An interventional study of Vincristine and Dactinomycin in Pleuropulmonary Blastoma, sponsored by Children's Hospitals and Clinics of Minnesota. Active, not recruiting at 1 site in United States. Open to participants aged Up to 21 Years. Per ClinicalTrials.gov, last updated 2026-10-23.
Sponsored by Children's Hospitals and Clinics of Minnesota · Not applicable, Interventional, and Health services research
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.
This Registry will collect family medical history and establishes a collection of specimens from PPB patients and their parents for future research. Primary goals of the biology research are (1) to identify new scientific ways to learn about PPB and the best way to treat it in the future and (2) to establish a collection of PPB biologic specimens for future research.
Background: PPB is a rare cancer of the lung presenting in early childhood, mostly from birth to age \~72 months. PPB occurs within the lung or between the lung and the chest wall. There are three forms of PPB called Types I, II, and III PPB. Children with Type I PPB require surgery and sometimes chemotherapy. Children with Types II and III PPB, require surgery, chemotherapy and sometimes radiation therapy. Because there has never been a consensus as to the best treatment for PPB, the Registry will provide treatment guidelines. It is the goal of the Treatment and Biology Registry that by offering specific regimens the Registry will be able to prospectively review and analyze the regimen for the first time.
Design:
Methods:
Analysis: Statistical data analysis will include only centrally confirmed cases. Additionally only patients with PPB Type II or Type III who were enrolled and treated with IVADo as per the regimen will be compared to historical controls. No statistical analysis is planned for patients with Type I PPB. Future treatment will be compared to the IVADo regimen in order to measure improvements.
Children's Hospitals and Clinics of Minnesota is the lead sponsor of 49 studies on the registry; 2 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Age : Patients from birth to \< 21 years of age at the time of diagnosis will be included in the Treatment and Biology Registry.
Patients of any age will be included in the Associated Diseases arm of this study.
Pathology Diagnosis: Patients with newly-diagnosed PPB Types I, II or III. Diagnosis is made by the local pathologist. Real-time central pathology review is encouraged but is not required. All cases must be submitted for central pathology review. Only centrally-reviewed cases confirmed as PPB will be analyzed prospectively.
Cases in which the initial diagnosis is "suggestive" or "supportive" of PPB, but not diagnostic, and in which later resection specimens, including resections following chemotherapy, confirm a PPB diagnosis will be included. Patients diagnosed by fine needle aspiration will be included only if a later resection specimen, including resections following chemotherapy, is diagnostic of PPB.
Diagnostic pathology for cases of diseases associated with PPB will also require registry central pathology review.
Prior Therapy: PPB Type I: All patients are eligible and will be followed in the study.
PPB Types II or III: Newly-diagnosed Types II and III PPB patients will be included in the Treatment and Biology Registry.
DICER1-related condition and DICER1 gene mutation: all patients are eligible and will be followed in the study.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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).
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.
Drug: Vincristine · Drug: Dactinomycin · Drug: Cyclophosphamide · Drug: Ifosfamide · Drug: Doxorubicin
≥ 3 years: 1.5 mg/m2 IV x 1 (maximum dose 2 mg)
Also known as: Vincristine; Oncovin
≥ 3 years: 0.045 mg/kg (maximum dose 2.5 mg) IV X 1
Also known as: Actinomycin-D
≥ 3 year: 1.2 gm/m2/dose IV as 1 hr infusion with IV fluids
Also known as: Cytoxan
≥ 3 years: 3 g/m2/dose IV over 3 hours on Days 1, 2, (6 g/m2/cycle)
Also known as: Ifos
≥ 3 years: 30 mg/m2/dose IV over 30 min, Days 1, 2 (60 mg/m2/cycle)
Also known as: Adriamycin
Event-free survival
The primary endpoint for statistical analysis will be time from start treatment to an event, defined as the occurrence of progression or recurrence of PPB, occurrence of a second malignant neoplasm, or death from any cause that is at least possibly related to the original disease or treatment.
Time frame: 5 years
Overall response to chemotherapy, and survival
Secondary endpoints will the best overall response to chemotherapy among patients with radiographically measurable tumor following initial surgery or biopsy, and time to death from any cause.
Time frame: 5 years
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
This study is active, not recruiting, as verified in Oct 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drugs tested here: Vincristine · Dactinomycin · Cyclophosphamide · Ifosfamide · Doxorubicin
Pleuropulmonary blastoma
Children's Hospitals and Clinics of Minnesota