Evaluating Our Potential Medicines

Clinical trials help physicians, investigators and regulators understand whether a potential treatment may safely and effectively treat a specific disease or condition and can provide patients an opportunity to access investigational treatments that they would otherwise not be able to use. Patient participation in clinical trials is vital to the successful development of medicines.

Horizon Clinical Trials

Click the link below for more information about clinical trials involving Horizon medicines and experimental treatments.

Find Clinical Trials

Featured Trials 

Frequently Asked Questions 

Clinical research studies, also known as clinical trials, look at a medicine or medical device to see if it is safe, how it works in the body and if it works to treat a specific disease. They also help us understand how a medicine may affect patients, what dosing is most effective and many more details. 

Clinical trials are conducted by doctors who are responsible for patients’ clinical trial-related care. Some clinical trials are sponsored, or conducted, by companies like Horizon. Other clinical studies are investigator-initiated trials, which are developed, sponsored and conducted by an independent researcher or academic institution. Horizon may support an investigator-initiated trial by providing an investigational medicine, but is not directly involved in the conception or management of the trial.

Clinical trials must be approved by an institutional review board or ethics committee who is responsible for helping to protect the rights and welfare of trial patients. In addition, every participant is monitored with study-related medical tests and exams before, during and sometimes even after the trial.

Clinical trials typically consist of three main phases before a medicine can be approved for use by the U.S. Food and Drug Administration (FDA). Each phase allows investigators to learn more about the safety and efficacy of a medicine and build upon knowledge gained from earlier phases. In certain situations, additional phases may be initiated after a medicine has been approved.

Phase 1
Phase 1 trials determine the safety and tolerability of a medicine in a small group. Frequent lab work and clinical exams may be required.

Phase 2
Phase 2 trials investigate how well a medicine works to treat a certain medical condition, as well as side effects in groups larger than Phase 1 trials. Sometimes, these trials examine whether different doses have different effects.

Phase 3
Phase 3 trials are larger studies that determine whether a medicine is safe and effective for others with the same condition and if it is better than the standard treatment, if one is available. Phase 3 studies are usually initiated after successful earlier phase trials.

Additional phases
In certain situations, additional phases may be initiated after a medicine has been approved. Patients in additional clinical trial phases help researchers further understand effects of the approved medicine.

Some requirements are similar for every trial, but each trial may have its own specific requirements. Clinical trials may require additional time and attention compared to standard treatment, including extra visits and tests. You should discuss participating in a clinical trial with your health care provider and caregiver to understand the requirements and time commitment needed for participation.

Before you can take part in any trial, you will first need to apply and then attend the screening visit(s) for initial tests and assessments, if applicable, to see if you are eligible. If you are eligible to take part, you may enter the trial and possibly receive the investigational medicine.

Participation in any clinical trial is completely voluntary, and patients may choose to leave the trial at any time for any reason. As a participant, your rights include informed consent, which is the process that ensures potential participants are aware of the key facts related to the trial before they decide to participate.

Each clinical trial location or site has a study coordinator who manages the process and provides information to potential participants. You will meet the clinical study doctor and study coordinator at your first visit to the office or hospital. You can reach out to the study coordinator with any questions you have about participation.

How long will my participation last?
Participation time is different depending on the trial.  Please reach out to your study coordinator to learn more.

The benefits and risks of clinical trials vary from one trial to another and will be explained to you by the study team before you decide to participate. Patients should discuss participating in a clinical trial with their health care provider to weigh the potential risks and benefits.

What are the benefits of participating?
Clinical trials provide patients an opportunity to access investigational treatments that they would otherwise not be able to use. Participation in clinical trials is also vital to the successful development of our medicines for those with rare, autoimmune and severe inflammatory diseases.

What are the risks of participating?
As with all clinical trials, using an investigational medicine involves potential risks and the possibility of complications and side effects, some of which are not currently known. The known and unknown risks of participating in a specific clinical trial will be explained to you by the study team during the informed consent process. If you qualify and choose to participate, your health will be closely monitored by the study team. In addition, you will be notified if there is any new information about side effects while you are participating in the study.

Participation in clinical trials is completely voluntary, and you may withdraw at any time, for any reason.

All clinical trials are designed differently, but it is common for some patients to receive a placebo (a substance that looks like an investigational medicine with no therapeutic effect) and not the experimental medicine. Researchers use a placebo to see if the investigational medicine works better or is safer than taking nothing.

You may be compensated for your participation in a trial and/or reimbursed for travel. Reach out to the study coordinator to learn more.

Trial locations differ depending on the study. There are often multiple locations for a given trial, and participation in a clinical trial may require travel to one of the locations. Patients may be compensated for their travel. Reach out to the study coordinator to learn more.

As a participant, your rights include informed consent, which is the process that ensures you are aware of the key facts related to the trial before you decide to participate. The study team will explain the informed consent document, which includes the trial purpose, time commitment, necessary procedures/tests and potential risks and benefits, as well as answer any questions you may have while deciding to participate. You can take as much time as you need to ask questions and review the materials in order to decide if the trial is right for you.

Participation in any clinical trial is completely voluntary, and patients may choose to leave the trial at any time for any reason. If you would like to leave the trial, you should discuss this with your study doctor, who will give you information about how to do this safely.

If you’re considering joining a clinical trial, it’s important to know what to expect. Download a list of questions to consider asking your doctor and clinical research team.

Our Commitment

Horizon is committed to the patient communities we serve and supporting their experiences through full transparency and clear communication during clinical trials and beyond. The development of our medicines would not be possible without the contributions of all past, current and future clinical trial participants.

Expanded Access

In circumstances when participating in a clinical trial is not possible, patients may seek special access to investigational medicines prior to regulatory approval. For more information, read our Expanded Access Policy Statement. 

Diversity in Clinical Trials

Horizon is committed to increasing clinical trial diversity. We are putting clinical trial diversity and inclusivity front and center in our development programs and actively working to ensure our trials reflect the patient populations affected by the disease. For more information, read our Diversity in Clinical Trials Statement.

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