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Molecular Profiling for Individualized Treatment Plan for DIPG

Information source: University of California, San Francisco
ClinicalTrials.gov processed this data on August 23, 2015
Link to the current ClinicalTrials.gov record.

Condition(s) targeted: Diffuse Intrinsic Pontine Glioma (DIPG)

Intervention: Specialized tumor board recommendation (Other); Standard radiation therapy (Radiation)

Phase: N/A

Status: Recruiting

Sponsored by: University of California, San Francisco

Overall contact:
Sabine Mueller, MD, Phone: 415-476-3831, Email: MuellerS@neuropeds.ucsf.edu

Summary

This is a single arm multi-center feasibility 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. The current study will test the feasibility of a specialized Tumor Board assigning a treatment plan within 21 business days of obtaining tissue. 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. At time of recurrence/progression, patients have the option to have molecular profiling repeated if fresh tissue is collected as part of their care. Molecular profiling will be performed similar to at time of initial diagnosis and a new treatment recommendation will be made based on the new molecular profile. Patients may then receive a second round of molecular based therapy with FDA approved drugs.

Clinical Details

Official title: A Pilot Trial Testing the Feasibility of Using Molecular Profiling to Guide an Individualized Treatment Plan in Children and Young Adults With Newly Diagnosed DIPG

Study design: Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Primary outcome: Number of patients for whom an individualized treatment plan is provided within 21 business days

Eligibility

Minimum age: N/A. Maximum age: 25 Years. Gender(s): Both.

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. Eligibility Criteria for Children with First Progression/Recurrence with DIPG:

- Patients with first recurrence/progressive DIPG who were enrolled in this study at

initial diagnosis who will undergo a repeat biopsy are eligible to participate in the 2nd part of this study. Patients with disseminated disease are eligible, and MRI of the spine must be performed if disseminated disease is suspected by the treating physician.

- Repeat biopsy should be performed within 21 days of the date of definite radiographic

diagnosis of first progression/recurrence.

- 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.

- Life expectancy > 8 weeks

- Organ Function Requirements:

- Adequate Bone Marrow Function Defined as:

- Peripheral absolute neutrophil count (ANC) ≥ 1000/mm3

- Platelet count ≥ 100,000/mm3 (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 GFR ≥ 70ml/min/1. 73 m2 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 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

- 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. Exclusion Criteria for Patients with First Progression/Recurrence with DIPG:

- Patients who are currently taking any anti-cancer directed therapy outside the

specialized tumor board recommendation. 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.

Locations and Contacts

Sabine Mueller, MD, Phone: 415-476-3831, Email: MuellerS@neuropeds.ucsf.edu

UCSF Helen Diller Family Comprehensive Cancer Center, San Francisco, California 94115, United States; Recruiting
Sabine Mueller, MD, Phone: 415-476-3831, Email: MuellerS@neuropeds.ucsf.edu
Additional Information

Starting date: September 2014
Last updated: June 2, 2015

Page last updated: August 23, 2015

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