A Phase 2 interventional study of vincristine and doxorubicin in Desmoplastic Small Round Cell Tumor, Ewing Sarcoma of Bone or Soft Tissue and Localized Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, sponsored by St. Jude Children's Research Hospital. Completed at 1 site in United States. Open to participants aged Up to 25 Years. Per ClinicalTrials.gov, last updated 2026-08-21.
Sponsored by St. Jude Children's Research Hospital · Phase 2, Interventional, and Treatment
This protocol will study treatment for Ewing sarcoma family of tumors (ESFT) and desmoplastic small round cell tumor (DSRCT). Participants with ESFT will be divided into two treatment groups, A or B, based on tumor characteristics.
Group A (standard risk) participants have tumor that is not in the pelvis, has not spread to other parts of the body, and are less than 14 years of age. Because previous clinical trials have shown that standard treatment is very effective for children whose tumors have these characteristics, these participants will receive standard treatment.
Group B (high risk) participants are 14 years of age or older or have tumor in the pelvis, or the tumor has spread to other parts of the body. Participants with DSRCT in the abdomen and/or pelvis or with tumor that cannot be removed by surgery alone or has spread to other parts of the body will be included in Group B. Participants in this group are considered high risk because there is a greater chance of tumor recurring following standard treatments currently in use.
All participants will be followed and evaluated for 10 years following completion of therapy.
PRIMARY OBJECTIVE:
SECONDARY OBJECTIVES:
Group A:
Participants will receive interval compressed (every 2 weeks) alternating courses of chemotherapy with vincristine, doxorubicin, and cyclophosphamide (VDC) and with ifosfamide and etoposide (IE). Doxorubicin will be omitted following a total cumulative dose of 375 mg/m\^2. Local control measures (surgery and/or radiation therapy) will be instituted after 6 courses of chemotherapy. Total duration of treatment is approximately 29 weeks.
Group B:
Participants eligible for the window therapy will receive two courses (21 days duration each) of mTOR inhibitor, temsirolimus, in combination with temozolomide and irinotecan. Irinotecan (20 mg/m\^2) will be administered IV on a protracted schedule of daily for 5 days, 2 days off, repeated daily x 5 [(qdx5)x2], temozolomide (100 mg/m\^2) PO daily x 5 days and temsirolimus 35 mg/m\^2 IV weekly on day 1 and 8. Following window treatment (weeks 1 - 6), participants will proceed to induction chemotherapy (weeks 7 - 33) consisting of interval compressed (every 2 weeks) alternating courses of chemotherapy with vincristine, doxorubicin, and cyclophosphamide (VDC) with ifosfamide and etoposide (IE). Doxorubicin will be omitted following a total cumulative dose of 375 mg/m\^2. Local control measures (surgery and/or radiation therapy) will be instituted after 6 courses of induction chemotherapy (week 19). Participants whose tumors respond to window therapy will receive temsirolimus, temozolomide and irinotecan at weeks 29 and 31 in place of ifosfamide and etoposide. Following induction therapy, participants will receive six 21-day courses of maintenance therapy consisting of bevacizumab IV on day 1 and daily oral sorafenib and low-dose cyclophosphamide day 1 through day 21.
59 studies on the registry are indexed under Desmoplastic Small Round Cell Tumor; 14 are open to participants now.
This study's enrollment of 24 is below the median of 44 across 47 interventional studies indexed under Desmoplastic Small Round Cell Tumor.
Browse Desmoplastic Small Round Cell Tumor studies →St. Jude Children's Research Hospital is the lead sponsor of 440 studies on the registry; 102 are open to participants now.
Of its 61 completed or terminated interventional studies of FDA-regulated products, 37 (61%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Additional criteria for Group B participants who will receive upfront window therapy (does not apply to participants who opt out of window therapy):
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The diagnosis was confirmed by looking at a sample under a microscope: tissue from a biopsy or surgery (histology), or cells from a fluid or needle sample (cytology).
Your pathology report is the document that shows this.
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
CYP3A is a group of enzymes, mainly CYP3A4, in the liver and gut that break down many medicines. Some medicines, supplements and foods (grapefruit, St John’s wort) slow them down or speed them up, which changes how much of a study drug stays in the body.
Studies list them so they can be avoided or switched; bring a list of everything you take, including supplements.
The amount of disease can be tracked with a defined measurement, so the study can see whether it shrinks or grows. It might be a tumour measured on a scan or, in some blood cancers such as myeloma, a marker measured in blood or urine.
The criteria or the study team say which measurement counts — having no tumour visible on a scan does not by itself rule you out.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Histologically or cytologically confirmed
The diagnosis was confirmed by looking at a sample under a microscope: tissue from a biopsy or surgery (histology), or cells from a fluid or needle sample (cytology).
Your pathology report is the document that shows this.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Metastases
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Absolute neutrophil count (ANC)
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
CYP3A (CYP3A4) inhibitors and inducers
CYP3A is a group of enzymes, mainly CYP3A4, in the liver and gut that break down many medicines. Some medicines, supplements and foods (grapefruit, St John’s wort) slow them down or speed them up, which changes how much of a study drug stays in the body.
Studies list them so they can be avoided or switched; bring a list of everything you take, including supplements.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Measurable disease
The amount of disease can be tracked with a defined measurement, so the study can see whether it shrinks or grows. It might be a tumour measured on a scan or, in some blood cancers such as myeloma, a marker measured in blood or urine.
The criteria or the study team say which measurement counts — having no tumour visible on a scan does not by itself rule you out.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants will receive vincristine, doxorubicin, cyclophosphamide, ifosfamide and etoposide. Doxorubicin will be omitted following a total cumulative dose of 375 mg/m\^2. Depending on the size and location of the participant's tumor, they will have surgery alone, radiation alone, or surgery followed by radiation. Local control measures (surgery and/or radiation therapy) will be instituted after 6 courses of chemotherapy. Total duration of treatment is approximately 29 weeks.
Drug: vincristine · Drug: doxorubicin · Drug: cyclophosphamide · Drug: ifosfamide · Drug: etoposide · Procedure: surgery · Radiation: radiation
Participants will receive vincristine, doxorubicin, cyclophosphamide, ifosfamide, etoposide, irinotecan, temozolomide, temsirolimus, bevacizumab, and sorafenib. Depending on the size and location of the participant's tumor, they will have surgery alone, radiation alone or surgery followed by radiation.
Drug: doxorubicin · Drug: cyclophosphamide · Drug: ifosfamide · Drug: etoposide · Drug: temozolomide · Drug: temsirolimus · Drug: bevacizumab · Drug: sorafenib · Procedure: surgery · Radiation: radiation
Dosage and route of administration: Infants \< 12 months of age: 0.05 mg/kg IV day 1; participants ≥ 12 months of age: 1.5 mg/m\^2 IV day 1 (max. dose 2 mg).
Also known as: Oncovin(R)
Dosage and route of administration: Infants \< 1 year 2.5 mg/kg continuous infusion (CI) over 48 hours, days 1-2; participants \> 1 year of age 75 mg/m\^2 CI over 48 hours, days 1-2.
Also known as: Adriamycin(R)
Dosage and route of administration: The dose and route are different in neo-adjuvant/adjuvant chemotherapy and maintenance therapy. Please see the Detailed Description for further information.
Also known as: Cytoxan(R)
Dosage and route of administration: Infants \< 1 year of age 60 mg/kg/day IV over 60 minutes days 1-5; participants \> 12 months of age 1800 mg/m\^2 IV over 60 minutes x 5 days, days 1-5.
Also known as: Ifex(R)
Dosage and route of administration: Infants \< 1 year of age 3.3 mg/kg/day IV over 60 minutes days 1-5; children \> 1 year 100 mg/m\^2 daily IV over 60 minutes days 1-5.
Also known as: VP-16, Vepesid(R)
Dosage and route of administration: Temozolomide 100 mg/m\^2 PO once daily, days 1-5.
Also known as: Temodar(R)
Dosage and route of administration: Temsirolimus 35 mg/m\^2 IV once day 1 and day 8.
Also known as: CCI-779, Torisel^TM
Dosage and route of administration: Bevacizumab 15 mg/kg IV on day 1 every 3 weeks.
Also known as: rhumab VEGF, Avastin(R)
Dosage and route of administration: 90 mg/m\^2/dose PO BID
Also known as: BAY-43-9006, Nexavar(R)
If participant meets the criteria, they will have surgical resection of their tumor.
Also known as: therapeutic conventional surgery
If the participant meets the criteria, participants will receive radiation therapy. Chemotherapy will continue during radiation.
Also known as: proton beam radiation therapy, external beam radiation therapy, brachytherapy
Response to Window Therapy (2 Courses) for Group B (High-risk) - ESFT Participants
Response rate will be defined as the proportion of patients who achieved complete response or partial response (CR+PR) using the World Health Organization (WHO) criteria evaluated after two initial courses of temsirolimus, temozolomide and irinotecan in previously untreated patients with high risk Ewing Sarcoma Family of Tumor (ESFT). Participants who are treated in Group B with Desmoplastic Small Round Cell Tumor (DSRCT) or those who do not receive window therapy will not be included in this analysis.
Time frame: at 6 weeks after start of therapy (after 2 initial courses)
Overall Survival
Overall survival (OS) is defined as the time interval from the date on study to the date of death or the date of last follow-up. OS will be estimated using the method of Kaplan-Meier for group A and B participants, respectively.
Time frame: Maximum of 11 years after the start of therapy
Progression-free Survival
Progression free survival (PFS) is defined as the time interval from the date on study to the date of disease progression or death or the date if last follow-up. PFS will be estimated using the method of Kaplan-Meier for group A and B participants, respectively.
Time frame: Maximum of 11 years after the start of therapy
Time to Progression
Median time to progression of group B patients will be estimated from the Kaplan-Meier curve.
Time frame: Maximum of 11 years after the start of therapy
Local Failure Rate
Loco-regional failure is defined as the time interval from date of start of local therapy to date of loco-regional failure. Distant failure or death prior to loco-regional failure will be considered competing events in the analyses. The cumulative incidence of loco-regional failure will be estimated using methods described in Kalbfleisch and Prentice.
Time frame: Maximum of 11 years after the start of therapy
Participants were enrolled at St. Jude Children's Research Hospital between December 2013 and June 2015.
| Milestone | Group A (Standard Risk) | Group B (High Risk) - ESFT | Group B (High Risk) - DSRCT |
|---|---|---|---|
| Started | 1 | 17 | 6 |
| Completed | 1 | 16 | 6 |
| Not completed | 0 | 1 | 0 |
| Withdrew: Physician decision | 0 | 1 | 0 |
| Milestone | Group A (Standard Risk) | Group B (High Risk) - ESFT | Group B (High Risk) - DSRCT |
|---|---|---|---|
| Started | 1 | 16 | 6 |
| Completed | 0 | 0 | 0 |
| Not completed | 1 | 16 | 6 |
| Withdrew: Some pts. still receiving study therapy | 1 | 16 | 6 |
Response rate will be defined as the proportion of patients who achieved complete response or partial response (CR+PR) using the World Health Organization (WHO) criteria evaluated after two initial courses of temsirolimus, temozolomide and irinotecan in previously untreated patients with high risk Ewing Sarcoma Family of Tumor (ESFT). Participants who are treated in Group B with Desmoplastic Small Round Cell Tumor (DSRCT) or those who do not receive window therapy will not be included in this analysis.
| participants | Group B (High Risk) - ESFT |
|---|---|
| Partial Response (PR) | 3 |
| Stable disease (no response) (NR) | 8 |
| Progressive Disease (PD) | 1 |
Overall survival (OS) is defined as the time interval from the date on study to the date of death or the date of last follow-up. OS will be estimated using the method of Kaplan-Meier for group A and B participants, respectively.
Results for this outcome have not been posted.
Progression free survival (PFS) is defined as the time interval from the date on study to the date of disease progression or death or the date if last follow-up. PFS will be estimated using the method of Kaplan-Meier for group A and B participants, respectively.
Results for this outcome have not been posted.
Median time to progression of group B patients will be estimated from the Kaplan-Meier curve.
Results for this outcome have not been posted.
Loco-regional failure is defined as the time interval from date of start of local therapy to date of loco-regional failure. Distant failure or death prior to loco-regional failure will be considered competing events in the analyses. The cumulative incidence of loco-regional failure will be estimated using methods described in Kalbfleisch and Prentice.
