A Study to Assess the Pharmacokinetics (absorption, distribution, metabolisation, excretion), Pharmacodynamics (biochemical and physiological effects of the drug), Efficacy, Safety, Tolerability, and Immunogenicity (Immune response) of a Single, Subcutaneous Dose of 100µg/kg XM22 in 21 Children with Ewing Family of Tumors or Rhabdomyosarcoma

Phase 1

Conditions: Children with Ewing Family of Tumors or Rhabdomyosarcoma receiving cytotoxic Chemotherapy for malignancy inducing neutropenia
Therapeutic area: Diseases [C] - Cancer [C04]

Registration Number: EUCTR2011-004742-18-CZ

Lead Sponsor: Merckle GmbH, a member of the ratiopharm group, a subsidiary of Teva Pharmaceutical Industries Ltd. Germany

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 21

Inclusion Criteria

1. Male or female children and adolescents aged 2 to <18 years
2. Written informed consent provided by parent(s)/legal
representative(s) of the pediatric patient and patient's assent if
appropriate
3. Able to understand and/or follow study instructions alone or with
parental assistance
4. Diagnosed with the Ewing family of tumors or rhabdomyosarcoma
5. Scheduled to receive 1 of the following chemotherapy regimens
(inpatient or outpatient)
• For the Ewing family of tumors:
- vincristine/ifosfamide/doxorubicin/etoposide (VIDE); concomitant
treatment with sodium 2-mercaptoethane sulfonate (MESNA) according
to local standards
- vincristine/doxorubicin/cyclophosphamide alternating with
ifosfamide/etoposide (VDC/IE); concomitant treatment with MESNA
according to local standards
• For rhabdomyosarcoma:
- vincristine/actinomycin/cyclophosphamide (VAC)
- vincristine/doxorubicin/cyclophosphamide alternating with
ifosfamide/etoposide (VDC/IE); concomitant treatment with MESNA
according to local standards
6. Chemotherapy-naïve
7. Body weight =15 kg
8. White blood cell (WBC) count >2.5 x 10(9)/L, absolute neutrophil
count (ANC) =1.5 x 10(9)/L, and platelet count =100 x 10(9)/L (at
screening and prior to CTX).
9. For patients aged =12 years, Eastern Cooperative Oncology Group
(ECOG) performance status =2
10. 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.
11. Female patients who have attained menarche must have a negative
urine pregnancy test at the screening visit.

Are the trial subjects under 18? yes
Number of subjects for this age range: 21
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Previous exposure to filgrastim, pegfilgrastim or lenograstim or
other G-CSFs in clinical development within 6 months prior to the XM22
administration
2. Known hypersensitivity to filgrastim, pegfilgrastim or lenograstim or
any other G-CSF in clinical development
3. History of congenital neutropenia or cyclic neutropenia
4. 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
5. Pregnant or nursing women
6. Fertile patients who do not agree to use highly reliable contraceptive
measures during the entire duration of the study
7. Prior bone marrow or stem cell transplant, or prior radiation to =
25% of bone marrow (eg, whole pelvic radiation) for any reason, or
any therapeutic radiation within the 3 weeks prior to the XM22 dose
8. Ongoing active infection or history of infectious disease within 2
weeks prior to the screening visit
9. Treatment with lithium at screening or planned during the study.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: The primary objective of the study is to assess the pharmacokinetics (PK) of a single subcutaneous (SC) injection of XM22, 100 µg/kg body weight (BW), in children with Ewing family of tumors or rhabdomyosarcoma.;Secondary Objective: The secondary objectives are to assess the pharmacodynamics (PD), efficacy, safety, tolerability, and immunogenicity of this single dose in the same patient population.;Primary end point(s): Assessment of pharmacokinetics of single dose XM22;Timepoint(s) of evaluation of this end point: Pharmacokinetic profile up to 240 hours postdose

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): Pharmacodynamic (ANC, CD34+), Efficacy, Safety, Tolerability,<br>Immunogenicity;Timepoint(s) of evaluation of this end point: Pharmacodynamic (ANC, CD34+) until day 15<br>Efficacy, Safety, Tolerability until day 21<br>Immunogenicity until day 21 and follow-up on day 180 and day 360

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