Phase 1 Dose Escalation Study Assessing the Safety and Pharmacokinetics of PTC518 in Healthy Subjects

Completed

• Conditions: Huntington's Disease; 10027664

• Registration Number: NL-OMON54972
• Lead Sponsor: PTC Therapeutics, Inc.

Brief Summary

Trial ended prematurely

Detailed Description

Not available

Study Timeline

• Results First Posted: 2022-07-01
• Last Update Posted: 9 April 2024

Recruitment & Eligibility

• Status: Completed
• Sex: Not specified
• Target Recruitment: 136

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 <=30.0 kg/m2 with a body weight
>=50.0 kg for male subjects and a body weight >=45.0 kg for female subjects at
Screening.
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 <= 450 msec for males and QTcF <= 470 ms for females) and
vital signs. Out of range values can be repeated once.
5. Male subjects and female subjects of childbearing potential must be willing
to use 2 methods of birth control for the duration of the study and for 30 days
after the last dosing.

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.

Study & Design

• Study Type: Interventional
• Study Design: Not specified