Ewing's Sarcoma/Soft Tissue SarcomaPhase 1/2NCT01288573
A Combined Study in Pediatric Cancer Patients for Dose Ranging and Efficacy/Safety of Plerixafor Plus Standard Regimens for Mobilization Versus Standard Regimens Alone
A Phase 1/2 study testing plerixafor in people with Ewing's Sarcoma/Soft Tissue Sarcoma and Neuroblastoma, run by Genzyme, a Sanofi Company at 27 sites in 12 countries.
Trial summary
This is a multi-site study with plerixafor in pediatric cancer patients. The study will be conducted in 2 stages: Stage 1 is a dose-escalation study. Stage 2 is an open-label, randomized, comparative study using the appropriate dosing regimen identified in the Stage 1 dose-escalation study. Read more about this study
- Who is it for?
- Ages 1 year to 18 years
- Conditions the study is for
- Ewing's Sarcoma/Soft Tissue Sarcoma, Neuroblastoma, Brain Tumors
- What is being tested?
- plerixafor
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 46 participants
- Where
- 27 sitesin 12 countries
- Study dates
- 3 Mar 2014to 9 May 2017
- Phase
- Phase 1/2
- 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 1 year to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Age 2 to < 18 years during stage 1 and 1 to < 18 years during stage 2
- Ewing's sarcoma, soft tissue sarcoma, lymphoma, neuroblastoma, brain tumors or other malignancy (excluding any form of leukemia) requiring treatment with high dose chemotherapy and autologous transplant as rescue therapy
- Eligible for autologous transplantation
- Recovered from all acute significant toxic effects of prior chemotherapy
- Adequate performance status (for patients ≥16 years of age, defined as Karnofsky score >60 and for patients <16 years of age, defined as Lansky score >60)
- Absolute neutrophil count >0.75 × 10^9/L
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- Platelet count >50 × 10^9/L
- Calculated creatinine clearance (using the Schwartz method): during study Stage 1, >80 mL/min/1.73m^2 and during study Stage 2, >60 mL/min/1.73m^2
- Aspartate aminotransferase(AST)/serum glutamic oxaloacetic transaminase(SGOT), alanine aminotransferase(ALT)/serum glutamic pyruvic transaminase (SGPT) and total bilirubin <3 × upper limit of normal
- The patient and/or their parent/legal guardian is willing and able to provide signed informed consent
- Patients who are sexually active must be willing to abstain from sexual intercourse or agree to use an approved form of contraception while receiving plerixafor and/or standard mobilization treatment and for at least 3 months following any plerixafor treatment
Who cannot join
- Any form of leukemia
- A co-morbid condition which, in the view of the Investigator, renders the patient at high-risk from treatment complications
- Previous stem cell transplantation
- Persistent high percentage marrow involvement prior to mobilization will be prohibited.
- On-going toxicities (excluding alopecia) Grade ≥2 resulting from prior chemotherapy
- Acute infection
Show all 12Show fewer
- Fever (temperature >38.5°C) - if fever is between 37°C and 38.5°C, infection must be excluded as a cause
- Known HIV seropositivity, AIDS, hepatitis C or active hepatitis B infections
- Positive pregnancy test in post pubertal girls
- History of clinically significant cardiac abnormality or arrhythmia
- Use of an investigational drug which is not approved in any indication either in adults or pediatrics within 2 weeks prior to the first dose of G-CSF to be administered as part of the patient's planned standard mobilization regimen, and/or during the study up until engraftment of the transplant. If patients are on investigational drugs as part of their anti-cancer regimen, this should be discussed with the Sponsor before screening. Drugs approved for other indications that are being used in a manner considered standard of care for this transplant procedure are allowed
- The patient (and/or their parent/legal guardian), in the opinion of the Investigator, is unable to adhere to the requirements of the study
Trial locations and contacts
27 sites in 12 countries.
