Study to Evaluate the Safety and Tolerability of EP0042

NCT04581512 | Recruiting Phase 1 FDA Drug

• 1 other identifier: EP0042-101
• Study Type: interventional
• Target: 70
• Locations: 3 countries
• Sites: 6

Brief Summary

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).

Conditions

Acute Myeloid Leukemia; Chronic Myelomonocytic Leukemia; Myelodysplastic Syndromes

Outcome Measures

Primary Outcomes (1)

• Incidence of dose-limiting toxicities (DLTs) from the first dose through the end of the DLT observation period.

  Incidence of dose-limiting toxicities (DLT)

  First cycle of treatment (28 Days)

Study Arms (2)

Module 1 EP0042

EXPERIMENTAL

Establishing the safety of EP0042 as a monotherapy and establishing an appropriate dose to take forward into subsequent modules.

Drug: EP0042

Module 2 EP0042

EXPERIMENTAL

* 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: EP0042; Drug: Venetoclax; Drug: Azacitidine (AZA)

Interventions

EP0042 DRUG

EP0042 Oral 20 mg 50 mg capsules

Module 1 EP0042; Module 2 EP0042

Venetoclax DRUG

Venetoclax

Module 2 EP0042

Azacitidine (AZA) DRUG

Azacitidine

Module 2 EP0042

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may not qualify if:

• Patients with any of the following will not be included in the study:
• Disease Under Study and Prior Anticancer Treatment
• Suspected brain and/or leptomeningeal metastases that are symptomatic or untreated or that require current therapy
• Acute promyelocytic leukemia (FAB:M3)
• 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)
• 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.
• Transplantation (allogeneic or autologous) within last 90 days, or on active immunosuppressive therapy for graft versus host disease in last 2 weeks
• Laboratory Parameters
• Patient with any out-of-range laboratory values defined as shown below.
• Creatinine clearance (calculated using Cockcroft-Gault formula, or measured) \< 50 mL/ min
• 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

Sponsors & Collaborators

• Ellipses Pharma: lead

Study Sites (6)

Royal Perth Hospital

Perth, Western Australia, 6000, Australia

RECRUITING

Amsterdam UMC

Amsterdam, Amsterdam, 1081HV Amsterdam, Netherlands

RECRUITING

Erasmus MC

Rotterdam, Rotterdam, 3000 CA Rotterdam, Netherlands

RECRUITING

University College London Hospital

London, London, W1T 7HA, United Kingdom

RECRUITING

The Royal Marsden

London, UK, United Kingdom

RECRUITING

The Christie Hospital

Manchester, M204BX, United Kingdom

RECRUITING

MeSH Terms

Conditions

Leukemia, Myeloid, Acute; Leukemia, Myelomonocytic, Chronic; Myelodysplastic Syndromes

Interventions

venetoclax; Azacitidine

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Myelodysplastic-Myeloproliferative Diseases; Bone Marrow Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Aza Compounds; Organic Chemicals; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Ribonucleosides

Study Officials

• David Taussig

  The Royal Marsden, UK

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Clinical Trials Team

CONTACT

+44 20 3743 0992; Enquiries@ellipses.life

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 16, 2020
• First Posted: October 9, 2020
• Study Start: November 2, 2020
• Primary Completion: October 1, 2025
• Study Completion (Estimated): December 1, 2026
• Last Updated: October 14, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will not share

Locations

AU (1); GB (3); NL (2)