International Randomised Phase III Clinical Trial in Children With Acute Myeloid Leukaemia
Condition(s) studied
Investigational drug(s) / intervention(s)
Gemtuzumab ozogamicin: Antibody-conjugated chemotherapy agent.
Liposomal daunorubicin: Anthracycline (Randomisation 1 (R1)) closed early to recruitment on 8th September 2017, due to liposomal daunorubicin manufacturing issues resulting in unavailability of the drug.
Mitoxantrone: DNA-reactive agent
Fludarabine: A water-soluble fluorinated nucleotide analogue of the antiviral agent vidarabine.
Cytarabine: Pyrimidine nucleoside analogue, an antineoplastic agent.
Busulfan: Alkylsulfonate
Cyclophosphamide: A nitrogen mustard alkylating agent from the oxazaphosphorine group
Study summary
The main purpose of this study is :
1. To establish which number of doses of gemtuzumab ozogamicin (up to a maximum of 3 doses) is tolerated and can be safety delivered in combination with cytarabine plus mitoxantrone or liposomal daunorubicin in induction
2. To compare mitoxantrone (anthracenedione) \& cytarabine with liposomal daunorubicin (anthracycline) \& cytarabine as induction therapy. (Randomisation 1 (R1) closed early to recruitment on 8th September 2017, due to liposomal daunorubicin manufacturing issues resulting in unavailability of the drug.)
3. To compare a single dose of gemtuzumab ozogamicin with the optimum tolerated number of doses of gemtuzumab ozogamicin (identified by the dose-finding study) when combined with induction chemotherapy.
4. To compare two consolidation regimens: high dose cytarabine (HD Ara-C) and fludarabine \& cytarabine (FLA) in standard risk patients.
5. To compare the toxicity and effectiveness of two haemopoietic stem cell transplant (HSCT) conditioning regimens of different intensity: conventional myeloablative conditioning (MAC) with busulfan/cyclophosphamide and reduced intensity conditioning (RIC) with fludarabine/busulfan.
Eligibility
Primary outcome measure(s)
- Incidence of dose limiting toxicities (DLTs). — Incidence of DLTs will be evaluated up to day 45 post course 1 and course 2 of induction chemotherapy.
- Event Free Survival (EFS). — Event free survival (EFS) will be evaluated as the time from randomisation one to the first event, up to 16 years.
The primary analysis will be carried out once the last patient has a minimum of 1 year follow up. EFS estimates will be presented at 24 months along with 95% confidence intervals. - Event Free Survival (EFS). — Event free survival (EFS) will be evaluated as the time from randomisation two to the first event, up to 16 years..
The primary analysis will be carried out once the last patient has a minimum of 1 year follow up. EFS estimates will be presented at 24 months along with 95% confidence intervals. - Relapse free survival (RFS). — Relapse free survival (RFS) will be evaluated as the time of randomisation three to the first relapse or death from any cause, up to 16 years.
The primary analysis will be carried out once the last patient has a minimum of 1 year follow up. RFS estimates will be presented at 24 months along with 95% confidence intervals. - Early treatment related adverse reactions. — Early treatment related adverse reactions will be evaluated at day 100 post-transplant.
Early treatment related adverse reactions defined as the incidence by day 100 post-transplant of grade 3-5 toxicity for the following systems using the National Cancer Institute (NCI) Common Terminology Criteria v4: * Cardiac (pericardial effusion/Left ventricular systolic dysfunction). * Respiratory, thoracic and mediastinal (hypoxia/pneumonitis). * Gastrointestinal (GI) (diarrhoea/typhlitis/upper and lower GI haemorrhage). * Investigations (bilirubin). * Renal and Urinary (acute kidney injury/haematuria). * Nervous system (seizure). - Relapse free survival (RFS). — Relapse free survival (RFS) will be evaluated as the time of randomisation four to the first relapse or death from any cause, up to 16 years.
The primary analysis will be carried out once the last patient has a minimum of 1 year follow up. RFS estimates will be presented at 12 months along with 95% confidence intervals.
Trial sites (68)
| Facility | City | Region | Status |
|---|---|---|---|
| Women and Children's Hospital Adelaide | Adelaide | Australia | |
| Queensland Children's Hospital | Brisbane | Australia | |
| Monash Children's Hospital | Melbourne | Australia | |
| Royal Childrens Hospital | Melbourne | Australia | |
| John Hunter Children's Hopsital | New Lambton Heights | Australia | |
| Perth Children's Hospital | Perth | Australia | |
| Sydney Children's Hospital | Sydney | Australia | |
| The Childrens Hospital At Westmead | Westmead | Australia | |
| Centre Hospitalier Universitaire Amiens - Picardie | Amiens | France | |
| Centre Hospitalier Universitaire D'angers | Angers | France | |
| Centre Hospitalier Regional Universitaire Besancon - Hopital Jean Minjoz | Besançon | France | |
| Centre Hospitalier Universitaire De Bordeaux - Hopital Pellegrin | Bordeaux | France | |
| Centre Hospitalier Regional Universitaire Brest - Hopital Morvan | Brest | France | |
| Centre Hospitalier Universitaire De Caen | Caen | France | |
| Centre Hospitalier Universitaire De Clermont-ferrand | Clermont-Ferrand | France | |
| Centre Hospitalier Universitaire Dijon Bourgogne - Hopital D'enfants | Dijon | France | |
| Centre Hospitalier Universitaire De Grenoble | Grenoble | France | |
| Hopital Jeanne Dr Flandre | Lille | France | |
| Centre Hospitalier Universitaire De Limoges | Limoges | France | |
| Centre Leon Berard | Lyon | France | |
| Hopital De La Timone | Marseille | France | |
| Centre Hospitalier Regional Universitaire Montpellier - Hopital Arnaud-de-villeneuve | Montpellier | France | |
| Centre Hospitalier Universitaire De Nancy | Nancy | France | |
| Centre Hospitalier Universitaire De Nantes | Nantes | France | |
| Centre Hospitalier Universitaire De NICE | Nice | France | |
| Hopital Armand Trousseau | Paris | France | |
| Hopital Robert Debre | Paris | France | |
| Hopital Saint Louis | Paris | France | |
| Centre Hospitalier Universitaire De Poitiers | Poitiers | France | |
| Chu De Reims | Reims | France | |
| Centre Hospitalier Universitaire De Rennes - Hopital Sud | Rennes | France | |
| Centre Hospitalier Universitaire De Rouen | Rouen | France | |
| Centre Hospitalier Universitaire Saint-etienne | Saint-Etienne | France | |
| Strasbourg Hautepierre | Strasbourg | France | |
| Centre Hospitalier Universitaire De Toulouse - Hopital Des Enfants | Toulouse | France | |
| Centre Hospitalier Regional Universitaire De Tours - Hopital Clocheville | Tours | France | |
| Our Lady's Hospital for Sick Children | Dublin | Ireland | |
| Starship Childrens Hospital | Auckland | New Zealand | |
| Christchurch Hospital | Christchurch | New Zealand | |
| Kantonsspital Aarau | Aarau | Switzerland |
+ 28 more sites — see the full list on the official registry below.
More University of Birmingham trials in France
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT02724163 on ClinicalTrials.gov ↗ ← All trials in France