Registration of Children With CML and Treatment With Imatinib

NCT ID: NCT00445822

Last Updated: 2012-02-29

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Total Enrollment

150 participants

Study Classification

OBSERVATIONAL

Study Start Date

2007-03-31

Study Completion Date

2012-12-31

Brief Summary

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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.

Detailed Description

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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

Conditions

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Myeloid Leukemia, Chronic

Keywords

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CML Children Imatinib bcr-abl Response Imatinib mesylate

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Eligibility Criteria

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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
Minimum Eligible Age

1 Day

Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Medical Academy of Wroclaw, Poland

UNKNOWN

Sponsor Role collaborator

University of Leiden, Netherlands

UNKNOWN

Sponsor Role collaborator

Nordic Society for Pediatric Hematology and Oncology

OTHER

Sponsor Role collaborator

Uniuversity of Genua, Italy

OTHER

Sponsor Role collaborator

Technische Universität Dresden

OTHER

Sponsor Role lead

Responsible Party

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Universitätskinderklinik Dresden

Principal Investigators

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Meinolf Suttorp, Prof. Dr.

Role: PRINCIPAL_INVESTIGATOR

University Children's Hospital, Dpt. Ped. Hemato-Oncology, Technical University of Dresden, Germany

Locations

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University Children's Hopsital, Technical University of Dresden, Fetscherstr. 74

Dresden, Saxony, Germany

Site Status RECRUITING

Countries

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Germany

Central Contacts

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Meinolf Suttorp, Prof. Dr.

Role: CONTACT

Phone: +49 351 458

Email: [email protected]

Xenia Graehlert, Dr.

Role: CONTACT

Phone: +49 351 458

Email: [email protected]

Facility Contacts

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Meinolf Suttorp, Prof. Dr.

Role: primary

References

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Millot F, Guilhot J, Nelken B, Leblanc T, De Bont ES, Bekassy 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. doi: 10.1038/sj.leu.2404051.

Reference Type BACKGROUND
PMID: 16341042 (View on PubMed)

Adler R, Viehmann S, Kuhlisch E, Martiniak Y, Rottgers 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.

Reference Type BACKGROUND
PMID: 19067742 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 18978743 (View on PubMed)

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. No abstract available.

Reference Type BACKGROUND
PMID: 19546438 (View on PubMed)

Bacher U, Klyuchnikov E, Zabelina T, Ottinger H, Beelen DW, Schrezenmeier H, Ehninger G, Muller C, Berger J, Suttorp M, Kolb HJ, Kroger 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.

Reference Type BACKGROUND
PMID: 19319532 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 21239821 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 21195300 (View on PubMed)

Suttorp M, Claviez A, Bader P, Peters C, Gadner H, Ebell W, Dilloo D, Kremens B, Kabisch H, Fuhrer M, Zintl F, Gobel 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.

Reference Type BACKGROUND
PMID: 19890786 (View on PubMed)

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

Reference Type BACKGROUND

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/haematol.2009.019224. Epub 2010 Feb 9. No abstract available.

Reference Type RESULT
PMID: 20145269 (View on PubMed)

Stiehler S, Sembill S, Schleicher O, Marx M, Rauh M, Krumbholz M, Karow A, Suttorp M, Woelfle J, Maj C, Metzler M. Imatinib treatment and longitudinal growth in pediatric patients with chronic myeloid leukemia: influence of demographic, pharmacological, and genetic factors in the German CML-PAED cohort. Haematologica. 2024 Aug 1;109(8):2555-2563. doi: 10.3324/haematol.2023.284668.

Reference Type DERIVED
PMID: 38497150 (View on PubMed)

Knofler R, Lange BS, Paul F, Tiebel O, Suttorp M. Bleeding signs due to acquired von Willebrand syndrome at diagnosis of chronic myeloid leukaemia in children. Br J Haematol. 2020 Mar;188(5):701-706. doi: 10.1111/bjh.16241. Epub 2019 Oct 15.

Reference Type DERIVED
PMID: 31617211 (View on PubMed)

Ulmer A, Tabea Tauer J, Glauche I, Jung R, Suttorp M. TK inhibitor treatment disrupts growth hormone axis: clinical observations in children with CML and experimental data from a juvenile animal model. Klin Padiatr. 2013 May;225(3):120-6. doi: 10.1055/s-0033-1343483. Epub 2013 May 28.

Reference Type DERIVED
PMID: 23716272 (View on PubMed)

Jaeger BA, Tauer JT, Ulmer A, Kuhlisch E, Roth HJ, Suttorp M. Changes in bone metabolic parameters in children with chronic myeloid leukemia on imatinib treatment. Med Sci Monit. 2012 Dec;18(12):CR721-8. doi: 10.12659/msm.883599.

Reference Type DERIVED
PMID: 23197234 (View on PubMed)

Other Identifiers

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EudraCT: 2007-001339-69

Identifier Type: -

Identifier Source: secondary_id

TUD-CML-paed 016

Identifier Type: -

Identifier Source: org_study_id