Newly Diagnosed Ewing SarcomaPhase 2NCT01864109
Irinotecan and Temozolomide in Combination With Existing High Dose Alkylator Based Chemotherapy for Treatment of Patients With Newly Diagnosed Ewing Sarcoma
A Phase 2 study testing Cyclophosphamide, Doxorubicin and Vincristine in people with Newly Diagnosed Ewing Sarcoma, run by Memorial Sloan Kettering Cancer Center at 7 sites in United States.
Trial summary
The purpose of this study is to find out what effects, good and/or bad, the combination of irinotecan and temozolomide has on Ewing sarcoma. Irinotecan and temozolomide are chemotherapy drugs that are used very often to treat pediatric patients at MSKCC. Read more about this study
- Who is it for?
- Ages 1 year to 40 years
- Conditions the study is for
- Newly Diagnosed Ewing Sarcoma
- What is being tested?
- Cyclophosphamide, Doxorubicin and Vincristine
- Type of treatment
- Pill or oral medicine, Injection or infusion, Medical device, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 83 participants
- Where
- 7 sitesUnited States
- Study dates
- May 2013to May 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 1 year to 40 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Age greater than or equal to one year and less than or equal to 40 years at the time of diagnosis
- Newly diagnosed, previously untreated patients with histologically or molecularly confirmed Ewing sarcoma
- Adequate hematologic function:
- Absolute neutrophil count ≥ 1,000/K/mcl
- Platelet count ≥ 100,000/Kmcl
- Adequate renal function:
Show all 17Show fewer
- Normal creatinine for age (See table below) OR
- Creatinine clearance or radioisotope GFR ≥ 70ml/min/1.73 m2 Age(Years) Maximum Serum Creatinine (mg/dL) ≤ 5 0.8 6 to ≤ 10 1 11 to ≤ 15 1.2 ≥ 16 1.5
- Adequate hepatic function:
- Total bilirubin ≤ 1.5 x the ULN
- AST ≤ 2.5 x the ULN [in the absence of hepatic involvement of tumor. Patients with hepatic involvement are considered eligibile for study]
- ALT ≤ 2.5 x the ULN [in the absence of hepatic involvement of tumor. Patients with hepatic involvement are considered eligibile for study]
- Normal cardiac function:
- Shortening fraction ≥ 28% by echocardiogram OR
- Left ventricular ejection fraction (LVEF) ≥ 50% on technetium- 99m pertechnetate radionuclide cineangiography (MUGA) or echocardiogram
- Patients must consent to an indwelling central venous catheter.
- Sexually active patients of reproductive potential must be willing to use an effective method of contraception.
Who cannot join
- Prior chemotherapy or radiotherapy (other than limited, emergent radiotherapy for treatment of eg. spinal cord compromise or threatened airway)
- Pregnant or breastfeeding females
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
United States7 sites
Memorial Sloan Kettering Cancer Center Basking Ridge
Basking Ridge, New Jersey, 07920
Memorial Sloan Kettering Commack
Commack, New York, 11725
Memorial Sloan Kettering Westchester
Harrison, New York, 10604
Memorial Sloan Kettering Monmouth
Middletown, New Jersey, 07748
Memorial Sloan Kettering Bergen
Montvale, New Jersey, 07645
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
Memorial Sloan Kettering Nassau
Uniondale, New York, 11553
About this study
Official title: A Phase II Trial of Irinotecan and Temozolomide in Combination With Existing High Dose Alkylator Based Chemotherapy for Treatment of Patients With Newly Diagnosed Ewing Sarcoma
The purpose of this study is to find out what effects, good and/or bad, the combination of irinotecan and temozolomide has on Ewing sarcoma.
Irinotecan and temozolomide are chemotherapy drugs that are used very often to treat pediatric patients at MSKCC. The investigators have used these two drugs for many years to treat patients with Ewing sarcoma whose cancer has relapsed.
Read more
For patients with newly diagnosed Ewing sarcoma the current standard of care at MSKCC is a five drug chemotherapy regimen in combination with surgery and/or radiation therapy. This standard regimen is called the EFT regimen. . Some patients with Ewing sarcoma do not have their cancer cured by the chemotherapy and surgery/radiation therapy.
This study adds the chemotherapy drugs called irinotecan and temozolomide to the standard EFT regimen. The investigators are trying to improve the success of standard therapy by adding these drugs. The use of irinotecan and temozolomide in this study is experimental because they have not been used before in patients with newly diagnosed Ewing sarcoma. However the investigators have found these drugs to be effective in patients with relapsed Ewing sarcoma. It is not known if adding these two drugs will improve the outcomes of patients treated for Ewing sarcoma.
- Conditions
- Newly Diagnosed Ewing Sarcoma
- Being tested
- Cyclophosphamide, Doxorubicin, Vincristine, Ifosfamide, Etoposide
- Keywords
- CYCLOPHOSPHAMIDE (CYTOXAN), DOXORUBICIN/ADRIAMYCIN, ETOPOSIDE (VP-16), IFOSFAMIDE, IRINOTECAN (CPT-11) CAMPTOSAR, TEMOZOLOMIDE, VINCRISTINE, 13-068
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CyclophosphamideDrug
DoxorubicinDevice
VincristineDrug
IfosfamideDrug
EtoposideDrug
SurgeryProcedure or surgery
Radiation Therapy*Radiation
If local control includes RT, RT should be given concurrently with chemotherapy cycles
TemozolomideDrug
IrinotecanDrug
MesnaDrug
Mesna 2,100 mg/m2 (or 70 mg/kg if < 10 yrs of age) administered intravenously in concordance with institutional pediatric administration guidelines.
DexrazoxaneDrug
Dexrazoxane 375 mg/m2 intravenously over approximately 15-30 minutes and as clinically indicated. To be administered immediately prior to doxorubicin.
G-CSFDrug
G-CSF-to be administered after the completion of appropriate chemotherapy cycles as per institutional guidelines.
| Study group | Role | What they receive |
|---|---|---|
| Patients with localized disease | Experimental | Patients with localized disease will receive six cycles of the combination as "maintenance" therapy following standard chemotherapy.
* Cycles 4-6 will include:
* Ifosfamide 2,800 mg/m2/day on days 1-5
* Etoposide 100 mg/m2/day on days 1-5
* Cycle 7 will include :
* Cyclophosphamide will be given on days 1 and 2 at a dose of 2,100 mg/m2/day, or for patients < 10 years of age at a dose of 70 mg/kg/day
* Doxorubicin will be given on days 1 and 2 at a dose of 37.5 mg/m2/day
* Vincristine will be given on day 1 at a dose of 2 mg/m2 or 0.067 mg/kg (whichever is lower, to a max dose of 2 mg)
* Cycles 8-13 will include:
* Irinotecan will be given on 10 days over weeks 1 and 2 of a cycle at a dose of 20 mg/m2/day intravenously
* Temozolomide will be given daily on the first 5 days of irinotecan administration at a dose of 100 mg/m2/day orally or intravenously Receives: Drug: Cyclophosphamide; Device: Doxorubicin; Drug: Vincristine; Drug: Ifosfamide; Drug: Etoposide; Procedure: Surgery; Radiation: Radiation Therapy*; Drug: Temozolomide; Drug: Irinotecan; Drug: Mesna; Drug: Dexrazoxane; Drug: G-CSF |
| Patients with metastatic disease | Experimental | Patients will get 10 cycles of the combination intercalated between the final 4 cycles of standard chemotherapy.
* Cycles 4, 5, 7, 8, 10, 11, 13, 14, 16, and 17 will include:
* Irinotecan will be given on 10 days over weeks 1 and 2 of a cycle at a dose of 20 mg/m2/day intravenously
* Temozolomide will be given daily on the first 5 days of irinotecan administration at a dose of 100 mg/m2/day orally or intravenously
* Cycles 6, 9, and 12 will include:
* Ifosfamide 2,800 mg/m2/day on days 1-5
* Etoposide 100 mg/m2/day on days 1-5
* Cycle 15 will include:
* Cyclophosphamide will be given on days 1 and 2 at a dose of 2,100 mg/m2/day, or for patients < 10 years of age at a dose of 70 mg/kg/day
* Doxorubicin will be given on days 1 and 2 at a dose of 37.5 mg/m2/day
* Vincristine will be given on day 1 at a dose of 2 mg/m2 or 0.067 mg/kg (whichever is lower, to a max dose of 2 mg) Receives: Drug: Cyclophosphamide; Device: Doxorubicin; Drug: Vincristine; Drug: Ifosfamide; Drug: Etoposide; Procedure: Surgery; Radiation: Radiation Therapy*; Drug: Temozolomide; Drug: Irinotecan; Drug: Mesna; Drug: Dexrazoxane; Drug: G-CSF |
What is being measured
The primary outcome is the main question the study is designed to answer.
event free survival of patients with localized disease
We will determine event free survival from the time of study entry for all patients. An event would include death from any cause, progression of tumor, recurrence of tumor, or second malignancy.
Progressive disease (PD) will be defined according to RECIST 1.1.
Measured: 4 years
Secondary outcomes (2)
event free survival of patients with metastatic disease
We will determine event free survival from the time of study entry for all patients. An event would include death from any cause, progression of tumor, recurrence of tumor, or second malignancy.
Progressive disease (PD) will be defined according to RECIST 1.1.
Measured: 4 years
adverse event profile
Toxicities are graded by the Common Toxicity Criteria (Version 4.0) developed by the National Cancer Institute (NCI) of the USA.
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 posted29 May 2013 (estimated)
- Study startMay 2013
- Primary completionMay 2027 (estimated)
- Study completionMay 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Newly Diagnosed Ewing 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
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 83 participants (actual)
Sponsor and investigators
- Lead sponsor
- Memorial Sloan Kettering Cancer Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Emily Slotkin, MD, Principal investigator, Memorial Sloan Kettering Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01864109
- Sponsor's protocol ID
- 13-068
- Status last verified
- Apr 2026
- First submitted
- 23 May 2013
- Last updated
- 17 Apr 2026
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Trial of Irinotecan and Temozolomide in Combination With Existing High Dose Alkylator Based Chemotherapy for Treatment of Patients With Newly Diagnosed Ewing Sarcoma
- ClinicalTrials.gov ID
- NCT01864109
- Acronym / sponsor ID
- 13-068
- Conditions
- Newly Diagnosed Ewing Sarcoma
- Keywords
- CYCLOPHOSPHAMIDE (CYTOXAN), DOXORUBICIN/ADRIAMYCIN, ETOPOSIDE (VP-16), IFOSFAMIDE, IRINOTECAN (CPT-11) CAMPTOSAR, TEMOZOLOMIDE, VINCRISTINE, 13-068
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 83 participants (actual)
- Lead sponsor
- Memorial Sloan Kettering Cancer CenterUniversity, hospital or other
- Study start
- May 2013
- Primary completion
- May 2027 (estimated)
- Study completion
- May 2027 (estimated)
- First posted
- 29 May 2013 (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.
The purpose of this study is to find out what effects, good and/or bad, the combination of irinotecan and temozolomide has on Ewing sarcoma.
Irinotecan and temozolomide are chemotherapy drugs that are used very often to treat pediatric patients at MSKCC. The investigators have used these two drugs for many years to treat patients with Ewing sarcoma whose cancer has relapsed.
For patients with newly diagnosed Ewing sarcoma the current standard of care at MSKCC is a five drug chemotherapy regimen in combination with surgery and/or radiation therapy. This standard regimen is called the EFT regimen. . Some patients with Ewing sarcoma do not have their cancer cured by the chemotherapy and surgery/radiation therapy.
This study adds the chemotherapy drugs called irinotecan and temozolomide to the standard EFT regimen. The investigators are trying to improve the success of standard therapy by adding these drugs. The use of irinotecan and temozolomide in this study is experimental because they have not been used before in patients with newly diagnosed Ewing sarcoma. However the investigators have found these drugs to be effective in patients with relapsed Ewing sarcoma. It is not known if adding these two drugs will improve the outcomes of patients treated for Ewing sarcoma.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Patients with localized disease | Experimental | Drug: Cyclophosphamide, Device: Doxorubicin, Drug: Vincristine, Drug: Ifosfamide, Drug: Etoposide, Procedure: Surgery, Radiation: Radiation Therapy*, Drug: Temozolomide, Drug: Irinotecan, Drug: Mesna, Drug: Dexrazoxane, Drug: G-CSFPatients with localized disease will receive six cycles of the combination as "maintenance" therapy following standard chemotherapy. * Cycles 4-6 will include: * Ifosfamide 2,800 mg/m2/day on days 1-5 * Etoposide 100 mg/m2/day on days 1-5 * Cycle 7 will include : * Cyclophosphamide will be given on days 1 and 2 at a dose of 2,100 mg/m2/day, or for patients < 10 years of age at a dose of 70 mg/kg/day * Doxorubicin will be given on days 1 and 2 at a dose of 37.5 mg/m2/day * Vincristine will be given on day 1 at a dose of 2 mg/m2 or 0.067 mg/kg (whichever is lower, to a max dose of 2 mg) * Cycles 8-13 will include: * Irinotecan will be given on 10 days over weeks 1 and 2 of a cycle at a dose of 20 mg/m2/day intravenously * Temozolomide will be given daily on the first 5 days of irinotecan administration at a dose of 100 mg/m2/day orally or intravenously |
| Patients with metastatic disease | Experimental | Drug: Cyclophosphamide, Device: Doxorubicin, Drug: Vincristine, Drug: Ifosfamide, Drug: Etoposide, Procedure: Surgery, Radiation: Radiation Therapy*, Drug: Temozolomide, Drug: Irinotecan, Drug: Mesna, Drug: Dexrazoxane, Drug: G-CSFPatients will get 10 cycles of the combination intercalated between the final 4 cycles of standard chemotherapy. * Cycles 4, 5, 7, 8, 10, 11, 13, 14, 16, and 17 will include: * Irinotecan will be given on 10 days over weeks 1 and 2 of a cycle at a dose of 20 mg/m2/day intravenously * Temozolomide will be given daily on the first 5 days of irinotecan administration at a dose of 100 mg/m2/day orally or intravenously * Cycles 6, 9, and 12 will include: * Ifosfamide 2,800 mg/m2/day on days 1-5 * Etoposide 100 mg/m2/day on days 1-5 * Cycle 15 will include: * Cyclophosphamide will be given on days 1 and 2 at a dose of 2,100 mg/m2/day, or for patients < 10 years of age at a dose of 70 mg/kg/day * Doxorubicin will be given on days 1 and 2 at a dose of 37.5 mg/m2/day * Vincristine will be given on day 1 at a dose of 2 mg/m2 or 0.067 mg/kg (whichever is lower, to a max dose of 2 mg) |
