Brain and Central Nervous System TumorsNCT01353300
Gene Mutation in Samples From Young Patients With Pleuropulmonary Blastoma Syndrome at Risk for Developing Cancer
An observational study of Brain and Central Nervous System Tumors and Hereditary Wilms Tumor, run by Children's Oncology Group.
Trial summary
RATIONALE: The identification of gene mutations in young patients with pleuropulmonary blastoma syndrome may allow doctors to better understand the genetic processes involved in the development of some types of cancer, and may also help doctors identify patients who are at risk for cancer. Read more about this study
- Who is it for?
- Ages Up to 120 years
- Conditions the study is for
- Brain and Central Nervous System Tumors, Hereditary Wilms Tumor, Kidney Cancer
- What is being tested?
- DNA analysis, cytogenetic analysis and gene expression analysis
- How many people
- 31 participants
- Where
- No sites listed yet
- Study dates
- May 2011to Jul 2016
- 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 Up to 120 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients with a diagnosis of pleuropulmonary blastoma syndrome.
DISEASE CHARACTERISTICS:
- Diagnosis of pleuropulmonary blastoma syndrome
- Normal tissue samples, if available
- Parental and sibling DNA samples, if available
PATIENT CHARACTERISTICS:
- Not specified
PRIOR CONCURRENT THERAPY:
- Not specified
About this study
Official title: Investigation of DICER1 in Cystic Nephroma and Cystic Partially Differentiated Nephroblastoma
RATIONALE: The identification of gene mutations in young patients with pleuropulmonary blastoma syndrome may allow doctors to better understand the genetic processes involved in the development of some types of cancer, and may also help doctors identify patients who are at risk for cancer.
PURPOSE: This research study studies gene mutations in samples from young patients with pleuropulmonary blastoma syndrome at risk for developing cancer.
Read the detailed description
OBJECTIVES:
- To determine if DICER1 mutations contribute to tumor pathogenesis in cystic nephromas and cystic partially differentiated nephroblastomas outside of families with pleuropulmonary blastoma (PPB) syndrome.
OUTLINE: Archived DNA samples are analyzed for DICER1 mutation by qPCR and directly sequenced using BigDye Terminator chemistry. Results are then compared against the single nucleotide polymorphism (SNP) database.
- Conditions
- Brain and Central Nervous System Tumors, Hereditary Wilms Tumor, Kidney Cancer, Liver Cancer, Neuroblastoma, Pleuropulmonary Blastoma
- Being tested
- DNA analysis, cytogenetic analysis, gene expression analysis, gene rearrangement analysis, mutation analysis
- Keywords
- embryonal childhood rhabdomyosarcoma, cystic nephroma, neuroblastoma, childhood medulloblastoma, childhood hepatoblastoma, stromal predominant Wilms tumor, hereditary Wilms tumor, pleuropulmonary blastoma
- Medical subject headings
- Central Nervous System Neoplasms, Kidney Neoplasms, Liver Neoplasms, Neuroblastoma, Pleuropulmonary blastoma, Sarcoma, Medulloblastoma, Hepatoblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DNA analysisGenetic
cytogenetic analysisGenetic
gene expression analysisGenetic
gene rearrangement analysisGenetic
mutation analysisGenetic
polymerase chain reactionGenetic
polymorphism analysisGenetic
laboratory biomarker analysisOther
medical chart reviewOther
What is being measured
The primary outcome is the main question the study is designed to answer.
Relationship between DICER1 mutations and tumor pathogenesis in cystic nephromas and cystic partially differentiated nephroblastomas outside of families with PPB nephroblastomas
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 May 2011 (estimated)
- Study startMay 2011
- Primary completionJul 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Brain and Central Nervous System TumorsStudies of the same condition, open and under way.
- More trials run by Children's Oncology GroupThe 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
- Case only
- Time perspective
- Retrospective
- Enrolment
- 31 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Children's Oncology Group
- Sponsor type
- Research network
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Sponsor
- Investigators
- Dana A. Hill, MD, Principal investigator, Children's National Research Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01353300
- Sponsor's protocol ID
- AREN11B2
- Other IDs
- COG-AREN11B2, NCI-2011-02854, AREN11B2
- Status last verified
- Jul 2016
- First submitted
- 12 May 2011
- Last updated
- 11 Jul 2016
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Jul 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Investigation of DICER1 in Cystic Nephroma and Cystic Partially Differentiated Nephroblastoma
- ClinicalTrials.gov ID
- NCT01353300
- Acronym / sponsor ID
- AREN11B2
- Conditions
- Brain and Central Nervous System Tumors, Hereditary Wilms Tumor, Kidney Cancer, Liver Cancer, Neuroblastoma, Pleuropulmonary Blastoma
- Keywords
- embryonal childhood rhabdomyosarcoma, cystic nephroma, neuroblastoma, childhood medulloblastoma, childhood hepatoblastoma, stromal predominant Wilms tumor, hereditary Wilms tumor, pleuropulmonary blastoma
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 31 participants (estimated)
- Lead sponsor
- Children's Oncology GroupResearch network
- Collaborators
- National Cancer Institute (NCI)
- Study start
- May 2011
- Primary completion
- Jul 2016 (actual)
- First posted
- 13 May 2011 (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.
RATIONALE: The identification of gene mutations in young patients with pleuropulmonary blastoma syndrome may allow doctors to better understand the genetic processes involved in the development of some types of cancer, and may also help doctors identify patients who are at risk for cancer.
PURPOSE: This research study studies gene mutations in samples from young patients with pleuropulmonary blastoma syndrome at risk for developing cancer.
Detailed description
OBJECTIVES:
- To determine if DICER1 mutations contribute to tumor pathogenesis in cystic nephromas and cystic partially differentiated nephroblastomas outside of families with pleuropulmonary blastoma (PPB) syndrome.
OUTLINE: Archived DNA samples are analyzed for DICER1 mutation by qPCR and directly sequenced using BigDye Terminator chemistry. Results are then compared against the single nucleotide polymorphism (SNP) database.
Study design
- Observational model
- Case only
- Time perspective
- Retrospective
Who may qualify
- Ages
- Up to 120 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with a diagnosis of pleuropulmonary blastoma syndrome.
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 |
|---|---|---|
| DNA analysis | Genetic | – |
| cytogenetic analysis | Genetic | – |
| gene expression analysis | Genetic | – |
| gene rearrangement analysis | Genetic | – |
| mutation analysis | Genetic | – |
| polymerase chain reaction | Genetic | – |
| polymorphism analysis | Genetic | – |
| laboratory biomarker analysis | Other | – |
| medical chart review | Other | – |
Primary outcome
Relationship between DICER1 mutations and tumor pathogenesis in cystic nephromas and cystic partially differentiated nephroblastomas outside of families with PPB nephroblastomas
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 11 Jul 2016.
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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.
Dana A. Hill, MD principal investigator
Children's National Research Institute
Questions about this trial
What is this clinical trial studying?
An observational study of Brain and Central Nervous System Tumors and Hereditary Wilms Tumor, run by Children's Oncology Group.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
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 11 Jul 2016. Status can change between updates, so confirm with the study contact before making plans.
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?
Relationship between DICER1 mutations and tumor pathogenesis in cystic nephromas and cystic partially differentiated nephroblastomas outside of families with PPB nephroblastomas. 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 11 Jul 2016.