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JOURNAL ONKOLOGIE – STUDIE
ICMLPed

International Chronic Myeloid Leukemia Pediatric Study

Rekrutierend

NCT-Nummer:
NCT01281735

Studienbeginn:
Januar 2011

Letztes Update:
17.01.2024

Wirkstoff:
-

Indikation (Clinical Trials):
Leukemia, Leukemia, Myeloid, Leukemia, Myelogenous, Chronic, BCR-ABL Positive

Geschlecht:
Alle

Altersgruppe:
Alle

Phase:
-

Sponsor:
Poitiers University Hospital

Collaborator:
CIC Inserm 1402, University of Milano Bicocca, University Medical Center Groningen, University of Liege, Imperial College London, University Hospital Dresden, International BFM Study Group,

Studienleiter

Frederic MILLOT, MD
Principal Investigator
Poitiers University Hospital

Kontakt

Studienlocations
(3 von 20)

University Hospital Dresden
01307 Dresden
(Sachsen)
GermanyRekrutierend» Google-Maps
Ansprechpartner:
Meinolf SUTTORP, Prof.
E-Mail: meinolf.suttorp@uniklinikum-dresden.de

Markus METZLER
E-Mail: Markus.Metzler@uk-erlangen.de» Ansprechpartner anzeigen
St. Anna Children's Cancer Research Institute and St. Anna Children's Hospital
1090 Vienna
AustriaRekrutierend» Google-Maps
Ansprechpartner:
Michael DWORZAK, Prof.
E-Mail: michael.dworzak@stanna.at» Ansprechpartner anzeigen
Belarussian Reseach Center for Pediatric Oncology and hematology (BRCPOH)
Minsk
BelarusRekrutierend» Google-Maps
Ansprechpartner:
Marina BORISEVICH, MD
E-Mail: borisevich10@mail.ru

Natalia SAVVA
E-Mail: n_savva@yahoo.com» Ansprechpartner anzeigen
Instituto de Oncologia Pediatrica
04023-062 Sao Paulo
BrazilRekrutierend» Google-Maps
Ansprechpartner:
Silvia Regina BRANDALISE, MD
E-Mail: silvia@boldrini.org.br

Julia YAJIMA
E-Mail: julia.yajima@gmail.com» Ansprechpartner anzeigen
Charles University Motol
Prague
CzechiaRekrutierend» Google-Maps
Ansprechpartner:
Petr SEDLACEK, PhD
Phone: +420 22443 6552
E-Mail: Petr.Sedlacek@fnmotol.cz

Ales LUKS
E-Mail: ales.luks@lfmotol.cuni.cz» Ansprechpartner anzeigen
Clinical Investigation Center CIC 1402
86021 Poitiers
FranceRekrutierend» Google-Maps
Ansprechpartner:
Violaine GOYEAU, Master
Phone: +33 (0)5 49 44 30 57
E-Mail: violaine.goyeau@chu-poitiers.fr» Ansprechpartner anzeigen
Agia Sophia Children's Hospital
11527 Athen
GreeceRekrutierend» Google-Maps
Ansprechpartner:
Mirella AMPATZIDOU, PhD
E-Mail: mirellaaba@yahoo.gr

Sophia POLYCHRONOPOULOU
E-Mail: sophpol@otenet.gr» Ansprechpartner anzeigen
Keio University School of Medicine
160-8582 Tokyo
JapanNoch nicht rekrutierend» Google-Maps
Ansprechpartner:
HIROYUKI Shimada, MD PhD
E-Mail: hshimada@a5.keio.jp

Shima HARUKO
E-Mail: sharuko@keio.jp» Ansprechpartner anzeigen
Dutch Childhood Oncology Group, Princess Maxima Center for pediatric oncology
Utrecht
NetherlandsRekrutierend» Google-Maps
Ansprechpartner:
Maaike LUESINK, Prof.
E-Mail: m.luesink@prinsesmaximacentrum.nl» Ansprechpartner anzeigen
Wroclaw Medical University Department
Wrocław
PolandRekrutierend» Google-Maps
Ansprechpartner:
Krzysztof KALWAK, PhD
Phone: +48717703170
E-Mail: krzysztof.kalwak@gmail.com

Malgorzata Janeczko
E-Mail: ml.janeczko@gmail.com» Ansprechpartner anzeigen
University Children's Hospital
813 72 Bratislava
SlovakiaRekrutierend» Google-Maps
Ansprechpartner:
Alexandra KOLENOVA, Prof
E-Mail: sasa.kolenova@gmail.com

Andrea HRASKOVA
E-Mail: hraskova.andrea@gmail.com» Ansprechpartner anzeigen
Alle anzeigen

Studien-Informationen

Detailed Description:

