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).

Phase 1

• Conditions: Acute myeloid leukaemia (AML), Chronic myelomonocytic leukaemia (CMML) and Myelodysplastic syndrome (MDS); MedDRA version: 21.1Level: PTClassification code 10000880Term: Acute myeloid leukaemiaSystem Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps); MedDRA version: 21.1Level: PTClassification code 10028533Term: Myelodysplastic syndromeSystem Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps); MedDRA version: 21.0Level: LLTClassification code 10054350Term: Chronic myelomonocytic leukemiaSystem Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps); Therapeutic area: Diseases [C] - Cancer [C04]

• Registration Number: EUCTR2020-000168-53-NL
• Lead Sponsor: Ellipses Pharma Limited

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-03-19
• Last Update Posted: 8 January 2024

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 75

Inclusion Criteria

Core Inclusion Criteria:
1. Male or female patients aged = 18 years of age, at the time of informed consent, with histological or cytological confirmation of leukemia.
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.
Are the trial subjects under 18? no
Number of subjects for this age range: 0
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 20
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 43

Exclusion Criteria

Core Exclusion Criteria:
1. Suspected brain and/or leptomeningeal metastases that are symptomatic or untreated or that require current therapy.
2. Ongoing toxic manifestations of previous treatments that have not reduced to at least Common Terminology Criteria for Adverse Events (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.
3. Creatinine clearance (calculated using Cockcroft-Gault formula, or measured) < 50 mL/min.
4. Receiving an investigational anti-cancer treatment concurrently or within 14 days or five half-lives of either the parent drug or any active metabolite prior to the start of treatment with EP0042. Patients with AML may receive hydroxyurea throughout the screening period and during the first 2 cycles of study treatment in the first module (FIH study).
5. Current refractory nausea and vomiting, malabsorption syndrome, disease significantly affecting gastrointestinal (GI) function, resection of the stomach, extensive small bowel resection that is likely to affect absorption, symptomatic inflammatory bowel disease, partial or complete bowel obstruction, or gastric restrictions and bariatric surgery such as gastric bypass.
6. Known active hepatitis B virus (HBV) or hepatitis C virus (HCV) infection per institutional protocol. Testing for HBV or HCV status is not necessary unless clinically indicated or the patient has a history of HBV or HCV infection.
7. Patients with active human immunodeficiency virus infection (HIV) infection (testing is not required). Patients living with HIV will be eligible if they have CD4+ T-cell count = 350 cells/µL, no history of AIDS-defining opportunistic infections in the past 12 months, and can be managed on a regimen consistent with this protocol's permitted concomitant medications
8. Malignant disease other than that being treated in this study, with the following exceptions:
a. Malignancies that were treated curatively and have not recurred within 2 years prior to study treatment.
b. Completely resected basal cell and squamous cell skin cancers.
c. Any malignancy considered to be indolent and that has never required therapy.
d. Completely resected carcinoma in situ of any type.
9. Any medical condition that would, in the investigator’s judgment, prevent the patient’s participation in the clinical study due to safety concerns, compliance with clinical study procedures, or interpretation of study results.
10. Any major surgical procedure (in the investigator’s judgement) within 2 weeks of the first dose of study drug.
11. Pregnant, likely to become pregnant, or lactating women (where pregnancy is defined as the state of a female after conception and until the termination of gestation).

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified