If you are a healthy male or female aged between 18 and 65, then you may be eligible to participate in a study investigating a new drug being developed for the potential treatment of a condition called Alpha-1 Antitrypsin Deficiency (AATD), which is a rare genetic condition that causes disease impacting the liver and lungs.
Study Payment* £4525 Plus Travel.
*Terms & Conditions apply.
Study Information
Simbec-Orion Clinical Pharmacology will soon be running a two-period study to investigate the study drug, WVE-006.
The overall objectives of this study are to determine the safety, tolerability (degree to which side effects of a drug can be tolerated) and concentration in the blood and urine of WVE-006 when WVE-006 is administered in different conditions i.e., single versus multiple doses and evaluating different dose strengths. Blood and urine samples will be taken at set timepoints throughout each period of the study in order to measure the levels of WVE-006 in the blood and urine. The results from each of the dosing groups and each study period will be analysed in order to understand how WVE-006 works in the body (including impact from single and multiple doses, and different dose strengths).
WVE-006 is being developed as a potential treatment for a condition called Alpha-1 Antitrypsin Deficiency (AATD), which is a rare genetic condition that causes diseases impacting the liver and lungs. Currently, there are limited treatments available for this condition and the majority of current treatments are intended to target the symptoms and diseases which arise as a result of the condition and therefore, there is an unmet need to develop more effective treatments that seek to target the underlying cause of AATD.
This study will be split into two separate periods (Period 1 & Period 2), and you will only participate in one period of the study.
Period 1 is now complete, and Period 2 is now in active recruitment.
Period 2
The purpose of Period 2 is to evaluate WVE-006 when it is administered at a selected dose strength once every other week for a period of 4 weeks (for a total of 3 doses).
Period 2 will consist of up to 8 participants and the dose selected for administration in Period 2 will be selected from a dose evaluated in Period 1 (evaluating different dose strengths of WVE-006 administered as a single dose on one occasion) which was considered to be safe and well tolerated (this may be the highest dose evaluated or a lower dose). In Period 2, the dose administration will include 2 participants that will be given the study drug or placebo approximately 48 hours before the rest of the participants; one will get the study drug and the other will get the placebo. These 2 participants are called “sentinels”. In Period 2, participants will either receive WVE-006 or a placebo (which contains no active drug) in the form of subcutaneous injection(s) which is a type of injection(s) into the tissue layer between the skin and muscle.
Screening Visit
Overnight Stay 1
Days: 4 (Days -1 to 3)
Return Visit
Day 8
Dose 2 Visit
Day 15
Return Visit
Day 21
Overnight Stay 2
Days: 4 (Days 29-31)
Return Visits
Visits: 9 (Days 36, 43, 50, 57, 64, 71, 78, 85 & 113)
