PK/PD of XM22 in Children With Ewing Family of Tumors or Rhabdomyosarcoma

NCT01585649 | Completed Phase 1

• 2 other identifiers: XM22-072011-004742-18
• Study Type: interventional
• Target: 21
• Locations: 6 countries
• Sites: 18

Brief 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)

Conditions

Ewing Family of Tumors, Rhabdomyosarcoma

Outcome Measures

Primary Outcomes (1)

• PK: Area under the curve, Maximum observed serum concentration (Cmax), Rate constant associated with terminal phase, Mean Residence Time, Time to reach Cmax, and Apparent volume of distribution during terminal phase after non-intravenous administration

  A total of 7 PK samples will be obtained at prespecified periods

  16 months

Secondary Outcomes (1)

• PD:Absolute Neutrophil Count

  16 months

Study Arms (1)

XM22, 100 μg/kg BW

EXPERIMENTAL

Drug: Lipegfilgrastim

Interventions

Lipegfilgrastim DRUG

Lipegfilgrastim 100ug/kg

XM22, 100 μg/kg BW

Eligibility Criteria

• Age: 2 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.)

You may not qualify if:

• 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.

Sponsors & Collaborators

• Merckle GmbH: lead

Study Sites (18)

Teva Investigational Site 0103

Plovdiv, Bulgaria

Location

Teva Investigational Site 0101

Sofia, Bulgaria

Location

Teva Investigational Site 0102

Varna, Bulgaria

Location

Teva Investigational Site 0201

Prague, Czechia

Location

Teva Investigational Site 0301

Budapest, Hungary

Location

Teva Investigational Site 0401

Lublin, Poland

Location

Teva Investigational Site 0501

Chelyabinsk, Russia

Location

Teva Investigational Site 0507

Krasnodar, Russia

Location

Teva Investigational Site 0505

Moscow, Russia

Location

Teva Investigational Site 0506

Moscow, Russia

Location

Teva Investigational Site 0508

Moscow, Russia

Location

Teva Investigational Site 0502

Saint Petersburg, Russia

Location

Teva Investigational Site 0504

Yekaterinburg, Russia

Location

Teva Investigational Site 0701

Dnipropetrovsk, Ukraine

Location

Teva Investigational Site 0705

Donetsk, Ukraine

Location

Teva Investigational Site 0702

Kharkiv, Ukraine

Location

Teva Investigational Site 0704

Kyiv, Ukraine

Location

Teva Investigational Site 0703

Lviv, Ukraine

Location

MeSH Terms

Conditions

Neuroectodermal Tumors, Primitive, Peripheral; Rhabdomyosarcoma

Interventions

pegfilgrastim

Condition Hierarchy (Ancestors)

Neuroectodermal Tumors, Primitive; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue; Myosarcoma; Neoplasms, Muscle Tissue; Neoplasms, Connective and Soft Tissue; Sarcoma

Study Officials

• Andreas Lammerich, MD

  Merckle GmbH, Teva Ratiopharm

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: SUPPORTIVE CARE
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 18, 2012
• First Posted: April 26, 2012
• Study Start: July 1, 2012
• Primary Completion: June 1, 2014
• Study Completion: April 1, 2015
• Last Updated: May 18, 2016

Record last verified: 2016-05

Locations

UA (5); RU (7); BU (3); PL (1); HU (1); CZ (1)