Pleuropulmonary BlastomaPPBNCT03044834
Review of the Paediatric Pleuropulmonary Blastoma French Series
An observational study of Pleuropulmonary Blastoma, run by Rennes University Hospital at 18 sites in France.
Trial summary
Pleuropulmonary blastoma is a rare embryonic malignant tumour that remains the most frequent malignant tumour of the lung in childhood. The International Pleuro pulmonary Blastoma Registry (IPPB) found only 220 cases in 2009 and 350 in 2015. In France, 20 cases were identified in 2009. Read more about this study
- Who is it for?
- Ages 1 year to 17 years
- Conditions the study is for
- Pleuropulmonary Blastoma
- What is being tested?
- PPB
- How many people
- 41 participants
- Where
- 18 sitesFrance
- Study dates
- 9 Jan 2017to 9 Jul 2018
- Phase
- Observational
- Are there results?
- None posted in the registry
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 1 year to 17 years
- All sexes
- No healthy volunteers
- Child
Study population: Patients born between 01/01/2000 and 01/01/2016, followed up for PPB, treated in a French department of paediatric oncology or paediatric surgery.
Who may be able to join
- Patients born between 01/01/2000 and 01/01/2016 ;
- Followed up for PPB
- Treated in a French department of paediatric oncology or paediatric surgery
- Study agreement
Who cannot join
- Part of the care out of France
- Study disagreement
Trial locations and contacts
18 sites in 1 country.
18 sites in 1 country
France18 sites
Angers University Hospital
Angers, 49000
Bordeaux University Hospital
Bordeaux, 33000
Caen University Hospital
Caen, 14000
Grenoble University Hospital
Grenoble, 38000
Kremlin-Bicêtre Hospital
Le Kremlin-Bicêtre, 94000
Lille University Hospital
Lille, 59000
Lyon Universty Hospital
Lyon, 69000
Marseille University Hospital
Marseille, 13000
Montpellier University Hospital
Montpellier, 34000
Nantes University Hospital
Nantes, 44000
Nice University Hospital
Nice, 06000
Necher Hospital
Paris, 75000
Poitiers University Hospital
Poitiers, 86000
Curie Institute
Saint-Cloud, 92000
Réunion University Hospital
Saint-Paul, 97000
Toulouse University Hospital
Toulouse, 31000
Tours University Hospital
Tours, 37000
Gustave Roussy Institute
Villejuif, 94000
About this study
Pleuropulmonary blastoma is a rare embryonic malignant tumour that remains the most frequent malignant tumour of the lung in childhood. The International Pleuro pulmonary Blastoma Registry (IPPB) found only 220 cases in 2009 and 350 in 2015. In France, 20 cases were identified in 2009.
Three histologies are described: type 1 purely cystic, type 2 combined and type 3 solid. Median age at diagnostic is 12 months, 35 months and 41 months respectively. Evolution is possible from type 1 to type 2 or 3 in 10% of the cases.
Read more
Since 2009, DICER 1 mutations research is proposed systematically to all families.
PPB symptoms are usually non-specific. Diagnostic is evoked when imaging work up shows bubbles or solid lesions, and confirmed by pathological analysis. However the diagnosis can be difficult because of the proximity with congenital cystic adenomatoid malformation.
The French society of paediatric oncology recommends surgery at first instance. PPB type 1 remains a problem because some are still misdiagnosed as CCAM, a benign lesion. Chemotherapy depends on the PPB type and the quality of the resection. There is a real interest to analyse the French series.
The prognosis of type 2 and 3 is low with a 5 years survival rate of 45-60%, whereas type 1 survival rate is 91%. The French experience reports a 100% survival rate in type 1 and 48% in type 2 and 3. Other prognostic factors are initial size of the tumour, extra pulmonary invasion and quality of surgery.
Early local relapses are possible and late ones concern more often type 2 and 3 with more cerebral metastasis.
In 2009, the french cases were collected, but no update has been performed since. The aim of this retrospective review of the cases since 2000, is to audit the care of PPB patients in France and update the French rare tumour database.
Evoking PPB diagnosis is difficult when imaging shows a neonatal cystic lesion. There are no radiologic criteria in the literature that differentiate congenital pulmonary cystic lesion and PPB type 1. Radiological presentation is however overlapping. Another aim of this study will be to look for a predictive sign of type 1 PPB.
Read the detailed description
Multicentre retrospective study
- Conditions
- Pleuropulmonary Blastoma
- Being tested
- PPB
- Medical subject headings
- Pleuropulmonary blastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PPBOther
Global current care
| Study group | Role | What they receive |
|---|---|---|
| Pleuropulmonary Blastoma | * Patients born between 01/01/2000 and 01/01/2016 ;
* Followed up for PPB
* Treated in a French department of paediatric oncology or paediatric surgery
* Study agreement Receives: Other: PPB |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall survival
Measured: Within 5 years from the diagnosis
Progression free survival
Characterize the evolution and the global care of PPB in the French series
Measured: Within 5 years from the diagnosis
Secondary outcomes (2)
State of PPB surgical care
Measured: Within 5 years from the diagnosis
Radiology sign
Identification of a type 1 PPB predictive radiological sign
Measured: Within 5 years from the diagnosis
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Feb 2017 (estimated)
- Study start9 Jan 2017
- Primary completion22 Feb 2018
- Study completion9 Jul 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pleuropulmonary BlastomaStudies of the same condition, open and under way.
