Treatment of Newly Diagnosed Rhabdomyosarcoma Using Molecular Risk Stratification and Liposomal Irinotecan Based Therapy in Children With Intermediate and High Risk Disease

Recruiting now · Phase 2

Conditions studied: Rhabdomyosarcoma

In brief

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.

Key facts

Study ID: NCT06023641
Run by: St. Jude Children's Research Hospital
People needed: 135
Starts: 2024-03-13
Expected to finish: 2037-10-01
Last updated by the study team: 2026-06-15

Who can join

Age: any, up to 22. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

Newly diagnosed participants with the diagnosis of rhabdomyosarcoma (RMS) of any subtype. This includes embryonal rhabdomyosarcoma (fusion negative), alveolar rhabdomyosarcoma (fusion positive), as well as spindle cell and sclerosing
Must have either low-, intermediate-risk or high-risk disease, defined as:
Low-risk: TP53 and MYOD1 negative AND
Embryonal, congenital/infantile spindle cell, or spindle cell/sclerosing FOXO1 fusion negative histology
Stage 1 Group I, Group II
Stage 1 Group III orbital only
Stage 2 Group I, Group II
Intermediate-risk: MYOD1 and TP53 negative AND
Embryonal, congenital/infantile spindle cell, or spindle cell/sclerosing FOXO1 fusion negative histology o Stage 1 Group III non orbit o Stage 3 Group I/II
o Stage 2/3 Group III
Stage 4 Group IV and Oberlin 0-1
Alveolar, spindle cell/sclerosing FOXO1 fusion positive histology
Stage 1-3, Group I-III N0
High-risk: All MYOD1 and TP53 mutant tumors regardless of stage and Group AND/OR
Embryonal, congenital/infantile spindle cell or spindle cell/sclerosing FOXO1 fusion negative o Group IV ≥ 10 year of age and Oberlin ≥ 2
Alveolar, spindle cell/sclerosing FOXO1 fusion positive
N1
Stage 4 Group IV
See Appendices I and II for Staging and Clinical Grouping.
Age < 22 years (eligible for enrollment until 22nd birthday)
Performance level corresponding to ECOG score of 0, 1, or 2. The Lansky performance score should be used for participants < 16 years (see Appendix VII).
Participant has received no prior radiotherapy or chemotherapy for rhabdomyosarcoma (excluding steroids) unless an emergency situation requires local tumor treatment (discuss with PI).
Initiation of chemotherapy is planned within 6 weeks (42 days) of the definitive biopsy or surgical resection.
Adequate bone marrow function defined as:
Peripheral absolute neutrophil count (ANC) ≥ 750/μL

You may not qualify if…

Patients who have received any chemotherapy (excluding steroids).
Patients who have received prior full course RT at the primary site of disease. This does not exclude patients that received emergent radiation.
Ongoing or history of non-infectious interstitial lung disease requiring significant medical intervention.
Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation and for at least 3 months after treatment is completed.
Female patients who are pregnant are not eligible since fetal toxicities or teratogenic effects have been noted for several of the study drugs. 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.
Lactating females who are or plan to breastfeed their infants are not eligible.

Where it is running

Stanford University — Palo Alto, California, United States (enrolling)
St. Jude Children's Research Hospital — Memphis, Tennessee, United States (enrolling)
Cook Children's Medical Center — Fort Worth, Texas, United States

Full record on ClinicalTrials.gov

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