SarcomaPhase 2NCT00077285
Irinotecan and Carboplatin as Upfront Window Therapy in Treating Patients With Newly Diagnosed Intermediate-Risk or High-Risk Rhabdomyosarcoma
A Phase 2 study testing filgrastim, carboplatin and cyclophosphamide in people with Sarcoma, run by Memorial Sloan Kettering Cancer Center at 1 site in United States.
Trial summary
RATIONALE: Drugs used in chemotherapy, such as irinotecan and carboplatin, work in different ways to stop tumor cells from dividing so they stop growing or die. Giving more than one drug may kill more tumor cells. Read more about this study
- Who is it for?
- Ages Up to 50 years
- Conditions the study is for
- Sarcoma
- What is being tested?
- filgrastim, carboplatin and cyclophosphamide
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 65 participants
- Where
- 1 siteUnited States
- Study dates
- Oct 2003to Oct 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 Up to 50 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Newly diagnosed, previously untreated histologically-proven rhabdomyosarcoma, undifferentiated sarcoma, or ectomesenchymoma. Histology must be confirmed by a MSKCC pathologist.
- Intermediate- or high-risk features as defined below:
- All patients with Stage 4 tumors (distant metastases).
- Intermediate Risk:
- All patients with non-metastatic undifferentiated sarcoma or alveolar RMS or ectomesenchymoma with alveolar features (regardless of age, site, size, stage, or degree of initial surgical resection);
- All patients < 1 year of age with non-metastatic embryonal RMS or ectomesenchymoma with embryonal features (regardless of site, stage, or degree of initial surgical resection).
Show all 21Show fewer
- Patients ≥ 1 year of age with Stage 2 or 3 (unfavorable site [see Appendix I] and either size > 5 cm, OR regional nodes positive, or both), Group III (gross residual disease post-biopsy or attempted resection) embryonal RMS or ectomesenchymoma with embryonal features
- Age: ≤ 50 years (inclusive) at the time of diagnosis.
- Biopsy or definitive surgery within 42 days of start of treatment.
- Organ function:
- Normal renal function: Normal serum creatinine for age or creatinine clearance or nuclear GFR of ≥ 80 ml/min/1.73m2 (in the absence of obstructive hydronephrosis, e.g., from pelvic or bladder/prostate tumor).
- Normal liver function: Total bilirubin, SGOT/SGPT < 2.5 times the upper limit of normal (in the absence of hepatic involvement by tumor)
- Normal cardiac function: echocardiogram shortening fraction ≥ 28% or resting left ventricular ejection fraction (LVEF) ≥ 50% on Technetium-99m pertechnetate radionuclide cineangiography (MUGA)
- Normal hematologic function: absolute neutrophil count (ANC) ≥ 1500/μL, hemoglobin ≥ 9 gm/dL, and platelet count ≥ 100,000/μL (in the absence of bone marrow infiltration by tumor or the presence of disseminated intravascular coagulation).
- Measurable disease is not required.
- Patients must consent to an indwelling central venous catheter.
- Sexually active patients of childbearing potential must be willing to use an effective method of contraception.
- Patient or guardian must be capable of providing informed consent.
- SUBJECT EXCLUSION CRITERIA:
- Prior chemotherapy or radiotherapy (other than limited, emergent radiotherapy for treatment of threatened airway or cord compromise).
- Pregnant or breast feeding females because the chemotherapy administered on this trial could have a detrimental effect on the developing fetus or newborn.
Who cannot join
None listed.
Trial locations and contacts
1 site in 1 country.
United States1 site
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
About this study
Official title: A Pilot Phase II Trial Of Irinotecan Plus Carboplatin, And Irinotecan Maintenance Therapy (High-Risk Patients Only), Integrated Into The Upfront Therapy Of Newly Diagnosed Patients With Intermediate - And High-Risk Rhabdomyosarcoma
RATIONALE: Drugs used in chemotherapy, such as irinotecan and carboplatin, work in different ways to stop tumor cells from dividing so they stop growing or die. Giving more than one drug may kill more tumor cells.
