Clinical Trials

We measure ourselves by the impact we have on people with cancer.
Let’s go big.

When there are no approved treatments for a condition, we must develop them. Clinical trials are studies that collect data to determine if a new drug is safe and effective. They are essential research practices for advancing science, enhancing medical knowledge and improving patient care.

Why join a clinical trial?

People’s involvement in clinical trials helps researchers uncover better ways to treat, prevent, diagnose, and understand different diseases and medical conditions. People take part in clinical trials for many reasons. For example, you could advance understanding of a disease and help others like yourself. You may also receive a new research treatment before it becomes widely available.

Clinical trials can also have risks. For example, the investigational medicine may not help you or it could have side effects. In some later-stage studies, it’s possible you may receive a placebo instead of the study medication. You would be made aware of this possibility before starting a research trial.

You should speak to your doctor to learn about the risks and benefits of a clinical trial before making the decision to participate.

 

Our clinical trials

We currently have a number of standalone and combination therapies in various phases in clinical trials. DeFi and ReNeu are our two most advanced programs.

Our Phase 3 DeFi trial is ongoing to evaluate nirogacestat, an oral, small molecule gamma secretase inhibitor (GSI) in adult patients with progressing desmoid tumors.
Our Phase 2b ReNeu trial is ongoing to evaluate mirdametinib, an oral, small molecule MEK inhibitor, in pediatric and adult patients with inoperable NF1-PN.
We are also conducting multiple clinical trials addressing highly prevalent, genetically defined cancers in collaboration with leaders in academia and industry.