What is the FERVENT-1 clinical research study?

Currently, we are working on developing potential treatments for non-transfusion dependent beta-thalassemia. FERVENT-1 is a clinical research study that aims to assess the efficacy and safety of an investigational treatment in reducing excess iron levels in adults with non-transfusion dependent beta-thalassemia.

What is the study treatment?

Investigational treatment:
This treatment is a monoclonal antibody that targets a protein in the body called TMPRSS6. This protein normally increases the amount of iron in the blood. Blocking this protein with the investigational treatment may lower excess iron levels in the body. Investigational means it is not approved for use outside of clinical studies.

Placebo:  
A placebo looks like the investigational treatment but does not contain any real medicine. Researchers can tell whether an investigational treatment is effective by understanding if it works better than a placebo.

Once the study doctor confirms that you qualify, you will be randomly assigned to receive the investigational treatment or a placebo. You will have a 4 in 5 chance of receiving the investigational treatment over the placebo.

The study treatment is given by subcutaneous injection (needle under the skin).

What can I expect if I participate?

Study participation is 100% voluntary (your choice). You will receive information on what to expect, as well as your role and responsibilities if you join the FERVENT-1 study. You may leave the study at any time without it affecting your regular health care.

If you join the study, there are certain things you will be expected to do. This includes attending study visits and undergoing certain assessments and procedures, such as imaging scans and blood draws. Your participation may last up to around a year and a half with up to 22 study visits.

Qualified participants may be reimbursed for travel to and from the study site for study visits. The study site staff will provide you with more information on reimbursement as approved in your country and study site.

How can I take part?

If you wish to take part in the FERVENT-1 clinical study, you can fill in a short pre-screener questionnaire to see if you may be eligible.

What else should I know?

As with all medicines, there are possible risks when taking the investigational treatment. If you qualify and choose to participate, you will be provided with an Informed Consent Form that explains any possible risks and side effects. It is also possible that the study treatment may affect you in unknown ways. Your health and safety are our top priorities and will be closely monitored throughout your participation.

You may benefit from additional check-ups on top of your regular care and have access to non-transfusion dependent beta-thalassemia specialists. There is no guarantee that you will receive a medical benefit from participating in this study. Your condition may get better, stay the same, or may even get worse. You are free to withdraw from the study for any reason and at any time.

This clinical trial will use an investigational drug product, the safety and efficacy of which has not been fully evaluated by Regulatory Authorities.

Find a study site near you

To find out more about the FERVENT-1 clinical study, and whether it may be right for you, please contact your closest study site – they will be more than happy to help.