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Looking for participantsPhase1/Phase2

Study to Evaluate the Safety and Tolerability of EP0042

Sponsor: Ellipses Pharma

NCT ID: NCT04581512

View on ClinicalTrials.gov ↗

At a glance

What the study gets you
Access to the study treatment being tested
Type of study
Interventional (receives a drug or procedure)
Time in hospital
In-person visits at study sites — visit count not specified by the sponsor
Drug or intervention
EP0042 (drug), Venetoclax (drug), Azacitidine (AZA) (drug)
How long the study runs
Study runs about 85 months (dates as stated)
About the drug or intervention
EP0042 — drug: EP0042 Oral 20 mg 50 mg capsules · Venetoclax — drug: Venetoclax · Azacitidine (AZA) — drug: Azacitidine
Patient visit burden
Not specified by the sponsor

In plain English

This study is testing a new medicine called EP0042 to see how safe it is and how well people cope with it. It is for adults with acute myeloid leukaemia (AML), chronic myelomonocytic leukaemia (CMML), or myelodysplastic syndromes (MDS) whose disease has come back or has not responded to previous treatment. The study is run by Ellipses Pharma.

Who can take part

  • Adults aged 18 or over with confirmed AML, MDS or CMML
  • Disease has come back (relapsed) or has not responded (refractory) to previous treatment, and other approved treatments are not an option
  • Able to give written informed consent and to swallow and keep down tablets
  • Well enough to complete at least one 28-day treatment cycle, with enough life expectancy to do so
  • Performance status (how well you can carry out daily activities) of 0 to 2 at screening
  • Other medical conditions must be well managed and stable for at least 28 days before starting the study drug
  • Known mutation status at the start of the trial, including AML without a FLT3 gene change (FLT3 wild type)
  • Previous treatment with venetoclax, hypomethylating agents or FLT3 inhibitors is allowed
  • People able to become pregnant, or male participants, must agree to use highly effective contraception during treatment and for 6 months after the last dose

Who may not be able to

  • Suspected cancer spread to the brain or the lining around it that is causing symptoms, is untreated, or needs treatment now
  • A specific type of AML called acute promyelocytic leukaemia
  • Anti-cancer treatment for this disease within 2 weeks of the first study dose (up to 3 weeks for some longer-lasting medicines). Hydroxyurea is allowed during screening and the first 6 treatment cycles
  • Ongoing side effects from previous treatment that have not improved enough, except hair loss or some milder side effects a doctor judges acceptable
  • A stem cell or bone marrow transplant (from yourself or a donor) in the last 90 days, or taking immunosuppressive drugs for graft versus host disease in the last 2 weeks
  • Kidney problems: creatinine clearance (a measure of kidney function) below 50 mL/min
  • Liver problems: bilirubin 1.5 times or more the upper limit of normal, or liver enzymes (ALT or AST) above set limits, or higher limits if leukaemia affects the liver

What taking part involves

  • • Taking EP0042 as a tablet by mouth
  • • Treatment is given in cycles, each lasting 28 days

Time commitment: Not stated — ask the trial team. Each treatment cycle lasts 28 days, and participants need to be able to complete at least one cycle.

Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.

Type of study
Testing a treatment
Ages
18 Years and over
Who
All
Number of participants
70
Started
2020-11-02
Last checked
2026-06

Plain English Summary

What is this study?

  • • Testing a new treatment for acute myeloid leukemia
  • • Phase1/Phase2 - 70 participants
  • • 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)

Who can take part?

  • • Ages 18 Years and over
  • • Diagnosed with acute myeloid leukemia

Where?

  • • London - The Royal Marsden
  • • London - University College London Hospital
  • • Manchester - The Christie Hospital

This is a simplified summary. Always discuss with your doctor before making any decisions.

About This Trial

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).

Acute Myeloid LeukemiaChronic Myelomonocytic LeukemiaMyelodysplastic Syndromes

How this trial compares with your answers

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What we know so far

Condition· Matched your search
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Gender· Tell us your sex for better matching

Still need:

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  • • Tell us your sex for better matching

Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.

Eligibility at a Glance

Key info

  • Age: 18 Years and over
  • Who can join: All genders

Biomarkers mentioned

FLT3

Treatment history

Treatments you must have had:

  • ✓ and have failed all (
  • ✓ current therapy

What the study is looking for

  • ✓General
  • ✓Male or female patients aged ≥ 18 years of age, at the time of agreement to take part, with histological or cytological...
  • ✓Ability to understand and provide written agreement to take part before any study-specific procedures, sampling, or...
  • ✓Ability to swallow and retain oral medication
  • ✓Sufficient life expectancy to allow the patient to complete at least 1 cycle (28 days) of the treatment period.
See the full criteria
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, midostaurin, 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

Where Is This Study? (3 UK sites)

The Royal Marsden

London, United Kingdom

Recruiting
Hospital R&D contact (matched)

Mark Brandon-Grove

research.development@rmh.nhs.uk020 3186 5416

University College London Hospital

London W1T 7HA, United Kingdom

Recruiting
Hospital R&D contact (matched)

Rajinder Sidhu - Associate Director, Research Governance and Operations

uclh.jro-communications@nhs.net020 3447 9825

The Christie Hospital

Manchester M204BX, United Kingdom

Recruiting
Hospital R&D contact (matched)

Research and Innovation Office

the-christie.ri@nhs.net---

How to Get in Touch

Clinical Trials Team

Sponsor contact

CONTACT

+44 20 3743 0992 Enquiries@ellipses.life
Data sourced from ClinicalTrials.gov · Last verified: 2026-06