Recruiting Adults With Alpha-1 Antitrypsin Deficiency (AATD).
Clinical trial summary
We are conducting a clinical trial for participants with genetically diagnosed alpha-1 antitrypsin deficiency (AATD). In this trial, we are recruiting participants with AATD (with a known or suspected PiZZ mutation) aged 18 to 70.
The clinical trial involves having single dose of the investigational drug via an intravenous (IV) infusion, safety assessments and repeated blood sampling to assess how safe the drug is, and to discover how your body processes the drug, how long the drug stays in your body and how the drug affects the body.
You will be reimbursed for your time and effort in this clinical trial.
If you are interested in participating in this trial:
- You will need to attend a Screening appointment so we can check if you are suitable to take part in the trial. At this appointment, we will take blood, check your heart, and carry out a physical examination to check your overall health. The appointment will be scheduled within 6 weeks (or shorter) before you receive the investigational drug.
- If results from Screening appointment show you are suitable for the trial, you will be invited to take part.
- This will involve an in-house stay of 4 days/3 nights in our clinical research unit.
- After discharge, you will be required to attend the unit 4 times (about once per week) to monitor your safety during a 28-day period. You will not receive additional doses of the drug during this time.
- In addition to the above, there are 8 follow-up visits to check your health at months 2, 3, 4, 5, 6, 9, 12, 18 and 24 at the clinical research unit.
- This means the whole trial takes up to approximately 25.5 months from the Screening appointment through to the final visit.
- In total, if you are included in the clinical trial, you will be required to come into the unit approximately 15 times.
Who can take part?
We will check your suitability for the clinical trial using the following criteria:
- You are aged between 18 to 70 years (inclusive).
- You have a Body Mass Index (BMI) < 30.
- You must have been diagnosed with alpha-1 antitrypsin deficiency (AATD) (with a known or suspected PiZZ mutation).
- You are willing to have genetic testing for mutations in the AAT gene (if it hasn’t previously been done).
- You must not have had any history or recent illnesses which may pose a risk and impact your safety.
- You must not have had a liver or lung transplant or are awaiting one.
- You must not be a regular smoker.
- You must not have had an active COVID infection within 7 day or have had a COVID vaccine within 1 month of enrolment.
- You must not test positive for HIV or Hepatitis B & C test.
- You must not have had prior treatment with another approved or experimental drug within 90 days of enrolment.
- You must not be pregnant or breastfeeding.
- You are able to understand and provide informed consent.
A full list of do’s and don’ts will be given to you by a member of our recruitment team if you are interested and want to find out more.
Duration of Stay
Clinical trial payment
In recognition of the time and effort involved in taking part in the trial, you will receive £2,550 (£1,700 clinical trial payment and £850 bonus) on completion of the clinical trial, provided you comply fully with all the clinical trial requirements. This amount is based on the anticipated clinical trial plan and will be adjusted pro-rata (i.e. same amount of money paid per unit of time), should we invite you to attend extra visits.
Location
Clinical trial dates
Admission: TBC
Dismissal: TBC
Follow-up visits: TBC
Please call a member of the recruitment team on 0207 042 5800 to discuss study dates.
Approval
The contents of this email have been approved by an ethics committee. Any response to this advertisement will be recorded. Your response will not place you under any obligation to participate in this trial.
You may be required to remain resident in the unit until resolution of any safety issues, after which you will be discharged from the unit.
Ref: C23018_June_2024 (email_website_advert text)
Speak to a member of our Volunteer Recruitment Team
Comments from volunteers
Thanks very much to the Volunteer Recruitment Team. I am so excited to be part of this trial. I can’t thank you and your colleagues enough for all your help, professionalism, and kindness.
Steve K
Unbelievable service! They looked after me so well, and I would recommend them to anyone interested in participating in a clinical trial. I want to thank all the staff at trials4us for their continued efforts and for looking after me so well.
Chas
Very impressive. Thank you for taking care of me and for the valuable service you offer.
Joann
Very professional environment in the heart of London to conduct studies that help discover the medicines of the future.
James
Recruiting people with type 2 diabetes
Study Summary
In this study, we are recruiting participants with Alpha 1 (with a known or suspected PiZZ mutation) aged 18 to 70. We will give you a tour of the clinical trials unit and conduct a number of clinical assessments to establish your current health status.
This study is to help participants with Alpha 1 decide if they want to participate in future gene editing clinical trials.
If you are interested in participating in this patient engagement study
- You will need to attend an in-person visit, we will give you a tour of the clinical trial unit and perform tests to check your general health to obtain a clinical baseline.
- The tests can be spread over multiple days (if required).
- You will have a follow-up call with a doctor a week after the in-person visit.
- This means the whole process takes approximately 1 week.
Who can take part?
- You are aged between 18 to 70 years (inclusive).
- You must have been diagnosed with alpha-1 antitrypsin deficiency (Alpha 1) (with a known or suspected PiZZ mutation).
- You must be able and willing to provide informed consent.
- You must be willing and able to comply with study assessments.
Study payment
Study dates
Approval
You may be required to remain resident in the unit until resolution of any safety issues, after which you will be discharged from the unit.
Ref: C23017_MAR_2024 (email_website_advert text)