NeuroblastomaNCT01355679
Molecular Guided Therapy for Refractory or Recurrent Neuroblastoma
A study testing Guided Therapy in people with Neuroblastoma, run by Giselle Sholler at 7 sites in United States.
Trial summary
The purpose of this study is to test the feasibility (ability to be done) of an experimental test to help plan your cancer treatment. This study plan is not studying the effectiveness of the proposed combinations of therapy for your cancer that you may receive after the experimental testing. Read more about this study
- Who is it for?
- Ages 1 year and older
- Conditions the study is for
- Neuroblastoma
- What is being tested?
- Guided Therapy
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 16 participants
- Where
- 7 sitesUnited States
- Study dates
- May 2011to Jan 2015
- Phase
- Interventional
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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 ≤ 21 at initial diagnosis.
- 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); tumor must be accessible for biopsy. Patients with bone marrow only disease expected to be > 75% are eligible to enroll.
- Current disease state must be one for which there is currently no known curative therapy
- Lansky or KarnofskyScore must be more than 50
Show all 14Show fewer
- 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 NCI-CTCAE V4.0
- 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 cannot 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 7 days prior to enrollment and 14 days prior to study treatment start date.
- Patients who have received any radiotherapy within the last 30 days must have another site of disease to follow.
- 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 V4.0), or active, serious infections requiring parenteral antibiotic therapy.
- 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
7 sites in 1 country.
7 sites in 1 country
United States7 sites
National Cancer Institute
Bethesda, Maryland, 20877
Levine Children's Hospital
Charlotte, North Carolina, 28204
Helen DeVos Children's Hospital
Grand Rapids, Michigan, 49503
Connecticut Children's Hospital
Hartford, Connecticut, 06106
Children's Mercy Hospitals and Clinics
Kansas City, Missouri, 64108
Arnold Palmer Hospital for Children- MD Anderson
Orlando, Florida, 32806
Cardinal Glennon Children's Medical Center
St Louis, Missouri, 63104
About this study
Official title: A Feasibility Trial Using Molecular-Guided Therapy for the Treatment of Patients With Refractory or Recurrent Neuroblastoma
The purpose of this study is to test the feasibility (ability to be done) of an experimental test to help plan your cancer treatment. This study plan is not studying the effectiveness of the proposed combinations of therapy for your cancer that you may receive after the experimental testing.
This study will look at an experimental technology to determine a tumor's molecular makeup (gene expression profile). This technology (called "OncInsights") is being used to discover new ways to understand cancers and potentially predict the best treatments for patients with cancer in the future. The experimental technology has not been approved by the U.S. Food and Drug Administration.
- Conditions
- Neuroblastoma
- Being tested
- Guided Therapy
- Medical subject headings
- Neuroblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Guided TherapyDevice
A total of 14 eligible neuroblastoma patients who are refractory or relapsed on conventional therapy will be treated. Guided therapy will allow the use of any therapeutic combination (up to 4 agents) provided it includes medications contained in the study report. All patients will be followed for disease response, progression and safety. All patients will be treated according to the discretion of the treating oncologist and study committee (minimum 3 oncologists and one pharmacist). Extent of disease will be measured and assessed for changes throughout the course of the study and at 6-8 week intervals (every 2 cycles).
| Study group | Role | What they receive |
|---|---|---|
| Guided therapy | Experimental | A total of 14 eligible neuroblastoma patients who are refractory or relapsed on conventional therapy will be treated. Guided therapy will allow the use of any therapeutic combination (up to 4 agents) provided it includes medications contained in the study report. All patients will be followed for survival, disease response, progression and safety. All patients will be treated according to the discretion of the treating oncologist and study committee (minimum 3 oncologists and one pharmacist). Extent of disease will be measured and assessed for changes throughout the course of the study and at 6-8 week intervals (every 2 cycles). Receives: Device: Guided Therapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants That Are Able to Meet Feasibility Parameters.
Feasibility parameter defined as: Enrollment onto study, quality mRNA obtained, gene chip completed, tumor board held, medical monitor review and approval, start of treatment by 21 days post biopsy/surgical resection date, and then completion of 1 cycle of therapy."
Measured: 1 year
Secondary outcomes (3)
Number of Participants With Adverse Events as a Measure of Safety
To determine the safety of allowing a molecular tumor board to determine individualized treatment plans
Measured: 1 year
Overall Response Rate (ORR) of Participants Using RECIST Criteria
Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), >=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR.
