical research unit staff. Monitoring the adherence to the intervention may include mouth checks.
Sponsors
CTI Clinical Trial and Consulting Services Australia Pty Ltd.
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 65 Years
Healthy volunteers
Yes
Inclusion criteria
1. Males and females aged 18 to 65 years inclusive at Screening 2. Participants able to provide informed consent 3. Body mass index of greater than or equal to 18.5 and less than or equal to 30.0 kg/m2 with a body weight of greater than or equal to 50.0 kg for male participants and a body weight of greater than or equal to 45.0 kg for female participants at Screening 4. Generally healthy as determined by the investigator based on medical evaluation, including medical history, physical examination, laboratory test results, ECG, and vital signs. 5. Male participants must be willing to use 2 acceptable contraceptive methods for the duration of the study and for a minimum of 6 months after the last dose, and female participants of childbearing potential must be willing to use 2 acceptable contraceptive methods for the duration of the study and for a minimum of 30 days after the last dose. 6. All female participants of childbearing potential must have a negative serum pregnancy test result at Screening and a negative urine pregnancy test on Day -1. 7. Male participants must agree to not donate sperm for the duration of the study and for at least 6 months after the last dose.
Exclusion criteria
1. Participants that participated in any drug or device clinical investigation within 60 days prior to Screening or who anticipate participating in any drug or device clinical investigation within the duration of this study. 2. Prior or ongoing medical condition (eg, concomitant illness, psychiatric condition), medical history, and/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. An abnormal general neurological examination. 4. Presence of any clinically significant abnormality during Screening. 5. Any psychological or emotional problems, 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. A positive hepatitis B surface antigen, positive hepatitis C antibody, or HIV antibody result at Screening. 7. Donation of plasma within 7 days prior to dosing. Donation or loss of blood (excluding volume drawn at Screening or menses) of 50 to 499 mL within 30 days or more than 499 mL within 56 days prior to dosing. 8. Excessive alcohol consumption (regular alcohol intake greater than or equal to 21 units per week for male participants and greater than or equal to 14 units per week for female participants) within 6 months prior to Screening. One unit (8 g) is equivalent to a half pint (280 mL) of beer, 1 measure (25 mL) of spirits, or 1 small glass (125 mL) of wine. 9. The participant is a smoker or uses other nicotine-containing products. Ex-smokers must have ceased smoking >3 months prior to Screening. Smokers who consume <4 tobacco products per week are allowed. 10. The participant has consumed grapefruit (or its juice), star fruit, pomegranate, pomelo, tangelo, or Seville orange-containing products in the 1 week before Screening. 11. A positive urine drug screen, cotinine screen, or alcohol breath test at Screening or on Day -1 of each treatment period. 12. Females who are pregnant or nursing. 13. Participant has previously received PTC607.
Outcome results
None listed