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New pill shows promise for Hard-to-Treat blood cancers

NCT ID NCT04278768

Disease control Sponsor: Curis, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jun 27, 2026

Summary

This study tests an oral drug called emavusertib (CA-4948) in adults with acute myeloid leukemia (AML) or a high-risk bone marrow disorder (MDS) that have not responded to or returned after prior treatment. The goal is to find the safest and most effective dose and to see if the drug can shrink or control the cancer. Participants must have specific genetic mutations (FLT3, SF3B1, or U2AF1) and have had no more than two prior treatments.

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 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 366 people

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

Started

Jul 2020

Expected to finish

Apr 2026

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.

Inclusion Criteria: 1. Males and females ≥18 years of age 2. Life expectancy of at least 3 months 3. Eastern Cooperative Oncology Group (ECOG) Performance Status of ≤1 4. Cytomorphology based confirmed diagnosis of MDS or AML (as per World Health Organisation \[WHO\] 2016 classification) with the following characteristics. Phase 1 Dose Escalation (Monotherapy) • AML (primary or secondary, including treatment-related) after failing at least 1 standard treatment (may include chemotherapy, re induction therapy or stem cell transplantation). OR • Higher-risk R/R MDS that are considered resistant/refractory following at least 2 to 3 cycles of hypomethylating agent (HMA) or evidence of early progression Phase 2a Dose Expansion (Monotherapy) Patients with: * R/R AMLwith FLT3 mutations who have been previously treated with a FLT3 inhibitor * R/R AML with spliceosome mutations of SF3B1 or U2AF1 * R/R hrMDS (IPSS-R score \> 3.5) with spliceosome mutations of SF3B1 or U2AF1 * Number of prior treatments: 1 or 2 5. Acceptable organ function at screening 6. Ability to swallow and retain oral medications 7. Negative serum pregnancy test in women of childbearing potential 8. Women of childbearing potential and men who partner with a woman of childbearing potential must agree to use highly effective contraceptive methods for the duration of the study and for 180 days after the last dose of emavusertib 9. Willing and able to provide written informed consent and comply with the requirements of the trial 10. Able to undergo serial bone marrow sampling and peripheral blood sampling Exclusion Criteria: 1. Diagnosed with acute promyelocytic leukemia (APL, M3) 2. Has known active central nervous system (CNS) leukemia 3. Allogeneic hematopoietic stem cell transplant (Allo-HSCT) within 60 days of the first dose of emavusertib, or clinically significant graft-versus-host disease (GVHD) requiring ongoing up titration of immunosuppressive medications prior to start of emavusertib 4. Chronic myeloid leukemia (CML) 5. Any prior systemic anti-cancer treatment such as chemotherapy, immunomodulatory drug therapy, etc., received within 3 weeks (or 5 half-lives) prior to start of emavusertib. Localized radiation or surgical resection of skin cancers allowed. 6. Use of any investigational agent within 3 weeks or 5 half-lives, whichever is shorter, prior to start of emavusertib 7. Presence of an acute or chronic toxicity resulting from prior anti-cancer therapy, with the exception of alopecia that has not resolved to Grade ≤ 1 within 7 days prior to start of emavusertib. 8. Known allergy or hypersensitivity to any component of the formulation of emavusertib 9. Major surgery, other than diagnostic surgery, \<28 days from the start of emavusertib; minor surgery \<14 days from the start of emavusertib 10. Patients with active advanced malignant solid tumors 11. Known to be human immunodeficiency virus (HIV) positive or have an acquired immunodeficiency syndrome-related illness 12. Hepatitis B virus (HBV) deoxyribonucleic acid (DNA) positive or Hepatitis C virus (HCV) infection \<6 months prior to start of emavusertib unless viral load is undetectable, or HCV with cirrhosis 13. Uncontrolled or severe cardiovascular disease including myocardial infarction or unstable angina within 6 months prior to CA-4948, New York Heart Association Class II or greater congestive heart failure, or left ventricular ejection fraction \< 50% by echocardiogram or multi-gated acquisition scan, serious arrhythmias uncontrolled on treatment, clinically significant pericardial disease, cardiac amyloidosis, congenital long QT syndrome, or QTc with Fridericia's correction (QTcF) that is unmeasurable or \> 450 milliseconds (msec) on Screening electrocardiogram (ECG) 14. Gastrointestinal disease or disorder that could interfere with the swallowing, oral absorption, or tolerance of emavusertib 15. Pregnant or lactating 16. Systemic fungal, bacterial, viral, or other infection that is not controlled 17. Any other severe, acute, or chronic medical, psychiatric or social condition, or laboratory abnormality that may increase the risk of trial participation or emavusertib administration