27 sites in 12 countries
Germany5 sites
Investigational Site Number 33
Frankfurt am Main, 60590
Investigational Site Number 34
Freiburg im Breisgau, 79106
Investigational Site Number 35
Hamburg, 20246
Investigational Site Number 31
Hanover, 30625
Investigational Site Number 36
München, 80337
Italy5 sites
Investigational Site Number 21
Genova, 16100
Investigational Site Number 24
Milan, 20133
Investigational Site Number 23
Padova, 35128
Investigational Site Number 22
Roma, 00165
Investigational Site Number 26
Torino, 10126
Czechia2 sites
Investigational Site Number 81
Brno, 62500
Investigational Site Number 82
Praha 5 - Motol, 15006
France2 sites
Investigational Site Number 42
Lyon, 69373
Investigational Site Number 43
Paris, 75248
Israel2 sites
Investigational Site Number 92
Petah Tikva, 4920235
Investigational Site Number 91
Tel Aviv, 64239
Netherlands2 sites
Investigational Site Number 72
Amsterdam, 1105 AZ
Investigational Site Number 71
Rotterdam, 3015 GJ
Poland2 sites
Investigational Site Number 85
Krakow, 30-663
Investigational Site Number 84
Wroclaw, 50-368
Spain2 sites
Investigational Site Number 94
Barcelona, 08035
Investigational Site Number 93
Madrid, 28009
United Kingdom2 sites
Investigational Site Number 11
Birmingham, B4 6NH
Investigational Site Number 13
Glasgow, G51 4TF
About this study
Official title: A Phase 1/2 Combined Dose Ranging and Randomized, Open-label, Comparative Study of the Efficacy and Safety of Plerixafor in Addition to Standard Regimens for Mobilization of Haematopoietic Stem Cells Into Peripheral Blood, and Subsequent Collection by Apheresis, Versus Standard Mobilization Regimens Alone in Pediatric Patients, Aged 1 to <18 Years, With Solid Tumours Eligible for Autologous Transplants.
This is a multi-site study with plerixafor in pediatric cancer patients. The study will be conducted in 2 stages:
- Stage 1 is a dose-escalation study.
- Stage 2 is an open-label, randomized, comparative study using the appropriate dosing regimen identified in the Stage 1 dose-escalation study.
Read more
All participating patients will receive a standard mobilization regimen as per study site practice guidelines (either chemotherapy plus once daily granulocyte-colony stimulating factor (G-CSF) or once daily G-CSF alone). The only change to the standard mobilization regimen is the addition of plerixafor treatment prior to apheresis for all patients in Stage 1 (dose escalation), and for those patients randomized to the plerixafor plus standard mobilization treatment arm in Stage 2 (randomized, comparative).
Stage 1 will enroll at least 27 patients. Stage 2 will enroll at least 40 patients.
- Being tested
- plerixafor
- Medical subject headings
- Neuroblastoma, Brain Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
plerixaforDrug
160 μg/kg subcutaneous (SC) injection
plerixaforDrug
240 μg/kg subcutaneous (SC) injection
plerixaforDrug
320 μg/kg subcutaneous (SC) injection
| Study group | Role | What they receive |
|---|---|---|
| Plerixafor 160 μg/kg | Experimental | Patients will receive subcutaneous (SC) injection of 160 μg/kg plerixafor in addition to their standard mobilization regimen. Each dose of plerixafor will be administered in the evening 9 to 11 hours prior to apheresis (up to a maximum of 5 apheresis sessions). Receives: Drug: plerixafor |
| Plerixafor 240 μg/kg | Experimental | Patients will receive subcutaneous (SC) injection of 240 μg/kg plerixafor in addition to their standard mobilization regimen. Each dose of plerixafor will be administered in the evening 9 to 11 hours prior to apheresis (up to a maximum of 5 apheresis sessions). Receives: Drug: plerixafor |
| Plerixafor 320 μg/kg | Experimental | Patients will receive subcutaneous (SC) injection of 320 μg/kg plerixafor in addition to their standard mobilization regimen. Each dose of plerixafor will be administered in the evening 9 to 11 hours prior to apheresis (up to a maximum of 5 apheresis sessions). Receives: Drug: plerixafor |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of patients achieving at least a doubling of peripheral blood CD34+ count during Stage 2
Measured: Up to 5 days
Secondary outcomes (14)
Number of days of apheresis required to reach ≥2 × 10^6 CD34+ cells/kg
During Stage 1 and Stage 2
Measured: Up to 5 days
Yield of CD34+ cells for each apheresis
During Stage 1 and Stage 2
Measured: Up to 5 days
Total CD34+ cell yield
During Stage 1 and Stage 2
Measured: Up to 5 days
Percentage of patients proceeding to transplant
During Stage 1 and Stage 2
Measured: Within 6 months of last apheresis
