Parkinson's Disease
Conditions
Interventions
PTC857 is available for clinical trial use in the form of capsules (compounded)
at 50 mg strength. Placebo is available as matching PTC857 for administration.
Sponsors
PTC Therapeutics, Inc.
Eligibility
Age
18 Years to 99 Years
Inclusion criteria
Inclusion criteria: Healthy male or female subjects aged from 18 to 55 years old, inclusive, at Screening. Subjects must understand the nature of the study and must provide signed and dated written informed consent before the conduct of any study-related procedures. BMI of * 19.0 kg/m2 and * 35.0 kg/m2 with a body weight * 50.0 kg for male subjects and a body weight * 45.0 kg for female subjects at Screening. Healthy as determined by the Investigator, based upon a medical evaluation including medical history, physical examination, laboratory tests, triplicate ECG recording (average QTcF * 450 msec) and vital signs. Out of range values can be repeated once.
Exclusion criteria
Exclusion criteria: History of coagulopathy. History of fat malabsorption. Dietary restrictions that preclude participation. Females who are pregnant or nursing. Subjects with a prior medical history of clinically significant gastrointestinal, renal, hepatic, neurologic, hematologic, endocrine, oncologic, pulmonary, immunologic,psychiatric, or cardiovascular disease or any other condition which, in the opinion of the Investigator, would jeopardize the safety of the subject or impact the validity of the study results.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Primary Endpoints: Part 1 * Single Ascending Dose The primary endpoint of the SAD part of the study is the overall safety profile characterized by type, frequency, severity, timing, and relationship to study treatment of any adverse events (AEs), vital signs, laboratory abnormalities, physical examination abnormalities, C-SSRS scores, or electrocardiogram (ECG) abnormalities. Part 2 * Multiple Ascending Dose The primary endpoint of the MAD part of the study is the overall safety profile characterized by type, frequency, severity, timing, and relationship to study treatment of any AEs, vital signs, laboratory abnormalities, physical examination abnormalities, C-SSRS scores, or ECG abnormalities. Part 3 * Food Effect The primary endpoint of the FE part of the study is the food effect on PK parameters including area under the curve (AUC) from time zero to the last quantifiable concentration (AUC0-t), maximum observed concentration (Cmax), and time corresponding to occurrence of Cmax (Tmax) after administration of a single dose of PTC857 in healthy subjects. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary Endpoints: Part 1 * Single Ascending Dose The secondary endpoints of the SAD part of the study are: PK parameters including AUC0-t, Cmax, Tmax, and dose normalized AUC (AUC0-t/D and AUC0-inf/D) and dose normalized Cmax (Cmax/D). The PTC857 dose range for Part 2 (MAD) of the study. Part 2 * Multiple Ascending Dose The secondary endpoints of the MAD part of the study are: PK parameters including AUC0-tau, Cmax, Tmax, tEHL, dose normalized AUC0-tau (AUC0-tau/D), Cmax/D, and the accumulation ratio based on AUC (Racc). The PTC857 dose range and regimen for subsequent Phase 2 studies. Part 3 * Food Effect The secondary endpoint of the FE part of the study is any food effect on the safety and tolerability of a single dose of PTC857 in healthy subjects characterized by type, frequency, severity, timing, and relationship to study treatment of any AEs, vital signs, laboratory abnormalities, physical examination abnormalities, C-SSRS scores or ECG abnormalities. | — |
Countries
Netherlands
Outcome results
None listed