Protocol Summary

Protocol No.: OCR13304

Sponsor Protocol No.: RMS13

Study Title
RMS13: Risk Adapted Focal Proton Beam Radiation and/or Surgery in Participants with Low, Intermediate and High Risk Rhabdomyosarcoma Receiving Standard or Intensified Chemotherapy

Principal Investigator(s)
Indelicato, Daniel

Objective
This study will treat participants with newly diagnosed, low, intermediate and high risk rhabdomyosarcoma (RMS) using multi-modality risk-adapted therapy with standard or intensified dose chemotherapy, radiation and surgical resection. Intermediate and high risk participants will receive an additional 18 weeks (6 cycles) of maintenance therapy with anti-angiogenic chemotherapy.

Description
Participants will be stratified based on both a pre-treatment staging system and a post-surgery surgico/pathologic clinical grouping system. Treatment for low-risk (subset 1) participants will consist of chemotherapy and radiation. Low-risk (subset 2) and intermediate-risk participants will chemotherapy and receive radiation and/or undergo surgery to destroy/remove the tumor. Intermediate-risk participants will also receive 16 weeks of maintenance chemotherapy. High-risk participants will receive chemotherapy and radiation therapy. High-risk participants will also receive additional maintenance therapy with anti-angiogenic chemotherapy.

Phase: Phase II (Cancer Control)

Age Group: Children

Scope: National

Treatment
Low-risk Stratum A:
Participants will receive chemotherapy and a total radiation dose of 36 Gy.

Low-risk Stratum B:
Participants will be treated with chemotherapy and a total radiation dose of 50.4 Gy.

Intermediate risk:
Participants with tumors ≥ 8 cm will receive chemotherapy and a total radiation dose of 59.4 Gy, plus maintenance chemotherapy.

Detailed Eligibility
INCLUSION CRITERIA:
1. Ages 0 to 21 years-old.
2. Newly diagnosed, previously untreated participants with localized rhabdomyosarcoma (RMS).
3. Must have either low-, intermediate-, or high-risk disease, defined as:
- Low-risk: Embryonal, botryoid, spindle cell tumors only (Subset 1: Stage 1, Group I; Stage 1 Group I; Stage 1 Group III orbital only; Stage 2 Group I; Stage 2 Group II) (Subset 2: Stage 1 Group III non orbit; Stage 3 Group I, II)
- Intermediate-risk: Embryonal, botryoid, or spindle cell RMS Stage 2 or 3 and Group III; Alveolar, undifferentiated, or anaplastic RMS: Stage 1-3, group I-I; I)
- High-risk: Embryonal, botryoid, spindle cell, alveolar, undifferentiated, or anaplastic RMS with metastatic disease at diagnosis (stage 4).
- Participants treated on this protocol in the low or intermediate risk arm who experience disease progression prior to week 13 will transfer to the high risk arm and proceed with high risk chemotherapy starting at week 1 of the protocol.
4. Performance level corresponding to ECOG score of 0, 1, or 2. The Lansky performance score should be used for participants 5. Participant has received no prior radiotherapy or chemotherapy (excluding steroids). Prior biopsy, surgical resection and lymph node sampling is allowed.
6. Initiation of chemotherapy is planned within 6 weeks (42 days) of the definitive biopsy or surgical resection.
7. Adequate bone marrow function defined as:
- Peripheral absolute neutrophil count (ANC) >= 750/μL
- Platelet count >= 75,000/μL (transfusion independent)
- Adequate liver function defined as total bilirubin =8. Adequate renal function defined as:
- Creatinine clearance or radioisotope GFR >= 70 mL/min/1.732 or
- Serum creatinine based on age and gender
- Participants with urinary tract obstruction by tumor must meet the renal function criteria listed above AND must have unimpeded urinary flow established via decompression of the obstructed portion of the urinary tract.
9. Patients requiring emergency radiation therapy are eligible for enrollment on this study.
10. Females of child-bearing potential cannot be pregnant or breast-feeding. Female participants >= 10 years of age or post-menarchal must have a negative serum or urine pregnancy test within 24 hours prior to beginning treatment. Female participants who are breast feeding must agree to stop breast feeding.
11. Sexually active patients of childbearing potential must be willing to use effective contraception during therapy and for at least 1 month after treatment is completed.
12. No evidence of active, uncontrolled infection.
13. All participants and/or their parents or legal guardians must sign a written informed consent.

EXCLUSION CRITERIA:
1. Participants who fail to meet one or more of the inclusion criteria will be excluded.

Applicable Disease Sites
Sarcoma

Participating Institutions
UF Gainesville : Ashley Williams
UF Jacksonville : Ashley Williams

Contact
Ashley Williams
Phone: +1 904-588-1451

Email: awilliams@floridaproton.org

More Information:
View study listing on ClinicialTrials.gov
http://www.clinicaltrials.gov/ct2/show/
NCT01871766