MedPath

Trial to Test the Effects of Adding 1 of 2 New Treatment Agents to Commonly Used Chemotherapy Combinations

Phase 2
Conditions
Acute Myeloid Leukaemia
Myelodysplastic Syndrome
Interventions
Drug: Arm E CPX-351 (200 V 300)
Drug: Arm D Small molecule or Not
Drug: Arm A Mylotarg plus DA Versus CPX-351
Drug: Arm B Vosaroxin and Decitabine
Drug: Arm F DA V IDAC
Drug: Arm C DA V FLAG-Ida V DAC
Registration Number
NCT02272478
Lead Sponsor
Cardiff University
Brief Summary

The AML18 Trial will evaluate several relevant therapeutic questions in Acute Myeloid Leukaemia (AML), as defined by the WHO, and High Risk Myelodysplastic Syndrome. The trial is primarily designed for patients over 60 years considered fit for an intensive chemotherapeutic approach, but younger patients who may not be considered suitable for the concurrent NCRI AML Trial for younger patients may also enter. Patients for whom intensive chemotherapy is not thought suitable may enter the concurrent NCRI trial of less intensive therapy (LI1). Approximately 1600 patients will be recruited.

At entry, a randomisation will compare a standard chemotherapy schedule DA (Daunorubicin/Ara-C) combined with 1 dose of Mylotarg (gemtuzumab ozogamicin, or GO) in course 1 against CPX-351. Patients who have known adverse risk cytogenetics (using Grimwade 2010 classification favourable/intermediate/adverse) at diagnosis may enter a Phase 2 evaluation of the combination of Vosaroxin plus Decitabine. Patients who achieve complete remission (CR) and who are MRD negative by flow cytometry after course one of DA will receive one further course of DA, with a randomisation to receive, either a course of DA or intermediate dose Cytarabine (IDAC) as a third course. Patients who are MRD negative by flow cytometry after course one of CPX-351 will receive up to 2 further course of CPX. Patients who fail to achieve a CR after course 1 of DA or who are MRD positive by flow cytometry or for whom MRD information is not available, are eligible to be randomised to compare DA with DA plus Cladribine (DAC) or FLAG-Ida for up to two courses of therapy. Patients who fail to achieve a CR after course 1 of CPX-351 or who are MRD positive by flow cytometry or for whom MRD information is not available are eligible to be randomised between a second course of standard dose CPX versus a repeat of the course 1 schedule. Patients receiving Vosaroxin and Decitabine are excluded from these post course 1 randomisations .

Following the outcome of course 1, patients who received DA chemotherapy on course 1 will be randomised to receive further chemotherapy with the 2nd generation FLT3 inhibitor AC220. Patients randomised to AC220 will be allocated a maximum of 3 courses (short AC220) or 3 courses plus maintenance for 1 year (long AC220). Patients receiving Vosaroxin and Decitabine are excluded from this randomisation.

Patients will be eligible for a non-intensive allogeneic stem cell transplant if a suitable HLA matched donor is available.

Detailed Description

AML18 is a trial primarily for older patients with AML and high risk Myelodysplastic Syndrome (MDS). It offers a randomised controlled Phase II/III trial which uses a factorial design for maximum efficiency to evaluate two induction options followed by treatment with small molecule beyond course 1, and dose intensification for patients without evidence of MRD negativity.

There are five randomised comparisons within the trial:

1. At diagnosis:

For patients not known to have adverse risk cytogenetics DA chemotherapy plus a single dose of 3 mg/m2 of Mylotarg versus CPX-351. Patients with abnormal LFTs can enter the randomisation but receive DA alone or CPX-351.

2. For patients who received DA chemotherapy but are not in CR or who are MRD +ve, or for whom MRD is not assessable.

DA versus DAC versus FLAG-Ida

3. All patients at second course who have received DA and have not received Vosaroxin and Decitabine induction AC220 versus no AC220 for a maximum of 3 cycles; then with or without maintenance for 1 year for patients allocated AC220

4. For patients who are in CR or CRi and MRD -ve post course1 and have completed 2 courses of DA DA versus intermediate dose Cytarabine (IDAC)

5. For patients who received CPX-351 chemotherapy but are not in CR or who are MRD +ve, or for whom MRD is not assessable CPX-351 100 units/m2 x 3 doses versus CPX-351 100 units/m2 x 2 doses

The trial will also assess:

* Non-intensive allogeneic stem cell transplant for patients with matched sibling or matched unrelated donors.

