Ewing Family of Tumors, RhabdomyosarcomaPhase 1NCT01585649
PK/PD of XM22 in Children With Ewing Family of Tumors or Rhabdomyosarcoma
A Phase 1 study testing Lipegfilgrastim in people with Ewing Family of Tumors, Rhabdomyosarcoma, run by Merckle GmbH at 18 sites in 6 countries.
Trial 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) Read more about this study
- Who is it for?
- Ages 2 to 17 years
- Conditions the study is for
- Ewing Family of Tumors, Rhabdomyosarcoma
- What is being tested?
- Lipegfilgrastim
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 21 participants
- Where
- 18 sitesin 6 countries
- Study dates
- Jul 2012to Apr 2015
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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:
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- 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.
Who cannot join
- 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
Show all 9Show fewer
- 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.
Trial locations and contacts
18 sites in 6 countries.
18 sites in 6 countries
Russia7 sites
Teva Investigational Site 0501
Chelyabinsk
Teva Investigational Site 0507
Krasnodar
Teva Investigational Site 0505
Moscow
Teva Investigational Site 0506
Moscow
Teva Investigational Site 0508
Moscow
Teva Investigational Site 0502
Saint Petersburg
Teva Investigational Site 0504
Yekaterinburg
Ukraine5 sites
Teva Investigational Site 0701
Dnipropetrovsk
Teva Investigational Site 0705
Donetsk
Teva Investigational Site 0702
Kharkiv
Teva Investigational Site 0704
Kyiv
Teva Investigational Site 0703
Lviv
Bulgaria3 sites
Teva Investigational Site 0103
Plovdiv
Teva Investigational Site 0101
Sofia
Teva Investigational Site 0102
Varna
About this study
Official title: Multicenter, Open-label Study to Assess the Pharmacokinetics (PK), Pharmacodynamics (PD), Efficacy, Safety, Tolerability, and Immunogenicity of a Single, Subcutaneous Dose of 100µg/kg XM22 in 21 Children With Ewing Family of Tumors or Rhabdomyosarcoma
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
- Being tested
- Lipegfilgrastim
- Medical subject headings
- Neuroectodermal Tumors, Primitive, Peripheral, Rhabdomyosarcoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LipegfilgrastimDrug
Lipegfilgrastim 100ug/kg
| Study group | Role | What they receive |
|---|---|---|
| XM22, 100 μg/kg BW | Experimental | Not described Receives: Drug: Lipegfilgrastim |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 16 months
Secondary outcomes (1)
PD:Absolute Neutrophil Count
Measured: 16 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Apr 2012 (estimated)
- Study startJul 2012
- Primary completionJun 2014
- Study completionApr 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Ewing Family of Tumors, RhabdomyosarcomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 21 participants (actual)
Sponsor and investigators
- Lead sponsor
- Merckle GmbH
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Andreas Lammerich, MD, Study director, Merckle GmbH, Teva Ratiopharm
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01585649
- Sponsor's protocol ID
- XM22-07
- Other IDs
- 2011-004742-18
- Status last verified
- May 2016
- First submitted
- 18 Apr 2012
- Last updated
- 18 May 2016
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 May 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multicenter, Open-label Study to Assess the Pharmacokinetics (PK), Pharmacodynamics (PD), Efficacy, Safety, Tolerability, and Immunogenicity of a Single, Subcutaneous Dose of 100µg/kg XM22 in 21 Children With Ewing Family of Tumors or Rhabdomyosarcoma
- ClinicalTrials.gov ID
- NCT01585649
- Acronym / sponsor ID
- XM22-07
- Conditions
- Ewing Family of Tumors, Rhabdomyosarcoma
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 21 participants (actual)
- Lead sponsor
- Merckle GmbHIndustry (pharma, biotech, devices)
- Study start
- Jul 2012
- Primary completion
- Jun 2014 (actual)
- Study completion
- Apr 2015 (actual)
- First posted
- 26 Apr 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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)
Study design
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| XM22, 100 μg/kg BW | Experimental | Drug: Lipegfilgrastim |
Who may qualify
- Ages
- 2 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (17 listed)
- 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)
Key exclusion criteria (9 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| LipegfilgrastimLipegfilgrastim 100ug/kg | Drug | XM22, 100 μg/kg BW |
Primary outcome
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
Time frame: 16 months
A total of 7 PK samples will be obtained at prespecified periods
1 secondary outcome
PD:Absolute Neutrophil Count
Time frame: 16 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 May 2016.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Andreas Lammerich, MD study director
Merckle GmbH, Teva Ratiopharm
18 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Lipegfilgrastim in people with Ewing Family of Tumors, Rhabdomyosarcoma, run by Merckle GmbH at 18 sites in 6 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 17 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 18 May 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 18 sites in Russia, Ukraine, Bulgaria, Czechia, Hungary and 1 more country. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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 (time frame: 16 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 May 2016.