SAD/MAD Study of AG-519 in Healthy Subjects (QCL117595)
Research type
Research Study
Full title
A Phase I Part-Randomized, Double-blind, Five-Part Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Ascending Oral Doses of AG-519, the Relative Bioavailability of a Capsule Formulation Compared with a Suspension Formulation, to Evaluate the Pharmacokinetics of the Capsule Formulation Under Fed and Fasted Conditions in Healthy Subjects, and to Assess the Pharmacokinetics, Safety and Tolerability of the Capsule Formulation in Healthy Subjects of Japanese Origin
IRAS ID
190836
Contact name
Sponsor organisation
Agios Pharmaceuticals
Eudract number
2015-003774-33
Clinicaltrials.gov Identifier
Duration of Study in the UK
0 years, 9 months, 7 days
Research summary
The Sponsor is developing the study drug, AG-519, for the potential treatment of pyruvate kinase deficiency (PK deficiency), a rare inherited disease that affects the body's red blood cells. Red blood cells are essential for carrying oxygen to tissues throughout the body. People with PK deficiency have mutations in an enzyme that results in the accelerated destruction of red blood cells (a condition known as hemolytic anemia) and further complications. The drug being tested in this study, AG-519, is a chemical compound that may help treat PK deficiency by restoring function in red blood cells.
The study will look at the safety and tolerability of the study drug, and will also look at the way the body takes up the drug and the effect the drug has on the body when given as a single dose and repeated doses. The study will consist of up to 3 parts involving up to 138 healthy male and female subjects . In Part 1, up to 32 subjects will receive a single dose of the study drug or placebo (dummy drug) given as a suspension (powder mixed with liquid). In Part 2, 32 subjects will receive repeated doses of the study drug or placebo, given as a suspension once or twice a day for 14 days. In both parts, 2 additional groups (8 subjects in each group) may be enrolled to look at different dose levels of the study drug. In Part 3, up to 18 subjects will receive a single daily dose of the study drug given as suspension or a tablet, either after food or on an empty stomach. Part 3 will be optional depending on the results from Part 2, and will consist of 4 periods. Part 3 will have a crossover design, meaning that all subjects will receive each treatment.
REC name
Wales REC 1
REC reference
15/WA/0354
Date of REC Opinion
13 Nov 2015
REC opinion
Further Information Favourable Opinion