Central Nervous System Tumor, PediatricNCT00010101
Genetic Study of Brain Tumors in Young Children
An observational study of Central Nervous System Tumor, Pediatric, run by Pediatric Brain Tumor Consortium at 4 sites in United States.
Trial summary
RATIONALE: Genetic studies may help in understanding the genetic processes involved in the development of some types of cancer. PURPOSE: Genetic study to understand how genes may be involved in the development of brain tumors in young children. Read more about this study
- Who is it for?
- Ages Up to 3 years
- Conditions the study is for
- Central Nervous System Tumor, Pediatric
- How many people
- 38 participants
- Where
- 4 sitesUnited States
- Study dates
- Mar 2001to Aug 2004
- 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 3 years
- All sexes
- No healthy volunteers
- Child
Study population: Tissue samples from infants (< 3 years old) with histologically confirmed primary intracranial CNS medulloblastoma/PNET, atypical teratoid/rhabdoid tumor, or choroid plexus carcinoma and with no prior chemotherapy, radiotherapy, or treatment from any other investigational agent will be used for this research study. Patients who meet the eligibility criteria, are treated at a PBTC institution that has IRB approval of this study, and consent to the usage of stored tumor specimens for the study objectives constitute the study population.
DISEASE CHARACTERISTICS:
- Histologically confirmed primary intracranial central nervous system tumor
- Medulloblastoma
- Primitive neuroectodermal tumor
- Atypical teratoid/rhabdoid tumor
- Choroid plexus carcinoma
- Potential enrollment on PBTC-001 therapeutic protocol
PATIENT CHARACTERISTICS:
Age:
- Under 3
Performance status:
- Not specified
Life expectancy:
- Not specified
Hematopoietic:
- Not specified
Hepatic:
- Not specified
Renal:
- Not specified
PRIOR CONCURRENT THERAPY:
Biologic therapy:
- Not specified
Chemotherapy:
- No prior chemotherapy
Endocrine therapy:
- Prior steroids allowed
Radiotherapy:
- No prior radiotherapy
Surgery:
- Not specified
Other:
- No concurrent investigational agents
Trial locations and contacts
4 sites in 1 country.
United States4 sites
Texas Children's Cancer Center
Houston, Texas, 77030-2399
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104-4318
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15213
Children's National Medical Center
Washington D.C., District of Columbia, 20010-2970
About this study
Official title: INI1 Mutation Analysis and Expression Profiling of Embryonal CNS Tumors
RATIONALE: Genetic studies may help in understanding the genetic processes involved in the development of some types of cancer.
PURPOSE: Genetic study to understand how genes may be involved in the development of brain tumors in young children.
Read the detailed description
OBJECTIVES:
- Determine the frequency and type of deletions and mutations of the INI1 gene in infants with embryonal central nervous system tumors.
- Compare the gene expression profiles in infants with atypical teratoid/rhabdoid tumors vs medulloblastoma or primitive neuroectodermal tumor.
OUTLINE: This is a multicenter study.
Tumor samples are analyzed by fluorescence in situ hybridization (FISH) for deletions of INI1 gene in chromosome band 22q11.2. Tumors without demonstration of deletions of INI1 gene by FISH are examined by polymerase chain reaction (PCR)-based microsatellite analysis for loss of heterozygosity using markers that map to 22q11.2.
DNA from tumor tissue is analyzed for mutations in the exons of the INI1 gene. Isolated matched normal DNA may be analyzed for identification of germline mutations. Parental DNA may be analyzed to identify inherited germline mutations of the INI1 gene.
The patient's physician may receive the results of the genetic testing. The results do not influence the type or duration of treatment.
PROJECTED ACCRUAL: A total of 50 patients will be accrued for this study within 25 months.
- Conditions
- Central Nervous System Tumor, Pediatric
- Keywords
- childhood choroid plexus tumor, untreated childhood supratentorial primitive neuroectodermal tumor, untreated childhood medulloblastoma, childhood atypical teratoid/rhabdoid tumor
- Medical subject headings
- Central Nervous System Neoplasms, Choroid Plexus Neoplasms, Rhabdoid Tumor
What is being measured
The primary outcome is the main question the study is designed to answer.
Deletions and mutations of the INI1 gene in infants with AT/RT, medulloblastoma, PNET, or choroid plexus carcinoma
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jan 2003 (estimated)
- Study startMar 2001
- Primary completionAug 2004
- Study completionAug 2004
Why it stopped: Poor accrual
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Central Nervous System Tumor, PediatricStudies of the same condition, open and under way.