Results for this outcome have not been posted.
Collected over Adverse events were collected from the on-study date through 05/03/2016.. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Group A (Standard Risk) | — | 0/1 (0%) | 1/1 (100%) |
| Group B (High Risk) - ESFT | — | 1/16 (6.3%) | 16/16 (100%) |
| Group B (High Risk) - DSRCT | — | 0/6 (0%) | 6/6 (100%) |
| Group B (High Risk) - Total | — | 1/22 (4.5%) | 22/22 (100%) |
| Event | Group A (Standard Risk) | Group B (High Risk) - ESFT | Group B (High Risk) - DSRCT | Group B (High Risk) - Total |
|---|---|---|---|---|
| DiarrheaGastrointestinal disorders | 0/1 | 1/16 | 0/6 | 1/22 |
| Event | Group A (Standard Risk) | Group B (High Risk) - ESFT | Group B (High Risk) - DSRCT | Group B (High Risk) - Total |
|---|---|---|---|---|
| Sinus tachycardiaCardiac disorders | 1/1 | 15/16 | 6/6 | 21/22 |
| Lymphocyte count decreasedInvestigations | 1/1 | 15/16 | 6/6 | 21/22 |
| Neutrophil count decreasedInvestigations | 1/1 | 15/16 | 6/6 | 21/22 |
| White blood cell decreasedInvestigations | 1/1 | 15/16 | 6/6 | 21/22 |
| HypertensionVascular disorders | 1/1 | 11/16 | 5/6 | 16/22 |
| Febrile neutropeniaBlood and lymphatic system disorders | 1/1 | 9/16 | 3/6 | 12/22 |
| AnemiaBlood and lymphatic system disorders | 1/1 | 16/16 | 6/6 | 22/22 |
| Wound infectionInfections and infestations | 1/1 | 0/16 | 1/6 | 1/22 |
| DiarrheaGastrointestinal disorders | 0/1 | 15/16 | 6/6 | 21/22 |
| Weight lossInvestigations | 0/1 | 13/16 | 5/6 | 18/22 |
| Age, Continuous(years) | Group A (Standard Risk) | Group B (High Risk) - ESFT | Group B (High Risk) - DSRCT | Total |
|---|---|---|---|---|
| Mean | 12.0 ± 0 | 13.0 ± 5.7 | 14.7 ± 4.1 | 13.4 ± 5.2 |
| Sex: Female, Male(Participants) | Group A (Standard Risk) | Group B (High Risk) - ESFT | Group B (High Risk) - DSRCT | Total |
|---|---|---|---|---|
| Female | 0 | 9 | 1 | 10 |
| Male | 1 | 7 | 5 | 13 |
| Race (NIH/OMB)(Participants) | Group A (Standard Risk) | Group B (High Risk) - ESFT | Group B (High Risk) - DSRCT | Total |
|---|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 | 0 |
| Asian | 0 | 1 | 0 | 1 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 | 0 |
| Black or African American | 0 | 0 | 2 | 2 |
| White | 1 | 14 | 3 | 18 |
| More than one race | 0 | 0 | 0 | 0 |
| Unknown or Not Reported | 0 | 1 | 1 | 2 |
| Primary disease site(participants) | Group A (Standard Risk) | Group B (High Risk) - ESFT | Group B (High Risk) - DSRCT | Total |
|---|---|---|---|---|
| Pelvis | 0 | 6 | 5 | 11 |
| Other | 1 | 10 | 1 | 12 |
| Disease stage(participants) | Group A (Standard Risk) | Group B (High Risk) - ESFT | Group B (High Risk) - DSRCT | Total |
|---|---|---|---|---|
| Metastases | 0 | 11 | 5 | 16 |
| Local | 1 | 5 | 1 | 7 |
Ran from Dec 2013 to primary completion in Aug 2015, 20 months.
Results were first posted in Oct 2016, 1 year 2 months after primary completion.
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This study is completed, as verified in Jul 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drugs tested here: Temozolomide · Temsirolimus · Bevacizumab · Sorafenib
Desmoplastic Small Round Cell Tumor →
St. Jude Children's Research Hospital →