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New hope for rare blood cancer: experimental drug targets mast cells

NCT ID NCT04996875

Disease control Sponsor: Cogent Biosciences, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Sep 16, 2026 · Updated 3 times

Summary

This study tests an experimental drug called CGT9486 (bezuclastinib) in people with advanced systemic mastocytosis, a rare and serious blood disorder. The drug aims to control the disease by blocking a key protein that drives abnormal mast cell growth. About 140 adults with aggressive forms of the disease will receive the drug to see if it is safe and effective.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 140 people

The number the study aims to enrol. It can still change while the study runs.

Started

Nov 2021

Expected to finish

Jul 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Key Inclusion Criteria for Main Study: 1. Diagnosed with one of the following advanced mastocytosis diagnoses by Eligibility Committee 1. Aggressive Systemic Mastocytosis (ASM) 2. Systemic Mastocytosis with an Associated Hematologic Neoplasm (SM-AHN) 3. Mast Cell Leukemia (MCL) 2. Measurable disease according to modified IWG-MRT-ECNM criteria. (A subset of patients inevaluble per mIWG-MRT-ECNM will be included in the study). 3. ECOG (0 to 3) 4. Have clinically acceptable local laboratory screening results (clinical chemistry, hematology) within certain limits Key Exclusion Criteria for Main Study: 1. Persistent toxicity from previous therapy for AdvSM that has not resolved to ≤ Grade 1 2. Associated hematologic neoplasm requiring immediate antineoplastic therapy 3. Clinically significant cardiac disease 4. Known positivity for the FIP1L1 PDGFRA fusion. Patients with eosinophilia without detectable KIT D816V mutation must demonstrate lack of PDGFRA fusion mutation prior to enrollment 5. Seropositive for human immunodeficiency virus (HIV) 1 or 2, or positive for hepatitis B surface antigen or hepatitis C virus (HCV) antibody 6. History of clinically significant bleeding event within 30 days before the first dose of study drug or need for therapeutic anticoagulation on study 7. Diagnosed with or treated for malignancy other than the disease under study within the prior 3 years before enrollment 8. Received any cytoreductive therapy or any investigational agent less than 14 days, and for cladribine, interferon alpha, pegylated interferon, and any antibody therapy less than 28 days, before screening bone marrow biopsy 9. Received hematopoietic growth factor support within 14 days before the first dose of study drug 10. Received strong CYP3A4 inhibitors or inducers within 14 days or 5 drug half-lives, whichever is longer, before the first dose of study drug 11. Need for treatment with high dose steroids Key Inclusion Criteria for Substudy Population: Rollover Cohort 1. Demonstrate AHN progression requiring immediate AHN-directed therapy while receiving bezuclastinib 2. Demonstrated clinical benefit from bezuclastinib therapy 3. Have clinically acceptable local laboratory screening results (clinical chemistry, hematology) within certain limits High-Risk Cohort 1. Receiving or indicated for AHN-directed therapy. 2. Diagnosed with one of the following pathologic diagnoses of SM-AHN: 1. Myelodysplastic syndrome (MDS) that is high- or very high-risk 2. Accelerated phase myeloproliferative neoplasm (MPN) 3. MDS with excessive blasts in bone marrow or peripheral blood 4. Chronic myelomonocytic leukemia-2 (CMML-2) 3. Have clinically acceptable local laboratory screening results (clinical chemistry, hematology) within certain limits. Key Exclusion Criteria for Substudy Population: 1. Diagnosis of Philadelphia chromosome-positive malignancy 2. Diagnosis of acute myeloid leukemia (AML) 3. Appropriate for allogenic hematopoietic stem cell transplantation 4. Any contraindication to selected concomitant therapy 5. Rollover Cohort: Have not demonstrated acceptable tolerability of previous bezuclastinib therapy 6. High-Risk Cohort: Previously treated with investigational therapy for AdvSM 7. High-Risk Cohort: Previously treated with cytoreductive therapy and discontinued due to treatment-related toxicity 8. High-Risk Cohort: Received any cytoreductive therapy or any investigational agent less than 14 days, and for cladribine, interferon alpha, pegylated interferon, and any antibody therapy less than 28 days, before screening or archival bone marrow biopsy

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Conditions

The condition(s) this trial relates to.

acute myeloid leukemia aggressive systemic mastocytosis allergic disease chronic myelomonocytic leukemia connective tissue neoplasm hematologic disorder hematopoietic and lymphoid system neoplasm immune system disorder leukemia mast cell leukemia mastocytosis Mastocytosis, Systemic myeloid leukemia Myeloproliferative Disorders neoplasm Neoplasms by Site skin disorder Soft Tissue Neoplasms systemic mastocytosis systemic mastocytosis with an associated clonal hematologic non-mast cell lineage disease type III hypersensitivity disease Urticaria Pigmentosa

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Advanced systemic mastocytosis (advsm) Aggressive systemic mastocytosis (ASM) Mast cell leukemia (MCL) SM with an associated hematologic neoplasm (SM-ahn)

