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ReclutamentoFASE2INTERVENTISTICO

(Apex) Bezuclastinib in Patients With Advanced Systemic Mastocytosis

This research study is looking at a new medication called bezuclastinib for adults suffering from advanced systemic mastocytosis (AdvSM). This includes specific types such as aggressive systemic mastocytosis (ASM), systemic mastocytosis with another blood cancer (SM-AHN), and mast cell leukemia (MCL). The main goal is to find out how safe the drug is and if it can help improve the condition. The study is called an 'open-label' study, which means both you and the study doctors will know you are receiving bezuclastinib. This is a Phase 2 study, meaning the drug has already been tested for safety in a small number of people, and now researchers want to learn more about its effects in a larger group of patients.

A colpo d'occhio

Stato
Reclutamento
Fase
FASE2
Sponsor
Cogent Biosciences, Inc.
Obiettivo di iscrizione
140
Inizio
09 Nov 2021
Completamento stimato
01 Jul 2027

Di cosa tratta questo studio?

This study is about an important new medicine called bezuclastinib, which is being tested for people who have advanced systemic mastocytosis (AdvSM). This is a rare condition where certain blood cells, called mast cells, build up too much in the body and can cause serious health problems. The study focuses on the more serious forms of this condition, including aggressive systemic mastocytosis, mastocytosis with another blood cancer, and a very rare type called mast cell leukemia.

Researchers want to see if bezuclastinib can help control these mast cells and improve symptoms for patients. Because mastocytosis is a complex condition, developing new treatments is really important to help improve patients' lives. This study is a 'Phase 2' study, which means that the medicine has already been found to be safe enough to test in a larger group of people, and now the scientists want to get a better idea of how well it works and if it has any side effects.

The study is 'open-label,' which means everyone involved – the patients and the doctors – will know that the study medicine being given is bezuclastinib. This type of study design helps researchers gather information efficiently about the drug's effects. The findings from this research could potentially lead to a new treatment option for people living with these challenging forms of mastocytosis.

Punti chiave

  • This study is for adults with advanced systemic mastocytosis.
  • It tests a new medicine called bezuclastinib.
  • The study aims to check how safe and effective the medicine is.
  • It's an 'open-label' study, meaning everyone knows you're getting the study drug.
  • Regular health checks and blood tests will be part of the study.
  • Puoi smettere di partecipare in qualsiasi momento.

Chi può essere idoneo?

To join this study, you need to be an adult (18 years or older) and have one of the more advanced forms of mastocytosis (aggressive systemic mastocytosis, mastocytosis with another blood cancer, or mast cell leukemia). Doctors will need to confirm your diagnosis. You also need to have signs of the disease that can be measured, and your general health, including your heart, should be stable enough to take part. Your recent blood test results also need to be within certain acceptable ranges.

There are also reasons why you might not be able to join. For example, if you still have serious side effects from previous mastocytosis treatments, or if you need immediate treatment for another blood cancer. You can't have certain heart conditions or have had serious bleeding recently. If you have HIV, Hepatitis B, or Hepatitis C, or certain other cancers, you wouldn't be able to participate. Also, if you've recently had other cancer treatments, certain medications, or specific types of growth factors, you might need to wait before joining.

Questo studio potrebbe fare al caso tuo?

Rispondi a queste domande rapide per vedere se potresti essere idoneo. Questo è solo una guida — il team di ricerca prende la decisione finale.

  1. Hai 18 anni o più?
  2. Have you been diagnosed with aggressive systemic mastocytosis, mastocytosis with another blood cancer, or mast cell leukemia?
  3. Is your general health, including your heart, currently stable?
  4. Have you spoken with your doctor about any recent treatments or serious medical conditions you have?
Rispondi a ogni domanda per vedere il tuo risultato.

Cosa comporta la partecipazione?

If you decide to take part in this study, you will be given the study medicine, bezuclastinib. The study is open-label, meaning everyone will know you are taking the study drug. You will have regular visits to the clinic so the study team can monitor your health, check how the medicine is working, and look for any side effects. This will involve regular blood tests, physical examinations, and possibly other tests to see how your mastocytosis is responding to the treatment. You might also be asked to complete questionnaires about how you are feeling.

The total duration of your participation in the study will depend on how you respond to the treatment and the study’s protocol. There will be an initial treatment period, followed by ongoing monitoring. You will be able to stop participating in the study at any time if you wish to, for any reason, without it affecting your usual medical care.

Rischi e benefici potenziali

Taking part in this study may offer potential benefits, such as receiving a new investigational treatment for your advanced mastocytosis that is not yet widely available. This treatment might help improve your symptoms and manage your condition. However, there are also potential risks involved. The study medicine, bezuclastinib, may cause side effects, some of which could be serious. The study team will closely monitor you for any side effects and will explain all known risks to you. You have the right to withdraw from the study at any time, for any reason, and this decision will not affect your future medical care.

Locations (42)

  • University of Alabama at Birmingham (UAB) Hospital
    Codice postale verificato
    Birmingham, Stati Uniti· Attivo ma non reclutante
  • Mayo Clinic Arizona
    Codice postale verificato
    Phoenix, Stati Uniti· Reclutamento
  • City of Hope Comprehensive Cancer Center
    Codice postale verificato
    Duarte, Stati Uniti· Reclutamento
  • UCLA Medical Center
    Codice postale verificato
    Los Angeles, Stati Uniti· Reclutamento
  • Stanford Cancer Institute
    Codice postale verificato
    Stanford, United States· Reclutamento
  • Galiz Research
    Codice postale verificato
    Hialeah, United States· Ritirato
  • Winship Cancer Institute - Emory University
    Codice postale verificato
    Atlanta, Stati Uniti· Reclutamento
  • Rush University Medical Center
    Codice postale verificato
    Chicago, Stati Uniti· Ritirato
  • Dana-Farber Cancer Institute
    Codice postale verificato
    Boston, Stati Uniti· Reclutamento
  • Columbia University Irving Medical Center
    Codice postale verificato
    New York, Stati Uniti· Ritirato
  • Cleveland Clinic Taussig Cancer Center
    Codice postale verificato
    Cleveland, Stati Uniti· Attivo ma non reclutante
  • MUSC Health University Medical Center
    Codice postale verificato
    Charleston, United States· Reclutamento

Domande comuni

What is systemic mastocytosis?

Systemic mastocytosis is a rare condition where too many mast cells, a type of immune cell, build up in your body's tissues and organs.

What is an 'advanced' form of mastocytosis?

Advanced forms of mastocytosis are more serious types of the condition, where the mast cells can cause more significant health problems or organ damage.

What does 'open-label' mean in a study?

Open-label means that both you and the study doctors will know that you are receiving the study medication, bezuclastinib.

What is a Phase 2 study?

A Phase 2 study tests a medicine in a larger group of patients after it has been shown to be safe in a smaller group. The goal is to see how well it works and to learn more about side effects.

Dovrò pagare per partecipare a questo studio?

No, you will not have to pay for the study drug or study-related tests and procedures. These costs are covered by the research study.

Come saperne di più

Cogent Biosciences, Inc.

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