Refractory NeuroblastomaNCT01109238
A Trial Using Molecular-Guided Therapy in Patients With Refractory or Recurrent Neuroblastoma
An observational study of Refractory Neuroblastoma and Relapsed Neuroblastoma, run by Giselle Sholler at 1 site in United States.
Trial summary
The investigators are studying new ways to make treatment decisions for these types of cancer. Technologies at the Van Andel Research Institute (VARI) are available to determine a tumor's molecular makeup (gene expression profile). Read more about this study
- Who is it for?
- Ages 12 months to 21 years
- Conditions the study is for
- Refractory Neuroblastoma, Relapsed Neuroblastoma
- How many people
- 5 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2010to Jul 2010
- 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 12 months to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Relapsed and refractory Neuroblastoma
Who may be able to join
- Patients must have histologically proven neuroblastoma and confirmation of refractory or recurrent disease with histologic confirmation at diagnosis or at the time of recurrence/progression
- Patients must be age >12 months and diagnosed before the age of 21
- Life expectancy must be more than 3 months
- If measurable disease, this must be demonstrated by residual abnormal tissue at a primary or metastatic site measuring more than 1 cm in any dimension by standardized imaging (CT or MRI). For patients with only skeletal sites of disease, there must be at least one persisting focus with increased activity on a pre-treatment meta-iodobenzylguanidine (MIBG) scan.
- Current disease state must be one for which there is currently no known curative therapy
- Lansky Play Score must be more than 30
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- Patients without bone marrow metastases must have an ANC > 750/μl and platelet count >50,000/μl
- Adequate liver function must be demonstrated, defined as:
- Total bilirubin ≤ 1.5 x upper limit of normal (ULN) for age AND
- SGPT (ALT) < 10 x upper limit of normal (ULN) for age
- No other significant organ toxicity defined as >Grade 2 by National Cancer Institute Common Toxicity Criteria for Adverse Events version 3 (NCI-CTCAE V3.0 (http://ctep.cancer.gov/forms/CTCAEv3.pdf))
- A negative serum pregnancy test is required for female participants of child bearing potential (≥13 years of age or after onset of menses)
- Both male and female post-pubertal study subjects need to agree to use one of the more effective birth control methods during treatment and for six months after treatment is stopped. These methods include total abstinence (no sex), oral contraceptives ("the pill"), an intrauterine device (IUD), levonorgestrol implants (Norplant), or medroxyprogesterone acetate injections (Depo-provera shots). If one of these can not be used, contraceptive foam with a condom is recommended.
- Informed Consent: All patients and/or legal guardians must sign informed written consent. Assent, when appropriate, will be obtained according to institutional guidelines. Voluntary consent for optional biology studies will be included.
Who cannot join
- Patients who have received any chemotherapy within the last 21 days.
- Patients receiving anti-tumor therapy for their disease or any investigational drug concurrently
- Patients with serious infection or a life-threatening illness (unrelated to tumor) that is > Grade 2 (NCI CTCAE V3.0), or active, serious infections requiring parenteral antibiotic therapy within 2 weeks prior to screening
- Patients with any other medical condition, including malabsorption syndromes, mental illness or substance abuse, deemed by the Investigator to be likely to interfere with the interpretation of the results or which would interfere with a patient's ability to sign or the legal guardian's ability to sign the informed consent, and patient's ability to cooperate and participate in the study
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Trial Testing the Feasibility of Using Molecular-Guided Therapy in Patients With Refractory or Recurrent Neuroblastoma
The investigators are studying new ways to make treatment decisions for these types of cancer. Technologies at the Van Andel Research Institute (VARI) are available to determine a tumor's molecular makeup (gene expression profile). This technology (called "Xenobase") is being used to discover new ways to understand cancers and potentially predict the best treatments for patients with cancer. The researchers at VARI have filed a patent on the Xenobase and the specific network analysis method that the investigators will be using as part of this study.
A specimen obtained from the tumor during a recent surgical, biopsy, or bone marrow procedure will be sent to the Van Andel Research Institute. Researchers will attempt to identify the molecular makeup within the specimen, as well as in blood and urine samples in patients with aggressive and/or refractory cancer. This additional testing is different than the routine tests currently performed at the hospital for the evaluation of cancer.
The goals of this part of the study are: To determine if the investigators tumor board committee (at minimum a panel of 3 oncologists and 1 pharmacist) can use patient specific cancer cells to make real-time treatment decision using patient specific genetic information, and predicted therapies generated in the Xenobase report.
- Conditions
- Refractory Neuroblastoma, Relapsed Neuroblastoma
- Keywords
- Refractory Neuroblastoma, Relapsed Neuroblastoma
- Medical subject headings
- Neuroblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Process Feasibility | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
Feasibility of using protocol process to make real time treatment decisions
To pilot 5 patients to evaluate the feasibility of using predictive modeling based on genome-wide mRNA expression profiles of bone marrow derived neuroblastoma cells or tumor biopsies to make real-time treatment decisions.
