Safety, Tolerability and PK of SAD of ISIS 598769 in Healthy Subjects
Research type
Research Study
Full title
A Blinded, Placebo-Controlled, Dose-Escalation, Phase 1 Study to Assess the Safety, Tolerability and Pharmacokinetics of Single Doses of ISIS 598769 Administered Subcutaneously in Healthy Volunteers
IRAS ID
152784
Contact name
Annelize Koch
Contact email
Sponsor organisation
Isis Pharmaceuticals, Inc.
Eudract number
2013-005352-15
ISRCTN Number
n/a
Research summary
The new medicine tested in this study is a compound called ISIS 598769. It is hoped that ISIS 598769 will be useful as a treatment for an inherited disease called myotonic dystrophy type 1 (DM1).
DM1 primarily causes problems with muscles including muscle wasting and weakness, but may also cause problems with the heart, eyes (early cataracts), glucose response (blood sugar regulation), hypersomnia (excessive sleepiness) and the gastrointestinal system (digestion).
This study will recruit healthy males and females (of non-childbearing potential) between the ages of 18 and 65 years. The study will take place at the PAREXEL Early Phase Clinical Unit (London), Northwick Park Hospital, Harrow, UK.
The primary objective of this study is to investigate the safety and tolerability of ISIS 598769 when given as single dose in healthy subjects. The study will also investigate how ISIS 598769 is absorbed, distributed, metabolized and eliminated by the body when given as a single dose in healthy subjects.
The length of study participation is expected to be approximately 8 weeks. This includes a 4-week screening period, a single dose and a 4-week post-treatment evaluation period.
Clinical safety assessments will be done throughout the study. The safety and tolerability of single doses of ISIS 598769 will be assessed by adverse events, vital signs, physical examinations and weight, clinical laboratory tests (serum chemistry, hematology, urinalysis, coagulation), ECGs and the use of concomitant medications. Pharmacokinetic analysis will be done to determine the levels of ISIS 598769 in plasma and urine.
REC name
London - Surrey Borders Research Ethics Committee
REC reference
14/LO/0579
Date of REC Opinion
19 May 2014
REC opinion
Further Information Favourable Opinion