Trial Information

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

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