None listed
Conditions
Brief summary
This will be a phase 1 first-in-human study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of multiple doses of PTC607 at various doses in healthy volunteers. Participants will be undergo screening for up to 28 days. Eligible participants will then be admitted to an inpatient unit for up to 19 days, where they will be taking study medication as directed by the unit staff and will undergo the following but not limited to procedures: vital sign measurements, physical examinations, collection of blood and urine samples, electrocardiograms. Once discharged from the unit, participants will have a follow up phone call approximately 30 days after the last dose of study drug.
Interventions
This is a multiple ascending dose study in healthy volunteers. It will consist of up to 3 sequential cohorts of 8 participants each. In each cohort, participants will be randomised to receive multiple doses of either PTC607 or placebo in tablet formulation in a 3:1 ration (6 participants will receive PTC607 and 2 will receive placebo) with food for 14 days. The following dose regimens have been selected:
- Cohort 3.1: 15 mg PTC607 or matching placebo once daily for 14 consecutive days
- Cohort 3
This is a multiple ascending dose study in healthy volunteers. It will consist of up to 3 sequential cohorts of 8 participants each. In each cohort, participants will be randomised to receive multiple doses of either PTC607 or placebo in tablet formulation in a 3:1 ration (6 participants will receive PTC607 and 2 will receive placebo) with food for 14 days. The following dose regimens have been selected: - Cohort 3.1: 15 mg PTC607 or matching placebo once daily for 14 consecutive days - Cohort 3.2: 30 mg PTC607 or matching placebo once daily for 14 consecutive days - Cohort 3.3: depending on pharmacokinetics data from cohorts 3.1 and 3.2, either 30 mg or 60 mg PTC607/placebo every other day for 14 days (7 doses in total). The Safety Review Committee will meet to review the results of each cohort prior to proceeding to the following cohort in this study. Healthy participants will stay in the clinical research unit for 18 consecutive nights and will take study drug with food between 8:00 am and 11:00 am under direct supervision of the clinical research unit staff. The study drug and matching placebo will be taken with 240 mL of water. 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. Healthy 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 kg/m2 with a body weight of greater than or equal to 50 kg for male participants and a body weight of greater than or equal to 45 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. Postmenopausal female participants must have had greater than or equal to 12 months of spontaneous amenorrhea (with follicle-stimulating hormone of greater than or equal to 30 mIU/mL at Screening). 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 equal to or greater than 21 units per week for male participants and equal to or greater than 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 equal to or less than 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