Percentage of patients successfully engrafting
During Stage 1 and Stage 2
Measured: 3, 6, 12 and 24 months post-transplant
Percentage of patients with durable engraftment
During Stage 1 and Stage 2
Measured: 3, 6, 12 and 24 months post-transplant
Summary of adverse events (AEs)
During Stage 1 and Stage 2
Measured: Up to 24 months after last transplant or 24 months after last dose (for patients that do not transplant within 6 months of last apheresis)
Duration of hospitalizations (planned or unplanned)
During Stage 1 and Stage 2
Measured: Throughout the duration of the study
Mobilization of tumor cells into peripheral blood
During Stage 1 and Stage 2
Measured: Up to 5 days
Relapse rates
During Stage 1 and Stage 2
Measured: 3, 6, 12 and 24 months post-transplant
Occurrence of secondary malignancies
During Stage 1 and Stage 2
Measured: 3, 6, 12 and 24 months post-transplant
Incidence of primary and secondary graft failure
During Stage 1 and Stage 2
Measured: 3, 6, 12 and 24 months post-transplant
Time to secondary graft failure
During Stage 1 and Stage 2
Measured: Up to 24 months post-transplant
Survival rates
During Stage 1 and Stage 2
Measured: 3, 6, 12 and 24 months post-transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Feb 2011 (estimated)
- Study start3 Mar 2014
- Primary completion9 May 2017
- Study completion9 May 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Ewing's Sarcoma/Soft Tissue SarcomaStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 46 participants (actual)
Sponsor and investigators
- Lead sponsor
- Genzyme, a Sanofi Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Sanofi
- Responsible party
- Sponsor
- Investigators
- Clinical Sciences & Operations, Study director, Sanofi
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01288573
- Sponsor's protocol ID
- DFI12860
- Other IDs
- 2010-019340-40, MOZ15609
- Status last verified
- May 2017
- First submitted
- 28 Jan 2011
- Last updated
- 16 May 2017
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Sebastien B, Cheverton P, Magnin C, Aouni J, Castan R. Development and validation of a predictive model to guide the use of plerixafor in pediatric population. Bone Marrow Transplant. 2022 Dec;57(12):1827-1832. doi: 10.1038/s41409-022-01831-2. Epub 2022 Sep 26.
PubMed 36163427Morland B, Kepak T, Dallorso S, Sevilla J, Murphy D, Luksch R, Yaniv I, Bader P, Rossler J, Bisogno G, Maecker-Kolhoff B, Lang P, Zwaan CM, Sumerauer D, Krivan G, Bernard J, Liu Q, Doyle E, Locatelli F. Plerixafor combined with standard regimens for hematopoietic stem cell mobilization in pediatric patients with solid tumors eligible for autologous transplants: two-arm phase I/II study (MOZAIC). Bone Marrow Transplant. 2020 Sep;55(9):1744-1753. doi: 10.1038/s41409-020-0836-2. Epub 2020 Mar 3.
PubMed 32127657
Study at a glance
From the registry record, last updated by the sponsor on 16 May 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2 Combined Dose Ranging and Randomized, Open-label, Comparative Study of the Efficacy and Safety of Plerixafor in Addition to Standard Regimens for Mobilization of Haematopoietic Stem Cells Into Peripheral Blood, and Subsequent Collection by Apheresis, Versus Standard Mobilization Regimens Alone in Pediatric Patients, Aged 1 to <18 Years, With Solid Tumours Eligible for Autologous Transplants.
- ClinicalTrials.gov ID
- NCT01288573
- Acronym / sponsor ID
- DFI12860
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 46 participants (actual)
- Lead sponsor
- Genzyme, a Sanofi CompanyIndustry (pharma, biotech, devices)
- Collaborators
- Sanofi
- Study start
- 3 Mar 2014
- Primary completion
- 9 May 2017 (actual)
- Study completion
- 9 May 2017 (actual)
- First posted
- 2 Feb 2011 (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 multi-site study with plerixafor in pediatric cancer patients. The study will be conducted in 2 stages:
- Stage 1 is a dose-escalation study.
- Stage 2 is an open-label, randomized, comparative study using the appropriate dosing regimen identified in the Stage 1 dose-escalation study.
All participating patients will receive a standard mobilization regimen as per study site practice guidelines (either chemotherapy plus once daily granulocyte-colony stimulating factor (G-CSF) or once daily G-CSF alone). The only change to the standard mobilization regimen is the addition of plerixafor treatment prior to apheresis for all patients in Stage 1 (dose escalation), and for those patients randomized to the plerixafor plus standard mobilization treatment arm in Stage 2 (randomized, comparative).
