A Study to Evaluate 5 μg/kg Tbo-filgrastim in Infants, Children and Adolescents With Solid Tumors Without Bone Marrow Involvement

A Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, Efficacy, and Immunogenicity of Daily Subcutaneous Administration of 5 ?g/kg Tbo-filgrastim in Infants, Children and Adolescents With Solid Tumors Without Bone Marrow Involvement

The purpose of the study is to evaluate the safety, pharmacokinetics, pharmacodynamics, and efficacy of daily subcutaneous administration of 5 μg/kg tbo-filgrastim in infants, children and adolescents with solid tumors without bone marrow involvement.

Inclusion:

1. Male or female infants, children and adolescents aged 1 month to <16 years.

2. Patients with solid tumors without bone marrow involvement, who are scheduled to receive myelosuppressive CTX.

3. Body weight ≥5 kg.

4. Patients must have an initial diagnosis and histologic proof of their malignancy. All enrolled subjects should have signed consent for a CTX regimen that is known to be myelotoxic, with counts expected to drop below the absolute neutrophil count (ANC) of 0.5 × 109/L for at least 3 days. These regimens would include at least one of the following:

   • Etoposide
   • doxorubicin
   • ifosfamide
   • cyclophosphamide

5. ANC and platelet count: Patients must have an ANC >1 × 109/L and a platelet count >100 × 109/L to be eligible for therapy at the start of CTX.

6. Normal cardiac, renal, and hepatic function.

7. All subjects must have a life expectancy of 12 weeks or more.

8. Performance Status: Lansky performance score >60 (age 1 to <16 years).

   • More criteria may apply, please contact the investigator for more information.

Exclusion:

1. Bone marrow involvement.

2. Active myelogenous leukemia or history of myelogenous leukemia.

3. Previous treatment with colony-stimulating factors (granulocyte colony-stimulating factor [G-CSF], granulocyte-macrophage colony-stimulating factor, interleukin 11 [IL-11]) less than 6 weeks prior to study entry.

4. History of congenital neutropenia or cyclic neutropenia.

5. Pregnant or nursing female patients.

6. Fertile patients who do not agree to use highly reliable contraceptive measures Prior bone marrow or stem cell transplant, or prior radiation to ≥25% of bone marrow within the 4 weeks prior to the first tbo-filgrastim dose.

7. Ongoing active infection or history of infectious disease within 2 weeks prior to the screening visit.

8. Treatment with lithium at screening or planned during the study

   • More criteria may apply, please contact the investigator for more information.