Registration of Children With CML and Treatment With Imatinib

Myeloid Leukemia, Chronic Clinical Trial

— CML-paed II  

Official title:

Protocol for Standardized Diagnostic Procedures, Registration, and Treatment Recommendations in Children and Adolescents With Philadelphia Chromosome-positive Chronic Myeloid Leukemia (CML)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT00445822
• Other study ID #: TUD-CML-paed 016
• Secondary ID: EudraCT: 2007-00
• Status: Recruiting
• Phase: N/A
• First received: March 8, 2007
• Last updated: February 28, 2012
• Start date: March 2007
• Est. completion date: December 2012

Study information

• Verified date: February 2012
• Source: Technische Universität Dresden
• Contact: Meinolf Suttorp, Prof. Dr.
• Phone: +49 351 458
• Email: meinolf.suttorp[@]uniklinikum-dresden.de
• Is FDA regulated: No
• Health authority: Germany: Ethics Commission
• Study type: Observational

Clinical Trial Summary

Newly diagnosed pediatric patients (age < 19 years) with bcr-abl-positive CML will be treated with imatinib. Serial monitoring of treatment response is performed in one month intervals during the first three months of treatment and in three months intervals thereafter. Patients with non-response, poor response (either molecular, cytogenetic, or hematologic non-/poor response) or progress of the disease while under imatinib treatment will stop imatinib and undergo stem cell transplantation. All responders to imatinib treatment with an HLA matched donor will undergo stem cell transplantation not later than 2 years after diagnosis.

Description:

Indication:

Newly diagnosed pediatric patients with bcr-abl-positive CML.

Design:

Multicenter, non-randomized, open, prospective clinical trial.

Objectives:

Primary:

• assessment of antileukemic activity of imatinib in children and adolescents with Philadelphia chromosome-positive chronic myeloid leukemia

Secondary:

• assessment of the time-to event-efficacy variables

• correlation of the quality of haematological, cytogenetical and molecular remission in children and adolescents with CML on ongoing imatinib therapy with survival

• safety of imatinib

Endpoints:

Primary:

• rate of haematological, cytogenetical and molecular remissions

Secondary:

• time to progression

• duration of chronic phase

• time to loss of response

• overall survival

• assessment of treatment-related toxicities in children and adolescents.

Inclusion criteria:

Newly diagnosed Ph+ or bcr-abl-positive CML- Male and female patients aged 0 to 18 years- Written informed consent

Exclusion criteria:

• CML without bcr-abl rearrangement detectable by PCR

• Pretreatment with Interferon alpha or any other cytostatic drug with the exception of hydroxyurea or anagrelide (Note: anagrelide is not approved in Germany for treatment of CML). However, these patients may be registered as observational patients.

• Any other severe underlying disease beside CML.

• Age > 18 years

• Pregnant or lactating women

• Subjects unlikely to comply with the requirements of the protocol

Number of patients to be enrolled: 150

Recruitment period: 5 years

Treatment period: 2 years

Planned start of study: 2007

Planned end of study: 2013

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 150
• Est. completion date: December 2012
• Est. primary completion date: July 2012
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A to 18 Years

Eligibility

Inclusion Criteria:

• Newly diagnosed Ph+ or bcr-abl-positive CML

• Male and female patients aged 0 to 18 years

• Written informed consent

Exclusion Criteria:

• CML without bcr-abl rearrangement detectable by PCR

• Pretreatment with Interferon alpha or any other cytostatic drug with the exception of hydroxyurea or anagrelide (Note: anagrelide is not approved in Germany for treatment of CML). However, these patients may be registered as observational patients.

• Any other severe underlying disease beside CML.