Measured: 1 year
Activity of Treatments Chosen Based on Progression Free Survival (PFS)
Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 May 2011 (estimated)
- Study startMay 2011
- Primary completionSep 2013
- Study completionJan 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 16 participants (actual)
Sponsor and investigators
- Lead sponsor
- Giselle Sholler
- Sponsor type
- University, hospital or other
- 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
- NCT01355679
- Sponsor's protocol ID
- NMTRC 001
- Status last verified
- Aug 2024
- First submitted
- 13 May 2011
- Last updated
- 6 Aug 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
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
- A Feasibility Trial Using Molecular-Guided Therapy for the Treatment of Patients With Refractory or Recurrent Neuroblastoma
- ClinicalTrials.gov ID
- NCT01355679
- Acronym / sponsor ID
- NMTRC 001
- Conditions
- Neuroblastoma
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 16 participants (actual)
- Lead sponsor
- Giselle ShollerUniversity, hospital or other
- Study start
- May 2011
- Primary completion
- Sep 2013 (actual)
- Study completion
- Jan 2015 (actual)
- First posted
- 18 May 2011 (estimated)
- Results
- Posted 28 Jul 2016
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to test the feasibility (ability to be done) of an experimental test to help plan your cancer treatment. This study plan is not studying the effectiveness of the proposed combinations of therapy for your cancer that you may receive after the experimental testing.
This study will look at an experimental technology to determine a tumor's molecular makeup (gene expression profile). This technology (called "OncInsights") is being used to discover new ways to understand cancers and potentially predict the best treatments for patients with cancer in the future. The experimental technology has not been approved by the U.S. Food and Drug Administration.
Study design
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Guided therapy | Experimental | Device: Guided TherapyA total of 14 eligible neuroblastoma patients who are refractory or relapsed on conventional therapy will be treated. Guided therapy will allow the use of any therapeutic combination (up to 4 agents) provided it includes medications contained in the study report. All patients will be followed for survival, disease response, progression and safety. All patients will be treated according to the discretion of the treating oncologist and study committee (minimum 3 oncologists and one pharmacist). Extent of disease will be measured and assessed for changes throughout the course of the study and at 6-8 week intervals (every 2 cycles). |
Who may qualify
- Ages
- 1 year and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
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 ≤ 21 at initial diagnosis.
- 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); tumor must be accessible for biopsy. Patients with bone marrow only disease expected to be > 75% are eligible to enroll.
- Current disease state must be one for which there is currently no known curative therapy
Key exclusion criteria (5 listed)
- Patients who have received any chemotherapy within the last 7 days prior to enrollment and 14 days prior to study treatment start date.
- Patients who have received any radiotherapy within the last 30 days must have another site of disease to follow.
- 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 V4.0), or active, serious infections requiring parenteral antibiotic therapy.
- 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.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Guided TherapyA total of 14 eligible neuroblastoma patients who are refractory or relapsed on conventional therapy will be treated. Guided therapy will allow the use of any therapeutic combination (up to 4 agents) provided it includes medications contained in the study report. All patients will be followed for disease response, progression and safety. All patients will be treated according to the discretion of the treating oncologist and study committee (minimum 3 oncologists and one pharmacist). Extent of disease will be measured and assessed for changes throughout the course of the study and at 6-8 week intervals (every 2 cycles). | Device | Guided therapy |
Primary outcome
Percentage of Participants That Are Able to Meet Feasibility Parameters.
Time frame: 1 year
Feasibility parameter defined as: Enrollment onto study, quality mRNA obtained, gene chip completed, tumor board held, medical monitor review and approval, start of treatment by 21 days post biopsy/surgical resection date, and then completion of 1 cycle of therapy."
3 secondary outcomes
Number of Participants With Adverse Events as a Measure of Safety
Time frame: 1 year
Overall Response Rate (ORR) of Participants Using RECIST Criteria
Time frame: 1 year
Activity of Treatments Chosen Based on Progression Free Survival (PFS)
Time frame: 1 year
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Aug 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Beat Childhood CancerLink provided by the sponsor.
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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.
Giselle Sholler, MD study chair
Beat Childhood Cancer at Atrium Health
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Guided Therapy in people with Neuroblastoma, run by Giselle Sholler at 7 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?
The sponsor lists 14 inclusion and 5 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 7 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?
Yes. Results are posted in the registry (first posted 28 Jul 2016). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Percentage of Participants That Are Able to Meet Feasibility Parameters. (time frame: 1 year). 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.