RecruitingNCT01247597
Respiratory
Pleuropulmonary Blastoma
Also known as PPB, pulmonary blastoma, DICER1 lung tumor
Pleuropulmonary blastoma is the most common primary malignant lung tumour in childhood and is strongly associated with germline or somatic mutations in the DICER1 gene. It is classified into three types based on tissue composition: Type I (
ORPHA:73034 ↗Gene DICER1Prevalence Fewer than 1 in 1,000,000; estimated 25–30 new cases per year in the USOnset Childhood (typically under age 6)
4
studies recruiting now
as of 7 Sept 2026
11
studies registered in total
as of 7 Sept 2026
3
countries with a recruiting site
as of 7 Sept 2026
18 Jul 2025
most recent study posted
among recruiting studies
Recruiting trials
RecruitingPhase 3NCT06647953
Testing a Standardized Approach to Surgery and Chemotherapy for Type I Pleuropulmonary Blastoma or the Addition of an Anti-cancer Drug, Topotecan, to the Usual Treatment for Types II and III Pleuropulmonary Blastoma
RecruitingNCT07072143
An International Study on Pediatric Patients With Rare Tumors.
RecruitingNCT03382158
International PPB/DICER1 Registry
Showing the 4 most recently updated recruiting studies, as recorded 7 Sept 2026. Live status on each study page.
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Countries with at least one recruiting site among the studies above, 7 Sept 2026. Tap a country to search trials there.
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About Pleuropulmonary Blastoma
Pleuropulmonary blastoma is the most common primary malignant lung tumour in childhood and is strongly associated with germline or somatic mutations in the DICER1 gene. It is classified into three types based on tissue composition: Type I (purely cystic), Type II (mixed cystic and solid), and Type III (purely solid), with higher types carrying a worse prognosis. PPB is part of the DICER1 tumour predisposition syndrome, which also includes thyroid tumours, cystic nephroma, and other neoplasms.
Common clinical features
Respiratory distress or recurrent pneumoniaChest painCystic or solid intrathoracic mass on imagingTension pneumothorax (Type I)Fever and malaiseHaemoptysis (less common)Metastatic spread to brain or bone (advanced disease)
From Orphanet’s phenotype annotations (CC BY 4.0). Not a complete list.
Before you apply
Things trial teams commonly ask about for Pleuropulmonary Blastoma. Not eligibility rules; those are set by each study.
- DICER1 germline mutation testing is central to eligibility for DICER1 syndrome studies; pursue genetic counselling and documented mutation status early.
- PPB type (I, II, or III) directly influences trial eligibility; confirm histopathological classification from a specialist paediatric pathology centre.
- Many paediatric oncology trials require enrolment at or referral to an affiliated centre; contact the International PPB/DICER1 Registry for guidance on participating sites.
Related conditions
Information, not medical advice. Trial listings are shown as recorded on ClinicalTrials.gov; whether any study is right for you is a decision for you and your clinicians, and eligibility is decided by each research team. Disease information from Orphanet (CC BY 4.0).