Chemotherapy + Surgery for Pleuropulmonary Blastoma

Recruiting in Palo Alto (17 mi)

Overseen byKris Ann Schultz, MD

Age: < 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Academic

Waitlist Available

Sponsor: Children's Hospitals and Clinics of Minnesota

No Placebo Group

Trial Summary

What is the purpose of this trial?Pleuropulmonary Blastoma (PPB) is very rare and there is no established "standard" or "best" therapy. For many years, children with PPB around the world have been treated according to decisions made case-by-case in many different hospitals by many different physicians. No treatment has been tested in a large group of PPB patients. The goal is to treat many children with one treatment program and to learn the results of the treatment.

Eligibility Criteria

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

Diagnostic pathology for cases of diseases associated with PPB will also require registry central pathology review

If you have PPB Type I, you can participate in the study.

I have a DICER1-related condition and a DICER1 gene mutation.

+8 more

Exclusion Criteria

I am unable to understand or give consent for treatment.

You have had central pathology review ruling out a diagnosis of pleuropulmonary blastoma.

Participant Groups

The trial tests a treatment program using drugs like Doxorubicin, Vincristine, Dactinomycin, Cyclophosphamide, and Ifosfamide on many patients to determine the effectiveness against PPB since there's no standard treatment due to its rarity.

2Treatment groups

Experimental Treatment

Group I: Types II and III PPB therapyExperimental Treatment5 Interventions

Combination chemotherapy with Ifosfamide, Vincristine, Dactinomycin and Doxorubicin ("IVADo"). Second look and possible 3rd look surgery may be required. Radiation therapy is recommended only for residual disease after maximum surgery.

Group II: Type I PPB therapyExperimental Treatment3 Interventions

PPB Type I therapy: All patients will be treated with surgery. Chemotherapy after surgery is per the treating physician(s) discretion. If chemotherapy is used the Registry will suggest that it be combination chemotherapy with Vincristine, Dactinomycin, Cyclophosphamide (VAC).

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

🇺🇸 Approved in United States as Cytoxan for: