Phase 1 study SAD-MAD of ACT-519276 in healthy subjects.
Research type
Research Study
Full title
A two-part single-center, Phase 1 study to assess the tolerability, safety, pharmacokinetics (including food interaction), and pharmacodynamics of ascending single and multiple doses of ACT-519276 in healthy subjects
IRAS ID
236613
Contact name
Sunu Valasseri
Contact email
Sponsor organisation
IDORSIA Pharmaceuticals Ltd
Eudract number
2017-004124-30
Duration of Study in the UK
1 years, 0 months, 1 days
Research summary
ACT-519276 is an Investigational Medicinal Product (IMP) being developed for the treatment of inherited genetic diseases affecting brain cells and nervous system such as Huntington’s disease. In such conditions cells lack capacity to handle certain kind of proteins, which leads to deposition of these proteins in the brain resulting in degeneration of nerve cells. It is thought that ACT-519276 will prevent build-up of these proteins and subsequent death of nerve cells.
The purposes of this first in man study are; to assess safety and tolerability when ACT-519276 is given as single and multiple oral doses to healthy volunteers, to understand how the body breaks down the drug, to assess effect of food on drug absorption and assess the effect on the electrical activity in the heart.
This study will be conducted in 2 parts. Part A will be done as a single dose study, starting with a low dose and subsequent groups getting a higher dose. There will be 6 groups in this part. The first two subjects in a group will be dosed 24 hours before the rest of the group gets the medication. Each group will have 8 volunteers, two of them will receive dummy (Placebo) drug. In group A4 a single dose will be administered in a fasted state in period 1 and in a fed state in period 2.
In part B, Subjects will receive multiple oral doses of ACT-519276 or placebo once daily in the morning in the fasted condition. The duration of administration will be at least 7 days.
Each dose level will be investigated in a new cohort of 10 healthy male and female subjects (4 male subjects on active drug, 1 on placebo, and 4 female subjects on active drug, 1 on placebo). Sentinel dosing wil be used in part B.REC name
North East - York Research Ethics Committee
REC reference
17/NE/0336
Date of REC Opinion
23 Nov 2017
REC opinion
Further Information Favourable Opinion