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Alkylating agents

Chemotherapy for Rhabdomyosarcoma

Phase 3
Recruiting
Led By Josephine H Haduong
Research Sponsored by Children's Oncology Group
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Patients must be =< 21 years at the time of enrollment
Patients must have a Lansky (for patients =< 16 years of age) or Karnofsky (for patients > 16 years of age) performance status score of >= 50
Must not have
Patients who have received prior chemotherapy and/or radiation therapy for cancer prior to enrollment. Surgical resection alone of previous cancer(s) is permitted
Female patients who are pregnant
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights

Summary

This trial is examining how well different types of chemotherapy work in treating patients with different levels of risk for RMS cancer.

Who is the study for?
This trial is for patients up to 21 years old with newly diagnosed very low-risk or low-risk rhabdomyosarcoma, a type of soft tissue cancer. They must have specific stages and groups of the disease, proper organ function, no prior cancer treatments except surgery, and not be on certain drugs that affect vincristine.Check my eligibility
What is being tested?
The study tests a chemotherapy regimen using vincristine and dactinomycin over 24 weeks for very low-risk patients. It also assesses standard chemo effectiveness in low-risk patients and intensifies therapy for those with DNA mutations to improve outcomes.See study design
What are the potential side effects?
Possible side effects include hair loss, nausea, vomiting, mouth sores from chemotherapy; potential nerve damage from vincristine; skin changes or fatigue due to radiation therapy; increased risk of infection; and blood count changes.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am 21 years old or younger.
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I can do most activities but need help with some, regardless of my age.
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I have been newly diagnosed with a specific type of muscle cancer.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I've had chemotherapy or radiation before for cancer, but surgery was only for removing it.
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I am currently pregnant.
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I cannot have radiation therapy if needed.
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I have an infection that is not responding to treatment.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Failure free survival (FFS) for low risk patients
Failure free survival (FFS) for very low risk patients
Secondary outcome measures
Feasibility of central molecular risk stratification of patients assessed by the percentage of patients who have molecular testing results returned by 6 weeks
Overall survival (OS) for low risk patients
Overall survival (OS) for very low risk patients
Other outcome measures
Methylation array profile of patients with fusion negative, low-risk rhabdomyosarcoma

Trial Design

3Treatment groups
Experimental Treatment
Group I: Regimen VAC/VA (VL RMS)Experimental Treatment8 Interventions
Patients with LR RMS receive vincristine IV on day 1 of each cycle and days 8 and 15 of cycles 1-3. Patients also receive dactinomycin IV over 1-5 minutes or 10-15 minutes and cyclophosphamide IV over 60 minutes on day 1 of each cycle. Treatment repeats every 21 days for 4 cycles in the absence of disease progression or unacceptable toxicity. Patients then receive vincristine IV on day 1 of each cycle and days 8 and 15 of cycles 5-7 and dactinomycin IV over 1-5 minutes or over 10-15 minutes on day 1 of each cycle. Treatment repeats every 21 days for 4 cycles in the absence of disease progression or unacceptable toxicity. Patients with MYOD1 or TP53 mutated tumors transition to Regimen M at cycle 2 (if mutation status is determined to be positive at week 3) or cycle 3 (if mutation status is determined to be positive after week 3). Radiation therapy (if needed) will be administered at cycle 5.Patients undergo CT scan, MRI, bone scan, PET scan and tumor biopsy throughout the study.
Group II: Regimen VA (VLR RMS)Experimental Treatment6 Interventions
Patients with VLR RMS receive vincristine intravenously (IV) on day 1 of each cycle and days 8 and 15 of cycles 1, 3, 5, and 7 and dactinomycin IV over 1-5 minutes or over 10-15 minutes on day 1 of each cycle. Treatment repeats every 21 days for 8 cycles in the absence of disease progression or unacceptable toxicity. Patients with MYOD1 or TP53 mutated tumors transition to Regimen M at cycle 2 (if mutation status is determined to be positive at week 3) or cycle 3 (if mutation status is determined to be positive after week 3). Patients undergo CT scan, MRI, bone scan, PET scan and tumor biopsy throughout the study.
Group III: Regimen M (positive mutation)Experimental Treatment9 Interventions
Patients receive vincristine IV on day 1 of each cycle and days 8 and 15 of cycles 2-4, 7-8, and 11-12 and dactinomycin IV over 1-5 minutes or 10-15 minutes on day 1 of cycles 2-5 and 8-14. Patients also receive cyclophosphamide IV over 60 minutes on day 1 of each cycle. Treatment repeats every 21 days for 12-13 cycles in the absence of disease progression or unacceptable toxicity. Patients may also undergo radiation therapy at cycle 5. Patients undergo CT scan, MRI, bone scan, PET scan and tumor biopsy throughout the study.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Cyclophosphamide
1995
Completed Phase 3
~3770
Positron Emission Tomography
2008
Completed Phase 2
~2210
Biopsy
2014
Completed Phase 4
~1090
Bone Scan
2015
Completed Phase 2
~50
Computed Tomography
2017
Completed Phase 2
~2720
Dactinomycin
2010
Completed Phase 3
~1310
Radiation Therapy
2017
Completed Phase 3
~7250
Vincristine
2003
Completed Phase 4
~2910

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.
Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
Vincristine and dactinomycin are commonly used treatments for Alveolar Rhabdomyosarcoma (ARMS). Vincristine inhibits microtubule formation, which is essential for cell division, thereby halting the mitotic process and leading to cell death. Dactinomycin intercalates into DNA and inhibits RNA synthesis, preventing the transcription necessary for protein synthesis and cell survival. These mechanisms are crucial for ARMS patients as they target the rapid and uncontrolled cell division characteristic of this cancer, potentially improving treatment outcomes.
Variable duration of vincristine-induced metaphase block in leukemic and nornal bone marrow cells of children.Evaluation of ABT-751 against childhood cancer models in vivo.

Find a Location

Who is running the clinical trial?

Children's Oncology GroupLead Sponsor
456 Previous Clinical Trials
239,582 Total Patients Enrolled
National Cancer Institute (NCI)NIH
13,748 Previous Clinical Trials
40,959,196 Total Patients Enrolled
Josephine H HaduongPrincipal InvestigatorChildren's Oncology Group

Media Library

Cyclophosphamide (Alkylating agents) Clinical Trial Eligibility Overview. Trial Name: NCT05304585 — Phase 3
Alveolar Rhabdomyosarcoma Research Study Groups: Regimen VA (VLR RMS), Regimen VAC/VA (VL RMS), Regimen M (positive mutation)
Alveolar Rhabdomyosarcoma Clinical Trial 2023: Cyclophosphamide Highlights & Side Effects. Trial Name: NCT05304585 — Phase 3
Cyclophosphamide (Alkylating agents) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05304585 — Phase 3
~137 spots leftby Dec 2030