* The combination of Vosaroxin and Decitabine for those with known adverse risk cytogenetics at diagnosis

Recruitment & Eligibility

Status
UNKNOWN
Sex
All
Target Recruitment
1600
Inclusion Criteria

Not provided

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Exclusion Criteria

Known serious cardiac illness or medical conditions, including but not limited to:

I. Clinically unstable cardiac disease, including unstable atrial fibrillation, symptomatic bradycardia, unstable congestive heart failure, active myocardial ischemia, or indwelling temporary pacemaker II. Ventricular tachycardia or a supraventricular tachycardia that requires treatment with a Class Ia antiarrhythmic drug (e.g., quinidine, procainamide, disopyramide) or Class III antiarrhythmic drug (e.g., sotalol, amiodarone, dofetilide). Use of other antiarrhythmic drugs is permitted.

III. Use of medications that have been linked to the occurrence of torsades de pointes (see Appendix for the list of such medications) IV. Second- or third-degree atrioventricular (AV) block unless treated with a permanent pacemaker V. Complete left bundle branch block (LBBB) VI. History of long QT Syndrome or a family member with this condition VII. Serum potassium, magnesium, and calcium levels not outside the laboratory's reference range VIII. QTc >450 ms (average of triplicate ECG recordings); a consistent method of QTc calculation must be used for each patient's QTc measurements. QTcF (Fridericia's formula) is preferred. Please see the trial website for QTcF calculator.

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Study & Design

Study Type
INTERVENTIONAL
Study Design
FACTORIAL
Arm && Interventions
GroupInterventionDescription
Arm EArm E CPX-351 (200 V 300)Prior to Course 2 for patients receiving CPX in course 1 and MRD positive PC1 Randomisation between CPX-351 100 units/m2 on days 1, and 3 (CPX 200) versus CPX-351 100 units/m2 on days 1, 3 and 5 (CPX 300)
Arm DArm D Small molecule or NotPrior to Course 2 - Patients that received DA plus GO in course 1 and MRD negative PC1 Randomisation to receive AC220 or not
Arm AArm A Mylotarg plus DA Versus CPX-351Patients not known adverse karyotype Randomise between Daunorubicin 60mg/m2 daily by i.v. infusion on days 1, 3 and 5 (3 doses) Cytosine Arabinoside 100mg/m2 12 hourly by i.v. push on days 1 - 10 inclusive (20 doses) Mylotarg (GO) 3mg/m2 on day 1 of DA chemotherapy Versus CPX-351 100 units/m2 on days 1, 3 and 5
Arm BArm B Vosaroxin and DecitabinePatients with known adverse karyotype 5 cycles of Vosaroxin and Decitabine therapy
Arm CArm D Small molecule or NotPrior to Course 2 - Patients receving DA plus GO in course 1 and MRD positive PC1 Randomise between Daunorubicin 50mg/2 daily by i.v. infusion on days 1, 3 and 5 (3 doses) Cytosine Arabinoside 100mg/m2 12 hourly by i.v.push on days 1 - 8 inclusive (16 doses) Versus Daunorubicin 50mg/m2 daily by i.v. infusion on days 1, 3 and 5 Cytosine Arabinoside 100mg/m2 12 hourly by i.v. push on days 1 - 8 inclusive Cladribine 5mg/m2 daily on days 1 - 5 inclusive Versus Patients aged 60-69 Fludarabine 30mg/m2 daily on i.v. on days 2 - 6 inclusive Cytosine Arabinoside 1g/m2 daily over 4 hours Fludarabine on days 2 - 6 inclusive Patients aged 70+ Fludarabine 25mg/m2 daily i.v. on days 2 - 5 inclusive Cytosine Arabinoside 1g/m2 daily over 4 hours Fludarabine on days 2 - 5 inclusive Idarubicin 5mg/m2 i.v. daily on days 3, 4 and 5 (3 doses) And Randomisation to receive AC220 or not
Arm FArm F DA V IDACPrior to Course 3 - Patients that received DA plus GO in course 1 and MRD negative PC1 Randomise between Daunorubicin 50 mg/m2 daily by i.v. infusion on days 1 and 3 (2 doses) Cytosine Arabinoside 100 mg/m2 12-hourly by i.v. push on days 1 - 5 inclusive (10 doses) versus Intermediate dose Cytarabine (IDAC) schedule Cytosine Arabinoside 1g/m2 daily by 4 hour infusion on days 1- 5 inclusive (5 doses)
Arm CArm C DA V FLAG-Ida V DACPrior to Course 2 - Patients receving DA plus GO in course 1 and MRD positive PC1 Randomise between Daunorubicin 50mg/2 daily by i.v. infusion on days 1, 3 and 5 (3 doses) Cytosine Arabinoside 100mg/m2 12 hourly by i.v.push on days 1 - 8 inclusive (16 doses) Versus Daunorubicin 50mg/m2 daily by i.v. infusion on days 1, 3 and 5 Cytosine Arabinoside 100mg/m2 12 hourly by i.v. push on days 1 - 8 inclusive Cladribine 5mg/m2 daily on days 1 - 5 inclusive Versus Patients aged 60-69 Fludarabine 30mg/m2 daily on i.v. on days 2 - 6 inclusive Cytosine Arabinoside 1g/m2 daily over 4 hours Fludarabine on days 2 - 6 inclusive Patients aged 70+ Fludarabine 25mg/m2 daily i.v. on days 2 - 5 inclusive Cytosine Arabinoside 1g/m2 daily over 4 hours Fludarabine on days 2 - 5 inclusive Idarubicin 5mg/m2 i.v. daily on days 3, 4 and 5 (3 doses) And Randomisation to receive AC220 or not
Primary Outcome Measures
NameTimeMethod
Overall survival1 year
Complete remission (CR + CRi) achievement and reasons for failure (for induction questions)1 month
Duration of remission, relapse rates and deaths in first CR1 month
Toxicity, both haematological and non-haematological1 month
Supportive care requirements (and other aspects of health economics)6 months
Secondary Outcome Measures
NameTimeMethod
The relevance of molecular characteristics and response to treatment1 month
The relevance of the presence of a cytogenetic abnormality in the bone marrow of patients in morphological remissionAt study end
To store diagnostic tissue for future research in the AML Tissue Bank6 years

