NeuroblastomaNCT01802567
Molecular-Guided Therapy for Relapsed and Refractory Childhood Cancer
A study testing Guided Therapy- Pediatric Gene Analysis Platform in people with Neuroblastoma and Medulloblastoma, run by Giselle Sholler at 13 sites in United States.
Trial summary
The purpose of this study is to test the feasibility (ability to be done) of experimental technologies to determine a tumor's molecular makeup (gene expression profile) and mutations. Read more about this study
- Who is it for?
- Ages 13 months and older
- Conditions the study is for
- Neuroblastoma, Medulloblastoma, Brain Tumors
- What is being tested?
- Guided Therapy- Pediatric Gene Analysis Platform
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 52 participants
- Where
- 13 sitesUnited States
- Study dates
- 4 Mar 2013to 17 Jan 2024
- 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 13 months and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Subjects must have histologically proven neuroblastoma, brain tumor, or rare tumor and confirmation of refractory or recurrent disease with histologic confirmation at diagnosis or at the time of recurrence/progression
- Subjects must be age >12 months at enrollment.
- Subjects must be age ≤ 21 years at initial diagnosis.
- Subjects must have measurable disease as 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% tumor are eligible to enroll.
- Current disease state must be one for which there is currently no known curative therapy
- Lansky or Karnofsky Score must be more than 50
Show all 13Show fewer
- Subjects without bone marrow metastases must have an ANC > 750/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
- 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 subjects 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
- Subjects who have received any cytotoxic chemotherapy within the last 7 days prior to enrollment and 14 days prior to study treatment start date.
- Subjects who have received any radiotherapy to the primary sample site within the last 14 days (radiation may be included in treatment decision after biopsy).
- Subjects receiving anti-tumor therapy for their disease or any investigational drug concurrently
- Subjects 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.
- Subjects 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 subject's ability to sign or the legal guardian's ability to sign the informed consent, and subject's ability to cooperate and participate in the study
Trial locations and contacts
13 sites in 1 country.
13 sites in 1 country
United States13 sites
Dell Children's Blood and Cancer Center
Austin, Texas, 78723
Medical University of South Carolina
Charleston, South Carolina, 29425
Levine Children's Hospital
Charlotte, North Carolina, 28204
Helen DeVos Children's Hospital
Grand Rapids, Michigan, 49503
Connecticut Children's Hospital
Hartford, Connecticut, 06106
Kapiolani Medical Center for Women and Children
Honolulu, Hawaii, 96813
Children's Mercy Hospitals and Clinics
Kansas City, Missouri, 64108
Vanderbilt-Ingram Cancer Center
Nashville, Tennessee, 37232
Arnold Palmer Hospital for Children- MD Anderson
Orlando, Florida, 32806
Phoenix Children's Hospital
Phoenix, Arizona, 85016
Primary Children's Hospital
Salt Lake City, Utah, 84113
Rady Children's Hospital
San Diego, California, 92123
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 Relapsed and Refractory Childhood Cancer
The purpose of this study is to test the feasibility (ability to be done) of experimental technologies to determine a tumor's molecular makeup (gene expression profile) and mutations. This technology called the "Pediatric Gene Analysis Platform" includes a genomic report (gene expression profile) and a DNA Mutation Panel Report that are being used to discover new ways to understand cancers and potentially predict the best treatments for patients with cancer in the future.
- Conditions
- Neuroblastoma, Medulloblastoma, Brain Tumors, Rare Tumors
- Being tested
- Guided Therapy- Pediatric Gene Analysis Platform
- Medical subject headings
- Neuroblastoma, Medulloblastoma, Brain Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Guided Therapy- Pediatric Gene Analysis PlatformDevice
Also called Molecular Guided Therapy
A total of 48 neuroblastoma, brain tumor, and rare tumor 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).
| Study group | Role | What they receive |
|---|---|---|
| Guided Therapy- Pediatric Gene Analysis Platform | Experimental | A total of 48 neuroblastoma, brain tumor, and rare tumor 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- Pediatric Gene Analysis Platform |
What is being measured
The primary outcome is the main question the study is designed to answer.
Determine Feasibility Using Days From the Date of Biopsy to Date of Start of Treatment
Days to treatment is one data point that will be used in order to determine feasibility. The definition of feasibility for this study will include: "Enrollment onto study, RNA expression profile completed, DNA Mutation Panel completed, genomic analysis and report generation, tumor board held with treatment decision, treatment review completed and start of treatment by 21 days post biopsy/surgical resection date, and then completion of 1 cycle of therapy."
Measured: Date from biopsy to completion of 1 cycle of therapy, generally about 30 days
Secondary outcomes (2)
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: Adverse Events were collected starting with the date of the first dose of study drug until 30 days after last dose of study drug, ongoing related adverse events were continued to be followed until resolution, on average of 3 years.
Overall Response Rate (ORR) of Participants by the Presence of Radiologically Assessable Disease by Cross-sectional CT or MRI Imaging and/or by MIBG or PET Scans.
