Molecular Profiling for Individualized Treatment Plan for DIPG
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ClinicalTrials.gov Identifier: NCT02274987 |
Recruitment Status :
Completed
First Posted : October 27, 2014
Last Update Posted : November 4, 2022
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Tracking Information | |||||||
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First Submitted Date ICMJE | October 21, 2014 | ||||||
First Posted Date ICMJE | October 27, 2014 | ||||||
Last Update Posted Date | November 4, 2022 | ||||||
Actual Study Start Date ICMJE | September 16, 2014 | ||||||
Actual Primary Completion Date | December 19, 2018 (Final data collection date for primary outcome measure) | ||||||
Current Primary Outcome Measures ICMJE |
To determine OS12 of children with newly diagnosed DIPG that are being treated based on a specialized tumor board recommendation which is based on RNA based expression analysis, WES and predictive modeling. [ Time Frame: 12 months ] | ||||||
Original Primary Outcome Measures ICMJE |
Number of patients for whom an individualized treatment plan is provided within 21 business days [ Time Frame: Within 21 business days of tumor tissue collection ] A specialized tumor board will make individualized treatment recommendations, using RNA based expression analysis, whole exome sequencing (WES) and predictive modeling
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Change History | |||||||
Current Secondary Outcome Measures ICMJE | Not Provided | ||||||
Original Secondary Outcome Measures ICMJE | Not Provided | ||||||
Current Other Pre-specified Outcome Measures | Not Provided | ||||||
Original Other Pre-specified Outcome Measures | Not Provided | ||||||
Descriptive Information | |||||||
Brief Title ICMJE | Molecular Profiling for Individualized Treatment Plan for DIPG | ||||||
Official Title ICMJE | A Pilot Trial Testing the Clinical Benefit of Using Molecular Profiling to Determine an Individualized Treatment Plan in Children With Newly Diagnosed DIPG | ||||||
Brief Summary | This is a single arm multi-center pilot trial within the Pacific Pediatric Neuro-Oncology Consortium (PNOC). The current study will use a new treatment approach based on each patient's tumor genomic profiling consisting of whole exome sequencing and RNA sequencing as well as predictive modeling. | ||||||
Detailed Description | The current study will test whether patients gain a clinical benefit from such a treatment approach by comparing overall survival at 12 months (OS12) to historical controls. Newly diagnosed patients will receive an individualized treatment recommendation including up to four FDA approved drugs based on the molecular profile of the patient's tumor as determined by gene expression analysis, Whole-exome sequencing (WES) and predictive modeling, age of the patient and other existing medical conditions. Initial therapy will consist of standard radiation therapy per institutional guidelines followed by molecular based therapy with FDA approved drugs. | ||||||
Study Type ICMJE | Interventional | ||||||
Study Phase ICMJE | Not Applicable | ||||||
Study Design ICMJE | Allocation: N/A Intervention Model: Single Group Assignment Masking: None (Open Label) Primary Purpose: Treatment |
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Condition ICMJE | Diffuse Intrinsic Pontine Glioma (DIPG) | ||||||
Intervention ICMJE |
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Study Arms ICMJE | Experimental: Treatment
The treatment plan for each patient is individualized and different depending on what the Specialized Tumor Board recommends depending on the molecular profile of the patient's tumor.
Interventions:
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Publications * | Not Provided | ||||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||||
Recruitment Status ICMJE | Completed | ||||||
Actual Enrollment ICMJE |
38 | ||||||
Original Estimated Enrollment ICMJE |
15 | ||||||
Actual Study Completion Date ICMJE | February 24, 2019 | ||||||
Actual Primary Completion Date | December 19, 2018 (Final data collection date for primary outcome measure) | ||||||
Eligibility Criteria ICMJE | Inclusion Criteria for Newly Diagnosed Patients with diffuse intrinsic pontine glioma (DIPG):
Creatinine clearance or radioisotope glomerular filtration rate (GFR) ≥ 70ml/min/1.73 m 2 OR a serum creatinine within normal limits based on age/gender as follows: Maximum Serum Creatinine (mg/dL) Age Male Female 3 to < 6 years 0.8 0.8 6 to < 10 years 1 1 10 to < 13 years 1.2 1.2 13 to < 16 years 1.5 1.4 ≥ 16 years 1.7 1.4 The threshold creatinine values in this table were derived from the Schwartz formula for estimating GFR utilizing child length and stature data published by the United States Centers for Disease Control and Prevention (CDC).
Exclusion Criteria for Newly Diagnosed Patients with DIPG:
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Sex/Gender ICMJE |
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Ages ICMJE | up to 25 Years (Child, Adult) | ||||||
Accepts Healthy Volunteers ICMJE | No | ||||||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||||
Listed Location Countries ICMJE | United States | ||||||
Removed Location Countries | |||||||
Administrative Information | |||||||
NCT Number ICMJE | NCT02274987 | ||||||
Other Study ID Numbers ICMJE | PNOC 003 14082 ( Other Identifier: University of California, San Francisco ) |
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Has Data Monitoring Committee | Yes | ||||||
U.S. FDA-regulated Product |
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IPD Sharing Statement ICMJE |
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Current Responsible Party | Sabine Mueller, MD, PhD, University of California, San Francisco | ||||||
Original Responsible Party | University of California, San Francisco | ||||||
Current Study Sponsor ICMJE | University of California, San Francisco | ||||||
Original Study Sponsor ICMJE | Same as current | ||||||
Collaborators ICMJE | Translational Genomics Research Institute | ||||||
Investigators ICMJE |
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PRS Account | University of California, San Francisco | ||||||
Verification Date | November 2022 | ||||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |