Study for Newly Diagnosed Rhabdomyosarcoma
This study is for children and young adults (up to 22 years old) newly diagnosed with rhabdomyosarcoma, a type of cancer. It's testing new ways to treat intermediate and high-risk forms of the disease. Researchers are combining standard chemotherapy drugs like vincristine, dactinomycin, and cyclophosphamide with a drug called liposomal irinotecan. The goal is to see how safe and effective these combinations are, especially when given with radiation therapy or surgery. The study aims to improve how long patients live without the cancer returning (event-free survival) and reduce how often the cancer comes back in the same area (local recurrence rate). This study is currently recruiting participants.
- Study design
- This is a Phase II study with an adaptive design, meaning it can be adjusted as it goes. It plans to enroll 135 participants and is a single-arm study, which means everyone receives the study treatment.
- What's involved
- Not specified in the trial record.
- Compensation
- Not stated in the trial record.
- Follow-up
- Participants will be followed for 2 years after completing their treatment to assess event-free survival and local recurrence rates.
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Treatment of Newly Diagnosed Rhabdomyosarcoma Using Molecular Risk Stratification and Liposomal Irinotecan Based Therapy in Children With Intermediate and High Risk Disease
At a glance
Conditions
Where it's being run
3 sites across 3 statesStudy leadership
- Alberto Pappo, MD · PRINCIPAL_INVESTIGATOR · St. Jude Children's Research Hospital
Who to contact
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Do you actually qualify for this trial?
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Inclusion
Exclusion
What this trial measures
- Maximum tolerated doses (MTDs)4 years
MTD is defined in the study as the highest treatment dose that would deliver desirable treatment effects without resulting in a target toxicity rate greater than 0.3. For each of three groups (intermediate-risk, high-risk, and intermediate-and-high-risk-with-early-radiation), we will employ the Bayesian optimal interval (BOIN) design to find the MTD.
- Event-free survival (EFS)2 years post, off therapy
We will estimate the 2-year event-free survival for intermediate-risk and high-risk patients, which is the estimated probability of a patient not having any events within the 2-year follow-up. If an event, including local failure, distant failure, death or loss to follow-up occurs for a patient within 2-year, we call it failure, otherwise call it response.
- Local recurrence rate (LRR)2 years
LRR is defined as a binary endpoint in the study. The local recurrence-free survival (LRFS) is defined as time from randomization to the earlier date of the first of local disease recurrence or death due to any cause. The distant failure will be considered to be the competing risk, patients for whom follow-up ended without clinical improvement will be censored. The goal of the local recurrence rate endpoint is to evaluate the 2-year LRR by comparing the administration of 59.4 GyRBE and 68 GyRBE for patients (pooled intermediate- and high-risk groups) with tumor size greater than or equal to 5cm meeting the eligibility criteria for randomization (no biliary tree or specific extremity cases). .