To determine the activity of treatments chosen based on Overall response rate (ORR) using RESIST criteria. The assessment of response will include the initial measurable targets and will be performed after cycle 2, then after every other cycle. Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1) for target lesions and assessed by CT or MRI imaging and/or by MIBG or PET scans: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), >=30% decrease in the sum of the longest diameter of target lesions; Progressive Disease, At least a 20% increase in the sum of the disease measurements for measurable lesions, Stable Disease, Neither sufficient decrease to qualify for PR or sufficient increase to qualify for PD from study entry. Overall Response (OR) = CR + PR.
Measured: Followed until off therapy, generally 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Mar 2013 (estimated)
- Study start4 Mar 2013
- Primary completion17 Jan 2024
- Study completion17 Jan 2024
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
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 52 participants (actual)
Sponsor and investigators
- Lead sponsor
- Giselle Sholler
- Sponsor type
- University, hospital or other
- Collaborators
- Dell, Inc.
- 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
- NCT01802567
- Sponsor's protocol ID
- NMTRC008
- Status last verified
- Aug 2024
- First submitted
- 26 Feb 2013
- 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 Relapsed and Refractory Childhood Cancer
- ClinicalTrials.gov ID
- NCT01802567
- Acronym / sponsor ID
- NMTRC008
- Conditions
- Neuroblastoma, Medulloblastoma, Brain Tumors, Rare Tumors
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 52 participants (actual)
- Lead sponsor
- Giselle ShollerUniversity, hospital or other
- Collaborators
- Dell, Inc.
- Study start
- 4 Mar 2013 (actual)
- Primary completion
- 17 Jan 2024 (actual)
- Study completion
- 17 Jan 2024 (actual)
- First posted
- 1 Mar 2013 (estimated)
- Results
- Posted 26 Apr 2024
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 experimental technologies to determine a tumor's molecular makeup (gene expression profile) and mutations. This technology called the "Pediatric Gene Analysis Platform" includes a genomic report (gene expression profile) and a DNA Mutation Panel Report that are being used to discover new ways to understand cancers and potentially predict the best treatments for patients with cancer in the future.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Guided Therapy- Pediatric Gene Analysis Platform | Experimental | Device: Guided Therapy- Pediatric Gene Analysis PlatformA total of 48 neuroblastoma, brain tumor, and rare tumor 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
- 13 months and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (13 listed)
- Subjects must have histologically proven neuroblastoma, brain tumor, or rare tumor and confirmation of refractory or recurrent disease with histologic confirmation at diagnosis or at the time of recurrence/progression
- Subjects must be age >12 months at enrollment.
- Subjects must be age ≤ 21 years at initial diagnosis.
- Subjects must have measurable disease as 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% tumor are eligible to enroll.
- Current disease state must be one for which there is currently no known curative therapy
Key exclusion criteria (5 listed)
- Subjects who have received any cytotoxic chemotherapy within the last 7 days prior to enrollment and 14 days prior to study treatment start date.
- Subjects who have received any radiotherapy to the primary sample site within the last 14 days (radiation may be included in treatment decision after biopsy).
- Subjects receiving anti-tumor therapy for their disease or any investigational drug concurrently
- Subjects 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.
- Subjects 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 subject's ability to sign or the legal guardian's ability to sign the informed consent, and subject'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 Therapy- Pediatric Gene Analysis PlatformAlso: Molecular Guided TherapyA total of 48 neuroblastoma, brain tumor, and rare tumor 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). | Device | Guided Therapy- Pediatric Gene Analysis Platform |
Primary outcome
Determine Feasibility Using Days From the Date of Biopsy to Date of Start of Treatment
Time frame: Date from biopsy to completion of 1 cycle of therapy, generally about 30 days
Days to treatment is one data point that will be used in order to determine feasibility. The definition of feasibility for this study will include: "Enrollment onto study, RNA expression profile completed, DNA Mutation Panel completed, genomic analysis and report generation, tumor board held with treatment decision, treatment review completed and start of treatment by 21 days post biopsy/surgical resection date, and then completion of 1 cycle of therapy."
2 secondary outcomes
Number of Participants With Adverse Events as a Measure of Safety
Time frame: Adverse Events were collected starting with the date of the first dose of study drug until 30 days after last dose of study drug, ongoing related adverse events were continued to be followed until resolution, on average of 3 years.
Overall Response Rate (ORR) of Participants by the Presence of Radiologically Assessable Disease by Cross-sectional CT or MRI Imaging and/or by MIBG or PET Scans.
Time frame: Followed until off therapy, generally 3 years
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 Cancer Consortium websiteLink provided by the sponsor.
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Genomic Structural Variation in Cancer Susceptibility
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Enasidenib in IDH2-Mutated Malignant Sinonasal and Skull Base Tumors
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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
13 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- Pediatric Gene Analysis Platform in people with Neuroblastoma and Medulloblastoma, run by Giselle Sholler at 13 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 13 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 13 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 26 Apr 2024). 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?
Determine Feasibility Using Days From the Date of Biopsy to Date of Start of Treatment (time frame: Date from biopsy to completion of 1 cycle of therapy, generally about 30 days). 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.