Condition category
Date applied
Date assigned
Last edited
Retrospectively registered
Overall trial status
Recruitment status
No longer recruiting

Contact information



Primary contact

Dr Pam Kearns


Contact details

Institute of Child Health
4th Floor
Whittal Street
B6 6NH
United Kingdom
+44 (0)121 333 8238

Additional identifiers

EudraCT number number

Protocol/serial number


Study information

Scientific title

Phase I escalation study of clofarabine (Clolar®) and liposomal daunorubicin (DaunoXome®) in childhood and adolescent acute myeloid leukaemia



Study hypothesis

To establish the maximum tolerated dose of clofarabine (Clolar®) when used in combination with DaunoXome®.

On 01/03/2011 the anticipated end date was changed from 01/06/2010 to 30/06/2011.

Ethics approval

Amended 11/02/2010: West Midlands Research Ethics Committee, 10/02/2009, ref: 08/H1208/36

Study design

Multicentre prospective non blinded open label phase I dose escalation study

Primary study design


Secondary study design

Randomised controlled trial

Trial setting


Trial type


Patient information sheet

Not available in web format, please use the contact details below to request a patient information sheet


Acute myeloid leukaemia


The calculation of dosage is based on the patients body surface area. The dose of DaunoXome® is fixed for all cohorts at 60 mg/m^2. DaunoXome® is given intravenously over 2 hours and start 4 hours after the start of Clofarabine (Clolar®) on days 1, 3 and 5. The starting dose of Clofarabine (Clolar®) will be 60% of the recommended single agent dose for the paediatric population. Clofarabine (Clolar®) is given intravenously over 2 hours on days 1, 2, 3, 4 and 5. Dose escalations in subsequent cohorts will approximate 33% increments.

The following dose levels will be studied:
Level -1: 20 mg/m^2/day x 5 days
Level 0: 30 mg/m^2/day/ x 5 days
Level 1: 40 mg/m^2/day x 5 days

Dose escalation will be capped at 40 mg/m^2/day. Patients will receive a single cycle of treatment and will be followed up until day 42.

Intervention type



Phase I

Drug names

Clofarabine (Clolar®), daunorubicin (DaunoXome®)

Primary outcome measures

To establish the maximum tolerated dose of clofarabine (Clolar®) when used in combination with DaunoXome®. The maximum tolerated dose will be defined by the number of dose-limiting toxicities during cycle 1 of therapy.

Secondary outcome measures

1. To characterise the safety and tolerability of the combination of clofarabine (Clolar®) and DaunoXome® including identification of the dose limiting toxicities
2. To document the overall response rate, including complete remission (CR), complete remission with incomplete blood count recovery (CRi) and partial remission (PR) in this population
3. To describe the durability fo response and follow up of these patients, including the number of patients that undergo stem-cell transplant after re-induction with clofarabine (Clolar®) and DaunoXome®

Measured as follows:
1. The nature, incidence and severity of the adverse events, collected throughout cycle 1 of therapy
2. Responses measured by bone marrow assessment between day 21 and 42 post first dose of clofarabine (Clolar®) and DaunoXome®

Overall trial start date


Overall trial end date


Reason abandoned


Participant inclusion criteria

1. Diagnosis of acute myelogenous leukaemia (AML)
2. Patients must be in first relapse within 12 months of initial diagnosis or refractory to induction therapy or be in second or subsequent relapse
3. Patients with refractory AML following induction must be greater than 20% blasts in the bone marrow
4. Age must be between 6 months to less than 18 years old, either sex
5. Karnofsky or Lansky score of greater than 50
6. Patients of childbearing potential must have a negative pregnancy test and agree to use an effective birth control or evidence of post-menopausal status. Post-menopausal status is defined as either radiation-induced oophorectomy with last menses greater than 1 year ago; chemotherapy induced menopause with 1 year interval since last menses.
7. Normal renal function
8. Normal hepatic function
9. Cardiac function defined as: shortening fraction of greater than 29% by echocardiogram left ventricular ejection fraction (LVEF) greater than 58%

Participant type


Age group




Target number of participants

12 - 18 patients

Participant exclusion criteria

1. First relapse greater than 1 year from their initial diagnosis of AML
2. Patients whose previous daunorubicin equivalent exposure equals or exceeds 450 mg/m^2
3. Isolated extramedullary leukaemia
4. Symptomatic central nervous system (CNS) involvement
5. Any evidence of severe or uncontrolled systemic conditions
6. Concurrent treatment or administration of any other experimental drug or with any other cancer therapy
7. Patients unable to be regularly followed up
8. Patient with expected non-compliance to toxicity management guidelines

Recruitment start date


Recruitment end date



Countries of recruitment

United Kingdom

Trial participating centre

Institute of Child Health
B6 6NH
United Kingdom

Sponsor information


University of Birmingham (UK)

Sponsor details

Research & Commercial Services
Aitchison Building
B15 2TT
United Kingdom

Sponsor type




Funder type


Funder name

Leukaemia Research Fund (UK)

Alternative name(s)

Funding Body Type

Funding Body Subtype


Results and Publications

Publication and dissemination plan

Not provided at time of registration

Intention to publish date

Participant level data

Not provided at time of registration

Results - basic reporting

Publication summary

Publication citations

Additional files

Editorial Notes