Trial Locations

Locations (87)

Roskilde Hospital

🇩🇰

Roskilde, Denmark

Belfast City Hospital

🇬🇧

Belfast, United Kingdom

Blackpool Victoria Hospital

🇬🇧

Blackpool, United Kingdom

Aalborg University Hospital

🇩🇰

Aalborg, Denmark

Herlev and Gentofte Hospital

🇩🇰

Copenhagen, Denmark

Aarhus University Hospital

🇩🇰

Aarhus, Denmark

Rigshospitalet

🇩🇰

Copenhagen, Denmark

Aberdeen Royal Infirmary

🇬🇧

Aberdeen, United Kingdom

Monklands Hospital

🇬🇧

Airdrie, United Kingdom

Ysbyty Gwynedd Hospital

🇬🇧

Bangor, United Kingdom

Royal United Hospital Bath

🇬🇧

Bath, United Kingdom

Birmingham Heartland Hospital

🇬🇧

Birmingham, United Kingdom

Queen Elizabeth Hospital

🇬🇧

Birmingham, United Kingdom

Ysbyty Glan Clwyd

🇬🇧

Bodelwyddan, United Kingdom

Pilgrim Hospital

🇬🇧

Boston, United Kingdom

Royal Bournemouth General Hospital

🇬🇧

Bournemouth, United Kingdom

Bradford Royal Infirmary

🇬🇧

Bradford, United Kingdom

Bristol Haematology & Oncology Centre

🇬🇧

Bristol, United Kingdom

Addenbrooke's Hospital

🇬🇧

Cambridge, United Kingdom

University Hospital of Wales

🇬🇧

Cardiff, United Kingdom

Countess of Chester Hospital

🇬🇧

Chester, United Kingdom

University Hospital of Coventry and Warwickshire

🇬🇧

Coventry, United Kingdom

St Richard's Hospital

🇬🇧

Chichester, United Kingdom

Russell Hall

🇬🇧

Dudley, United Kingdom

Ninewells Hospital

🇬🇧

Dundee, United Kingdom

Hairmyres Hospital

🇬🇧

Glasgow, United Kingdom

Royal Free Hospital

🇬🇧

Hamstead, United Kingdom

The New Victoria Hospital

🇬🇧

Glasgow, United Kingdom

Raigmore Hospital

🇬🇧

Inverness, United Kingdom

Kettering General Hospital

🇬🇧

Kettering, United Kingdom

St Jame's University Hospital

🇬🇧

Leeds, United Kingdom

Leicester Royal Infirmary

🇬🇧

Leicester, United Kingdom

Lincoln County Hospital

🇬🇧

Lincoln, United Kingdom

Aintree University Hospital

🇬🇧

Liverpool, United Kingdom

The Royal Liverpool University Hospital

🇬🇧

Liverpool, United Kingdom

The Royal Marsden

🇬🇧

London, United Kingdom

St George's Hospital

🇬🇧

London, United Kingdom

Maidstone District General Hospital

🇬🇧

Maidstone, United Kingdom

The Christie Hospital

🇬🇧

Manchester, United Kingdom

The James Cook University Hospital

🇬🇧

Middlesbrough, United Kingdom

Freeman Hospital

🇬🇧

Newcastle, United Kingdom

Milton Keynes

🇬🇧

Milton Keynes, United Kingdom

Northampton General Hospital

🇬🇧

Northampton, United Kingdom

