Clinical trial · Interventional
International Pleuropulmonary Blastoma (PPB) Treatment and Biology Registry
International Pleuropulmonary Blastoma (PPB) Treatment and Biology Registry Protocol
NCT01464606CI-TRIAL-00079991active not recruitingN/AClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
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.
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Pleuropulmonary Blastoma | Pleuropulmonary Blastoma | ONTOLOGY_EXACT | 0.98 |
Interventions
Interventions (5)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Cyclophosphamide | Drug | Cyclophosphamide | ALIAS |
| Dactinomycin | Drug | Dactinomycin | ALIAS |
| Doxorubicin | Drug | Doxorubicin | ALIAS |
| Ifosfamide | Drug | Ifosfamide | ALIAS |
| Vincristine | Drug | Vincristine | ALIAS |
Design
Arms and outcomes
Arms (2)
- type
- EXPERIMENTAL
- label
- Type I PPB therapy
- description
- 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).
- interventionNames
- Drug: Vincristine
- Drug: Dactinomycin
- Drug: Cyclophosphamide
- type
- EXPERIMENTAL
- label
- Types II and III PPB therapy
- description
- 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.
- interventionNames
- Drug: Vincristine
- Drug: Dactinomycin
- Drug: Cyclophosphamide
- Drug: Ifosfamide
Eligibility
Eligibility (as posted)
- Sex
- All
- Maximum age
- 21 Years
Show eligibility criteria text
Inclusion Criteria: 1. 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. 2. 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. 3. 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. 4. Prior corticosteroid therapy is allowed. 5. Patients who have received other chemotherapy regimens or radiation therapy will not be statistical analysis. 6. 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. 7. Informed consent by patient or parent/guardian. (also, where appropriate: assent and HIPPA consent) Exclusion Criteria: 1. Inability of patient, or parent/guardian to obtain informed consent. 2. Patients who have their PPB diagnosed ruled out by Registry central pathology review.
References
Publications (1)
- BACKGROUNDHill 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. PMID 19556464