PURPOSE: This phase II trial is studying how well giving irinotecan together with carboplatin as upfront window therapy (first-line therapy) works in treating patients with newly diagnosed intermediate-risk or high-risk rhabdomyosarcoma.
Read the detailed description
OBJECTIVES:
Primary
- Determine the response rate in patients with newly diagnosed intermediate- or high-risk rhabdomyosarcoma treated with upfront window therapy comprising irinotecan and carboplatin.
- Determine the acute toxic effects of this regimen combined with radiotherapy in these patients.
- Determine the safety and feasibility of this regimen in these patients.
- Determine the rate of local control achieved in patients treated with this regimen in combination with intensity-modulated radiotherapy.
- Determine the safety and feasibility of administering maintenance therapy comprising irinotecan to patients with high-risk rhabdomyosarcoma treated with this regimen.
Secondary
- Correlate, preliminarily, in vitro measurements of angiogenesis with clinical features (extent of disease), response to therapy, and outcome in patients treated with this regimen.
- Determine, preliminarily, the efficacy of this regimen, in terms of improved outcomes, in these patients.
OUTLINE: This is a pilot study.
- Courses 1 and 2: Patients receive carboplatin IV over 1 hour on day 1 and irinotecan IV over 1 hour on days 1-5 and 8-12. Treatment repeats every 21 days for a total of 2 courses.
- Courses 3-5: Patients receive vincristine IV on days 1, 8, and 15; dexrazoxane IV over 15-30 minutes, doxorubicin IV over 15-30 minutes, and cyclophosphamide IV over 1 hour on days 1 and 2; and filgrastim (G-CSF) subcutaneously (SC) once daily beginning on approximately day 3 and continuing until blood counts recover. Treatment repeats every 21 days for a total of 3 courses.
Some patients may undergo surgical resection of the tumor after completion of course 5. After course 5, patients undergo radiotherapy once daily, 5 days a week, for 4-5.5 weeks.
- Courses 6 and 7*: Patients receive vincristine IV and carboplatin IV over 1 hour on day 1; irinotecan IV over 1 hour on days 1-5 and 8-12; and G-CSF SC once daily beginning on approximately day 13 and continuing until blood counts recover. Treatment repeats every 21 days for a total of 2 courses.
NOTE: *Patients who develop disease progression during courses 1 or 2 do not receive further irinotecan and carboplatin. Instead, patients receive ifosfamide and etoposide as in courses 8 and 9.
- Courses 8 and 9: Patients receive vincristine IV on day 1; etoposide IV over 1 hour and ifosfamide IV over 2 hours on days 1-5; and G-CSF SC once daily beginning on approximately day 6 and continuing until blood counts recover. Treatment repeats every 21 days for a total of 2 courses.
- Course 10: Patients receive vincristine IV on days 1, 8, 15, 22, 29, 36, and 43; dexrazoxane IV over 15-30 minutes, doxorubicin IV over 15-30 minutes, and cyclophosphamide IV over 1 hour on days 1 and 2; and filgrastim SC beginning on approximately day 3 and continuing until blood counts recover (1 course).
- Course 11 and 12: Patients receive etoposide IV over 1 hour and ifosfamide IV over 2 hours on days 1-5 and G-CSF SC once daily beginning on approximately day 6 and continuing until blood counts recover. Treatment repeats every 21 days for a total of 2 courses.
Patients with high-risk disease proceed to maintenance therapy.
- Maintenance therapy*: Patients receive irinotecan IV over 1 hour on days 1-5 and 8-12. Treatment repeats every 21 days for a total of 6 courses.
NOTE: *Patients who develop disease progression during courses 1 or 2 do not receive further irinotecan.
In all courses, treatment continues in the absence of unacceptable toxicity or disease progression or recurrence after initial response.
