Study to Evaluate the Safety and Tolerability of EP0042

October 10, 2025 updated by: Ellipses Pharma

A Modular, Multipart, Multi-arm, Open-label, Phase I/IIa Study to Evaluate the Safety and Tolerability of EP0042 Alone and in Combination With Anti-cancer Treatments in Patients With Advanced Malignancies

A research study looking at a new treatment for patients with advanced cancer, to investigate different doses of the experimental study drug, EP0042, in order to determine a dose, which is safe, well-tolerated and likely to be effective in treating AML (acute myeloid leukaemia).

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

70

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

General

  1. Male or female patients aged ≥ 18 years of age, at the time of informed consent, with histological or cytological confirmation of AML
  2. Ability to understand and provide written informed consent before any study-specific procedures, sampling, or analyses, including access to archival tumor tissue
  3. Ability to swallow and retain oral medication
  4. Sufficient life expectancy to allow the patient to complete at least 1 cycle (28 days) of the treatment period.
  5. Eastern Cooperative Oncology Group (ECOG) Performance Status of 0-2 at screening
  6. In the opinion of the investigator, all other relevant medical conditions must be well-managed and stable for at least 28 days prior to first administration of study drug
  7. Patients with pathologically confirmed/documented AML or MDS, as defined by the 2022 European LeukaemiaNet (ELN) recommendations, or CMML, as defined by World Health Organization (WHO) criteria, who have relapsed from or are refractory to previous therapy and have failed all (or are not eligible for/intolerant to) available approved therapies for their disease.
  8. Patients with relapsed/refractory FLT3 WT AML.
  9. Previous exposure to venetoclax, hypomethylating agent and/or FLT3 inhibitors (gilteritinib, midostorin, quizartinib, sorafenib) is allowed for relapsed/refractory AML patients.
  10. Mutation status of patients must be known at trial entry.
  11. Female patients should either be of non-child-bearing potential or must agree to use highly effective methods of contraception from Screening until 6 months following administration of the last dose of study drug
  12. Male patients must use double barrier contraception from enrolment through treatment and for 6 months following administration of the last dose of study drug

5.2 Core Exclusion Criteria

Patients with any of the following will not be included in the study:

Disease Under Study and Prior Anticancer Treatment

  1. Suspected brain and/or leptomeningeal metastases that are symptomatic or untreated or that require current therapy
  2. Acute promyelocytic leukemia (FAB:M3)
  3. Systemic anti-cancer therapy for the disease under study within 2 weeks of the first dose of study treatment. If the previous anti-cancer therapy has a very long half-life and may interact with EP0042, e.g. a strong CYP3A4 inhibitor, the washout period may need to be increased for safety reasons but will be no longer than 3 weeks (Concomitant hydroxyurea is acceptable and will be permitted throughout the screening period and during first 6 cycles of study treatment)
  4. Ongoing toxic manifestations of previous treatments that have not reduced to at least CTCAE Grade 1. Exceptions to this are alopecia or certain Grade 2 treatment related toxicities, which in the opinion of the Investigator should not exclude the patient.

  5. Transplantation (allogeneic or autologous) within last 90 days, or on active immunosuppressive therapy for graft versus host disease in last 2 weeks

    Laboratory Parameters

  6. Patient with any out-of-range laboratory values defined as shown below.

    • Creatinine clearance (calculated using Cockcroft-Gault formula, or measured) < 50 mL/ min

  7. Inadequate liver function as demonstrated by

    • total serum bilirubin ≥ 1.5 times the upper limits of normal range (ULN) or
    • ALT ≥3 times the ULN or
    • AST ≥3 times the ULN or
    • AST or ALT ≥5 times the ULN in the presence of liver involvement by leukemia

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Module 1 EP0042
Establishing the safety of EP0042 as a monotherapy and establishing an appropriate dose to take forward into subsequent modules.
Drug: EP0042
EP0042 Oral 20 mg 50 mg capsules
Experimental: Module 2 EP0042
  • Part A: To evaluate the safety, tolerability, of EP0042 + a Bcl-2 inhibitor (venetoclax) in patients with R/R FLT3 wildtype (WT) AML.
  • Part B: To evaluate the safety, tolerability, of EP0042 in combination with a Bcl 2 inhibitor (venetoclax) + a hypomethylating agent (azacitidine) in patients with R/R FLT3 WT or newly diagnosed AML.
Drug: Venetoclax
Venetoclax
Drug: EP0042
EP0042 Oral 20 mg 50 mg capsules
Drug: Azacitidine (AZA)
Azacitidine

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Incidence of dose-limiting toxicities (DLTs) from the first dose through the end of the DLT observation period.
Time Frame: First cycle of treatment (28 Days)
Incidence of dose-limiting toxicities (DLT)
First cycle of treatment (28 Days)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Ellipses Pharma

Investigators

  • Principal Investigator: David Taussig, The Royal Marsden, UK

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 2, 2020

Primary Completion (Estimated)

October 1, 2025

Study Completion (Estimated)

December 1, 2026

Study Registration Dates

First Submitted

September 16, 2020

First Submitted That Met QC Criteria

October 5, 2020

First Posted (Actual)

October 9, 2020

Study Record Updates

Last Update Posted (Estimated)

October 14, 2025

Last Update Submitted That Met QC Criteria

October 10, 2025

Last Verified

October 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • EP0042-101

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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