Stanford University, Stanford, CA
Dana Farber Cancer Institute, Boston, MA
Herbert Irving Comprehensive Cancer Center, New York, NY
Huntsman Cancer Institute, Salt Lake City, UT
Introducing PATHFINDER - a clinical trial of avapritinib for people with advanced systemic mastocytosis.
PATHFINDER is a phase 2 clinical trial that will test the safety and effectiveness of an investigational drug called avapritinib (formerly known as BLU-285) in people with advanced systemic mastocytosis (SM).
Advanced SM is a rare condition caused by the accululation of mast cells in the body's organs, including the bone marrow. Mast cells are an important type of immune cell that normally play a role in the body to help fight infection and trigger the allergic response. In advanced systemic mastocytosis, mast cells are inappropriately released causing symptoms in multiple organs around the body.
There are three different subtypes of advanced SM (advSM) - aggressive SM (ASM), SM with an associated hematological neoplasm (SM-AHN), and mast cell leukemia (MCL). Across all subtypes, up to 95% of people have a specific mutation (genetic alteration) in a gene called KIT. The mutation, known as KIT D816V, plays a central role in the activation and accumulation of mast cells in people with systemic mastocytosis. Avapritinib is an investigational drug that was specifically designed to selectively target the KIT D816V mutation.
You may be able to take part in the PATHFINDER clinical trial if you:
Have one of several types of advanced systemic mastocytosis, including aggressive systemic mastocytosis, systemic mastocytosis with associated hematologic neoplasm,* or mast cell leukemia
Have a measurable clinical finding, also known as C-Finding, that is determined to be related to SM (not applicable to patients with mast cell leukemia)*
Have elevated levels of serum tryptase, a marker of mast cell activity
Are at least 18 years old
*SM-related C findings are defined by international criteria for the diagnosis of SM and the measurement of clinical response to treatment. Using these criteria, the PATHFINDER clinical trial will measure the effect of avapritinib on one or more C findings. Talk to your doctor or contact us to learn more about C findings.
You are not eligible to take part in the PATHFINDER clinical trial if you:
Have acute myeloid leukemia
Have indolent or smoldering SM*
*Contact us for more information regarding a separate clinical trial planned by Blueprint Medicines.
Other eligibility criteria apply and these will be assessed at your screening.
If you take part in the PATHFINDER clinical trial, you will receive the investigational drug avapritinib. Avapritinib is designed to selectively target KIT D816V, the underlying driver of systemic mastocytosis. Avapritinib comes in a tablet and should be swallowed whole with water once a day.
During screening, certain tests will be done to assess if your disease meets the criteria for the study. The screening period varies and could take up to 8 weeks in some cases (from start of screening to your first dose of avapritinib).
As a single-arm trial, there is no placebo (sugar-pill) group and all eligible participants are guaranteed to receive the investigational drug.
All participants must have a follow-up visit 14 days after their last dose of investigational drug. Follow-up calls and site visits may be required periodically thereafter.
*There is no maximum duration for this trial. You will continue with your cycles of treatment until:
During the trial, a variety of health assessments will be conducted to see how you are responding to the investigational drug. In most cases, these assessments will be done at a clinical site.
Health assessments will vary between visits, but may include:
More information about health assessments and visit length will be found in the informed consent form, which you will need to read and sign if you want to take part in the PATHFINDER clinical trial.
How often will these visits take place?*
Cycle 3 onwards
Every 2-3 months
*Please note that these numbers are approximate. One treatment cycle is 28 days.
Please enter your city, ZIP Code, or country to find a trial site near you, or see the full site list.
A travel support program is available for participants and caregivers at most trial sites to reduce out-of-pocket expenses and help ensure travel to the trial site is simple and comfortable. More information about travel support is provided by the trial site during the screening process.