Desmoplastic Small Round Cell Tumor Clinical Trial
Official title:
Therapeutic Trial for Patients With Ewing Sarcoma Family of Tumor and Desmoplastic Small Round Cell Tumors
Verified date | July 2023 |
Source | St. Jude Children's Research Hospital |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
Status | Active, not recruiting |
Enrollment | 24 |
Est. completion date | July 2026 |
Est. primary completion date | August 2015 |
Accepts healthy volunteers | No |
Gender | All |
Age group | N/A to 25 Years |
Eligibility | Inclusion Criteria: - Group A participants must be <14 years of age at time of diagnosis of histologically proven non-pelvic localized Ewing sarcoma family of tumor (ESFT) involving the bone or soft tissue. - Group B participants must have newly diagnosed of histologically proven ESFT involving the bone or soft tissue and at least one of the following: metastatic disease (must be biopsy proven), or pelvic primary, or =14 years of age at the time of diagnosis. - OR Group B participants must be newly diagnosed with intra-abdominal, unresectable or metastatic desmoplastic small round cell tumor. Metastatic site must be biopsy proven. - Age must be =25 years. - Adequate bone marrow function defined as a peripheral absolute neutrophil count (ANC) =750/m^3 and platelet count =75,000/m^3 (no transfusion within 7 days of study enrollment). Patients with Ewing sarcoma metastatic to the bone marrow are not required to meet bone marrow criteria for study eligibility and are not evaluable for hematologic toxicity. - Must have adequate renal function based on age. - Must not have had prior chemotherapy or radiation therapy. Emergent radiotherapy to preserve vital organ function is permitted. Participants who receive emergent radiation will not be eligible for window therapy. - Must have adequate hepatic function defined as total bilirubin =3.0 mg/dL. - Must have adequate cardiac function defined as shortening fraction =28%. - Females of childbearing potential and males able to father a child must be willing to practice acceptable methods of birth control to prevent pregnancy. - Additional criteria for Group B participants who will receive upfront window therapy (does not apply to participants who opt out of window therapy): - Cytochrome P450 CYP3A4 active agents: Must not be taking any of the following potent CYP3A4 inducers or inhibitors within 1 week prior to study entry: azole antifungals (such as fluconazole, voriconazole, itraconazole, ketoconazole), rifampin, phenytoin, phenobarbitol, carbamazepine, grapefruit juice and St. John's wort. - Must have measurable disease. - Must not have received emergent radiation therapy. - Serum triglyceride level = 300 mg/dL and serum cholesterol = 300 mg/dL. - Random or fasting glucose within the upper limits of normal for age. If random glucose is elevated, fasting glucose must be within normal range. - SGOT (AST) and SGPT (ALT) =3.0 x upper limit of normal for age. Exclusion Criteria: - Participant is pregnant or breastfeeding. - Inability or unwillingness of research participant or legal guardian/representative to give written informed consent. - Participant has a prior history of malignancy, with the exception of non-melanoma skin cancer. Participants with history of skin cancer must have 5 years elapse since that diagnosis, be in remission, and must not have received chemotherapy, immunotherapy, or radiation therapy. |
Country | Name | City | State |
---|---|---|---|
United States | St. Jude Children's Research Hospital | Memphis | Tennessee |
Lead Sponsor | Collaborator |
---|---|
