Chemotherapy + Surgery for Pleuropulmonary Blastoma
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to find an effective treatment plan for Pleuropulmonary Blastoma (PPB), a rare childhood cancer, by testing chemotherapy and surgery. It examines how specific chemotherapy combinations work alongside surgery for different PPB types. For Type I PPB, surgery is followed by optional chemotherapy, while Types II and III receive more intensive chemotherapy (including drugs like Cyclophosphamide, Dactinomycin, Doxorubicin, Ifosfamide, and Vincristine) and possible additional surgeries. Children newly diagnosed with PPB or related conditions can participate. The trial seeks to establish a standard treatment approach and improve outcomes for children with this rare disease. As an unphased trial, it offers a unique opportunity to contribute to groundbreaking research that could shape future PPB treatments.
Will I have to stop taking my current medications?
The trial protocol does not specify whether you need to stop taking your current medications. However, prior corticosteroid therapy is allowed, so you may be able to continue some medications.
Is there any evidence suggesting that this trial's treatments are likely to be safe?
Research has shown that the VAC regimen, which includes Vincristine, Dactinomycin, and Cyclophosphamide, treats various cancers, such as rhabdomyosarcoma (a cancer affecting muscle tissue). Several studies indicate that patients generally tolerate this combination well. However, like most chemotherapy treatments, it can cause some side effects.
The IVADo regimen, which includes Ifosfamide, Vincristine, Dactinomycin, and Doxorubicin, has shown promising results in studies for children with type II and III pleuropulmonary blastoma. Research indicates that this treatment offers favorable event-free and overall survival rates. While side effects are possible, its use since 2007 and recommendation in specific cases suggest confidence in its safety.
Both regimens use drugs common in chemotherapy. Although side effects can occur, these regimens have been part of treatment programs for many years. Discuss potential side effects and benefits with a healthcare provider.12345Why are researchers excited about this trial's treatments?
Researchers are excited about these treatments for pleuropulmonary blastoma (PPB) because they offer a tailored approach depending on the type of PPB. For Type I PPB, the treatment combines surgery with optional chemotherapy, specifically suggesting a combination of vincristine, dactinomycin, and cyclophosphamide (VAC). This approach allows for flexibility in treatment based on the physician's discretion. For Types II and III PPB, the unique combination of ifosfamide, vincristine, dactinomycin, and doxorubicin (IVADo) is used, potentially with additional surgeries and radiation if needed. This comprehensive and targeted strategy aims to address the aggressive nature of these cancer types more effectively than standard treatments.
What evidence suggests that this trial's treatments could be effective for Pleuropulmonary Blastoma?
Research has shown that certain chemotherapy treatments are often used for children with pleuropulmonary blastoma (PPB), a rare lung tumor. In this trial, participants with Type I PPB will undergo surgery, and the treating physician may use the VAC combination (vincristine, dactinomycin, and cyclophosphamide) for chemotherapy. For Types II and III PPB, participants will receive the IVADo combination (ifosfamide, vincristine, dactinomycin, and doxorubicin), which has shown promising results. Studies suggest that this treatment may lead to better outcomes compared to past cases without a standard treatment plan. Although no perfect treatment exists for everyone yet, these drug combinations are currently the best-supported options for managing the disease.36789
Who Is on the Research Team?
Kris Ann Schultz, MD
Principal Investigator
Children's Hospitals and Clinics of Minnesota
Are You a Good Fit for This Trial?
This trial is for children and young adults up to 21 years old with a rare lung tumor called Pleuropulmonary Blastoma (PPB). It includes those newly diagnosed, or with prior PPB, confirmed by central pathology review. Patients must be able to give consent through a guardian and may have had previous corticosteroid therapy.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Surgery
Surgery is necessary for all types of PPB. For Type I, surgery is followed by optional chemotherapy. For Types II and III, surgery is followed by chemotherapy and possibly radiation therapy.
Chemotherapy
For Type I, optional chemotherapy with Vincristine, Dactinomycin, Cyclophosphamide (VAC) may be used. For Types II and III, combination chemotherapy with Ifosfamide, Vincristine, Dactinomycin, and Doxorubicin (IVADo) is recommended.
Radiation Therapy
Radiation therapy is recommended only for residual disease after maximum surgery in Types II and III PPB.
Follow-up
Participants are monitored for overall response to chemotherapy and survival, with a primary focus on event-free survival over a 5-year period.
What Are the Treatments Tested in This Trial?
Interventions
- Cyclophosphamide
- Dactinomycin
- Doxorubicin
- Ifosfamide
- Vincristine
Cyclophosphamide is already approved in United States, European Union, Canada, Japan for the following indications:
- Breast cancer
- Ovarian cancer
- Multiple myeloma
- Leukemia
- Lymphoma
- Rheumatoid arthritis
- Breast cancer
- Ovarian cancer
- Multiple myeloma
- Leukemia
- Lymphoma
- Rheumatoid arthritis
- Breast cancer
- Ovarian cancer
- Multiple myeloma
- Leukemia
- Lymphoma
- Rheumatoid arthritis
- Breast cancer
- Ovarian cancer
- Multiple myeloma
- Leukemia
- Lymphoma
Find a Clinic Near You
Who Is Running the Clinical Trial?
Children's Hospitals and Clinics of Minnesota
Lead Sponsor