DeFi Trial Overview
What is the DeFi trial?
The DeFi (Desmoid/Fibromatosis) trial is a Phase 3 clinical trial designed to evaluate the safety, efficacy, and tolerability of an investigational medicine called nirogacestat in people over the age of 18 with progressing desmoid tumors. Nirogacestat is an oral, small molecule inhibitor of gamma secretase – in other words, a pill that is designed to inhibit a protein in the body that is known to contribute to desmoid tumor growth.
The DeFi trial is a double-blind, placebo-controlled study, meaning that people are separated into two groups: one that receives nirogacestat, and one that receives a placebo, a pill or substance with no active ingredients that is not expected to have any medical effect on a study participant. Neither the study participant nor the doctor knows the group to which the person is assigned.
The trial is being conducted in approximately 50 sites in the U.S., Canada and Europe.
The DeFi trial is fully enrolled and therefore the study is no longer accepting new patients. For medical professionals seeking additional information about the DeFi trial, please email firstname.lastname@example.org.
How long the study lasts:
The double-blind phase of the DeFi trial will run for approximately 2 years. If a desmoid tumor is progressing while participating in the double-blind phase or the double-blind phase has completed, there is an optional open-label extension phase of the study. Those who qualify for the open-label extension will receive treatment with nirogacestat.
How often you need to be seen as part of the trial:
Following initial evaluation and visits to record your health at the start, most participants will begin taking a pill and be asked to return to the clinic for study visits on days 8, 15 and 22 of the first month, day 28 of the second month, day 1 of the fourth month, and then every 3 months after that.
What happens at each clinic visit:
Each visit will include a routine evaluation such as a physical exam and a check of your vital signs.
As part of the study, participants will also be asked to complete questionnaires, have scans (CT or MRI) to measure their tumor(s), and have blood drawn.