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Trial registered on ANZCTR


Registration number
ACTRN12605000152628
Ethics application status
Approved
Date submitted
12/08/2005
Date registered
15/08/2005
Date last updated
15/09/2023
Date data sharing statement initially provided
15/09/2023
Type of registration
Retrospectively registered

Titles & IDs
Public title
A phase II trial of high dose cytarabine and fludarabine without anthracycline for patients with core binding factor acute myeloid leukaemia, measuring efficacy, safety and monitoring minimal residual disease
Scientific title
A phase II trial of high dose cytarabine and fludarabine without anthracycline for patients with core binding factor acute myeloid leukaemia, measuring efficacy, safety and monitoring minimal residual disease
Secondary ID [1] 116 0
Australasian Leukaemia and Lymphoma Group (ALLG): ALLG AMLM13
Secondary ID [2] 117 0
National Clinical Trials Registry: NCTR555
Universal Trial Number (UTN)
Trial acronym
CBF-AML
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Core binding factor acute myeloid leukaemia 245 0
Condition category
Condition code
Cancer 276 276 0 0
Leukaemia - Acute leukaemia

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Patients with CBF leukaemia will be treated with high dose Cytarabine and Fludarabine chemotherapy. An induction course will be followed by 6 consolidation cycles 3 of which are Cytarabine alone and 3 of which are fludarabine & cytarabine in combination but in lower total doses than in the induction cycle. Total treatment time will be 7 to 9 months. In addition, minimal residual disease will be monitored using sensitive laboratory methods at 1 and 3 monthly intervals for blood and bone marrow respectively.
Intervention code [1] 182 0
Treatment: Drugs
Comparator / control treatment
there is no compatator/control treatment in this trial
Control group
Uncontrolled

Outcomes
Primary outcome [1] 325 0
To establish the failure-free (FFS) rates of patients with newly diagnosed core binding factor (CBF) acute myeloid leukaemia (AML) treated with high-dose cytarabine (Ara-C) containing regimens exclusive of anthracyclines.
Timepoint [1] 325 0
The first analysis of FFS will take place 12 months after the last-registered patient has commenced treatment. If appropriate, the final analysis will take place after all patients have had a minimum potential follow-up of 4 years from the attainment of a complete response (CR).
Primary outcome [2] 326 0
To establish the overall survival (OS) rates of patients with newly diagnosed core binding factor (CBF) acute myeloid leukaemia (AML) treated with high-dose cytarabine (Ara-C) containing regimens exclusive of anthracyclines.
Timepoint [2] 326 0
The first analysis of OS will take place 12 months after the last-registered patient has commenced treatment. If appropriate, the final analysis will take place after all patients have had a minimum potential follow-up of 4 years from the attainment of a complete response (CR).
Secondary outcome [1] 724 0
1. To estimate the percentage of patients achieving CR with this treatment approach.
Timepoint [1] 724 0
For each patient, this will be assessed 50 days post commencement of induction therapy.
Secondary outcome [2] 725 0
2. To estimate the leukaemia-free survival (LFS) rate for patients who achieve CR with protocol treatment.
Timepoint [2] 725 0
The first analysis of LFS will take place 12 months after the last-registered patient has commenced treatment. If appropriate, the final analysis will take place after all patients have had a minimum potential follow-up of 4 years from the attainment of a complete response (CR).
Secondary outcome [3] 726 0
3. To determine the safety and tolerability of the protocol treatment.
Timepoint [3] 726 0
Monitored continuously and independently reviewed every 6 months.
Secondary outcome [4] 727 0
4. To identify the proportion of patients with CBF AML with cryptic rearrangements (cytogenetically silent).
Timepoint [4] 727 0
Assessed at diagnosis.
Secondary outcome [5] 728 0
5. To monitor the levels of CBF fusion transcripts in PB and BM by quantitative real time polymerase chain reaction (Q-PCR) during therapy and remission and hence to establish the kinetics of leukaemia eradication and the threshold or incremental change in transcript levels which correlate with risk of subsequent relapse.
Timepoint [5] 728 0
Measured after each treatment and 1 to 3 monthly thereafter for 2 years.
Secondary outcome [6] 729 0
6. To compare the utility of Q-PCR with interphase fluorescent in-situ hybridisation (i-FISH), conventional cytogenetics, and, where applicable, flow cytometry in detection of minimal residual disease and prediction of relapse risk.
Timepoint [6] 729 0
Measured after each treatment and 1 to 3 monthly thereafter for 2 years.

Eligibility
Key inclusion criteria
1. A morphologic diagnosis of AML by WHO criteria; note that patients with t(8;21) and less than 20% blasts will be included.2. Confirmation of CBF subtype by cytogenetic finding of t(8;21) or inv(16) or t(16;16) (either alone or in combination with other cytogenetic abnormalities) or PCR evidence of a CBF fusion transcript 3. ECOG performance status 0 to 3; 4. Written informed consent prior to registration 5. Potentially childbearing patients must use effective contraception 6. Patients must be registered prior to the commencement of induction therapy.
Minimum age
15 Years
Maximum age
65 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Serious cardiac or pulmonary dysfunction precluding the delivery of the proposed therapy2. Severe renal dysfunction3. Severe hepatic dysfunction including bilirubin > 2.5 ULN unless attributable to leukaemia4. Prior treatment for AML5. Contraindication to the use of study drugs6. Known HIV infection7. Pregnancy and lactation8. Inability to comply with study requirements.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint/s
Safety/efficacy
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Date of first participant enrolment
Anticipated
14/12/2004
Actual
11/02/2005
Date of last participant enrolment
Anticipated
Actual
19/06/2009
Date of last data collection
Anticipated
Actual
31/12/2012
Sample size
Target
50
Accrual to date
Final
53
Recruitment in Australia
Recruitment state(s)

