Cytarabine; Daunorubicin for Acute myeloid leukemia (AML)|Relapsed/refractory AML

Inclusion criteria

• {"criterion_text":"- Any ≥ 2nd relapse of AML\n- Refractory AML (defined as ≥ 20% blasts in the bone marrow after standard (re-) induction therapy)\n- Early 1st relapse (defined as relapse within one year from initial diagnosis) of AML\n- Any relapse of AML after prior allogenic HSCT\n- Any relapse of AML with high risk cytogenetic characteristics\n- Complete initial work-up within 7 days prior to study entry, including bone-marrow aspiration, lumbar puncture (without intrathecal therapy)\n- Lansky play score ≥ 60 for patients <16 years of age; or Karnofsky performance status ≥ 60 for patients ≥ 16 years of age\n- Life expectancy > 6 weeks\n- a calculated GFR ≥ 70mL/min/1.73 m2\n- Liver function: total serum bilirubin ≤ 3 mg/dl or 50 µmol/L and aspartate transaminase (AST) and alanine transaminase (ALT) ≤200 U/L\n- Adequate cardiac function (defined as shortening fraction ≥28% or ejection fraction ≥50%)"}

Exclusion criteria

• {"criterion_text":"- evidence of a currently uncontrolled bacterial, viral or parasitic infection\n- evidence of a fungal infection, defined as either: - Pulmonary infiltrates suggestive of a fungal infection at HR-CT (within 3 weeks prior to enrollment) - Positive Aspergillus serum test (galactomannan), according to local laboratory practice (within 3 weeks prior to enrollment)\n- evidence of isolated extramedullary relapse, including isolated CNS-relapse\n- evidence of CNS3 or symptomatic CNS leukemia\n- Down Syndrome\n- evidence of relapsed/refractory acute promyelocytic leukemia (APL)\n- use of any anticancer therapy within 2 weeks before study entry. The patient must have recovered from all acute toxicities from any previous therapy (note: hematological toxicities do not need to be considered since the patient has overt leukemia)\n- history of prior veno-occlusive disease (VOD)\n- known hypersensitivity to cytarabine, clofarabine or liposomal daunorubicin\n- known copper metabolism deficiency, such as Wilson's disease."}

Primary endpoints

• {"endpoint_text":"- Frequency of Dose-limiting toxicities (DLTs) during the first course of therapy","definition_or_measurement_approach":"Measured as the frequency of DLTs occurring during the first course of therapy (DLT assessment during first treatment course)."}

Secondary endpoints

• {"endpoint_text":"- 1. Safety and tolerability: frequency of AEs, frequency of clinically significant laboratory abnormalities and number of toxic deaths\n- 2. Measures of anti-leukemic activity\n- 3. Overall patient survival and relapse-free survival\n- 4. Number of patients undergoing HSCT after treatment.\n- 5. Serum and intracellular pharmacokinetic parameters\n- 6. Relationship between response (ORR) and Ara-CTP accumulation\n- 7.Correlation between duration of response and measurable residual disease assessed by flow-cytometry","definition_or_measurement_approach":"Safety/tolerability: frequency of adverse events, clinically significant lab abnormalities, and number of toxic deaths. Anti-leukemic activity: response rate (ORR) determined by morphology with flow cytometric confirmation (including CR, CRi, PR). Survival endpoints: overall survival and relapse-free survival measured over follow-up. HSCT: count of patients undergoing hematopoietic stem cell transplant after treatment. PK: serum and intracellular PK parameters measured. Relationship analyses: correlation of ORR with Ara-CTP accumulation and of duration of response with measurable residual disease assessed by flow cytometry."}

Austria

Latest Decision Or Authorization Date

13-05-2025

Number Of Sites

1

Number Of Participants

7

Denmark

Latest Decision Or Authorization Date

15-04-2025

Number Of Sites

1

Number Of Participants

3

Germany

Latest Decision Or Authorization Date

15-04-2025

Number Of Sites

5

Number Of Participants

3

Italy

Latest Decision Or Authorization Date

16-04-2025

Number Of Sites

2

Number Of Participants

5

Netherlands

Latest Decision Or Authorization Date

23-07-2025

Number Of Sites

1

Number Of Participants

3

Spain

Latest Decision Or Authorization Date

21-10-2025

Number Of Sites

3

Number Of Participants

4

Primary sponsor

Full Name

Prinses Maxima Centrum voor Kinderoncologie B.V.

Organisation Type

Hospital/Clinic/Other health care facility

Country Of Registered Address

Netherlands

Contract research organisations

Name

Julius Clinical International B.V.

Responsibilities

Sponsor duties (code 1) as listed in the record; contact/liaison for trial conduct (email: ella.wijnia@juliusclinical.com)

Third parties

• {"country":"Netherlands","full_name":"Julius Clinical International B.V.","duties_or_roles":"Sponsor duties (code 1) listed in record; contact: ella.wijnia@juliusclinical.com","organisation_type":"Pharmaceutical company (CRO service provider listed as third party)"}