Patients are followed monthly for 1 year, every 3 months for 1 year, every 6 months for 1 year, and then annually thereafter.
*Starting with A(8), patients will undergo PET Scans instead of Bone Scans, at the discretion of the PI.
- Conditions
- Sarcoma
- Being tested
- filgrastim, carboplatin, cyclophosphamide, dexrazoxane hydrochloride, doxorubicin hydrochloride
- Keywords
- previously untreated childhood rhabdomyosarcoma, embryonal childhood rhabdomyosarcoma, alveolar childhood rhabdomyosarcoma, adult rhabdomyosarcoma, stage IV adult soft tissue sarcoma, metastatic childhood soft tissue sarcoma, nonmetastatic childhood soft tissue sarcoma, childhood malignant mesenchymoma, adult malignant mesenchymoma, stage III adult soft tissue sarcoma, stage II adult soft tissue sarcoma, stage I adult soft tissue sarcoma
- Medical subject headings
- Sarcoma, Rhabdomyosarcoma, Malignant mesenchymal tumor
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
filgrastimBiological
carboplatinDrug
cyclophosphamideDrug
dexrazoxane hydrochlorideDrug
etoposideDrug
ifosfamideDrug
conventional surgeryProcedure or surgery
radiation therapyRadiation
| Study group | Role | What they receive |
|---|---|---|
| pts with intermediate- and high-risk rhabdomyosarcoma | Experimental | Not described Receives: Biological: filgrastim; Drug: carboplatin; Drug: cyclophosphamide; Drug: dexrazoxane hydrochloride; Drug: doxorubicin hydrochloride; Drug: etoposide; Drug: ifosfamide; Drug: irinotecan hydrochloride; Drug: vincristine sulfate; Procedure: conventional surgery; Radiation: radiation therapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Response rate
Measured: 2 years
Toxicity
Measured: 2 years
Safety and feasibility
Measured: 2 years
Rate of local control
Measured: 2 years
Secondary outcomes (2)
Correlation of in vitro measurements of angiogenesis with clinical features (extent of disease), response to therapy, and outcome
Measured: 2 years
Efficacy in terms of improved outcomes
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Feb 2004 (estimated)
- Study startOct 2003
- Primary completionOct 2027 (estimated)
- Study completionOct 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for SarcomaStudies of the same condition, open and under way.
- More trials run by Memorial Sloan Kettering Cancer CenterThe sponsor's other registered studies.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 65 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Memorial Sloan Kettering Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Sponsor
- Investigators
- Leonard H. Wexler, MD, Principal investigator, Memorial Sloan Kettering Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00077285
- Sponsor's protocol ID
- 03-099
- Other IDs
- MSKCC-03099
- Status last verified
- May 2026
- First submitted
- 10 Feb 2004
- Last updated
- 22 May 2026
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Phase II Trial Of Irinotecan Plus Carboplatin, And Irinotecan Maintenance Therapy (High-Risk Patients Only), Integrated Into The Upfront Therapy Of Newly Diagnosed Patients With Intermediate - And High-Risk Rhabdomyosarcoma
- ClinicalTrials.gov ID
- NCT00077285
- Acronym / sponsor ID
- 03-099
- Conditions
- Sarcoma
- Keywords
- previously untreated childhood rhabdomyosarcoma, embryonal childhood rhabdomyosarcoma, alveolar childhood rhabdomyosarcoma, adult rhabdomyosarcoma, stage IV adult soft tissue sarcoma, metastatic childhood soft tissue sarcoma, nonmetastatic childhood soft tissue sarcoma, childhood malignant mesenchymoma
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 65 participants (estimated)
- Lead sponsor
- Memorial Sloan Kettering Cancer CenterUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- Oct 2003
- Primary completion
- Oct 2027 (estimated)
- Study completion
- Oct 2027 (estimated)
- First posted
- 11 Feb 2004 (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.
