Study to Evaluate the Safety and Tolerability of EP0042

NCT ID: NCT04581512

Last Updated: 2025-10-14

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

70 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-11-02

Study Completion Date

2026-12-31

Brief Summary

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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).

Detailed Description

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Conditions

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Acute Myeloid Leukemia Chronic Myelomonocytic Leukemia Myelodysplastic Syndromes

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Module 1 EP0042

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

Group Type EXPERIMENTAL

EP0042

Intervention Type DRUG

EP0042 Oral 20 mg 50 mg capsules

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.

Group Type EXPERIMENTAL

EP0042

Intervention Type DRUG

EP0042 Oral 20 mg 50 mg capsules

Venetoclax

Intervention Type DRUG

Venetoclax

Azacitidine (AZA)

Intervention Type DRUG

Azacitidine

Interventions

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EP0042

EP0042 Oral 20 mg 50 mg capsules

Intervention Type DRUG

Venetoclax

Venetoclax

Intervention Type DRUG

Azacitidine (AZA)

Azacitidine

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

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

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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Ellipses Pharma

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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David Taussig

Role: PRINCIPAL_INVESTIGATOR

The Royal Marsden, UK

Locations

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Royal Perth Hospital

Perth, Western Australia, Australia

Site Status RECRUITING

Amsterdam UMC

Amsterdam, Amsterdam, Netherlands

Site Status RECRUITING

Erasmus MC

Rotterdam, Rotterdam, Netherlands

Site Status RECRUITING

University College London Hospital

London, London, United Kingdom

Site Status RECRUITING

The Royal Marsden

London, UK, United Kingdom

Site Status RECRUITING

The Christie Hospital

Manchester, , United Kingdom

Site Status RECRUITING

Countries

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Australia Netherlands United Kingdom

Central Contacts

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Clinical Trials Team

Role: CONTACT

+44 20 3743 0992

Facility Contacts

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Kanako Ohara

Role: primary

Jeroen Janssen, Dr

Role: primary

Mojca Jongen-Lavrencic, Dr

Role: primary

Jenny O'Nions, Dr

Role: primary

David Taussig, Dr

Role: primary

Role: backup

Emma Searle

Role: primary

Other Identifiers

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EP0042-101

Identifier Type: -

Identifier Source: org_study_id

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