NCT03382158Recruiting
International PPB/DICER1 Registry
International Pleuropulmonary Blastoma/DICER1 Registry (for PPB, DICER1 and Associated Conditions)
Source: ClinicalTrials.gov · Last updated 26 Oct 2026
Brief summary (registry, in English)
PPB has strong genetic implications, with a unique set of conditions in an individual or their family, occurring mostly in childhood and adolescence. The International PPB/DICER1 Registry first reported DICER1 germline loss-of-function variations in individuals with PPB and their family members. Conditions associated with DICER1 include Sertoli-Leydig cell tumor (SLCT), gynandroblastoma, ovarian sarcoma, peritoneal PPB, lung cysts, renal cysts, cystic nephroma (CN), anaplastic sarcoma of the kidney (ASK), Wilms tumor, primary DICER1-associated central nervous system (CNS) sarcoma, pineoblastoma, pituitary blastoma, multinodular goiter, thyroid carcinoma, nasal chondromesenchymal hamartoma, ciliary body medulloepithelioma (CBME), juvenile hamartomatous intestinal polyps, embryonal rhabdomyosarcoma (ERMS) and undifferentiated sarcoma. DICER1 variations may also be associated with macrocephaly and overgrowth syndrome. The International PPB/DICER1 Registry estimates that 30-50 cases of Type I and Ir PPB and 40-60 cases of Types II and III PPB are diagnosed annually worldwide. In 2009, the International Pleuropulmonary Blastoma Registry began a prospective study to collect treatment data on individuals for whom therapy decisions were made by their treating institution.
- Status (registry)
- Recruiting
- Study type
- Observational
- Enrollment
- 3400 (estimated)
- First submitted
- 11 Oct 2017
- Start date
- 6 Dec 2016
- Primary completion date
- 6 Dec 2031
- Lead sponsor
- Children's Hospitals and Clinics of Minnesota
- NCT number
- NCT03382158
What you commit to
- Study type: Observational
- Planned participants: 3400
- From 6 Dec 2016 to the primary completion date, 6 Dec 2031
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Other
- Absence of appropriate consent for Registry participation
Who the study is looking for
- Known or suspected PPB or related thoracic tumor
- Known or suspected sex-cord stromal tumor including Sertoli-Leydig cell tumor and gynandroblastoma (males or females)
- Other known or suspected DICER1-related condition including ovarian sarcoma, cystic nephroma, renal sarcoma, pineoblastoma, pituitary blastoma, nasal chondromesenchymal hamartoma, ciliary body medulloepithelioma and others
- Individuals with known or suspected DICER1 pathogenic variation regardless of whether they have an established DICER1-associated condition
- Informed consent by patient/ or parent/guardian (also, where appropriate: assent and HIPAA consent)
Age in the registry: 0 Minutes to 100 years · Sex: All · Accepts healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Children's Minnesota Coordinator of this site? Confirm its status | Minneapolis | United States | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.