Registry for rare lung tumors and DICER1-related conditions
International Pleuropulmonary Blastoma/DICER1 Registry (for PPB, DICER1 and Associated Conditions)
This study is collecting information about rare lung tumors in children, like pleuropulmonary blastoma, to help doctors understand how these conditions develop and how to treat them better.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 3400 (estimated) |
| Ages | 0 Minutes to 100 Years |
| Sex | All |
| Sponsor | Children's Hospitals and Clinics of Minnesota Academic / other |
| Drugs / interventions | chemotherapy, radiation, cyclophosphamide, doxorubicin |
| Locations | 1 site (Minneapolis, Minnesota) |
| Trial ID | NCT03382158 on ClinicalTrials.gov |
What this trial studies
The International PPB/DICER1 Registry aims to collect and analyze data on pleuropulmonary blastoma (PPB) and other DICER1-associated conditions in children. This observational registry will enroll individuals with known or suspected PPB, Sertoli-Leydig cell tumors, and other related tumors to better understand their clinical course and treatment outcomes. The study builds on previous research and encourages collaboration with international rare tumor groups, while treatment decisions remain at the discretion of the treating institution. The registry will also facilitate retrospective and real-time pathology reviews to enhance the understanding of these rare conditions.
Who should consider this trial
Good fit: Ideal candidates include children with known or suspected pleuropulmonary blastoma or other DICER1-related tumors.
Not a fit: Patients without a diagnosis of PPB or related DICER1 conditions may not benefit from this registry.
Why it matters
Potential benefit: If successful, this registry could lead to improved understanding and treatment strategies for rare tumors associated with DICER1 mutations.
How similar studies have performed: Previous studies have shown promise in understanding rare tumors through registries, indicating potential for success in this approach.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: 1. Known or suspected PPB or related thoracic tumor 2. Known or suspected sex-cord stromal tumor including Sertoli-Leydig cell tumor and gynandroblastoma (males or females) 3. 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 4. Individuals with known or suspected DICER1 pathogenic variation regardless of whether they have an established DICER1-associated condition 5. Informed consent by patient/ or parent/guardian (also, where appropriate: assent and HIPAA consent) Exclusion criteria: Absence of appropriate consent for Registry participation
Where this trial is running
Minneapolis, Minnesota
- Children's Minnesota — Minneapolis, Minnesota, United States (Recruiting)
Study contacts
- Principal investigator: Kris Ann P Schultz, MD — Children's Minnesota
- Study coordinator: Kris Ann P Schultz, MD
- Email: krisann.schultz@childrensmn.org
- Phone: 612-813-7121
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.