• Age > 18 years- Pregnant or lactating women

• Subjects unlikely to comply with the requirements of the protocol

Study Design

Observational Model: Cohort, Time Perspective: Prospective

Related Conditions & MeSH terms

• Leukemia, Myelogenous, Chronic, BCR-ABL Positive
• Leukemia, Myeloid
• Myeloid Leukemia, Chronic

Locations

Country	Name	City	State
Germany	University Children's Hopsital, Technical University of Dresden, Fetscherstr. 74	Dresden	Saxonia

Sponsors (5)

Lead Sponsor	Collaborator
Technische Universität Dresden	Medical Academy of Wroclaw, Poland, Nordic Society for Pediatric Hematology and Oncology, Uniuversity of Genua, Italy, University of Leiden, Netherlands

Country where clinical trial is conducted

Germany, 

References & Publications (10)

Adler R, Viehmann S, Kuhlisch E, Martiniak Y, Röttgers S, Harbott J, Suttorp M. Correlation of BCR/ABL transcript variants with patients' characteristics in childhood chronic myeloid leukaemia. Eur J Haematol. 2009 Feb;82(2):112-8. doi: 10.1111/j.1600-0609.2008.01170.x. Epub 2008 Nov 10. — View Citation

Bacher U, Klyuchnikov E, Zabelina T, Ottinger H, Beelen DW, Schrezenmeier H, Ehninger G, Müller C, Berger J, Suttorp M, Kolb HJ, Kröger N, Zander AR. The changing scene of allogeneic stem cell transplantation for chronic myeloid leukemia--a report from the German Registry covering the period from 1998 to 2004. Ann Hematol. 2009 Dec;88(12):1237-47. doi: 10.1007/s00277-009-0737-3. Epub 2009 Mar 25. — View Citation

Millot F, Guilhot J, Nelken B, Leblanc T, De Bont ES, Békassy AN, Gadner H, Sufliarska S, Stary J, Gschaidmeier H, Guilhot F, Suttorp M. Imatinib mesylate is effective in children with chronic myelogenous leukemia in late chronic and advanced phase and in relapse after stem cell transplantation. Leukemia. 2006 Feb;20(2):187-92. — View Citation

Schmid H, Jaeger BA, Lohse J, Suttorp M. Longitudinal growth retardation in a prepuberal girl with chronic myeloid leukemia on long-term treatment with imatinib. Haematologica. 2009 Aug;94(8):1177-9. doi: 10.3324/haematol.2009.008359. Epub 2009 Jun 22. — View Citation

Suttorp M, Claviez A, Bader P, Peters C, Gadner H, Ebell W, Dilloo D, Kremens B, Kabisch H, Führer M, Zintl F, Göbel U, Klingebiel T. Allogeneic stem cell transplantation for pediatric and adolescent patients with CML: results from the prospective trial CML-paed I. Klin Padiatr. 2009 Nov-Dec;221(6):351-7. doi: 10.1055/s-0029-1239529. Epub 2009 Nov 4. — View Citation

Suttorp M, Millot F. Treatment of pediatric chronic myeloid leukemia in the year 2010: use of tyrosine kinase inhibitors and stem-cell transplantation. Hematology Am Soc Hematol Educ Program. 2010;2010:368-76. doi: 10.1182/asheducation-2010.1.368. Review. — View Citation

Suttorp M, Tauer JT, Millot F: Chronic myeloid leukemia in children: diagnostics and management Onkologie (CZ) 5(2):12-15, 2011

Suttorp M, Thiede C, Tauer JT, Range U, Schlegelberger B, von Neuhoff N. Impact of the type of the BCR-ABL fusion transcript on the molecular response in pediatric patients with chronic myeloid leukemia. Haematologica. 2010 May;95(5):852-3. doi: 10.3324/h — View Citation

Suttorp M, Yaniv I, Schultz KR. Controversies in the treatment of CML in children and adolescents: TKIs versus BMT? Biol Blood Marrow Transplant. 2011 Jan;17(1 Suppl):S115-22. doi: 10.1016/j.bbmt.2010.09.003. — View Citation

Suttorp M. Innovative approaches of targeted therapy for CML of childhood in combination with paediatric haematopoietic SCT. Bone Marrow Transplant. 2008 Oct;42 Suppl 2:S40-6. doi: 10.1038/bmt.2008.282. Review. — View Citation