RATIONALE: Drugs used in chemotherapy, such as irinotecan and carboplatin, work in different ways to stop tumor cells from dividing so they stop growing or die. Giving more than one drug may kill more tumor cells.
PURPOSE: This phase II trial is studying how well giving irinotecan together with carboplatin as upfront window therapy (first-line therapy) works in treating patients with newly diagnosed intermediate-risk or high-risk rhabdomyosarcoma.
Detailed description
OBJECTIVES:
Primary
- Determine the response rate in patients with newly diagnosed intermediate- or high-risk rhabdomyosarcoma treated with upfront window therapy comprising irinotecan and carboplatin.
- Determine the acute toxic effects of this regimen combined with radiotherapy in these patients.
- Determine the safety and feasibility of this regimen in these patients.
- Determine the rate of local control achieved in patients treated with this regimen in combination with intensity-modulated radiotherapy.
- Determine the safety and feasibility of administering maintenance therapy comprising irinotecan to patients with high-risk rhabdomyosarcoma treated with this regimen.
Secondary
- Correlate, preliminarily, in vitro measurements of angiogenesis with clinical features (extent of disease), response to therapy, and outcome in patients treated with this regimen.
- Determine, preliminarily, the efficacy of this regimen, in terms of improved outcomes, in these patients.
OUTLINE: This is a pilot study.
- Courses 1 and 2: Patients receive carboplatin IV over 1 hour on day 1 and irinotecan IV over 1 hour on days 1-5 and 8-12. Treatment repeats every 21 days for a total of 2 courses.
- Courses 3-5: Patients receive vincristine IV on days 1, 8, and 15; dexrazoxane IV over 15-30 minutes, doxorubicin IV over 15-30 minutes, and cyclophosphamide IV over 1 hour on days 1 and 2; and filgrastim (G-CSF) subcutaneously (SC) once daily beginning on approximately day 3 and continuing until blood counts recover. Treatment repeats every 21 days for a total of 3 courses.
Some patients may undergo surgical resection of the tumor after completion of course 5. After course 5, patients undergo radiotherapy once daily, 5 days a week, for 4-5.5 weeks.
- Courses 6 and 7*: Patients receive vincristine IV and carboplatin IV over 1 hour on day 1; irinotecan IV over 1 hour on days 1-5 and 8-12; and G-CSF SC once daily beginning on approximately day 13 and continuing until blood counts recover. Treatment repeats every 21 days for a total of 2 courses.
NOTE: *Patients who develop disease progression during courses 1 or 2 do not receive further irinotecan and carboplatin. Instead, patients receive ifosfamide and etoposide as in courses 8 and 9.
- Courses 8 and 9: Patients receive vincristine IV on day 1; etoposide IV over 1 hour and ifosfamide IV over 2 hours on days 1-5; and G-CSF SC once daily beginning on approximately day 6 and continuing until blood counts recover. Treatment repeats every 21 days for a total of 2 courses.
- Course 10: Patients receive vincristine IV on days 1, 8, 15, 22, 29, 36, and 43; dexrazoxane IV over 15-30 minutes, doxorubicin IV over 15-30 minutes, and cyclophosphamide IV over 1 hour on days 1 and 2; and filgrastim SC beginning on approximately day 3 and continuing until blood counts recover (1 course).
- Course 11 and 12: Patients receive etoposide IV over 1 hour and ifosfamide IV over 2 hours on days 1-5 and G-CSF SC once daily beginning on approximately day 6 and continuing until blood counts recover. Treatment repeats every 21 days for a total of 2 courses.
Patients with high-risk disease proceed to maintenance therapy.
- Maintenance therapy*: Patients receive irinotecan IV over 1 hour on days 1-5 and 8-12. Treatment repeats every 21 days for a total of 6 courses.
NOTE: *Patients who develop disease progression during courses 1 or 2 do not receive further irinotecan.
