pk/pd of xm22 in children with ewing family of tumors or rhabdomyosarcoma

Conditions: Ewing Family of Tumors, Rhabdomyosarcoma

Summary: this is a phase i, open label study aimed at assessing the pharmacokinetics, pharmacodynamics, the efficacy, safety, and tolerability of a single injection of xm22 in children with ewing family of tumors or rhabdomyosarcoma scheduled to receive chemotherapy (ctx)


Eligibility criteria:
inclusion criteria: * male or female children and adolescents aged 2 to \<18 years * written informed consent provided by parent(s)/legal representative(s) of the pediatric patient and patient's assent if appropriate * able to understand and/or follow study instructions alone or with parental assistance * diagnosed with the ewing family of tumors or rhabdomyosarcoma * scheduled to receive 1 of the following ctx regimens (inpatient or outpatient) * for the ewing family of tumors: * vincristine/ifosfamide/doxorubicin/etoposide (vide); with concomitant sodium 2-mercaptoethane sulfonate (mesna) according to local standards * vincristine/doxorubicin/cyclophosphamide alternating with ifosfamide/etoposide (vdc/ie); with concomitant mesna treatment according to local standards * for rhabdomyosarcoma: * vincristine/actinomycin/cyclophosphamide (vac) * vincristine/doxorubicin/cyclophosphamide alternating with ifosfamide/etoposide (vdc/ie); with concomitant mesna treatment according to local standards * chemotherapy-naïve * body weight ≥15 kg * white blood cell (wbc) count \>2.5 x 109/l, absolute neutrophil count (anc) ≥1.5 x 109/l, and platelet count ≥100 x 109/l (at screening and prior to ctx) * for patients aged ≥12 years, eastern cooperative oncology group (ecog) performance status ≤2 (see appendix a.) * fertile patients (male or female) must use highly reliable contraceptive measures (i.e. two of the following: oral contraception, implants, injections, barrier contraception, and intrauterine device, or vasectomized/sterilized partners, or sexual abstinence). for purposes of this study, a fertile female patient is any female patient who has experienced menarche and who has not undergone tubal ligation. * female patients who have attained menarche must have a negative urine pregnancy test at the screening visit. exclusion criteria: * previous exposure to filgrastim, pegfilgrastim or lenograstim or other g-csfs in clinical development within 6 months prior to the xm22 administration * known hypersensitivity to filgrastim, pegfilgrastim or lenograstim or any other g-csf in clinical development * history of congenital neutropenia or cyclic neutropenia * any illness or condition that in the opinion of the investigator may affect the safety of the patient or the evaluation of any study endpoint * pregnant or nursing women * fertile patients who do not agree to use highly reliable contraceptive measures during the entire duration of the study * prior bone marrow or stem cell transplant, or prior radiation to ≥25% of bone marrow (e.g. whole pelvic radiation) for any reason, or any therapeutic radiation within the 3 weeks prior to the xm22 dose * ongoing active infection or history of infectious disease within 2 weeks prior to the screening visit * treatment with lithium at screening or planned during the study.