Clinical trial comparing Lipegfilgrastim to Filgrastim in Pediatric Patients Diagnosed with Ewing Family of Tumors or Rhabdomyosarcoma Receiving Chemotherapy.

An Open-Label, Randomized, Active-Controlled, Multicenter Study to Evaluate the Efficacy, Pharmacokinetics, Pharmacodynamics, Safety, Tolerability, and Immunogenicity of Lipegfilgrastim 100 µg/kg Body Weight in Comparison to Filgrastim 5 µg/kg Body Weight in Pediatric Patients Diagnosed with Ewing Family of Tumors or Rhabdomyosarcoma Receiving Chemotherapy

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2015-000087-34-HU

Enrollment

Registered

2015-04-22

Start date

2015-06-05

Completion date

Unknown

Last updated

2019-02-28

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Neutropenia in pediatric patients diagnosed with Ewing family of Tumors or rhabdomyosarcoma receiving chemotherapy MedDRA version: 19.0 Level: PT Classification code 10029354 Term: Neutropenia System Organ Class: 10005329 - Blood and lymphatic system disorders MedDRA version: 19.0 Level: PT Classification code 10016288 Term: Febrile neutropenia

Interventions

Trade Name: LONQUEX Product Name: Lipegfilgrastim Product Code: XM22 Pharmaceutical Form: Solution for injection INN or Proposed INN: Li

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: a. Male or female children and adolescents aged 2 to 2.5 × 10^9/L, absolute neutrophil count (ANC) =1.5 × 10^9/L, and platelet count =100 × 10^9/L (at screening and prior to CTX). i. For patients aged =12 years, Eastern Cooperative Oncology Group (ECOG) performance status =2. j. Fertile patients (male or female) must use highly reliable contraceptive measures for the entire duration of the study. k. 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: 42 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

Exclusion criteria: a. Previous exposure to filgrastim, pegfilgrastim, lenograstim or other granulocyte colony stimulating factors (G-CSFs) / granulocyte macrophage colony-stimulating factors (GM-CSFs) in clinical development within 6 weeks prior to the first lipegfilgrastim/filgrastim administration in this study. b. Known hypersensitivity to filgrastim, pegfilgrastim, lenograstim, any other G-CSF in clinical development or any LONQUEX (lipegfilgrastim) excipients. c. History of congenital neutropenia or cyclic neutropenia. d. Patients with a clinically significant or unstable medical or surgical condition that would preclude safe and complete study participation, as determined by medical history, physical examinations, electrocardiogram (ECG), laboratory tests, or imaging. e. Pregnancy or breast feeding (if a patient becomes pregnant during the study she will be withdrawn from the study). f. 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 first dose of study drug (lipegfilgrastim/filgrastim). g. Major surgery or serious infection within 3weeks before first administration of study drug (lipegfilgrastim/filgrastim), serious trauma or compound medical procedure within the 4 weeks prior to the first study drug dose. h. Treatment with lithium at screening or planned during the study. i. Participation in a clinical study with an investigational product within 30 days or 5 half-lives before randomization, whichever is longer.

Design outcomes

Primary

Measure	Time frame
Secondary Objective: The secondary objectives of this study are to assess pharmacodynamics, pharmacokinetics, safety, tolerability, and immunogenicity of a single sc dose of 100 µg/kg BW of lipegfilgrastim per cycle compared to daily doses of 5 µg/kg BW of filgrastim.;Primary end point(s): The primary efficacy endpoint is the duration of severe neutropenia (DSN) in cycle 1, defined as the number of days with severe neutropenia in cycle 1 (from start of CTX until day 15). Severe neutropenia is defined as grade 4 neutropenia, with ANC <0.5 × 10^9/L.;Timepoint(s) of evaluation of this end point: Samples for ANC assessments in cycles 1 to 4 will be obtained on day 1 prior to study drug administration, on days 2, 4, 5, 6, 7, 8, 9, 10, 12, and 15 and at EOS/(or ET) visit.;Main Objective: The primary objective of this study is to assess the efficacy of a single subcutaneous (sc) dose of 100 µg/kg body weight (BW) of lipegfilgrastim per cycle compared to daily 5 µg/kg BW of filgrastim doses in children receiving CTX	—

Measure

Time frame

Secondary Objective: The secondary objectives of this study are to assess pharmacodynamics, pharmacokinetics, safety, tolerability, and immunogenicity of a single sc dose of 100 µg/kg BW of lipegfilgrastim per cycle compared to daily doses of 5 µg/kg BW of filgrastim.;Primary end point(s): The primary efficacy endpoint is the duration of severe neutropenia (DSN) in cycle 1, defined as the number of days with severe neutropenia in cycle 1 (from start of CTX until day 15). Severe neutropenia is defined as grade 4 neutropenia, with ANC <0.5 × 10^9/L.;Timepoint(s) of evaluation of this end point: Samples for ANC assessments in cycles 1 to 4 will be obtained on day 1 prior to study drug administration, on days 2, 4, 5, 6, 7, 8, 9, 10, 12, and 15 and at EOS/(or ET) visit.;Main Objective: The primary objective of this study is to assess the efficacy of a single subcutaneous (sc) dose of 100 µg/kg body weight (BW) of lipegfilgrastim per cycle compared to daily 5 µg/kg BW of filgrastim doses in children receiving CTX

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Secondary

Measure	Time frame
Secondary end point(s): Secondary Efficacy Variables and Endpoints: The secondary efficacy endpoints for this study are as follows: - Incidence of severe neutropenia (ANC 38.3°C or 2 consecutive readings higher than 37.8°C measured at axilla or external ear for 2 hours and ANC 1.0 × 10^9/L and ANC >2.0 × 10^9/L) from first day of CTX. - Time to ANC recovery (ANC >1.0 × 10^9/L and ANC >2.0 × 10^9/L) from nadir per cycle. ; Timepoint(s) of evaluation of this end point: Samples for PD assessments (ANC) in cycles 1 to 4 will be obtained on day 1 prior to study drug administration, on days 2, 4, 5, 6, 7, 8, 9, 10, 12, and 15 and at EOS/(or ET) visit. Continous tempareture measurements during the days 1-21 of each cycle.	—

Measure

Time frame

Secondary end point(s): Secondary Efficacy Variables and Endpoints: The secondary efficacy endpoints for this study are as follows: - Incidence of severe neutropenia (ANC 38.3°C or 2 consecutive readings higher than 37.8°C measured at axilla or external ear for 2 hours and ANC 1.0 × 10^9/L and ANC >2.0 × 10^9/L) from first day of CTX. - Time to ANC recovery (ANC >1.0 × 10^9/L and ANC >2.0 × 10^9/L) from nadir per cycle. ; Timepoint(s) of evaluation of this end point: Samples for PD assessments (ANC) in cycles 1 to 4 will be obtained on day 1 prior to study drug administration, on days 2, 4, 5, 6, 7, 8, 9, 10, 12, and 15 and at EOS/(or ET) visit. Continous tempareture measurements during the days 1-21 of each cycle.

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Countries

Belgium, Bulgaria, Croatia, Czech Republic, Germany, Hungary, Lithuania, Poland, Romania, Russian Federation, Slovakia, Spain, Ukraine

Contacts

Public ContactDr. med. Andreas Lammerich

Merckle GmbH

andreas.lammerich@ratiopharm.de497314023891

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026