- 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
- Cross-sectional
- Enrolment
- 38 participants (actual)
Sponsor and investigators
- Lead sponsor
- Pediatric Brain Tumor Consortium
- Sponsor type
- Research network
- Collaborators
- National Cancer Institute (NCI)
- Investigators
- Jaclyn A. Biegel, PhD, Study chair, Children's Hospital of Philadelphia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00010101
- Sponsor's protocol ID
- CDR0000068445
- Other IDs
- PBTC-N03
- Status last verified
- Jul 2011
- First submitted
- 2 Feb 2001
- Last updated
- 20 Jul 2011
- Results posted
- No
- Data monitoring committee
- No
Publications
Pomeroy SL, Tamayo P, Gaasenbeek M, Sturla LM, Angelo M, McLaughlin ME, Kim JY, Goumnerova LC, Black PM, Lau C, Allen JC, Zagzag D, Olson JM, Curran T, Wetmore C, Biegel JA, Poggio T, Mukherjee S, Rifkin R, Califano A, Stolovitzky G, Louis DN, Mesirov JP, Lander ES, Golub TR. Prediction of central nervous system embryonal tumour outcome based on gene expression. Nature. 2002 Jan 24;415(6870):436-42. doi: 10.1038/415436a.
PubMed 11807556
Study at a glance
From the registry record, last updated by the sponsor on 20 Jul 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- INI1 Mutation Analysis and Expression Profiling of Embryonal CNS Tumors
- ClinicalTrials.gov ID
- NCT00010101
- Acronym / sponsor ID
- CDR0000068445
- Conditions
- Central Nervous System Tumor, Pediatric
- Keywords
- childhood choroid plexus tumor, untreated childhood supratentorial primitive neuroectodermal tumor, untreated childhood medulloblastoma, childhood atypical teratoid/rhabdoid tumor
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: Poor accrual
- Enrolment
- 38 participants (actual)
- Lead sponsor
- Pediatric Brain Tumor ConsortiumResearch network
- Collaborators
- National Cancer Institute (NCI)
- Study start
- Mar 2001
- Primary completion
- Aug 2004 (actual)
- Study completion
- Aug 2004 (actual)
- First posted
- 27 Jan 2003 (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: Genetic studies may help in understanding the genetic processes involved in the development of some types of cancer.
PURPOSE: Genetic study to understand how genes may be involved in the development of brain tumors in young children.
Detailed description
OBJECTIVES:
- Determine the frequency and type of deletions and mutations of the INI1 gene in infants with embryonal central nervous system tumors.
- Compare the gene expression profiles in infants with atypical teratoid/rhabdoid tumors vs medulloblastoma or primitive neuroectodermal tumor.
OUTLINE: This is a multicenter study.
Tumor samples are analyzed by fluorescence in situ hybridization (FISH) for deletions of INI1 gene in chromosome band 22q11.2. Tumors without demonstration of deletions of INI1 gene by FISH are examined by polymerase chain reaction (PCR)-based microsatellite analysis for loss of heterozygosity using markers that map to 22q11.2.
DNA from tumor tissue is analyzed for mutations in the exons of the INI1 gene. Isolated matched normal DNA may be analyzed for identification of germline mutations. Parental DNA may be analyzed to identify inherited germline mutations of the INI1 gene.
The patient's physician may receive the results of the genetic testing. The results do not influence the type or duration of treatment.
PROJECTED ACCRUAL: A total of 50 patients will be accrued for this study within 25 months.
Study design
- Observational model
- Case only
- Time perspective
- Cross-sectional
Who may qualify
- Ages
- Up to 3 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Tissue samples from infants (< 3 years old) with histologically confirmed primary intracranial CNS medulloblastoma/PNET, atypical teratoid/rhabdoid tumor, or choroid plexus carcinoma and with no prior chemotherapy, radiotherapy, or treatment from any other investigational agent will be used for this research study. Patients who meet the eligibility criteria, are treated at a PBTC institution that has IRB approval of this study, and consent to the usage of stored tumor specimens for the study objectives constitute the study population.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Deletions and mutations of the INI1 gene in infants with AT/RT, medulloblastoma, PNET, or choroid plexus carcinoma
Results and safety
The study was stopped early (reason given: Poor accrual). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Jul 2011.
- Results publication: PubMed 11807556Pomeroy SL, Tamayo P, Gaasenbeek M, Sturla LM, Angelo M, McLaughlin ME, Kim JY, Goumnerova LC, Black PM, Lau C, Allen JC, Zagzag D, Olson JM, Curran T, Wetmore C, Biegel JA, Poggio T, Mukherjee S, Rifkin R, Califano A, Stolovitzky G, Louis DN, Mesirov JP, Lander ES, Golub TR. Prediction of central nervous system embryonal tumour outcome based on gene expression. Nature. 2002 Jan 24;415(6870):436-42. doi: 10.1038/415436a.
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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.
Jaclyn A. Biegel, PhD study chair
Children's Hospital of Philadelphia
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Central Nervous System Tumor, Pediatric, run by Pediatric Brain Tumor Consortium at 4 sites in United States.
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: stopped early, as of the sponsor's last update on 20 Jul 2011. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States. 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 was stopped early (reason given: Poor accrual). Results are not available in the source record.
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?
Deletions and mutations of the INI1 gene in infants with AT/RT, medulloblastoma, PNET, or choroid plexus carcinoma. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00010101, which also lists 1 linked publication.
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 20 Jul 2011.