Status:

RECRUITING

Vyxeos® With Clofarabine for Pediatric AML

Lead Sponsor:

Princess Maxima Center for Pediatric Oncology

Collaborating Sponsors:

Jazz Pharmaceuticals

Conditions:

Relapsed Pediatric AML

Refractory Pediatric AML

Eligibility:

All Genders

1-21 years

Phase:

PHASE1

Brief Summary

Treatment with intensive chemotherapy in AML results in approximately 70% survival in newly diagnosed patients. Prognosis at relapse is worse and is in the 30-40% range. Relapse treatment generally co...

Eligibility Criteria

Inclusion

  • We will include pediatric patients ≥1 year and ≤21 years with:
  • Any ≥ 2nd relapse of AML
  • Refractory AML (defined as ≥ 20% blasts in the bone marrow after standard (re-) induction therapy)
  • Early 1st relapse (defined as relapse within one year from initial diagnosis) of AML
  • Any relapse of AML after prior allogenic HSCT
  • Any relapse of AML with high risk cytogenetic characteristics (as defined in Appendix V)
  • In order to be eligible to participate in this study, a subject must meet all of the following criteria:
  • Initial work-up:
  • • Complete initial work-up within 7 days prior to study entry, including bone-marrow aspiration, lumbar puncture (without intrathecal therapy)
  • General condition:
  • Lansky play score ≥ 60 for patients \<16 years of age; or Karnofsky performance status ≥ 60 for patients ≥ 16 years of age (see Appendix I for Performance scales).
  • Life expectancy \> 6 weeks
  • The patient must have a calculated GFR ≥ 70mL/min/1.73 m2.
  • Liver function: total serum bilirubin ≤ 3 mg/dl or 50 μmol/L and aspartate transaminase (AST) and alanine transaminase (ALT) ≤200 U/L
  • Adequate cardiac function (defined as shortening fraction ≥28% or ejection fraction ≥50%)
  • No evidence of a currently uncontrolled bacterial, viral or parasitic infection
  • No 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)
  • No evidence of isolated extramedullary relapse, including isolated CNS-relapse
  • No evidence of CNS3 or symptomatic CNS leukemia
  • No Down Syndrome
  • No evidence of relapsed/refractory acute promyelocytic leukemia (APL)
  • No 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)
  • No history of prior veno-occlusive disease (VOD)
  • No known hypersensitivity to cytarabine, clofarabine or liposomal daunorubicin
  • No known copper metabolism deficiency, such as Wilson's disease.
  • Other:
  • For female patients with childbearing potential, a negative test for pregnancy is to be performed before entry on study.
  • Male and female patients must use a highly effective contraceptive method according to the CTFG 2014-guidelines during the study and for a minimum of 6 months after study treatment.
  • NL72866.041.20 / Vyxeos liposomal and Clofarabine in R/R pediatric AML - ITCC-092 Protocol version: 2.2, 08-04-2021 38 of 80
  • Female patients may not breast feed during the study and for a minimum of 3 months after study treatment.
  • Absence of any psychological, familial, sociological or geographical condition potentially hampering compliance with the study protocol and follow-up schedule is required; those conditions should be discussed with the patient before registration in the trial.
  • Before patient registration/randomization, written informed consent must be given according to ICH/GCP, and national/local regulations.
  • Concomitant treatments:
  • Concomitant administration of any other experimental drug under investigation, or concurrent treatment with any other anti-cancer therapy other than specified in the protocol is not allowed.
  • GCSF will not be used for priming and no routine GCSF support is allowed during the 1st course, except for life-threatening infections.
  • Additional criteria:
  • • At least 6 patients must be enrolled with an M3 or a WBC count \>10x109/L with blasts.

Exclusion

    Key Trial Info

    Start Date :

    November 6 2020

    Trial Type :

    INTERVENTIONAL

    Allocation :

    ESTIMATED

    End Date :

    December 31 2028

    Estimated Enrollment :

    25 Patients enrolled

    Trial Details

    Trial ID

    NCT07156435

    Start Date

    November 6 2020

    End Date

    December 31 2028

    Last Update

    September 5 2025

    Active Locations (13)

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    Page 1 of 4 (13 locations)

    1

    St. Anna Kinderspital

    Vienna, Austria

    2

    Rigshospitalet

    Copenhagen, Denmark

    3

    Universitätsklinikum Augsburg

    Augsburg, Germany

    4

    Charité Berlin

    Berlin, Germany