NCT05024994 · REGISTRY CONDITION SEARCH

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

Found through the registry condition index, not selected as a treatment recommendation. This discovery collection was restricted to studies with posted results.

Phase: PHASE2
Status at capture: COMPLETED
Registry last update: 2026-06-17

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).

Open the original ClinicalTrials.gov record → · Download preserved record

What did the study report?

Outcomes, safety and baseline populations are separate source sections. Quality-of-life measures appear under their original outcome titles.

flow

Participant flow

→

baseline

Baseline characteristics

→

outcome · POSTED

Median Overall Survival

→

adverse events

Adverse events

→

more info

Limitations, agreements, and source contact

→

Who could take part

eligibility Criteria: Inclusion Criteria: 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. a. 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. b. 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. ii. 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-). e. 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. 4\. Subject has an Eastern Cooperative Oncology Group (ECOG) performance status of 0-3 5. Subject has adequate organ function defined as: 1. 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) 2. Serum direct bilirubin \< 1.5 x ULN. 3. Creatinine clearance ≥ 60 mL/min based on the Cockroft-Gault glomerular filtration rate (GFR) estimation. 4. 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. Exclusion Criteria: 1. Patients with acute promyelocytic leukemia 2. 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 3. 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. 4. 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. 5. Subject is known to have dysphagia, short-gut syndrome, gastroparesis, or other conditions that limit the ingestion or gastrointestinal absorption of drugs administered orally. 6. 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). 7. 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. 8. Female subject who is pregnant or lactating. 9. Subject with known hypersensitivity to sulfa medications
healthy Volunteers: false
minimum Age: 18 Years
sex: ALL
std Ages: ADULT
OLDER_ADULT

Treatment arms and interventions

arm Groups

description
Each 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.
intervention Names
Drug: E7820
label
E7820
type
EXPERIMENTAL

interventions

arm Group Labels
E7820
description
100mg of E7820 QD
name
E7820
type
DRUG

Study design

allocation

intervention Model

SINGLE_GROUP

intervention 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 Info

masking: NONE

primary Purpose

TREATMENT

Enrollment

count: 12
type: ACTUAL

Registered outcome plans (not posted results)

primary Outcomes

description
To assess the 1 year Overall Survival (OS)
measure
Median Overall Survival
time Frame
1 year

Full study description

brief 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).

Source references

references

citation
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.
pmid
42013378
type
DERIVED
citation
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.
pmid
37814121
type
DERIVED