Paediatric patients (6 months to 18 years) diagnosed with AML who are refractory to front line therapy, early first relapse (less than 1 year from diagnosis) and second and subsequent relapse patients. MedDRA version: 9.1 Level: LLT Classification code 10000880 Term: Acute myeloid leukaemia MedDRA version: 9.1 Level: LLT Classification code 10066764 Term: Acute myelo
Conditions
Interventions
Trade Name: DaunoXome injection (liposomal daunorubicin)
Product Name: DaunoXome
Pharmaceutical Form: Intravenous infusion
INN or Proposed INN: DAUNORUB
Sponsors
University of Birmingham
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Diagnosis of acute myelogenous leukaemia (AML). - Patients must be in first relapse within 12 months of initial diagnosis or refractory to induction therapy or be in second or subsequent relapse. - Patients in first or subsequent relapse must have = 5% blasts in the bone marrow, with or without extramedullary disease AND immunophentypic confirmation of AML. - Patients with refractory AML following induction must have > 20% blasts in the bone marrow. - Age must be between 6 months to 18 years old. - Karnofsky or Lansky score of = 50 - Patients of childbearing potential must have a negative pregnancy test and agree to use an effective birth control method or evidence of post-menopausal status. Post-menopausal status is defined as either radiation induced oophorectomy with last menses >1 year ago; chemotherapy induced menopause with 1 year interval since last menses. - Normal renal function defined as Serum creatinine based on normal range for age/gender - Normal hepatic function defined as: Total bilirubin NORMAL for age. ALT OR AST 58% Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: - First relapse > 1 year from their initial diagnosis of AML - Patients whose previous daunorubicin equivalent exposure equals or exceeds 450mg/m2. - Isolated extramedullary leukemia. - Symptomatic CNS-involvement. - Any evidence of severe or uncontrolled systemic conditions (e.g., systemic infection) or current unstable or uncompensated respiratory or cardiac conditions which makes it undesirable for the patient to participate in the study or which could jeopardize compliance with the protocol. - Concurrent treatment or administration of any other experimental drug or with any other anti-cancer therapy. - Patients unable to be regularly followed up for psychological, social, family or geographic reasons. - Patient with expected non-compliance to toxicity management guidelines
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The main objective of the trial is to establish the maximum tolerated dose of clofarabine when used in combination with daunoXome.;Primary end point(s): The primary endpoint of this trial will be the appearance of Dose Limiting Toxicities. The Maximum Tolerated Dose will be defined by the number of Dose Limiting Toxicities during Cycle 1 of therapy.; Secondary Objective: - To characterise the safety and tolerability of the combination of clofarabine and daunoXome including identification of the dose-limiting toxicities. - To document the overall response rate, including (CR and CRi and PR) in this population. - To describe the durability of response and follow-up of these patients, including the number of patients that undergo stem-cell transplant after re-induction with clofarabine and daunoXome. | — |
Countries
United Kingdom
Outcome results
None listed