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Trial record 3 of 58 for:    DIPG

Molecular Profiling for Individualized Treatment Plan for DIPG

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02274987
Recruitment Status : Recruiting
First Posted : October 27, 2014
Last Update Posted : March 14, 2017
Sponsor:
Collaborator:
Information provided by (Responsible Party):

Study Description
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.

Condition or disease Intervention/treatment
Diffuse Intrinsic Pontine Glioma (DIPG) Other: Specialized tumor board recommendation Radiation: Standard radiation therapy

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, 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 Design

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 27 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Pilot Trial Testing the Clinical Benefit of Using Molecular Profiling to Determine an Individualized Treatment Plan in Children With Newly Diagnosed DIPG
Study Start Date : September 2014
Estimated Primary Completion Date : December 2017
Estimated Study Completion Date : May 2018

Resource links provided by the National Library of Medicine

U.S. FDA Resources

Arms and Interventions

Arm Intervention/treatment
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.
Other: Specialized tumor board recommendation
A combination of up to four FDA approved drugs based on the molecular profile of the patient's tumor as determined by gene expression analysis, WES and predictive modeling.
Radiation: Standard radiation therapy
Initial therapy will consist of standard radiation therapy per institutional guidelines followed by molecular based therapy with FDA approved drugs.


Outcome Measures

Primary Outcome Measures :
  1. 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 ]

Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 25 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria for Newly Diagnosed Patients with diffuse intrinsic pontine glioma (DIPG):

  • Diagnosis: Patients with newly diagnosed DIPG, defined as tumors with a pontine epicenter and diffuse involvement of the pons who undergo a biopsy are eligible. Patients with disseminated disease are not eligible, and MRI of the spine must be performed if disseminated disease is suspected by the treating physician.
  • Enrollment within 28 days of the date of radiographic diagnosis.
  • Age ≤ 25 years
  • Karnofsky score ≥ 50 for patients ≥ 16 years of age and Lansky score ≥ 50 for patients ≤15 years of age. Patients who are unable to walk because of paralysis but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score .
  • Organ Function Requirements:
  • Adequate Bone Marrow Function Defined as:
  • Peripheral absolute neutrophil count (ANC) ≥ 1000/mm 3
  • Platelet count ≥ 100,000/mm 3 (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment)
  • Hemoglobin ≥ 8 g/dl (can be transfusion dependent)
  • Adequate Renal Function Defined as:

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).

  • Organ Function Requirements cont.
  • Adequate Liver Function Defined as:
  • Bilirubin (sum of conjugated + unconjugated) ≤ 1.5 x upper limit of normal (ULN) for age
  • Serum glutamic pyruvic transaminase (SGPT) (ALT) ≤ 110 U/L. For the purpose of this study, the ULN for SGPT is 45 U/L.
  • Serum albumin ≥ 2 g/dL
  • The effects of the current treatment paradigm on the developing human fetus are unknown. For this reason, women of child-bearing potential and men must agree to use adequate contraception: hormonal or barrier method of birth control; abstinence prior to study entry and for the duration of study participation, and 30 days after completion of study drug administration. Should a female become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. Men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of study participation, and 30 days after completion of study drug administration
  • Adequate Neurologic Function Defined as:
  • Patients with seizure disorder may be enrolled if seizures are well controlled.
  • Ability by patient or parent/legal guardian (for patients under 18 years of age) to understand a written informed consent document, and the willingness to sign it.

Exclusion Criteria for Newly Diagnosed Patients with DIPG:

  • Patients who are currently taking any anti-cancer directed therapy. Steroids are not considered anti-cancer therapy.
  • Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
  • Female patients of childbearing potential must not be pregnant or breast-feeding. Female patients of childbearing potential must have a negative serum or urine pregnancy test prior to the start of therapy.
  • Patients with inability to return for follow-up visits or obtain follow-up studies required to assess toxicity to therapy.
Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02274987


Contacts
Contact: Sabine Mueller, MD 415-476-3831 sabine.mueller@ucsf.edu
Contact: Jenna Weight 415-502-1600 jenna.weight@ucsf.edu

Locations
United States, California
Rady Children's Hospital Recruiting
San Diego, California, United States, 92123
Contact: John Crawford, MD    858-966-4939    jrcrawford@rchsd.org   
Contact: Jennifer Elster, MD       jelster@rchsd.org   
UCSF Helen Diller Family Comprehensive Cancer Center Recruiting
San Francisco, California, United States, 94158
Contact: Sabine Mueller, MD    415-476-3831    sabine.mueller@ucsf.edu   
United States, District of Columbia
Children's National Medical Center Recruiting
Washington, District of Columbia, United States, 20010
Contact: Lindsay Kilburn, MD    202-476-2800    lkilburn@cnmc.edu   
Contact: Roger Packer, MD       rpacker@cnmc.edu   
United States, Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago Recruiting
Chicago, Illinois, United States, 60611
Contact: Stewart Goldman, MD    312-227-4873    sgoldman@luriechildrens.org   
United States, Utah
University of Utah Recruiting
Salt Lake City, Utah, United States, 84113
Contact: Richard Lemons, MD, PhD       richard.lemons@imail.org   
Contact: Carol Bruggers, MD       carol.bruggers@imail.org   
Sponsors and Collaborators
University of California, San Francisco
Translational Genomics Research Institute
Investigators
Principal Investigator: Sabine Mueller, MD University of California, San Francisco
More Information

Responsible Party: University of California, San Francisco
ClinicalTrials.gov Identifier: NCT02274987     History of Changes
Other Study ID Numbers: PNOC 003
14082 ( Other Identifier: University of California, San Francisco )
First Posted: October 27, 2014    Key Record Dates
Last Update Posted: March 14, 2017
Last Verified: March 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No