Pleuropulmonary BlastomaNCT01464606
International Pleuropulmonary Blastoma (PPB) Treatment and Biology Registry
A study testing Vincristine, Dactinomycin and Cyclophosphamide in people with Pleuropulmonary Blastoma, run by Children's Hospitals and Clinics of Minnesota at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages Up to 21 years
- Conditions the study is for
- Pleuropulmonary Blastoma
- What is being tested?
- Vincristine, Dactinomycin and Cyclophosphamide
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 156 participants
- Where
- 1 siteUnited States
- Study dates
- 22 Dec 2009to 31 Dec 2035
- Phase
- Interventional
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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 biopsy 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.
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- 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.
- Prior corticosteroid therapy is allowed.
- Patients who have received other chemotherapy regimens or radiation therapy will not be statistical analysis.
- Types II and III PPB patients with PRIOR Type I PPB diagnosis: Types II and III PPB cases which are recurrences of an earlier Type I PPB are included.
- Informed consent by patient or parent/guardian. (also, where appropriate: assent and HIPPA consent)
Who cannot join
- Inability of patient, or parent/guardian to obtain informed consent.
- Patients who have their PPB diagnosed ruled out by Registry central pathology review.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: International Pleuropulmonary Blastoma (PPB) Treatment and Biology Registry Protocol
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.
Read the detailed description
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:
- Diagnostic central pathology review will be required for patients who will be prospectively evaluated.
- This Treatment Registry will offer surgery and chemotherapy regimen guidelines, and optional radiation therapy guidelines for prospectively enrolled patients.
- The Treatment and Biology Registry will create a PPB biologic specimens reference bank for tumor.
Methods:
- For Type I PPB, surgery is necessary. Some doctors use chemotherapy after surgery. If chemotherapy is used the Registry will suggest that it be combination chemotherapy with vincristine, dactinomycin, cyclophosphamide (VAC ).
- For Types II and III PPB patients surgery is necessary, followed by chemotherapy. The Registry will suggest that subjects receive combination chemotherapy with Ifosphamide, Vincristine, Actinomycin-D and Doxorubicin (IVADo). Second look and possible 3rd look surgery may be required. Radiation therapy is recommended only for residual disease after maximum surgery.
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.
- Conditions
- Pleuropulmonary Blastoma
- Being tested
- Vincristine, Dactinomycin, Cyclophosphamide, Ifosfamide, Doxorubicin
- Keywords
- pleuropulmonary blastoma, pediatric, lung cysts
- Medical subject headings
- Pleuropulmonary blastoma, Cystic Disease Of Lung
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VincristineDrug
Also called Vincristine; Oncovin
≥ 3 years: 1.5 mg/m2 IV x 1 (maximum dose 2 mg)
DactinomycinDrug
Also called Actinomycin-D
≥ 3 years: 0.045 mg/kg (maximum dose 2.5 mg) IV X 1
CyclophosphamideDrug
Also called Cytoxan
≥ 3 year: 1.2 gm/m2/dose IV as 1 hr infusion with IV fluids
IfosfamideDrug
Also called Ifos
≥ 3 years: 3 g/m2/dose IV over 3 hours on Days 1, 2, (6 g/m2/cycle)
DoxorubicinDrug
Also called Adriamycin
≥ 3 years: 30 mg/m2/dose IV over 30 min, Days 1, 2 (60 mg/m2/cycle)
| Study group | Role | What they receive |
|---|---|---|
| Type I PPB therapy | Experimental | 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). |
| Types II and III PPB therapy | Experimental | 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. Receives: Drug: Vincristine; Drug: Dactinomycin; Drug: Cyclophosphamide; Drug: Ifosfamide; Drug: Doxorubicin |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 5 years
Secondary outcomes (1)
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.
Measured: 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Nov 2011 (estimated)
- Study start22 Dec 2009
- Primary completion31 Dec 2030 (estimated)
- Study completion31 Dec 2035 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Pleuropulmonary BlastomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Health services research
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 156 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospitals and Clinics of Minnesota
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Kris Ann Schultz, Children's Hospitals and Clinics of Minnesota
- Investigators
- Kris Ann P Schultz, MD, Principal investigator, Children's Hospitals and Clinics of Minnesota
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01464606
- Sponsor's protocol ID
- PPB Reg Tx-Biol-001
- Status last verified
- Oct 2026
- First submitted
- 29 Sep 2011
- Last updated
- 23 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Hill DA, Ivanovich J, Priest JR, Gurnett CA, Dehner LP, Desruisseau D, Jarzembowski JA, Wikenheiser-Brokamp KA, Suarez BK, Whelan AJ, Williams G, Bracamontes D, Messinger Y, Goodfellow PJ. DICER1 mutations in familial pleuropulmonary blastoma. Science. 2009 Aug 21;325(5943):965. doi: 10.1126/science.1174334. Epub 2009 Jun 25.
PubMed 19556464