Study 36129

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.

Plus travel expenses

*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

The purpose of Period 1 is to evaluate WVE-006 when is administered as a single dose at increasing dose strengths.

Period 1 will consist of up to 5 planned groups of up to 8 participants (with the option to include a maximum of one additional group of up to 8 participants): each group will evaluate a different dose of WVE-006 starting at the lowest dose and gradually increasing the dose level in each group. In Period 1, each group 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”. This is known as a single ascending dose (SAD) study. Each group 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.

If you participate in this period of the study, you will be required to stay at the Simbec-Orion clinical unit for 4 days (3 overnight stays – Day -1 to Day 3) for the in-house stay, followed by 12 return visits (Days 8, 15, 22, 29, 36, 43, 50, 57, 64, 71, 78 & 85 – defined as the end of study visit).You should therefore ensure that you are able to commit to completing all visits before agreeing to participate in this study.

All of the visits from Day 8 onwards are single day visits i.e. you will not be required to stay overnight at the clinical unit. However, you should ensure that you are able to commit to all of the relevant study dates for each visit and commit to the 3 overnight stays required as part of the in-house stay (Day -1 to Day 3) as these visits are not flexible.

If you complete this period of the study, you will receive a maximum inconvenience payment of £3975. The payment will be split into 4 parts; £1000 after completion of the Day 15 visit, £800 after completion of the Day 29 visit, £800 after completion of the Day 57 visit, the remaining £1375 after completion of the end of study visit (Day 85).

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.

If you participate in this period of the study, you will be required to stay at the Simbec-Orion clinical unit for 4 days for in-house stay 1 (Dose 1: 3 overnight stays – Day -1 to Day 3) and for 3 days for in-house stay 2 (Dose 3: 2 overnight stays – Day 29 to Day 31); Dose 2 will be administered on Day 15 and will require at least 4 hours of follow up following administration. This period of the study also includes 11 return visits (Days 8, 21, 36, 43, 50, 57, 64, 71, 78, 85 & 113 – defined as the end of study visit). You should therefore ensure that you are able to commit to completing all visits before agreeing to participate in this study.

All of the visits with the exception of the two in-house stays (Day -1 to Day 3 and Day 29 to Day 31) are single day visits i.e., you will not be required to stay overnight at the clinical unit. However, you should ensure that you are able to commit to all of the relevant study dates for each visit and commit to the overnight stays required as part of the treatment period (3 overnight stays for in-house stay 1 – Day -1 to Day 3 and 2 overnight stays for in-house stay 2 – Day 29 to Day 31) as these visits are not flexible.

If you complete this period of the study, you will receive a maximum inconvenience payment of £4525. The payment will be split into 4 parts; £900 after completion of the Day 15 visit, £900 after completion of the Day 31 visit, £900 after completion of the Day 57 visit, the remaining £1825 after completion of the end of study visit (Day 113).

Study Payment: £3975 to £4525 Apply. Be amazing

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