Actively Recruiting
Treatment of Newly Diagnosed Rhabdomyosarcoma Using Molecular Risk Stratification and Liposomal Irinotecan Based Therapy in Children With Intermediate and High Risk Disease
Led by St. Jude Children's Research Hospital · Updated on 2025-08-19
135
Participants Needed
3
Research Sites
707 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This is a phase II study to determine safety and efficacy of combining liposomal irinotecan with vincristine alternating with VAC in intermediate-risk patients, liposomal irinotecan with temozolomide and vincristine alternating with VAC in high-risk patients and the chemotherapy combinations when given with concomitant radiation therapy in intermediate and high risk patients. Primary Objective * Estimate event-free survival for intermediate-risk participants treated with VAC and vincristine and liposomal irinotecan (VLI) with the addition of maintenance therapy with vinorelbine and cyclophosphamide. * Estimate the event-free survival for high-risk patients treated with VAC and vincristine, liposomal irinotecan, and temozolomide with the addition of maintenance therapy with vinorelbine and cyclophosphamide. Secondary Objectives * To assess the relation between pharmacogenetic variation in CEP72 genotype and vinca alkaloid (vincristine; vinorelbine) disposition in children with rhabdomyosarcoma. * To assess the relation between the pharmacogenetic variation in drug metabolizing enzymes and drug transporters, and the pharmacokinetics of vinca alkaloids, liposomal irinotecan, and cyclophosphamide in children with rhabdomyosarcoma. * To assess the extent of inter-patient variability in the pharmacokinetics of vinca alkaloids, liposomal irinotecan, and cyclophosphamide in children with rhabdomyosarcoma, and explore possible associations between drug disposition and patient specific covariates (e.g., age, sex, race, weight). * Estimate the cumulative incidence of local recurrence and overall 3-year event-free survival in patients with low-risk disease, intermediate-risk disease or high-risk disease treated with either no adjuvant radiation or minimal volume radiation and compare these outcomes with the outcomes achieved on RMS13.
CONDITIONS
Official Title
Treatment of Newly Diagnosed Rhabdomyosarcoma Using Molecular Risk Stratification and Liposomal Irinotecan Based Therapy in Children With Intermediate and High Risk Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Newly diagnosed with rhabdomyosarcoma of any subtype
- Have low-, intermediate-, or high-risk disease as defined by specific genetic and staging criteria
- Age less than 22 years old
- Performance status ECOG 0, 1, or 2 (Lansky score used if younger than 16 years)
- No prior radiotherapy or chemotherapy for rhabdomyosarcoma except emergency local treatment
- Planned start of chemotherapy within 6 weeks of biopsy or surgery
- Adequate bone marrow function (ANC 750/bcL, platelets 75,000/bcL)
- Adequate liver function (total bilirubin less than 1.5 times upper limit of normal)
- Adequate kidney function based on creatinine clearance or serum creatinine thresholds by age and sex
- No evidence of dyspnea at rest and pulse oximetry over 94% if clinically indicated
- No active, uncontrolled infection
- Signed informed consent by participant or guardian
You will not qualify if you...
- Prior chemotherapy for rhabdomyosarcoma (excluding steroids)
- Prior full course radiation at primary tumor site (except emergency radiation)
- History or ongoing non-infectious interstitial lung disease requiring significant treatment
- Sexually active patients of reproductive potential not agreeing to effective contraception during and 3 months after study
- Pregnant females or those over 10 years/post-menarchal without negative pregnancy test
- Lactating females who are breastfeeding or planning to breastfeed infants
AI-Screening
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Trial Site Locations
Total: 3 locations
1
Stanford University
Palo Alto, California, United States, 94304
Actively Recruiting
2
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105
Actively Recruiting
3
Cook Children's Medical Center
Fort Worth, Texas, United States, 76104-2796
Not Yet Recruiting
Research Team
A
Alberto Pappo, MD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
3
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