Huntington's Disease
Conditions
Interventions
PTC518 as 5 mg and 50 mg tablets.
Sponsors
PTC Therapeutics, Inc.
Eligibility
Age
18 Years to 64 Years
Inclusion criteria
Inclusion criteria: 1. For Part 1, Part 2, Part 4, and Part 5: Healthy male or female subjects aged from 18 to 65 years old, inclusive, at Screening. For Part 3: healthy male of female subjects aged 50 to 65 years old, inclusive, at Screening. 2. 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. 3. Body Mass Index (BMI) of >=18.5 kg/m2 and =50.0 kg for male subjects and a body weight >=45.0 kg for female subjects at Screening. 4. Healthy as determined by the Investigator, based upon a medical evaluation including medical history, physical examination, laboratory test results, ECG recording (e.g. QTcF
Exclusion criteria
Exclusion criteria: 1. Subjects 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 (e.g., concomitant illness, psychiatric condition), medical history, physical findings that, in the Investigator*s opinion, could adversely affect the safety of the subject 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, emotional problems, any disorders or resultant therapy that is likely to invalidate informed consent or limit the ability of the subject to comply with the protocol requirements.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Part 1 - Single Ascending Dose: Type, frequency, severity, timing, and relationship to study treatment of any adverse events (AEs), laboratory abnormalities, vital signs abnormalities, physical examination abnormalities or ECG abnormalities when PTC518 is administered as a single dose. Part 2 - Multiple Ascending Dose: Type, frequency, severity, timing, and relationship to study treatment of any AEs, laboratory abnormalities, vital signs abnormalities, physical examination abnormalities, ECG abnormalities, and C-SSRS scores when PTC518 is administered as multiple doses. Part 3 - CSF PTC518 concentrations in blood and CSF when PTC518 is administered for 7 days. Part 4 - Food Effect: PK parameters, eg, Tmax, Cmax, AUC, elimination half-life (t1/2), apparent total body clearance (CL/F), and apparent volume of distribution (Vd/F) when PTC518 is administered in fed (Part 4) and fasted (Part 1) states. Part 5 (MD28D): Type, frequency, severity, timing, and relationship to study treatment of any AEs, laboratory abnormalities, vital signs abnormalities, physical examination abnormalities, ECG abnormalities, and C-SSRS scores when PTC518 is administered as multiple doses. | — |
Secondary
| Measure | Time frame |
|---|---|
| Part 1 - Single Ascending Dose: PK parameters, eg, Tmax, Cmax, AUC, elimination half-life (t1/2), apparent total body clearance (CL/F), and apparent volume of distribution (Vz/F) when PTC518 is administered as a single dose. Part 2 - Multiple Ascending Dose: PK parameters, eg, Tmax, Cmax, AUC, elimination half-life (t1/2), apparent total body clearance (CL/F), and apparent volume of distribution (Vz/F) when PTC518 is administered as multiple doses. QT, and the corrected QT interval by Fridericia*s formula (QTcF) extracted from Holter monitoring and PTC518 plasma concentration-QTc effects. Part 3 - CSF Type, frequency, severity, timing, and relationship to study treatment of any AEs, laboratory abnormalities, vital signs abnormalities, physical examination abnormalities, ECG abnormalities and C-SSRS scores when PTC518 is administered for 7 days. Part 4 - Food Effect: Type, frequency, severity, timing, and relationship to study treatment of any AEs, laboratory abnormalities, vital signs abnormalities, physical examination abnormalities or ECG abnormalities when PTC518 is administered in fed state and compare with those in part 1 (fasted). Part 5 (MD28D): PK parameters, eg, Tmax, Cmax, AUC, T1/2, CL/F, and V z/F when PTC518 is administered as multiple doses. QT, and the corrected QT interval by Fridericia*s formula (QTcF) extracted from Holter monitoring and PTC518 plasma concentration-QTc effects. | — |
Countries
Netherlands
Outcome results
None listed