Norfolk & Norwich University

🇬🇧

Norwich, United Kingdom

Royal Oldham Hospital

🇬🇧

Oldham, United Kingdom

Churchill Hospital

🇬🇧

Oxford, United Kingdom

Derriford Hospital

🇬🇧

Plymouth, United Kingdom

Queen Alexandra Hospital

🇬🇧

Portsmouth, United Kingdom

Whiston Hospital & St Helens

🇬🇧

Prescot, United Kingdom

Salisbury District Hospital

🇬🇧

Salisbury, United Kingdom

Southampton General Hospital

🇬🇧

Southampton, United Kingdom

Salford Royal Hospital

🇬🇧

Salford, United Kingdom

Wexham Park Hospital

🇬🇧

Slough, United Kingdom

Stafford Hospital

🇬🇧

Stafford, United Kingdom

St Helier Hospital

🇬🇧

Sutton, United Kingdom

Sunderland Royal Hospital

🇬🇧

Sunderland, United Kingdom

Singleton Hospital

🇬🇧

Swansea, United Kingdom

Torbay District General Hospital

🇬🇧

Torquay, United Kingdom

Royal Cornwall Hospital

🇬🇧

Truro, United Kingdom

Sandwell Hospital

🇬🇧

West Bromwich, United Kingdom

Pinderfields Hospital

🇬🇧

Wakefield, United Kingdom

Arrowe Park Hospital

🇬🇧

Wirral, United Kingdom

New Cross Hospital

🇬🇧

Wolverhampton, United Kingdom

Wishaw General Hospital

🇬🇧

Wishaw, United Kingdom

Worcestershire Royal Hospital

🇬🇧

Worcester, United Kingdom

Worthing Hospital

🇬🇧

Worthing, United Kingdom

Odense University Hospital

🇩🇰

Odense, Denmark

UHW

🇬🇧

Cardiff, United Kingdom

Cheltenham General Hospital

🇬🇧

Cheltenham, United Kingdom

Derby Teaching Hospital

🇬🇧

Derby, United Kingdom

Western General Hospital

🇬🇧

Edinburgh, United Kingdom

Royal Devon & Exeter Hospital

🇬🇧

Exeter, United Kingdom

Beatson West of Scotland Cancer Centre

🇬🇧

Glasgow, United Kingdom

Gloucestershire Royal Hospital

🇬🇧

Gloucester, United Kingdom

Ipswich Hospital

🇬🇧

Ipswich, United Kingdom

Crosshouse & Ayr Hospital

🇬🇧

Irvine, United Kingdom

Victoria Hospital

🇬🇧

Kirkcaldy, United Kingdom

Forth Valley Royal Hospital

🇬🇧

Larbert, United Kingdom

St Bartholomew's Hospital

🇬🇧

London, United Kingdom

University College London Hospital

🇬🇧

London, United Kingdom

Nottingham University Hospital

🇬🇧

Nottingham, United Kingdom

Manchester Royal Infirmary

🇬🇧

Manchester, United Kingdom

Queen's Hospital

🇬🇧

Romford, United Kingdom

University Hospital of Royal Stoke

🇬🇧

Stoke-on-Trent, United Kingdom

Hillingdon Hospital

🇬🇧

Uxbridge, United Kingdom

York Hospital

🇬🇧

York, United Kingdom

Guy's Hospital

🇬🇧

London, United Kingdom

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