Active not recruitingPHASE2INTERVENTIONAL

(Summit) A Study to Evaluate the Efficacy and Safety of CGT9486 Versus Placebo in Patients With Indolent or Smoldering Systemic Mastocytosis

This research study, called Summit, is looking at a new medicine called bezuclastinib for people with a rare blood condition known as systemic mastocytosis, specifically the 'indolent' or 'smoldering' types. These are forms of mastocytosis where symptoms haven't been severe enough to cause major organ damage but are still troublesome and not fully controlled by usual care. The study aims to see if bezuclastinib is safe and effective in reducing these symptoms. Participants will receive either bezuclastinib or a dummy pill (placebo) alongside their usual supportive care. The study also includes people whose symptoms are not well controlled by another medicine called avapritinib.

At a glance

Status

Active not recruiting

Phase

PHASE2

Sponsor

Cogent Biosciences, Inc.

Enrolment target

237

Start

27 Jun 2022

Estimated completion

01 Apr 2030

SSM Mastocytosis, Indolent Mastocytosis, Systemic Mastocytosis ISM BMM Smoldering Systemic Mastocytosis Bone Marrow Mastocytosis

What is this study about?

This study is investigating a new medicine called bezuclastinib for specific types of a rare blood disorder called systemic mastocytosis. This condition happens when too many special immune cells, called mast cells, build up in different parts of your body. The study is focusing on 'indolent' (ISM) and 'smoldering' (SSM) systemic mastocytosis, which are forms where symptoms can be disruptive but are not usually life-threatening like more advanced types. The main goal is to find out if bezuclastinib can safely and effectively reduce uncomfortable symptoms that haven't been properly managed with typical care.

To do this, some people in the study will receive bezuclastinib, while others will get a placebo (a dummy pill that looks like the real medicine but has no active ingredients). This is a common way to test new medicines, as it helps researchers understand if any improvements are truly due to the study medicine or something else. All participants will also continue to receive their usual supportive care for their symptoms. The study will look at how well the medicine works and also track any side effects.

This research is important because current treatments for mastocytosis often focus on managing symptoms rather than addressing the underlying cause. If successful, bezuclastinib could offer a new option for people whose symptoms are still troubling despite available care, potentially improving their quality of life. There's also a smaller part of the study looking at people who haven't found enough relief from another mastocytosis medicine called avapritinib.

Key takeaways

This study is for people with specific types of systemic mastocytosis (ISM, SSM, BMM).
It tests a new medicine called bezuclastinib against a dummy pill (placebo) to manage symptoms.
The goal is to see if bezuclastinib is safe and effective in reducing symptoms not controlled by usual care.
Participation involves taking pills and regular clinic visits for checks.
You might get the active medicine or a placebo, and neither you nor your doctor will know which.
You can stop participating at any time if you wish.

Who may be eligible?

To be able to take part in this study, you must have been diagnosed with certain types of systemic mastocytosis: either 'indolent systemic mastocytosis' (ISM), 'smoldering systemic mastocytosis' (SSM), or 'bone marrow mastocytosis' (BMM). Your symptoms must be at a moderate to severe level, and despite trying at least two different symptom-reducing treatments for at least two weeks, your symptoms are still not well controlled. You also need to be generally well enough to participate, as assessed by your doctor.

However, there are reasons you might not be able to join. For example, if you have certain more aggressive types of mastocytosis, such as aggressive systemic mastocytosis or mast cell leukaemia, this study isn't for you. You also can't take part if you only have mastocytosis affecting your skin without it affecting your wider body. If you've recently had other treatments for mastocytosis, or have certain other medical conditions like significant bleeding problems, you also wouldn't be able to participate. Your doctor will carefully review all these criteria to determine if this study is suitable for you.

Quick self-check

Have I been diagnosed with indolent, smoldering, or bone marrow mastocytosis?
Are my mastocytosis symptoms moderate to severe and not well controlled by my current treatments?
Am I generally well enough to take part in a study?
Have I had other mastocytosis treatments recently that might prevent me from joining?
Do I need to take more than 10mg of prednisone (or similar steroid) per day?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you decide to take part in this study, you will be given either the new medicine, bezuclastinib, or a placebo (a dummy pill). You won't know which one you're receiving, nor will your study doctor, as this is a 'double-blind' study design. You'll take the tablets by mouth. The study is divided into several parts. You'll initially receive either bezuclastinib or placebo, and if you complete this part, you might have the option to continue into a later part of the study where everyone receives bezuclastinib.

Throughout your participation, you'll have regular visits to the clinic for assessments. These will include check-ups, blood tests, and discussions about how you're feeling and any symptoms you're experiencing. You'll also be asked about any side effects. The total length of time you'll be in the study will depend on which part of the study you join and how long you continue treatment, but it will involve multiple visits over several months.

Potential risks and benefits

Participating in a clinical trial offers potential benefits, such as access to a new medicine before it's widely available, and close medical monitoring. You might find your symptoms improve or that your quality of life gets better. However, there are also potential risks; the new medicine might cause side effects, some of which could be serious, or it might not work for you. The placebo treatment also means you might not receive any active medicine for a period of time. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (57)

University of Alabama at Birmingham
Birmingham, United States
Mayo Clinic Arizona
Phoenix, United States
One of a Kind Clinical Research Center
Scottsdale, United States
Modena Allergy and Asthma Clinical
La Jolla, United States
Innovative Research of West Florida
Clearwater, United States
Mayo Clinic - Jacksonville
Jacksonville, United States
University of South Florida
Tampa, United States
Emory University
Atlanta, United States
Rush University
Chicago, United States
Walter Reed National Military Medical Center
Bethesda, United States
Allervie Clinical Research
Glenn Dale, United States
Institute for Asthma and Allergy
Wheaton, United States

+45 more sites — see the official record for the full list.

Common questions

What is systemic mastocytosis?

It's a rare condition where special immune cells called mast cells build up in different parts of your body, which can cause various symptoms like skin rashes, stomach problems, or bone pain.

What does 'indolent' or 'smoldering' mean in mastocytosis?

These are types of systemic mastocytosis where the disease is not usually life-threatening, even though it can cause troublesome symptoms. They are less aggressive than other forms of the condition.

What is a 'placebo'?

A placebo is a dummy pill that looks exactly like the study medicine but contains no active ingredients. It's used in research to help scientists figure out if any changes are truly due to the new medicine.

Will I know if I'm getting the real medicine or the placebo?

No, neither you nor your study doctor will know whether you are receiving bezuclastinib or the placebo. This is called a 'double-blind' study.

Can I stop participating in the study at any time?

Yes, you are free to leave the study at any point and for any reason, without it affecting your medical care.