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    18 sites in 5 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • AKH Wien, Universitatsklinikum

    ACTIVE_NOT_RECRUITING

    Vienna, 1090, Austria

  • AOU San Giovanni di Dio e Ruggi dAragonia

    ACTIVE_NOT_RECRUITING

    Salerno, 84131, Italy

  • Azienda Ospedaliero Universitaria Careggi

    WITHDRAWN

    Florence, 50134, Italy

  • Azienda Ospidaleira Universitaria Integrata Verona

    WITHDRAWN

    Verona, 37124, Italy

  • CHU de Liege

    WITHDRAWN

    Liège, 4000, Belgium

  • Centre Hospitalier Universitaire (CHU) de Poitiers

    ACTIVE_NOT_RECRUITING

    Poitiers, 86000, France

  • Centre Hospitalier Universitaire (CHU) de Toulouse

    ACTIVE_NOT_RECRUITING

    Toulouse, 31300, France

  • City of Hope Comprehensive Cancer Center

    RECRUITING

    Duarte, California, 91010, United States

  • Cleveland Clinic Taussig Cancer Center

    ACTIVE_NOT_RECRUITING

    Cleveland, Ohio, 44106, United States

  • Columbia University Irving Medical Center

    WITHDRAWN

    New York, New York, 10032, United States

  • Dana-Farber Cancer Institute

    RECRUITING

    Boston, Massachusetts, 02215, United States

  • Galiz Research

    WITHDRAWN

    Hialeah, Florida, 33016, United States

  • Gold Coast University Hospital

    RECRUITING

    Southport, Queensland, 4215, Australia

  • Guy's Hospital - NHS Foundation Trust

    RECRUITING

    London, SE1 9RT, United Kingdom

  • Hospital Universitario Ramón y Cajal

    ACTIVE_NOT_RECRUITING

    Madrid, 28034, Spain

  • Hospital Universitario Vall d'Hebron

    ACTIVE_NOT_RECRUITING

    Barcelona, 08740, Spain

  • Huntsman Cancer Institute - University of Utah Health

    RECRUITING

    Salt Lake City, Utah, 84112, United States

  • IRCCS Azienda Ospedaliero Universitaria di Bologna

    TERMINATED

    Bologna, 40138, Italy

  • Institut Català d'Oncologia - Hospital Duran i Reynals

    ACTIVE_NOT_RECRUITING

    Barcelona, 08908, Spain

  • Leeds Teaching Hospitals NHS Trust

    RECRUITING

    Leeds, LS9 7TF, United Kingdom

  • MUSC Health University Medical Center

    RECRUITING

    Charleston, South Carolina, 29425, United States

  • Mayo Clinic Arizona

    RECRUITING

    Phoenix, Arizona, 85054, United States

  • Necker-Enfants Malades Hospital

    ACTIVE_NOT_RECRUITING

    Paris, 75015, France

  • Nepean Hospital

    RECRUITING

    Kingswood, New South Wales, 2747, Australia

  • Oslo University Hospital

    ACTIVE_NOT_RECRUITING

    Oslo, 0450, Norway

  • Peter MacCallum Cancer Centre

    RECRUITING

    Melbourne N., Victoria, 3051, Australia

  • Public University Hospital No. 1 in Lublin

    ACTIVE_NOT_RECRUITING

    Lublin, 20-400, Poland

  • Rush University Medical Center

    WITHDRAWN

    Chicago, Illinois, 60612, United States

  • St. Michael's Hospital - Unity Health Toronto

    RECRUITING

    Toronto, Ontario, M5B 1W8, Canada

  • Stanford Cancer Institute

    RECRUITING

    Stanford, California, 94305, United States

  • The University of Texas MD Anderson Cancer Center

    RECRUITING

    Houston, Texas, 77030, United States

  • UCLA Medical Center

    RECRUITING

    Los Angeles, California, 90095, United States

  • UKSH Campus Lubeck

    WITHDRAWN

    Lübeck, 23562, Germany

  • University College London Hospital - NHS Foundation Trust

    RECRUITING

    London, NW1 2BU, United Kingdom

  • University Hospital Aachen

    ACTIVE_NOT_RECRUITING

    Aachen, 52074, Germany

  • University Medical Center Groningen

    ACTIVE_NOT_RECRUITING

    Groningen, 9713, Netherlands

  • University of Alabama at Birmingham (UAB) Hospital

    ACTIVE_NOT_RECRUITING

    Birmingham, Alabama, 35233, United States

  • University of Alberta Hospital

    RECRUITING

    Edmonton, Alberta, T6G 2G3, Canada

  • Universitätsklinikum Freiburg

    ACTIVE_NOT_RECRUITING

    Freiburg im Breisgau, 79104, Germany

  • Universitätsklinikum Mannheim

    ACTIVE_NOT_RECRUITING

    Mannheim, 68167, Germany

  • Universitätsspital Basel

    RECRUITING

    Basel, 4031, Switzerland

  • Winship Cancer Institute - Emory University

    RECRUITING

    Atlanta, Georgia, 30322, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.