What are trials and observational studies?

Clinical trials test a drug, device, or intervention to see if it changes the course of the disease.
Observational clinical studies, on the other hand, do not include drugs, treatments, or devices. Some of these observational clinical studies can help researchers understand how FA progresses over time. An example is the UNIFAI natural history study, in which many people in our community generously participate (thank you!). In this and other observational clinical studies, no treatment is given—the focus is data collection and a chance to contribute to the bigger picture.
You can usually only be in one trial at a time—but you can be in an observational study and a trial.
Both are incredibly important. Both move us forward.
Do you know the difference between a

What are the phases of a clinical trial?

Each phase plays a distinct role in evaluating a potential treatment’s safety and efficacy on the path to approval.
  • Phase 1 focuses on safety and dosing in a small group. Often, this is the first time a potential treatment is being tested in humans.
  • Phase 2 continues safety monitoring, refines dosing, and looks for early signs of efficacy.
  • Phase 3 involves more people and evaluates efficacy.
  • Phase 4 (postmarketing) takes place after approval to track long-term impact.
In rare diseases, phases may be combined to speed progress. Sometimes early-phase data can allow a potential treatment to be considered for accelerated approval. However, a Phase 3 trial is still required to confirm efficacy before full approval can be granted.
We are deeply grateful to the members of our community who step forward to make progress possible during all phases of clinical trials.
Phases of Trials

How to use FARA's Clinical Trial Finder

If you are interested in more information about a clinical trial or observational study, FARA’s website is a good place to start. This tool allows you to search active and recruiting trials, view eligibility criteria and study locations, and learn who to contact for more information.
Everyone with FA is eligible to participate in the UNIFAI Natural History study, and other opportunities are added as they become available. Anyone considering participation should first talk with their doctor to see if a study or trial is a good fit. FARA does not endorse or recommend any particular studies.
We’re grateful to our research partners for their urgency and commitment to developing FA treatments and to every volunteer whose participation helps move progress forward.
Clinical Trial Finder
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What is Informed Consent?

Understanding what to expect in a clinical trial is an important part of deciding whether to participate. The informed consent process is your opportunity to ask questions about the drug being studied, potential risks and benefits of participation, and what would be required of you as a participant — from different tests and procedures to the study schedule and travel arrangements. The informed consent discussion also provides an opportunity to self-reflect on why you are interested in participating in the trial and how different outcomes of the trial may impact you.
We’re sharing our Informed Consent Guide to help you think through these questions.
Download the guide before meeting with the study coordinator to feel more confident and informed.
Informed Consent Guide
Copy of Informed Constent

Clinical Trial Etiquette

Being in a clinical trial can bring up a lot of questions. You might wonder what you’re allowed to share with family and friends, the difference between research and clinical care visits, or what to do if you encounter something unexpected.

We’ve put together a short guide to help you feel more prepared and confident during your trial experience. Download the full Clinical Trials 101 Guide for Participants below.

Clinical Trials 101 Guide
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