Clinical Trials & COVID-19

About Clinical Trials

What are clinical trials?

Clinical trials (or research studies) help scientists and doctors explore whether a medical strategy, device, or medication is safe and effective for people. Before any medication can be approved by the FDA or other regulatory agencies and made available to the public, it must go through several phases of clinical research.

As a Phase 2b NF1 clinical trial, the ReNeu study is researching the effectiveness of the study drug at improving a plexiform neurofibroma (PN) that is causing significant issues in people with Neurofibromatosis type 1 (NF1).

Why do people participate in clinical trials?

People participate in clinical trials for different reasons. Some may participate because they want to learn more about their disease. Others volunteer to participate because they want to help researchers learn more about a disease to potentially help them and others in the future.

Are clinical trials safe?

Clinical trials have risks but are designed to follow a specific set of standards called Good Clinical Practice. They are closely regulated and overseen by clinical researchers and doctors to help keep all participants safe.

Can someone leave a clinical trial once it has started?

Yes. Participation in clinical trials is completely voluntary. Participants in the ReNeu study can end their involvement at any time, for any reason.

Participants should talk to the study doctor if they want to stop participating in the study.

If a participant decides to leave the study early, the study doctor may ask that the participant return to the study clinic for a final visit to ensure the participant’s health and safety. There will be no negative impact on future care that the participant receives.

Can participants see their regular doctors during the ReNeu study?

Yes. Participants can visit any doctor to meet the participant’s health needs during the ReNeu study.

Participants should let their study doctor know that they will be seeing another doctor and if any other medication has been prescribed. Participants should also let their other doctors know that they have joined a clinical research study.

What if I have questions during the ReNeu study?

You can ask questions of the study team at any time before, during, and after the ReNeu study.

Before you or your child agree to participate in the study, make sure that you understand the responsibilities of study participants. If you have any concerns about study participation, you should feel comfortable discussing them with a member of the study team at any time.

Find out if you or your child qualifies