St. Jude Children's Research Hospital | Nemours Children's Clinic, University of Florida, University of Tennessee |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | 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. | at 6 weeks after start of therapy (after 2 initial courses) | |
Secondary | 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. | Maximum of 11 years after the start of therapy | |
Secondary | 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. | Maximum of 11 years after the start of therapy | |
Secondary | Time to Progression | Median time to progression of group B patients will be estimated from the Kaplan-Meier curve. | Maximum of 11 years after the start of therapy | |
Secondary | 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. | Maximum of 11 years after the start of therapy |
Status | Clinical Trial | Phase | |
---|---|---|---|
Not yet recruiting |
NCT06456359 -
Pasireotide as Maintenance Treatment in Synovial Sarcoma and Desmoplastic Small Round Cell Tumor
|
Phase 2 | |
Active, not recruiting |
NCT04095221 -
A Study of the Drugs Prexasertib, Irinotecan, and Temozolomide in People With Desmoplastic Small Round Cell Tumor and Rhabdomyosarcoma
|
Phase 1/Phase 2 | |
Active, not recruiting |
NCT02834169 -
French National Registry of Rare Peritoneal Surface Malignancies
|
N/A | |
Active, not recruiting |
NCT03600649 -
Clinical Trial of SP-2577 (Seclidemstat) in Patients With Relapsed or Refractory Ewing or Ewing-related Sarcomas
|
Phase 1 | |
Recruiting |
NCT03618381 -
EGFR806 CAR T Cell Immunotherapy for Recurrent/Refractory Solid Tumors in Children and Young Adults
|
Phase 1 | |
Recruiting |
NCT04897321 -
B7-H3-Specific Chimeric Antigen Receptor Autologous T-Cell Therapy for Pediatric Patients With Solid Tumors (3CAR)
|
Phase 1 | |
Recruiting |
NCT05918640 -
Lurbinectedin in FET-Fused Tumors
|
Phase 1/Phase 2 | |
Terminated |
NCT04901806 -
Study of PBI-200 in Subjects With NTRK-Fusion-Positive Solid Tumors
|
Phase 1 | |
Recruiting |
NCT04022213 -
A Study of the Drug I131-Omburtamab in People With Desmoplastic Small Round Cell Tumors and Other Solid Tumors in the Peritoneum
|
Phase 2 | |
Active, not recruiting |
NCT04483778 -
B7H3 CAR T Cell Immunotherapy for Recurrent/Refractory Solid Tumors in Children and Young Adults
|
Phase 1 | |
Active, not recruiting |
NCT04145349 -
CAMPFIRE: A Study of Ramucirumab (LY3009806) in Children and Young Adults With Desmoplastic Small Round Cell Tumor
|
Phase 1/Phase 2 | |
Suspended |
NCT01125449 -
Study of High Dose Intravenous (IV) Ascorbic Acid in Measurable Solid Tumor Disease
|
Phase 2 | |
Enrolling by invitation |
NCT05266196 -
A Rollover Protocol to Allow for Continued Access to the LSD1 Inhibitor Seclidemstat (SP-2577)
|
Phase 1/Phase 2 | |
Not yet recruiting |
NCT06277154 -
MASCT-I Combined With Doxorubicin and Ifosfamide for First-line Treatment of Advanced Soft Tissue Sarcoma
|
Phase 2 | |
Completed |
NCT00436657 -
Continuous Hyperthermic Peritoneal Perfusion (CHPP) With Cisplatin for Children With Peritoneal Cancer
|
Phase 1 | |
Recruiting |
NCT04690374 -
Registry to Collect Health Information About Desmoplastic Small Round Cell Tumor
|
||
Not yet recruiting |
NCT02982486 -
A Phase II of Nivolumab Plus Ipilimumab in Non-resectable Sarcoma and Endometrial Carcinoma
|
Phase 2 | |
Completed |
NCT02982941 -
Enoblituzumab (MGA271) in Children With B7-H3-expressing Solid Tumors
|
Phase 1 | |
Recruiting |
NCT04530487 -
Donor Stem Cell Transplant After Chemotherapy for the Treatment of Recurrent or Refractory High-Risk Solid Tumors in Pediatric and Adolescent-Young Adults
|
Phase 2 | |
Recruiting |
NCT03967834 -
Multimodal Immune Characterization of RAre Soft Tissue Sarcoma - MIRAS Project From SARRA (SARcome RAre) Project of the French Sarcoma Group
|
N/A |