International study of CML in children and adolescents

1. Rational and objectives 1.1 Rational: Chronic myeloid leukaemia (CML) is a malignant

disease of the hematopoietic system characterized by the presence a reciprocal

translocation between chromosome 9 and 22. CML is a very rare disease in children and

adolescents, accounting for 2% to 3% of leukemias in this age range, with an annual

incidence of 1 case per million children (1,2,3). A recent report of the French National

Registry of childhood leukemia and lymphoma revealed an incidence of 0.6 case per

million children under 15 years of age between 1990 and 1999 in France. Characteristics

of CML seem to differ in this age range compared with adults (5). In Europe, very few

children and adolescents with CML is included in national trials nor are they recorded

in regional or national registries. A European CML-Registry was established to improve

the knowledge concerning this disease but only patients aged 18 years and over are

registered. In Europe, a national registry was recently set up in UK to collect

prospectively data in patients less than 15 years of age with CML. However, limited

information is available concerning the epidemiology and the characteristics of CML in

childhood.

There are currently two main treatment options in children with CML. The first option is

allogeneic hematopoietic transplantation which is a potentially curative therapy in

children with a suitable donor. Transplantation with a matched unrelated donor resulted

in an overall survival rate of 66% at 3 years in children with CML in first chronic

phase (6). A 5-year overall survival rate of 73 % was reported in those transplanted

with a matched sibling donor (7). The second option is treatment with imatinib mesylate,

an orally administered antityrosine kinase. Imatinib mesylate is well tolerated and

cytogenetic and molecular remissions can be achieved in a significant percentage of

children with CML (8, 9). However, the ability of imatinib mesylate to cure the disease

remains unknown.

In adults with CML, Sokal and Hasford reported 2 distinct staging systems able to

distinguish patient groups with different survival. These scoring systems were

determined in cohorts of patients including adults and children, but the data of the

younger patients were not analysed separately. Moreover, the Sokal score was determined

in patients treated with busulfan or hydroxyurea and the Hasford score in patients

receiving IFN therapy. Identification of prognosis factors and determination of a

prognostic scoring system in children and adolescents with CML are essential to optimize

individual treatment strategy in this age group. All these points justify the need for

an international network to better describe this rare disease in children and

adolescents and to optimize individual treatment strategy.

1.2 Objectives.

The main objectives of the study are the followings:

- To describe the characteristics of CML in a large cohort of patients less than 18

years of age

- To describe the treatment policies.

- To identify prognostic factors in this age-group

- To determine prognostic scoring systems in this population in order to optimize

individual treatment choices.

- To determine side effects and long term effects of treatments, mainly the tyrosine

kinase inhibitor effect, on growth and development of a pediatric population

2. Population. All patients less than 18 years of age with newly diagnosed Philadelphia

positive and/or bcr-abl positive CML are eligible whatever the phase of the disease, the

type of treatment and the enrollment or not in a clinical study.

However their data will be collected only if requirements concerning ethics and policy

agreement are fulfilled (see section 9)

3. Method The study is strictly observational. Retrospective and prospective data will be

collected from patient flow charts and/or existing databases.

A steering committee (SC) including A Biondi, E De Bont, MF Dresse, J de la Fuente, M

Suttorp, J Guilhot and F Millot will promote the international study of CML in children

and adolescents. The SC will include representatives of each country involved in the

study. The SC will agree on policy, support its implementation, initiate research, and

raise public awareness about the study by presentations and publications.A scientific

committee (SciC) will be set up. By laws regarding data property,analysis and

publication will be decided by the scientific committee and the steering committee. For

each objective of the study, there will be a working party (WP),which will be open to

all interested participants in the study.Data will be provided by clinicians and

biologists involved in pediatric haematology.The data base of the study will be set up

in the Clinical Investigation Center(CIC INSERM 802)of the University Hospital of

Poitiers(France)which will be the International Central Data Center(ICDC) of the study

in charge of and centralizing the data. Sophie Zin Ka Yeu will be the responsible for

the monitoring,E Ducasse for data base management and J Guilhot for the statistical

analysis. The study is international.A National Coordinating Center and a leader will be

identified in each participating country. The National Coordinating Center will be in

charge of collecting data for his country. Each leader of the National Coordinating

Center will be in charge of registering the patients and sending the data to the ICDC

(CIC INSERM 802, Poitiers, France). To avoid double entry, the location of the medical

center where the CML of the patient was diagnosed will be recorded in the case report

form. Moreover, notification of new cases may be cross checked by national coordinators

with their own national registry when available (for example with the Registre national

des hémopathies malignes de l'enfant in France).

4. Origin and type of data:

Data will include:

- Identification number

- Demographic data

- Date of birth

- Sex

- Medical data Ethnicity will be collected because of variation in terms of clinical

characteristics (height for example) and pharmacokinetic of medication of the

different groups of children potentially involved. Identifying biological results

will not be collected.

