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New treatment option for Rhabdomyosarcoma
Official title Treatment of Newly Diagnosed Rhabdomyosarcoma Using Molecular Risk Stratification and Liposomal Irinotecan Based Therapy in Children With Intermediate and High Risk Disease
ClinicalTrials.gov ID: NCT06023641
What this study is testing
What is Vincristine?
Vincristine is an investigational medicine, given as an once-weekly infusion into a vein, being studied as a potential treatment for rhabdomyosarcoma.
Also referred to as Oncovin.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
- Time commitment: about 4 years
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages up to 22
You may be able to join if
- Newly diagnosed participants with the diagnosis of rhabdomyosarcoma (RMS) of any subtype. This includes embryonal rhabdomyosarcoma (fusion negative)...
- 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
You likely can't join 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...
- Female patients who are pregnant are not eligible since fetal toxicities or teratogenic effects have been noted for several of the study drugs...
- Lactating females who are or plan to breastfeed their infants are not eligible.
See the full eligibility criteria
- 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...
- 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
- 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
- Platelet count ≥ 75,000/μL (transfusion independent)
- Adequate liver function defined as total bilirubin \ 70 mL/min/1.732 or serum creatinine based on age as follows: Age Maximum serum creatinine (mg/dL) Male Female 1 month to \< 6 months 0.4 0.4 6 months to \< 1 year 0.5...
- to \< 2 years 0.6 0.6
- to \ 16 years 1.7 1.4 The threshold creatinine values in this table were derived from the Schwartz formula for estimating GFR25 utilizing child length and stature. Data published by the CDC. Participants with urinary...
- Adequate pulmonary function defined as: no evidence of dyspnea at rest and a pulse oximetry \> 94% if there is a clinical indication for determination. Pulmonary function tests are not required.
- Patients requiring emergency radiation therapy are eligible for enrollment on this trial. See Section 4.11 for radiation therapy guidelines.
- No evidence of active, uncontrolled infection. All participants and/or their parents or legal guardians must sign a written informed consent.
- 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...
- Lactating females who are or plan to breastfeed their infants are not eligible.
The study team makes the final eligibility decision.
Where it's taking place
- Palo Alto, California, United States
- Memphis, Tennessee, United States
- Fort Worth, Texas, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The study runs about 4 years per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, up to 22 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Palo Alto, California, United States; Memphis, Tennessee, United States; Fort Worth, Texas, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.