In all courses, treatment continues in the absence of unacceptable toxicity or disease progression or recurrence after initial response.
Patients are followed monthly for 1 year, every 3 months for 1 year, every 6 months for 1 year, and then annually thereafter.
*Starting with A(8), patients will undergo PET Scans instead of Bone Scans, at the discretion of the PI.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| pts with intermediate- and high-risk rhabdomyosarcoma | Experimental | Biological: filgrastim, Drug: carboplatin, Drug: cyclophosphamide, Drug: dexrazoxane hydrochloride, Drug: doxorubicin hydrochloride, Drug: etoposide, Drug: ifosfamide, Drug: irinotecan hydrochloride, Drug: vincristine sulfate, Procedure: conventional surgery, Radiation: radiation therapy |
Who may qualify
- Ages
- Up to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (21 listed)
- Newly diagnosed, previously untreated histologically-proven rhabdomyosarcoma, undifferentiated sarcoma, or ectomesenchymoma. Histology must be confirmed by a MSKCC pathologist.
- All patients with Stage 4 tumors (distant metastases).
- All patients with non-metastatic undifferentiated sarcoma or alveolar RMS or ectomesenchymoma with alveolar features (regardless of age, site, size, stage, or degree of initial surgical resection);
- All patients < 1 year of age with non-metastatic embryonal RMS or ectomesenchymoma with embryonal features (regardless of site, stage, or degree of initial surgical resection).
- Patients ≥ 1 year of age with Stage 2 or 3 (unfavorable site [see Appendix I] and either size > 5 cm, OR regional nodes positive, or both), Group III (gross residual disease post-biopsy or attempted resection) embryonal RMS or ectomesenchymoma with embryonal features
Key exclusion criteria (0 listed)
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 |
|---|---|---|
| filgrastim | Biological | pts with intermediate- and high-risk rhabdomyosarcoma |
| carboplatin | Drug | pts with intermediate- and high-risk rhabdomyosarcoma |
| cyclophosphamide | Drug | pts with intermediate- and high-risk rhabdomyosarcoma |
| dexrazoxane hydrochloride | Drug | pts with intermediate- and high-risk rhabdomyosarcoma |
| doxorubicin hydrochloride | Drug | pts with intermediate- and high-risk rhabdomyosarcoma |
| etoposide | Drug | pts with intermediate- and high-risk rhabdomyosarcoma |
| ifosfamide | Drug | pts with intermediate- and high-risk rhabdomyosarcoma |
| irinotecan hydrochloride | Drug | pts with intermediate- and high-risk rhabdomyosarcoma |
| vincristine sulfate | Drug | pts with intermediate- and high-risk rhabdomyosarcoma |
| conventional surgery | Procedure or surgery | pts with intermediate- and high-risk rhabdomyosarcoma |
| radiation therapy | Radiation | pts with intermediate- and high-risk rhabdomyosarcoma |
Primary outcome
Response rate
Time frame: 2 years
Toxicity
Time frame: 2 years
Safety and feasibility
Time frame: 2 years
Rate of local control
Time frame: 2 years
2 secondary outcomes
Correlation of in vitro measurements of angiogenesis with clinical features (extent of disease), response to therapy, and outcome
Time frame: 2 years
Efficacy in terms of improved outcomes
Time frame: 2 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 May 2026.
- Memorial Sloan Kettering Cancer CenterLink provided by the sponsor.
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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.
Leonard H. Wexler, MD principal investigator
Memorial Sloan Kettering Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing filgrastim, carboplatin and cyclophosphamide in people with Sarcoma, run by Memorial Sloan Kettering Cancer Center at 1 site in United States.
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 21 inclusion and 0 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: under way, not looking for participants, as of the sponsor's last update on 22 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. 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 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?
This study is still under way. Results are not available in the source record yet.
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?
Response rate (time frame: 2 years). The study lists 4 primary outcomes in all. 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 22 May 2026.