AzTrials
AzTrials

Acute Myeloid LeukemiaPhase 2NCT05024994

A Study of E7820 in People With Bone Marrow (Myeloid) Cancers

A Phase 2 study testing E7820 in people with Acute Myeloid Leukemia and Myelodysplastic Syndromes, run by Memorial Sloan Kettering Cancer Center at 7 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

The researchers are doing this study to find out whether E7820 is an effective treatment for people with relapsed/refractory myeloid cancers with mutations in splicing factor genes. Participants will have acute myeloid leukemia (AML), myelodysplastic syndromes (MDS), or chronic myelomonocytic leukemia (CMML). Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
E7820
Could I receive a placebo?
No, everyone receives an active treatment
How many people
12 participants
Where
7 sitesUnited States
Study dates
13 Aug 2021to 19 Jun 2025
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 1. Subject is ≥ 18 years of age at the time of signing informed consent 2. Subject is willing and able to adhere to the study visit schedule and other protocol requirements. 3. Subject has relapsed or refractory MDS, AML or CMML with a previously defined hotspot splicing factor mutation in SF3B1, SRSF2, U2AF1, or U2AF2 (with hotspot mutations as defined by OncoKB) or a nonsense or frameshift mutation in ZRSR2. A splicing factor mutation is required to be detected on next generation sequencing from bone marrow aspirate or peripheral blood at any timepoint within the 6 months prior to screening for the study.
  • Relapsed AML is defined as: i. The appearance of 5% or greater myeloblasts in the bone marrow or peripheral blood after achieving a CR (MRD positive or negative), CRh, or CRi
  • 1. Patients with mutations in FLT3, IDH1 or IDH2 must have failed or been intolerant of an FDA approved FLT3, IDH1 or IDH2 inhibitor before enrolling on study.
  • Refractory AML is defined as failure to achieve a CR, CRh, or CRi after one of the following regimens: i. Two cycles of intensive induction chemotherapy with a cytarabine containing regimen (e.g. 7+3, MEC, HIDAC, etc.) ii. Two cycles of HMA/venetoclax or LDAC/glasdegib iii. 4 cycles of HMA monotherapy c. Relapsed MDS is defined as: i. Any relapse after achieving an IWG defined response. d. Refractory MDS is defined as: i. For patients with intermediate, high or very high risk disease by IPSS-R, the failure to achieve a response (as per IWG 2006 criteria) after 4 cycles of HMA monotherapy or 2 cycles of HMA + venetoclax.
  • For patients with very low and low risk disease by IPSS-R failure to achieve hematologic improvement or loss of hematologic improvement after treatment with standard of care agents such as ESAs, Luspatercept (for MDS with ringed sideroblasts) and lenalidomide (for pts with a 5q-).
  • Relapsed CMML is defined as: i. Any relapse after achieving an IWG defined response. f. Refractory CMML is defined as: i. Failure to achieve a response (as per IWG 2006 criteria) after 4 cycles of HMA monotherapy or 2 cycles of HMA + venetoclax.
Show all 13Show fewer
  • 4. Subject has an Eastern Cooperative Oncology Group (ECOG) performance status of 0-3 5. Subject has adequate organ function defined as:
  • Serum aspartate aminotransferase/serum glutamic oxaloacetic transaminase (AST/SGOT) and alanine aminotransferase (ALT/SGPT) ≤ 3 x ULN, unless considered due to organ involvement by the patient's myeloid malignancy (in that case a cut off of ≤ 5 x ULN will be used)
  • Serum direct bilirubin < 1.5 x ULN.
  • Creatinine clearance ≥ 60 mL/min based on the Cockroft-Gault glomerular filtration rate (GFR) estimation.
  • Females of childbearing potential may participate provided they have a negative serum pregnancy test at screening and a negative serum OR urine pregnancy test within 72 hours of starting on treatment. Females and male participants with female partners of childbearing potential also must agree to either abstain from sexual intercourse or use a highly effective method of contraception while on study and for 4 months after completing the study treatment.
  • 6. There are no limits on transfusion and/or growth factor support for enrollment.
  • 7. In case of leukemic organ involvement, patients with creatinine clearance > 30 ml/min and bilirubin ≤ 2.0 x ULN will be eligible to be included.

Who cannot join

  • Patients with acute promyelocytic leukemia
  • Subject has immediate life-threatening, severe complications of their myeloid malignancy such as uncontrolled bleeding, pneumonia with hypoxia or shock, and/or disseminated intravascular coagulation
  • Subject has significant active cardiac disease within 6 months prior to the start of study treatment, including New York Heart Association (NYHA) class III or IV congestive heart failure; acute coronary syndrome (ACS); and/or stroke or left ventricular ejection fraction (LVEF) <40% by echocardiogram (ECHO) or multi-gated acquisition (MUGA) scan obtained within 28 days prior to the start of study treatment.
  • Subject has active viral infection with human immunodeficiency virus (HIV), or active infection with hepatitis B virus (HBV) or hepatitis C virus (HCV). Patients with HIV that is controlled with HAART are eligible to participate.
  • Subject is known to have dysphagia, short-gut syndrome, gastroparesis, or other conditions that limit the ingestion or gastrointestinal absorption of drugs administered orally.
  • Subject has active uncontrolled systemic fungal, bacterial, or viral infection (defined as ongoing signs/symptoms related to the infection without improvement despite appropriate antibiotics, antiviral therapy, and/or other treatment).
Show all 9Show fewer
  • Subject has QTc interval (i.e., Fridericia's correction [QTcF]) ≥ 480 ms or other factors that increase the risk of QT prolongation or arrhythmic events (e.g., heart failure family history of long QT interval syndrome) at screening. Patients with left bundle branch block or right bundle branch block with prolonged QTc will be allowed to enroll on the trial with medical monitor approval.
  • Female subject who is pregnant or lactating.
  • Subject with known hypersensitivity to sulfa medications