- More trials run by Rennes University HospitalThe sponsor's other registered studies.
- 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
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 41 participants (actual)
Sponsor and investigators
- Lead sponsor
- Rennes University Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Alexis ARNAUD, MD, Principal investigator, Rennes University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03044834
- Sponsor's protocol ID
- 35RC15_3009_PPB
- Other IDs
- 15.18, 15.468, 915465
- Acronym
- PPB
- Status last verified
- Aug 2020
- First submitted
- 16 Jan 2017
- Last updated
- 12 Aug 2020
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Aug 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Review of the Paediatric Pleuropulmonary Blastoma French Series
- ClinicalTrials.gov ID
- NCT03044834
- Acronym / sponsor ID
- PPB / 35RC15_3009_PPB
- Conditions
- Pleuropulmonary Blastoma
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 41 participants (actual)
- Lead sponsor
- Rennes University HospitalUniversity, hospital or other
- Study start
- 9 Jan 2017 (actual)
- Primary completion
- 22 Feb 2018 (actual)
- Study completion
- 9 Jul 2018 (actual)
- First posted
- 7 Feb 2017 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Pleuropulmonary blastoma is a rare embryonic malignant tumour that remains the most frequent malignant tumour of the lung in childhood. The International Pleuro pulmonary Blastoma Registry (IPPB) found only 220 cases in 2009 and 350 in 2015. In France, 20 cases were identified in 2009.
Three histologies are described: type 1 purely cystic, type 2 combined and type 3 solid. Median age at diagnostic is 12 months, 35 months and 41 months respectively. Evolution is possible from type 1 to type 2 or 3 in 10% of the cases.
Since 2009, DICER 1 mutations research is proposed systematically to all families.
PPB symptoms are usually non-specific. Diagnostic is evoked when imaging work up shows bubbles or solid lesions, and confirmed by pathological analysis. However the diagnosis can be difficult because of the proximity with congenital cystic adenomatoid malformation.
The French society of paediatric oncology recommends surgery at first instance. PPB type 1 remains a problem because some are still misdiagnosed as CCAM, a benign lesion. Chemotherapy depends on the PPB type and the quality of the resection. There is a real interest to analyse the French series.
The prognosis of type 2 and 3 is low with a 5 years survival rate of 45-60%, whereas type 1 survival rate is 91%. The French experience reports a 100% survival rate in type 1 and 48% in type 2 and 3. Other prognostic factors are initial size of the tumour, extra pulmonary invasion and quality of surgery.
Early local relapses are possible and late ones concern more often type 2 and 3 with more cerebral metastasis.
In 2009, the french cases were collected, but no update has been performed since. The aim of this retrospective review of the cases since 2000, is to audit the care of PPB patients in France and update the French rare tumour database.
Evoking PPB diagnosis is difficult when imaging shows a neonatal cystic lesion. There are no radiologic criteria in the literature that differentiate congenital pulmonary cystic lesion and PPB type 1. Radiological presentation is however overlapping. Another aim of this study will be to look for a predictive sign of type 1 PPB.
Detailed description
Multicentre retrospective study
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Pleuropulmonary Blastoma | – | Other: PPB* Patients born between 01/01/2000 and 01/01/2016 ; * Followed up for PPB * Treated in a French department of paediatric oncology or paediatric surgery * Study agreement |
Who may qualify
- Ages
- 1 year to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Patients born between 01/01/2000 and 01/01/2016, followed up for PPB, treated in a French department of paediatric oncology or paediatric surgery.
Key inclusion criteria (4 listed)
- Patients born between 01/01/2000 and 01/01/2016 ;
- Followed up for PPB
- Treated in a French department of paediatric oncology or paediatric surgery
- Study agreement
Key exclusion criteria (2 listed)
- Part of the care out of France
- Study disagreement
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PPBGlobal current care | Other | Pleuropulmonary Blastoma |
Primary outcome
Overall survival
Time frame: Within 5 years from the diagnosis
Progression free survival
Time frame: Within 5 years from the diagnosis
Characterize the evolution and the global care of PPB in the French series
2 secondary outcomes
State of PPB surgical care
Time frame: Within 5 years from the diagnosis
Radiology sign
Time frame: Within 5 years from the diagnosis
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Aug 2020.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Alexis ARNAUD, MD principal investigator
Rennes University Hospital
18 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Pleuropulmonary Blastoma, run by Rennes University Hospital at 18 sites in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 12 Aug 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 18 sites in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall survival (time frame: Within 5 years from the diagnosis). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Aug 2020.