Who may qualify
- Ages
- 1 year to 40 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (17 listed)
- Age greater than or equal to one year and less than or equal to 40 years at the time of diagnosis
- Newly diagnosed, previously untreated patients with histologically or molecularly confirmed Ewing sarcoma
- Adequate hematologic function:
- Absolute neutrophil count ≥ 1,000/K/mcl
- Platelet count ≥ 100,000/Kmcl
Key exclusion criteria (2 listed)
- Prior chemotherapy or radiotherapy (other than limited, emergent radiotherapy for treatment of eg. spinal cord compromise or threatened airway)
- Pregnant or breastfeeding females
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 |
|---|---|---|
| Cyclophosphamide | Drug | Patients with localized disease, Patients with metastatic disease |
| Doxorubicin | Device | Patients with localized disease, Patients with metastatic disease |
| Vincristine | Drug | Patients with localized disease, Patients with metastatic disease |
| Ifosfamide | Drug | Patients with localized disease, Patients with metastatic disease |
| Etoposide | Drug | Patients with localized disease, Patients with metastatic disease |
| Surgery | Procedure or surgery | Patients with localized disease, Patients with metastatic disease |
| Radiation Therapy*If local control includes RT, RT should be given concurrently with chemotherapy cycles | Radiation | Patients with localized disease, Patients with metastatic disease |
| Temozolomide | Drug | Patients with localized disease, Patients with metastatic disease |
| Irinotecan | Drug | Patients with localized disease, Patients with metastatic disease |
| MesnaMesna 2,100 mg/m2 (or 70 mg/kg if < 10 yrs of age) administered intravenously in concordance with institutional pediatric administration guidelines. | Drug | Patients with localized disease, Patients with metastatic disease |
| DexrazoxaneDexrazoxane 375 mg/m2 intravenously over approximately 15-30 minutes and as clinically indicated. To be administered immediately prior to doxorubicin. | Drug | Patients with localized disease, Patients with metastatic disease |
| G-CSFG-CSF-to be administered after the completion of appropriate chemotherapy cycles as per institutional guidelines. | Drug | Patients with localized disease, Patients with metastatic disease |
Primary outcome
event free survival of patients with localized disease
Time frame: 4 years
We will determine event free survival from the time of study entry for all patients. An event would include death from any cause, progression of tumor, recurrence of tumor, or second malignancy. Progressive disease (PD) will be defined according to RECIST 1.1.
2 secondary outcomes
event free survival of patients with metastatic disease
Time frame: 4 years
adverse event profile
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 17 Apr 2026.
- Memorial Sloan-Kettering Cancer CenterLink provided by the sponsor.
Related trials for Newly Diagnosed Ewing Sarcoma
Active, not recruitingPhase 3
Combination Chemotherapy With or Without Ganitumab in Treating Patients With Newly Diagnosed Metastatic Ewing Sarcoma
Metastatic Ewing Sarcoma, Metastatic Malignant Neoplasm in the Bone
Active, not recruitingPhase 2
Cabozantinib-S-Malate in Treating Younger Patients With Recurrent, Refractory, or Newly Diagnosed Sarcomas, Wilms Tumor, or Other Rare Tumors
Adrenal Cortical Carcinoma, Alveolar Soft Part Sarcoma
Active, not recruitingPhase 1
Phase Ib / Regorafenib With Conventional Chemotherapy/Newly Diagnosed Patients/ Multimetastatic Ewing Sarcoma
Ewing Sarcoma
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.
Emily Slotkin, MD principal investigator
Memorial Sloan Kettering Cancer Center
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Cyclophosphamide, Doxorubicin and Vincristine in people with Newly Diagnosed Ewing Sarcoma, run by Memorial Sloan Kettering Cancer Center at 7 sites 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 17 inclusion and 2 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 17 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites 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?
event free survival of patients with localized disease (time frame: 4 years). 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 17 Apr 2026.