- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01355679
Molecular Guided Therapy for Refractory or Recurrent Neuroblastoma
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.
Study Overview
Study Type
Enrollment (Actual)
Phase
- Not Applicable
Contacts and Locations
Study Locations
-
-
Connecticut
-
Hartford, Connecticut, United States, 06106
- Connecticut Children's Hospital
-
-
Florida
-
Orlando, Florida, United States, 32806
- Arnold Palmer Hospital for Children- MD Anderson
-
-
Maryland
-
Bethesda, Maryland, United States, 20877
- National Cancer Institute
-
-
Michigan
-
Grand Rapids, Michigan, United States, 49503
- Helen DeVos Children's Hospital
-
-
Missouri
-
Kansas City, Missouri, United States, 64108
- Children's Mercy Hospitals and Clinics
-
Saint Louis, Missouri, United States, 63104
- Cardinal Glennon Children's Medical Center
-
-
North Carolina
-
Charlotte, North Carolina, United States, 28204
- Levine Children's Hospital
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Description
Inclusion Criteria:
- 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
- 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.
Exclusion Criteria:
- 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
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Diagnostic
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Guided therapy
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).
|
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).
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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."
|
1 year
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Number of Participants With Adverse Events as a Measure of Safety
Time Frame: 1 year
|
To determine the safety of allowing a molecular tumor board to determine individualized treatment plans
|
1 year
|
Overall Response Rate (ORR) of Participants Using RECIST Criteria
Time Frame: 1 year
|
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.
|
1 year
|
Activity of Treatments Chosen Based on Progression Free Survival (PFS)
Time Frame: 1 year
|
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
|
1 year
|
Collaborators and Investigators
Sponsor
Investigators
- Study Chair: Giselle Sholler, MD, Beat Childhood Cancer at Atrium Health
Publications and helpful links
Helpful Links
Study record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimated)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- NMTRC 001
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
product manufactured in and exported from the U.S.
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Neuroblastoma
-
Children's Oncology GroupNational Cancer Institute (NCI)Active, not recruitingStage 4S Neuroblastoma | Ganglioneuroblastoma | Stage 2A Neuroblastoma | Stage 2B Neuroblastoma | Stage 3 Neuroblastoma | Stage 4 Neuroblastoma | Stage 1 Neuroblastoma | Stage 2 NeuroblastomaUnited States, Canada, Australia, New Zealand
-
Children's Oncology GroupNational Cancer Institute (NCI)CompletedRecurrent Neuroblastoma | Disseminated Neuroblastoma | Localized Resectable Neuroblastoma | Localized Unresectable Neuroblastoma | Regional Neuroblastoma | Stage 4S NeuroblastomaUnited States
-
Children's Oncology GroupNational Cancer Institute (NCI)Active, not recruitingLocalized Resectable Neuroblastoma | Localized Unresectable Neuroblastoma | Regional Neuroblastoma | Stage 4S Neuroblastoma | Ganglioneuroblastoma | Stage 4 NeuroblastomaUnited States
-
Children's Oncology GroupNational Cancer Institute (NCI)Active, not recruitingRecurrent Neuroblastoma | Stage 4S Neuroblastoma | Stage 2A Neuroblastoma | Stage 2B Neuroblastoma | Stage 3 Neuroblastoma | Stage 4 NeuroblastomaUnited States, Canada, Australia, New Zealand
-
National Cancer Institute (NCI)Active, not recruitingRecurrent Neuroblastoma | Localized Resectable Neuroblastoma | Localized Unresectable Neuroblastoma | Regional Neuroblastoma | Stage 4S Neuroblastoma | Stage 4 NeuroblastomaUnited States, Canada, Australia, New Zealand, Puerto Rico
-
Children's Oncology GroupNational Cancer Institute (NCI)CompletedRecurrent Neuroblastoma | Localized Resectable Neuroblastoma | Localized Unresectable Neuroblastoma | Regional Neuroblastoma | Stage 4S Neuroblastoma | Stage 4 NeuroblastomaUnited States, Canada, Australia, New Zealand, Puerto Rico, Switzerland
-
Children's Oncology GroupNational Cancer Institute (NCI)RecruitingLocalized Resectable Neuroblastoma | Localized Unresectable Neuroblastoma | Regional Neuroblastoma | Stage 4S Neuroblastoma | Ganglioneuroblastoma | Stage 4 NeuroblastomaUnited States, Puerto Rico, Canada, Australia, New Zealand, Netherlands, Saudi Arabia, Switzerland
-
Children's Oncology GroupNational Cancer Institute (NCI)CompletedLocalized Resectable Neuroblastoma | Localized Unresectable Neuroblastoma | Regional Neuroblastoma | Stage 4S NeuroblastomaUnited States
-
Children's Oncology GroupNational Cancer Institute (NCI)CompletedRecurrent Neuroblastoma | Localized Resectable Neuroblastoma | Localized Unresectable Neuroblastoma | Regional Neuroblastoma | Stage 4 NeuroblastomaUnited States
-
Children's Oncology GroupNational Cancer Institute (NCI)CompletedRecurrent Neuroblastoma | Disseminated Neuroblastoma | Localized Resectable Neuroblastoma | Localized Unresectable Neuroblastoma | Stage 4S NeuroblastomaUnited States
Clinical Trials on Guided Therapy
-
Giselle ShollerDell, Inc.Active, not recruitingMedulloblastoma | Neuroblastoma | Brain Tumors | Rare TumorsUnited States
-
Jonsson Comprehensive Cancer CenterActive, not recruitingProstate AdenocarcinomaUnited States
-
British Columbia Cancer AgencyCompletedRadiation Therapy for Primary Breast CancerCanada
-
Giselle ShollerTranslational Genomics Research Institute; Dell, Inc.CompletedGlioma | Renal Cell Carcinoma | Osteosarcoma | Ependymoma | Medulloblastoma | Neuroblastoma | Meningioma | Craniopharyngioma | Choroid Plexus Neoplasms | Rhabdomyosarcoma | Wilms Tumor | Ewings Sarcoma | Malignant Rhabdoid Tumor | Liver Tumors | Germ Cell Tumors | Dysembryoplastic Neuroepithelial Tumor | Clear Cell Sarcoma | ... and other conditionsUnited States, Lebanon
-
Careggi HospitalCompletedCoronary Artery | Stent Thrombosis | Platelet | ThrombusItaly
-
University of SheffieldUnknown
-
Mansoura UniversityCompletedUrologic Diseases | Stone, Kidney | Stone, Urinary | ESWL | Radiolucent Calculus of Urinary Tract | DissolutionEgypt
-
University Hospital, MontpellierEURO BIO ScientificRecruiting
-
Ministry of Health, SpainCompleted