First in human clinical study of a novel drug PTC607 to assess its safety and tolerability in healthy volunteers
- Conditions
- Huntington's DiseaseNeurological - Neurodegenerative diseasesHuman Genetics and Inherited Disorders - Other human genetics and inherited disorders
Recruitment & Eligibility
- Status
- Completed
- Sex
- All
- Target Recruitment
- 40
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.
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.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method