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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|Condition or disease||Intervention/treatment||Phase|
|Diffuse Intrinsic Pontine Glioma (DIPG)||Other: Specialized tumor board recommendation Radiation: Standard radiation therapy||Not Applicable|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||38 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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|
|Actual Study Start Date :||September 16, 2014|
|Actual Primary Completion Date :||December 19, 2018|
|Actual Study Completion Date :||February 24, 2019|
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.
- 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 ]
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
|Ages Eligible for Study:||up to 25 Years (Child, Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
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.
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
|United States, California|
|Rady Children's Hospital|
|San Diego, California, United States, 92123|
|University of California, San Francisco|
|San Francisco, California, United States, 94158|
|United States, District of Columbia|
|Children's National Medical Center|
|Washington, District of Columbia, United States, 20010|
|United States, Illinois|
|Ann & Robert H. Lurie Children's Hospital of Chicago|
|Chicago, Illinois, United States, 60611|
|United States, Utah|
|University of Utah|
|Salt Lake City, Utah, United States, 84113|
|Principal Investigator:||Sabine Mueller, MD||University of California, San Francisco|
|Responsible Party:||Sabine Mueller, MD, PhD, Associate Adjunct Professor, University of California, San Francisco|
|Other Study ID Numbers:||
14082 ( Other Identifier: University of California, San Francisco )
|First Posted:||October 27, 2014 Key Record Dates|
|Last Update Posted:||November 4, 2022|
|Last Verified:||November 2022|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||Yes|
|Plan Description:||Individual participant data after de-identification.|
|Studies a U.S. FDA-regulated Drug Product:||No|
|Studies a U.S. FDA-regulated Device Product:||No|
Diffuse Intrinsic Pontine Glioma
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Brain Stem Neoplasms
Central Nervous System Neoplasms
Nervous System Neoplasms
Neoplasms by Site
Central Nervous System Diseases
Nervous System Diseases