The ICDC will collect only data which is strictly anonymized by the national

coordinating centers. Patients will be identified by a code including 2 to 3 letters for

the country and 4 digits for the patient number (ex: FR/0001 for the first registered

Patient in France). No initial of patients will be used. As country coordinator for

France, F Millot will also collect initials of French patients in order to avoid double

registration. These initials will not be computerized. The international ID number will

be immediately provided.

5. Circulation of the data The data is centralized in the ICDC of the study. The ICDC is

located in the Clinical Research Investigation Center of the University Hospital of

Poitiers (France).

Retrospective data:the data will be collected in a one step procedure. Prospective

data:The data will be collected using a two step procedures.

1. Notification of the cases:

New cases will be notified by physicians and biologists involved in pediatric

hematology to their National Coordinating Center; at regular intervals these cases

will then be referred by this coordinating center to the ICDC.

2. Full data collection:

The data will be collected from the clinical chart of the patients. The complete data

will be send to each National Coordinating Center and then centralized at regular

intervals subsequently in the ICDC. Follow up will be required twice a year. For each

case of CML, a yet to be fixed sum of money will be transferred from the ICDC to the

National Coordinating Center at notification of the case and when complete data transfer

will be performed.

To avoid extra work, data already computerized will be accepted if it fulfilled the

requirements of ethics, confidentiality and partnership rules as mentioned in paragraph

7.

When available agreement between parties, data may come from case report form of

patients included in clinical studies or other Registries. Data will be sent by regular

mail, fax or electronic file by the national coordinators to the ARC of the study at the

ICDC. Data received by mail will be transferred by copy using disks (CD-Rom or external

disk) to the data-manager and destroyed from the computer with mail box. Data will be

entered in a Microsoft ACCESS database in the ICDC. Data base will be implemented in a

PC (I) (Windows) located in a room dedicated for the purpose of the work. A second PC

(II) in the same place will be used for duplication, backup, quality control and

analysis of the data. None of these 2 PCs will offer connections such as Internet, or

Intranet. The use of an electronic case Report form (e-CRF) is not planned. A secure

access building and rooms of the ICDC is organized; specific password and login for the

computers are used. At regular interval a secure storage of the data will be performed

and located in the secure IDCD room organized for this purpose. Access to the data will

be restricted to the coordinator of the study, the ARC, the data manager, the

biostatistician, and Health Authorities, if needed.

6. Study duration and organization The expected start date is August 1, 2009. The planned

study duration is about 5 years. Study prolongation will depend on the funding linked to

the effective participation of the centres in the study.

7. Analysis of the data Data will be analysed using mainly SAS software (SAS institute,

CARY, NC, USA). Final analyses will be provided within 5 years from start of the study.

Analysis will be mainly descriptive. Date of inclusion in the study is defined as the

date of reception of the notification of case by the ICDC. For the purpose of

descriptive analysis, all parameters will be analysed at regular intervals: number of

registered cases, baseline characteristics, follow-up and outcome variables. Categorical

data will be presented with frequency, percentage and 95 % confidence interval.

Quantitative variable will be presented with median and range, mean and standard

deviation when relevant. Survival data and issue to events (time to response or

side-effects, loss and duration of response…); will be analysed by the Kaplan Meier and

competing risks methods. For prognosis factors identification, exploratory analyses will

be performed as appropriate. The final statistical plan will be validated by the

steering committee and the scientific committee.

Reports will be provided every 6 months and by the end of the study by the ARC, the data

manager, the biostatistician and Dr F. Millot, coordinator of the study. These reports

will be sent to the national coordinators and to the participants. Results could be

presented in Workshops, meetings and could be published with the agreement of the

steering and scientific committees. All reports and publications will remain anonymous.

8. Number of Patients :

A proximately 100 to 150 patients per year are expected. As the number of cases of

children and adolescent with CML being low, no strict calculation was made. The

objective is to collect a maximum of cases to improve the power of the study.

9. Ethical Considerations The children, according to their age, and their parents will be

informed about the study and its procedures, according to the European policy regarding

collection and transfer data for research purpose. Informed consent (parents and, when

possible, children) will have to be provided.

Anonymized data will be collected and presented in this study. Rules will be in accordance

with the Principles and Guideline of the European Community concerning clinical studies and

data collection and the French Law "Informatique, fichiers et liberté" (January 6, 1978,

updated.It is the responsibility of each national coordinator to provide insurance concerning

the participation of the members of his groups according these rules and the local rules

required by his own country.

Ein-/Ausschlusskriterien

Inclusion Criteria:

- patients less than 18 years of age

- diagnosed Philadelphia positive and/or BCR-ABL positive Chronic Myeloid Leukemia (CML)

all phases

Exclusion Criteria:

- patients more than 18 years of age at diagnosis of CML

Quelle: ClinicalTrials.gov


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