Active not recruitingPHASE2INTERVENTIONAL

(PIONEER) Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, Versus Placebo in Patients With Indolent Systemic Mastocytosis

This study, called PIONEER, is for people in the UK with indolent systemic mastocytosis (ISM) whose symptoms aren't fully controlled by their current treatment. We're looking at a new drug called avapritinib. This drug aims to block a specific protein involved in ISM. Researchers want to see how effective and safe avapritinib is compared to a dummy pill (placebo). Participants will start with either avapritinib or placebo, alongside their usual care. Later, everyone in the study will get avapritinib. This helps scientists understand if the new drug can make a real difference to people's symptoms and well-being.

At a glance

Status

Active not recruiting

Phase

PHASE2

Sponsor

Blueprint Medicines Corporation

Enrolment target

251

Start

16 Apr 2019

Estimated completion

23 Jun 2027

Indolent Systemic Mastocytosis

What is this study about?

This study is called PIONEER and it's looking into a health condition called indolent systemic mastocytosis (ISM). ISM is a rare condition where certain cells, called mast cells, build up in different parts of the body. This can cause various symptoms like skin rashes, stomach problems, and tiredness, which can really affect daily life. For some people, these symptoms are hard to control even with their usual treatments.

The main goal of this study is to test a new medicine called avapritinib. This drug works by targeting a specific problem within the mast cells, which scientists hope will reduce the number of mast cells and help ease symptoms. The study will compare avapritinib with a 'placebo', which is a dummy pill that looks just like the study drug but contains no active medicine. This comparison helps researchers scientifically prove if avapritinib is truly effective and safe.

Throughout the study, all participants will also receive what's called 'best supportive care'. This means you'll continue to get the treatments and care your doctor usually recommends to manage your symptoms. The study is divided into three parts. In the first part, people will receive either avapritinib or the placebo. In the third part, everyone in the study will get to take avapritinib, including those who were initially on the placebo. This setup ensures that everyone eventually has the chance to try the new treatment.

Key takeaways

Tests a new medicine (avapritinib) for indolent systemic mastocytosis (ISM).
Compares avapritinib to a dummy pill (placebo) first, then everyone gets the new drug.
Aims to relieve symptoms not well controlled by current treatments.
Involves regular health checks and symptom assessments.
Open to adults aged 18 and over with confirmed ISM.
Your usual medical care continues alongside the study.

Who may be eligible?

To join this study, you must be 18 years or older and have a confirmed diagnosis of indolent systemic mastocytosis (ISM). Your ISM symptoms should be moderate to severe, and your current treatment shouldn't be fully controlling at least one of your main symptoms. You'll also need to be generally well enough to take part in the study activities.

There are some reasons why you wouldn't be able to join. For example, if you have a different type of mastocytosis (like aggressive mastocytosis or mast cell leukaemia), or if you've recently used avapritinib or certain other treatments. Your doctor will carefully check all the requirements to see if this study is suitable for you.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you 18 years of age or older?
Do you have a confirmed diagnosis of indolent systemic mastocytosis (ISM)?
Are your ISM symptoms moderate to severe and not fully controlled by your current treatment?
Have you avoided certain treatments recently, as checked by your doctor?
Do you feel well enough for regular clinic visits and tests?
Have you not previously taken this specific study drug, avapritinib?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will be randomly assigned to either receive the study drug, avapritinib, or a placebo (dummy pill) for the first part of the study, lasting several months. Both groups will also continue with their usual care. After this initial period, everyone in the study will receive avapritinib for the third part of the study.

Participation will involve regular visits to the study clinic for health checks, blood tests, and to assess your symptoms. You will also complete questionnaires about your symptoms and how you are feeling. The exact number of visits and the total duration aren't specified but are typical for a Phase 2 study. Your doctor will explain how often you'll need to attend and what each visit will involve.

Potential risks and benefits

Taking part in a clinical trial offers potential benefits, such as gaining access to a new treatment like avapritinib before it's widely available, and closely monitored medical care. However, there are also potential risks, including side effects from the study drug, which will be carefully monitored. The placebo group might not experience direct benefits from the study drug during the initial phase. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual care.

Locations (49)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

University of Alabama at Birmingham
Verified postcode
Birmingham, United States
Mayo Clinic Hospital
Verified postcode
Phoenix, United States
Stanford Cancer Institute
Verified postcode
Stanford, United States
Mayo Clinic Florida
Verified postcode
Jacksonville, United States
H. Lee Moffitt Cancer Center
Verified postcode
Tampa, United States
Winship Cancer Institute, Emory University
Verified postcode
Atlanta, United States
Rush University Medical Center
Verified postcode
Chicago, United States
University of Kansas Hospital
Verified postcode
Kansas City, United States
Brigham & Women's Hospital
Verified postcode
Boston, United States
Dana Farber Cancer Institute
Verified postcode
Boston, United States
Michigan Medicine, University of Michigan
Verified postcode
Ann Arbor, United States
Mayo Clinic
Verified postcode
Rochester, United States

Common questions

What is indolent systemic mastocytosis (ISM)?

ISM is a rare condition where special immune cells, called mast cells, build up in different body tissues, causing a range of symptoms.

What is avapritinib?

Avapritinib is a new drug being tested that aims to block a specific protein signal that contributes to the growth of mast cells in ISM.

What is a placebo?

A placebo is a 'dummy' pill that looks just like the real medicine but contains no active drug. It helps researchers understand if the new drug is truly effective.

Will I definitely get the new drug?

In the first part of the study, you'll have a 50/50 chance of getting avapritinib or the placebo. However, everyone will get avapritinib in the third part of the study.

Who is running this study?

This study is called PIONEER and is being conducted by Blueprint Medicines, working with doctors and hospitals.