Funding & Sponsors
Funding source category [1] 330 0
Charities/Societies/Foundations
Name [1] 330 0
Leukaemia Foundation of Australia
Country [1] 330 0
Australia
Funding source category [2] 331 0
Commercial sector/Industry
Name [2] 331 0
Amgen Australia
Country [2] 331 0
Australia
Primary sponsor type
Other Collaborative groups
Name
Australasian Leukaemia and Lymphoma Group
Address
Level 2, 10 St Andrews Place
East Melbourne Vic 3002
Country
Australia
Secondary sponsor category [1] 263 0
None
Name [1] 263 0
nil
Address [1] 263 0
Country [1] 263 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 1265 0
Canberra
Ethics committee address [1] 1265 0
Ethics committee country [1] 1265 0
Australia
Date submitted for ethics approval [1] 1265 0
Approval date [1] 1265 0
Ethics approval number [1] 1265 0
Ethics committee name [2] 1266 0
Gosford
Ethics committee address [2] 1266 0
Ethics committee country [2] 1266 0
Australia
Date submitted for ethics approval [2] 1266 0
Approval date [2] 1266 0
12/11/2004
Ethics approval number [2] 1266 0
Ethics committee name [3] 1267 0
Hobart
Ethics committee address [3] 1267 0
Ethics committee country [3] 1267 0
Australia
Date submitted for ethics approval [3] 1267 0
Approval date [3] 1267 0
Ethics approval number [3] 1267 0
Ethics committee name [4] 1268 0
Mater Brisbane
Ethics committee address [4] 1268 0
Ethics committee country [4] 1268 0
Australia
Date submitted for ethics approval [4] 1268 0
Approval date [4] 1268 0
Ethics approval number [4] 1268 0
Ethics committee name [5] 1269 0
Mater Newcastle
Ethics committee address [5] 1269 0
Ethics committee country [5] 1269 0
Australia
Date submitted for ethics approval [5] 1269 0
Approval date [5] 1269 0
Ethics approval number [5] 1269 0
Ethics committee name [6] 1270 0
Princess Alexandra
Ethics committee address [6] 1270 0
Ethics committee country [6] 1270 0
Australia
Date submitted for ethics approval [6] 1270 0
Approval date [6] 1270 0
Ethics approval number [6] 1270 0
Ethics committee name [7] 1271 0
Royal Melbourne
Ethics committee address [7] 1271 0
Ethics committee country [7] 1271 0
Australia
Date submitted for ethics approval [7] 1271 0
Approval date [7] 1271 0
Ethics approval number [7] 1271 0
Ethics committee name [8] 1272 0
Royal Perth
Ethics committee address [8] 1272 0
Ethics committee country [8] 1272 0
Australia
Date submitted for ethics approval [8] 1272 0
Approval date [8] 1272 0
Ethics approval number [8] 1272 0
Ethics committee name [9] 1273 0
St Vincent's Sydney
Ethics committee address [9] 1273 0
Ethics committee country [9] 1273 0
Australia
Date submitted for ethics approval [9] 1273 0
Approval date [9] 1273 0
Ethics approval number [9] 1273 0
Ethics committee name [10] 1274 0
Westmead
Ethics committee address [10] 1274 0
Ethics committee country [10] 1274 0
Australia
Date submitted for ethics approval [10] 1274 0
Approval date [10] 1274 0
Ethics approval number [10] 1274 0

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 35869 0
Address 35869 0
Country 35869 0
Phone 35869 0
Fax 35869 0
Email 35869 0
Contact person for public queries
Name 9371 0
Dr Paula Marlton
Address 9371 0
Oncology/Haematology Unit
Princess Alexandra Hospital
Ipswich Road
Woolloongabba QLD 4102
Country 9371 0
Australia
Phone 9371 0
+61 7 32407036
Fax 9371 0
+61 7 32402252
Email 9371 0
[email protected]
Contact person for scientific queries
Name 299 0
Dr Paula Marlton
Address 299 0
Oncology/Haematology Unit
Princess Alexandra Hospital
Ipswich Road
Woolloongabba QLD 4102
Country 299 0
Australia
Phone 299 0
+61 7 32407036
Fax 299 0
+61 7 32402252
Email 299 0
[email protected]

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
De-identified IPD data, for all data collected during the trial
When will data be available (start and end dates)?
Data available 3 months following publication, for an indefinite period
Available to whom?
Data are potentially available to:
• Researchers from not-for-profit organisations
• Commercial organisations
• Other
Based in:
• Any location
Further information:
All data requests will be considered by the primary sponsor on a case by case basis. Requests must include a methodologically sound proposal. Specific conditions of use may apply and will be specified in a data sharing agreement (or similar) that the requester must agree to before access is granted.
Available for what types of analyses?
Any type of analysis
Assessed on a case-by-case basis
How or where can data be obtained?
Access can be requested via the Health Data Australia catalogue (https://researchdata.edu.au/health/). Search for the ACTRN number in the catalogue to find datasets associated with this trial or email enquiries to [email protected]


What supporting documents are/will be available?

Doc. No.TypeCitationLinkEmailOther DetailsAttachment
20360Study protocol  [email protected] Access can be requested via the Health Data Austra... [More Details]



Results publications and other study-related documents

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