Trial locations and contacts

7 sites in 1 country.

7 sites in 1 country

United States7 sites

  • Memorial Sloan Kettering Basking Ridge

    Basking Ridge, New Jersey, 07920

  • Memorial Sloan Kettering Commack

    Commack, New York, 11725

  • Memorial Sloan Kettering Westchester (All protocol activities)

    Harrison, New York, 10604

  • University of Miami (Data Collection AND Specimen Analysis Only)

    Miami, Florida, 33136

  • Memorial Sloan Kettering Monmouth

    Middletown, New Jersey, 07748

  • Memorial Sloan Kettering Bergen

    Montvale, New Jersey, 07645

  • Memorial Sloan Kettering Nassau

    Uniondale, New York, 11553

Other trials in United States

About this study

Official title: A Phase II Clinical Trial of E7820 for Patients With Relapsed/Refractory Myeloid Malignancies With Mutations in Splicing Factor Genes.

The researchers are doing this study to find out whether E7820 is an effective treatment for people with relapsed/refractory myeloid cancers with mutations in splicing factor genes. Participants will have acute myeloid leukemia (AML), myelodysplastic syndromes (MDS), or chronic myelomonocytic leukemia (CMML).

Being tested
E7820
Keywords
E7820, Bone Marrow cancers, Myeloid, Relapsed/Refractory, Mutations in Splicing Factor Genes, 21-159
Medical subject headings
Leukemia, Myeloid, Acute, Myelodysplastic Syndromes, Leukemia, Myelomonocytic, Chronic, Recurrence

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • E7820Drug

    100mg of E7820 QD

Study groups in NCT05024994
Study groupRoleWhat they receive
E7820ExperimentalEach patient will receive daily administration of E7820. The starting dose for every patient will be 100 mg daily but the dose can subsequently be reduced if excessive toxicity is encountered.
Receives: Drug: E7820

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Median Overall Survival

    To assess the 1 year Overall Survival (OS)

    Measured: 1 year

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted27 Aug 2021
  2. Study start13 Aug 2021
  3. Primary completion19 Jun 2025
  4. Study completion19 Jun 2025

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Model description
This is a phase II study exploring the efficacy of E7820 in patients with relapsed and refractory myeloid malignancies with mutations in splicing factors.
Masking
None (open label)
Enrolment
12 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05024994
Sponsor's protocol ID
21-159
Status last verified
Jun 2025
First submitted
23 Aug 2021
Last updated
17 Jun 2026
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: Memorial Sloan Kettering Cancer Center supports the international committee of medical journal editors (ICMJE) and the ethical obligation of responsible sharing of data from clinical trials. The protocol summary, a statistical summary, and informed consent form will be made available on clinicaltrials.gov when required as a condition of Federal awards, other agreements supporting the research and/or as otherwise required. Requests for deidentified individual participant data can be made beginning 12 months after publication and for up to 36 months post publication. Deidentified individual participant data reported in the manuscript will be shared under the terms of a Data Use Agreement and may only be used for approved proposals. Requests may be made to: crdatashare@mskcc.org.

Publications

  • Maron MI, Abdel-Wahab O. Outstanding Questions to Understand and Target Splicing Factor-Mutant Blood Cancers. Blood Cancer Discov. 2026 May 5;7(3):348-352. doi: 10.1158/2643-3230.BCD-26-0047.

    PubMed 42013378

  • Bewersdorf JP, Stahl M, Taylor J, Mi X, Chandhok NS, Watts J, Derkach A, Wysocki M, Lu SX, Bourcier J, Hogg SJ, Rahman J, Chaudhry S, Totiger TM, Abdel-Wahab O, Stein EM. E7820, an anti-cancer sulfonamide, degrades RBM39 in patients with splicing factor mutant myeloid malignancies: a phase II clinical trial. Leukemia. 2023 Dec;37(12):2512-2516. doi: 10.1038/s41375-023-02050-4. Epub 2023 Oct 9. No abstract available.

    PubMed 37814121

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing E7820 in people with Acute Myeloid Leukemia and Myelodysplastic Syndromes, run by Memorial Sloan Kettering Cancer Center at 7 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 13 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 17 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 7 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 17 Jun 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Median Overall Survival (time frame: 1 year). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05024994, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Jun 2026.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in