Measured: 4 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Apr 2010 (estimated)
- Study startApr 2010
- Primary completionJul 2010
- Study completionJul 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Refractory NeuroblastomaStudies 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
- Prospective
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- Giselle Sholler
- Sponsor type
- University, hospital or other
- Collaborators
- Van Andel Research Institute
- Responsible party
- Sponsor-investigator, Giselle Sholler, Milton S. Hershey Medical Center
- Investigators
- Giselle Sholler, MD, Study chair, Beat Childhood Cancer at Atrium Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01109238
- Sponsor's protocol ID
- NMTRC 001P
- Status last verified
- Aug 2024
- First submitted
- 21 Apr 2010
- Last updated
- 6 Aug 2024
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Trial Testing the Feasibility of Using Molecular-Guided Therapy in Patients With Refractory or Recurrent Neuroblastoma
- ClinicalTrials.gov ID
- NCT01109238
- Acronym / sponsor ID
- NMTRC 001P
- Conditions
- Refractory Neuroblastoma, Relapsed Neuroblastoma
- Keywords
- Refractory Neuroblastoma, Relapsed Neuroblastoma
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 5 participants (actual)
- Lead sponsor
- Giselle ShollerUniversity, hospital or other
- Collaborators
- Van Andel Research Institute
- Study start
- Apr 2010
- Primary completion
- Jul 2010 (actual)
- Study completion
- Jul 2010 (actual)
- First posted
- 23 Apr 2010 (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.
The investigators are studying new ways to make treatment decisions for these types of cancer. Technologies at the Van Andel Research Institute (VARI) are available to determine a tumor's molecular makeup (gene expression profile). This technology (called "Xenobase") is being used to discover new ways to understand cancers and potentially predict the best treatments for patients with cancer. The researchers at VARI have filed a patent on the Xenobase and the specific network analysis method that the investigators will be using as part of this study.
A specimen obtained from the tumor during a recent surgical, biopsy, or bone marrow procedure will be sent to the Van Andel Research Institute. Researchers will attempt to identify the molecular makeup within the specimen, as well as in blood and urine samples in patients with aggressive and/or refractory cancer. This additional testing is different than the routine tests currently performed at the hospital for the evaluation of cancer.
The goals of this part of the study are: To determine if the investigators tumor board committee (at minimum a panel of 3 oncologists and 1 pharmacist) can use patient specific cancer cells to make real-time treatment decision using patient specific genetic information, and predicted therapies generated in the Xenobase report.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Process Feasibility | – | – |
Who may qualify
- Ages
- 12 months to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Relapsed and refractory Neuroblastoma
Key inclusion criteria (14 listed)
- Patients must have histologically proven neuroblastoma and confirmation of refractory or recurrent disease with histologic confirmation at diagnosis or at the time of recurrence/progression
- Patients must be age >12 months and diagnosed before the age of 21
- Life expectancy must be more than 3 months
- If measurable disease, this must be demonstrated by residual abnormal tissue at a primary or metastatic site measuring more than 1 cm in any dimension by standardized imaging (CT or MRI). For patients with only skeletal sites of disease, there must be at least one persisting focus with increased activity on a pre-treatment meta-iodobenzylguanidine (MIBG) scan.
- Current disease state must be one for which there is currently no known curative therapy
Key exclusion criteria (4 listed)
- Patients who have received any chemotherapy within the last 21 days.
- Patients receiving anti-tumor therapy for their disease or any investigational drug concurrently
- Patients with serious infection or a life-threatening illness (unrelated to tumor) that is > Grade 2 (NCI CTCAE V3.0), or active, serious infections requiring parenteral antibiotic therapy within 2 weeks prior to screening
- Patients with any other medical condition, including malabsorption syndromes, mental illness or substance abuse, deemed by the Investigator to be likely to interfere with the interpretation of the results or which would interfere with a patient's ability to sign or the legal guardian's ability to sign the informed consent, and patient's ability to cooperate and participate in the study
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
Feasibility of using protocol process to make real time treatment decisions
Time frame: 4 months
To pilot 5 patients to evaluate the feasibility of using predictive modeling based on genome-wide mRNA expression profiles of bone marrow derived neuroblastoma cells or tumor biopsies to make real-time treatment decisions.
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 6 Aug 2024.
- Related InfoLink provided by the sponsor.
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Neuroblastoma
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.
Giselle Sholler, MD study chair
Beat Childhood Cancer at Atrium Health
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Refractory Neuroblastoma and Relapsed Neuroblastoma, run by Giselle Sholler at 1 site 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?
The sponsor lists 14 inclusion and 4 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 6 Aug 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 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?
Feasibility of using protocol process to make real time treatment decisions (time frame: 4 months). 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 6 Aug 2024.