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Conditions

The condition(s) this trial relates to.

acute myeloid leukemia hereditary sensory and autonomic neuropathy myelodysplastic syndrome Myelodysplastic Syndromes Recurrence

As listed by the trial registrant

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

Acute myelogenous leukemia Myelodysplastic syndrome

How to take part

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

  1. The places running it

    32 sites in 8 countries. The list below names each one and where it is.

  2. The official record

    The full official record for this study. This one lists no contact details, but it is the first place any would appear.

    Open the record ↗

  3. 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

Locations

  • APHP - Hopital Saint Louis

    Paris, 75475, France

  • APHP - Sorbonne Universite

    Paris, 75012, France

  • Albert Einstein Medical College

    The Bronx, New York, 10461, United States

  • Azienda Ospedaliera Santa Croce e Carle

    Cuneo, Italy

  • Dana Farber Cancer Institute

    Boston, Massachusetts, 02215, United States

  • Edith Wolfson Medical Center

    Holon, 5822012, Israel

  • Hadassah University MC

    Jerusalem, 9112001, Israel

  • Hospital Universitario Virgen del Rocio

    Seville, Spain

  • Hospital Universitaro del a Princesa

    Madrid, 28006, Spain

  • Hospital de la Santa Creu I Sant Pau (Neuvo Hospital)

    Barcelona, Spain

  • Instituto Romagnolo per lo Studio dei Tumori "Dino Amadori"

    Meldola, Italy

  • Karmanos Cancer Institute

    Detroit, Michigan, 48201, United States

  • Klinikum rechts der Isar der Technischen Universitat Munchen

    München, 81675, Germany

  • MD Anderson Cancer Center Madrid

    Madrid, Spain

  • Marien Hospital Dusseldorf; Klinik fur Onkologie und Hamatologie, Palliativmedizin

    Düsseldorf, 40479, Germany

  • Moffitt Cancer Center

    Tampa, Florida, 33612, United States

  • Northwestern Memorial Hospital

    Chicago, Illinois, 60611, United States

  • Novant Health Hematology - Forsyth

    Winston-Salem, North Carolina, 27103, United States

  • Oncology Hematology West, PC dba Nebraska Cancer Specialists

    Omaha, Nebraska, 68130, United States

  • Service d'hématologie clinique CHU de Nice

    Nice, 6200, France

  • Soroka University MC

    Beersheba, 84100, Israel

  • The Ohio State University Wexner Medical Center - James Cancer Hospital

    Columbus, Ohio, 43210, United States

  • The University of Texas MD Anderson Cancer Center

    Houston, Texas, 77030, United States

  • Universitatsklinikum Leipzig; Medizinische Klinik und Poliklinik I

    Leipzig, 04103, Germany

  • Universitatsklinikum Munster

    Münster, 48149, Germany

  • University Hospital in Krakow

    Krakow, 31 501, Poland

  • University of Chicago Medical Center

    Chicago, Illinois, 60637, United States

  • University of Rochester Medical Center

    Rochester, New York, 14642, United States

  • Universitätsklinikum Hamburg-Eppendorf (UKE)

    Hamburg, 20246, Germany

  • Uniwersyteckie Centrum Kliniczne Osrodek Badan Klinicznych Wczesnych Faz

    Gdansk, 80-214, Poland

  • Vseobecna Fakultni nemocnice v Praze

    Prague, 12 808, Czechia

  • Winship Cancer Institute

    Atlanta, Georgia, 30322, United States

More trials for these conditions

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