Stage 1 will enroll at least 27 patients. Stage 2 will enroll at least 40 patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Plerixafor 160 μg/kg | Experimental | Drug: plerixaforPatients will receive subcutaneous (SC) injection of 160 μg/kg plerixafor in addition to their standard mobilization regimen. Each dose of plerixafor will be administered in the evening 9 to 11 hours prior to apheresis (up to a maximum of 5 apheresis sessions). |
| Plerixafor 240 μg/kg | Experimental | Drug: plerixaforPatients will receive subcutaneous (SC) injection of 240 μg/kg plerixafor in addition to their standard mobilization regimen. Each dose of plerixafor will be administered in the evening 9 to 11 hours prior to apheresis (up to a maximum of 5 apheresis sessions). |
| Plerixafor 320 μg/kg | Experimental | Drug: plerixaforPatients will receive subcutaneous (SC) injection of 320 μg/kg plerixafor in addition to their standard mobilization regimen. Each dose of plerixafor will be administered in the evening 9 to 11 hours prior to apheresis (up to a maximum of 5 apheresis sessions). |
Who may qualify
- Ages
- 1 year to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (11 listed)
- Age 2 to < 18 years during stage 1 and 1 to < 18 years during stage 2
- Ewing's sarcoma, soft tissue sarcoma, lymphoma, neuroblastoma, brain tumors or other malignancy (excluding any form of leukemia) requiring treatment with high dose chemotherapy and autologous transplant as rescue therapy
- Eligible for autologous transplantation
- Recovered from all acute significant toxic effects of prior chemotherapy
- Adequate performance status (for patients ≥16 years of age, defined as Karnofsky score >60 and for patients <16 years of age, defined as Lansky score >60)
Key exclusion criteria (12 listed)
- Any form of leukemia
- A co-morbid condition which, in the view of the Investigator, renders the patient at high-risk from treatment complications
- Previous stem cell transplantation
- Persistent high percentage marrow involvement prior to mobilization will be prohibited.
- On-going toxicities (excluding alopecia) Grade ≥2 resulting from prior chemotherapy
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 |
|---|---|---|
| plerixafor160 μg/kg subcutaneous (SC) injection | Drug | Plerixafor 160 μg/kg |
| plerixafor240 μg/kg subcutaneous (SC) injection | Drug | Plerixafor 240 μg/kg |
| plerixafor320 μg/kg subcutaneous (SC) injection | Drug | Plerixafor 320 μg/kg |
Primary outcome
Proportion of patients achieving at least a doubling of peripheral blood CD34+ count during Stage 2
Time frame: Up to 5 days
14 secondary outcomes
Number of days of apheresis required to reach ≥2 × 10^6 CD34+ cells/kg
Time frame: Up to 5 days
Yield of CD34+ cells for each apheresis
Time frame: Up to 5 days
Total CD34+ cell yield
Time frame: Up to 5 days
Percentage of patients proceeding to transplant
Time frame: Within 6 months of last apheresis
Percentage of patients successfully engrafting
Time frame: 3, 6, 12 and 24 months post-transplant
Percentage of patients with durable engraftment
Time frame: 3, 6, 12 and 24 months post-transplant
Summary of adverse events (AEs)
Time frame: Up to 24 months after last transplant or 24 months after last dose (for patients that do not transplant within 6 months of last apheresis)
Duration of hospitalizations (planned or unplanned)
Time frame: Throughout the duration of the study
Mobilization of tumor cells into peripheral blood
Time frame: Up to 5 days
Relapse rates
Time frame: 3, 6, 12 and 24 months post-transplant
Occurrence of secondary malignancies
Time frame: 3, 6, 12 and 24 months post-transplant
Incidence of primary and secondary graft failure
Time frame: 3, 6, 12 and 24 months post-transplant
Time to secondary graft failure
Time frame: Up to 24 months post-transplant
Survival rates
Time frame: 3, 6, 12 and 24 months post-transplant
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 16 May 2017.
- Derived from this study: PubMed 36163427Sebastien B, Cheverton P, Magnin C, Aouni J, Castan R. Development and validation of a predictive model to guide the use of plerixafor in pediatric population. Bone Marrow Transplant. 2022 Dec;57(12):1827-1832. doi: 10.1038/s41409-022-01831-2. Epub 2022 Sep 26.
- Derived from this study: PubMed 32127657Morland B, Kepak T, Dallorso S, Sevilla J, Murphy D, Luksch R, Yaniv I, Bader P, Rossler J, Bisogno G, Maecker-Kolhoff B, Lang P, Zwaan CM, Sumerauer D, Krivan G, Bernard J, Liu Q, Doyle E, Locatelli F. Plerixafor combined with standard regimens for hematopoietic stem cell mobilization in pediatric patients with solid tumors eligible for autologous transplants: two-arm phase I/II study (MOZAIC). Bone Marrow Transplant. 2020 Sep;55(9):1744-1753. doi: 10.1038/s41409-020-0836-2. Epub 2020 Mar 3.
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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.
Clinical Sciences & Operations study director
Sanofi
27 sites in 12 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing plerixafor in people with Ewing's Sarcoma/Soft Tissue Sarcoma and Neuroblastoma, run by Genzyme, a Sanofi Company at 27 sites in 12 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 11 inclusion and 12 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 16 May 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 27 sites in Germany, Italy, Czechia, France, Israel and 7 more countries. 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 combined 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.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
Proportion of patients achieving at least a doubling of peripheral blood CD34+ count during Stage 2 (time frame: Up to 5 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01288573, which also lists 2 linked publications.
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 16 May 2017.