205 Participants Needed

Chemotherapy for Rhabdomyosarcoma

Recruiting at 173 trial locations
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Pivotal Trial (Near Approval)This treatment is in the last trial phase before FDA approval
Prior Safety DataThis treatment has passed at least one previous human trial

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial uses chemotherapy drugs to treat patients with very low-risk and low-risk rhabdomyosarcoma. The goal is to maintain good outcomes while reducing treatment intensity. The study also examines if patients with specific DNA mutations benefit from more intensive therapy.

Will I have to stop taking my current medications?

The trial requires that you stop taking any drugs that are moderate to strong inhibitors or inducers of CYP3A4 (a liver enzyme) at least 7 days before enrolling. If you are on chemotherapy for non-cancer conditions, you must stop it before starting the trial treatment.

Is chemotherapy for rhabdomyosarcoma safe for humans?

The chemotherapy drugs vincristine, dactinomycin, and cyclophosphamide have been used in treating rhabdomyosarcoma and other conditions, showing low toxic effects in studies. These drugs are generally considered safe for humans, with no increased toxicity observed in certain dosing regimens.12345

How is the drug combination of Cyclophosphamide, Dactinomycin, and Vincristine unique for treating rhabdomyosarcoma?

This drug combination is unique because it uses a shorter-duration therapy with lower doses of cyclophosphamide, aiming to maintain effectiveness while reducing treatment length and potential side effects for low-risk rhabdomyosarcoma patients.24567

What data supports the effectiveness of the drug combination of Cyclophosphamide, Dactinomycin, and Vincristine for treating rhabdomyosarcoma?

Research shows that more than 70% of children and adolescents with rhabdomyosarcoma are cured with modern treatments, including the combination of vincristine, dactinomycin, and cyclophosphamide. Studies also confirm that this combination is effective, with major responses observed in 67% to 70% of patients, and it is feasible with low toxic effects.24578

Who Is on the Research Team?

JH

Josephine H Haduong

Principal Investigator

Children's Oncology Group

Are You a Good Fit for This Trial?

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.

Inclusion Criteria

All patients and/or their parents or legal guardians must sign a written informed consent
All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met
Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) =< 135 U/L (within 7 days prior to enrollment)
See 9 more

Exclusion Criteria

I've had chemo or radiation for a non-cancer condition but will stop before starting the trial.
I've had chemotherapy or radiation before for cancer, but surgery was only for removing it.
I am currently pregnant.
See 5 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Patients receive chemotherapy with vincristine and dactinomycin for VLR RMS or vincristine, dactinomycin, and cyclophosphamide for LR RMS. Treatment is adjusted based on mutation status.

24 weeks
Visits every 21 days for 8 cycles

Follow-up

Participants are monitored for safety and effectiveness after treatment

Up to 5 years

Regimen M

Patients with positive mutation status receive intensified therapy with vincristine, dactinomycin, and cyclophosphamide, potentially including radiation therapy.

12-13 cycles

What Are the Treatments Tested in This Trial?

Interventions

  • Cyclophosphamide
  • Dactinomycin
  • Vincristine
Trial Overview 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.
How Is the Trial Designed?
3Treatment groups
Experimental Treatment
Group I: Regimen VAC/VA (VL RMS)Experimental Treatment8 Interventions
Group II: Regimen VA (VLR RMS)Experimental Treatment6 Interventions
Group III: Regimen M (positive mutation)Experimental Treatment9 Interventions

Cyclophosphamide is already approved in United States, European Union, Canada, Japan for the following indications:

๐Ÿ‡บ๐Ÿ‡ธ
Approved in United States as Cytoxan for:
๐Ÿ‡ช๐Ÿ‡บ
Approved in European Union as Endoxan for:
๐Ÿ‡จ๐Ÿ‡ฆ
Approved in Canada as Neosar for:
๐Ÿ‡ฏ๐Ÿ‡ต
Approved in Japan as Endoxan for:

Find a Clinic Near You

Who Is Running the Clinical Trial?

Children's Oncology Group

Lead Sponsor

Trials
467
Recruited
241,000+

National Cancer Institute (NCI)

Collaborator

Trials
14,080
Recruited
41,180,000+

Published Research Related to This Trial

With modern treatments, over 70% of children and adolescents with rhabdomyosarcoma can be cured, highlighting the importance of accurate diagnosis and multidisciplinary therapy for maximizing cure rates.
Current research is focusing on new therapies, including topoisomerase-I inhibitors and molecular characterization of tumors, which may lead to more effective treatments for high-risk patients who currently have poor outcomes.
Rhabdomyosarcoma: new windows of opportunity.Breitfeld, PP., Meyer, WH.[2022]
In a study using a laboratory model of childhood rhabdomyosarcoma (RMS) with seven patient-derived xenografts, the conventional chemotherapy agent vincristine was found to be the most effective treatment.
L-phenylalanine mustard (L-PAM) showed remarkable efficacy, causing complete tumor regressions in six out of seven RMS lines, including those resistant to cyclophosphamide, indicating its potential as a powerful treatment option.
Childhood rhabdomyosarcoma xenografts: responses to DNA-interacting agents and agents used in current clinical therapy.Houghton, JA., Cook, RL., Lutz, PJ., et al.[2019]
In a clinical trial involving 92 children with rhabdomyosarcoma, both high single doses and divided doses of dactinomycin (ACT-D) combined with vincristine and cyclophosphamide showed similar effectiveness, with major response rates of 67% and 70%, respectively.
The study found that administering high, single doses of ACT-D resulted in low toxicity and did not increase adverse effects compared to the divided dosing, suggesting it is a feasible treatment option that requires less hospitalization for patients.
Tumor response and toxicity after single high-dose versus standard five-day divided-dose dactinomycin in childhood rhabdomyosarcoma.Carli, M., Pastore, G., Perilongo, G., et al.[2017]

Citations

Rhabdomyosarcoma: new windows of opportunity. [2022]
Childhood rhabdomyosarcoma xenografts: responses to DNA-interacting agents and agents used in current clinical therapy. [2019]
Tumor response and toxicity after single high-dose versus standard five-day divided-dose dactinomycin in childhood rhabdomyosarcoma. [2017]
Shorter-duration therapy using vincristine, dactinomycin, and lower-dose cyclophosphamide with or without radiotherapy for patients with newly diagnosed low-risk rhabdomyosarcoma: a report from the Soft Tissue Sarcoma Committee of the Children's Oncology Group. [2022]
PAX3-FKHR sensitizes human alveolar rhabdomyosarcoma cells to camptothecin-mediated growth inhibition and apoptosis. [2021]
Feasibility of combining temsirolimus to vincristine, dactinomycin, cyclophosphamide, and vincristine and irinotecan chemotherapy for children with intermediate-risk rhabdomyosarcoma: A report from Children's Oncology Group. [2023]
Rising drug cost impacts on cost-effectiveness of 2 chemotherapy regimens for intermediate-risk rhabdomyosarcoma: A report from the Children's Oncology Group. [2023]
The Intergroup Rhabdomyosarcoma Study: update, november 1978. [2007]
Unbiased ResultsWe believe in providing patients with all the options.
Your Data Stays Your DataWe only share your information with the clinical trials you're trying to access.
Verified Trials OnlyAll of our trials are run by licensed doctors, researchers, and healthcare companies.
Back to top
Terms of ServiceยทPrivacy PolicyยทCookiesยทSecurity