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Trial record 58 of 77 for:    DIPG

Clinical Benefit of Using Molecular Profiling to Determine an Individualized Treatment Plan for Patients With High Grade Glioma (PNOC008)

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ClinicalTrials.gov Identifier: NCT03739372
Recruitment Status : Recruiting
First Posted : November 12, 2018
Last Update Posted : July 11, 2019
Sponsor:
Collaborator:
Pacific Pediatric Neuro-Oncology Consortium
Information provided by (Responsible Party):
Sabine Mueller, MD, PhD, University of California, San Francisco

Brief Summary:

This is a 2 strata pilot trial within the Pacific Pediatric Neuro-Oncology Consortium (PNOC).

The study will use a new treatment approach based on each patient's tumor gene expression, whole-exome sequencing (WES), targeted panel profile (UCSF 500 gene panel), and RNA-Seq. The current study will test the efficacy of such an approach in children with High-grade gliomas HGG.


Condition or disease Intervention/treatment Phase
Glioma Glioma of Brain Cancer Pediatric Cancer Pediatric Brain Tumor Astrocytoma Glioblastoma Hemispheric Other: Specialized tumor board recommendation Not Applicable

Detailed Description:
For children with High-grade gliomas (HGG) including HGG presenting within the midline structures of the brain and spine, outcome remains poor and the majority of children die from this disease. The current study will use a new treatment approach based on each patient's tumor gene expression, whole-exome sequencing (WES), targeted panel profile (UCSF 500 gene panel), and RNA-Seq. This treatment strategy has shown promising results in adult patients with solid tumors and is currently being explored in children with DIPG, neuroblastoma and other solid tumors. The current study will test the efficacy of such an approach in children with HGG for which outcomes remain dismal.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 44 participants
Allocation: Non-Randomized
Intervention Model: Parallel 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 and Young Adults With High Grade Glioma (Excluding Diffuse Intrinsic Pontine Glioma)
Actual Study Start Date : November 6, 2018
Estimated Primary Completion Date : November 1, 2020
Estimated Study Completion Date : November 1, 2021


Arm Intervention/treatment
Experimental: Newly diagnosed HGG (Stratum A)
Children and young adults with newly diagnosed HGG receive an individualized treatment plan. Each treatment is different and depends on what the Specialized Tumor Board recommends depending on the molecular profile of the patient's tumor.
Other: Specialized tumor board recommendation
Based on the molecular profile, the specialized tumor board will determine an individualized treatment recommendation for each patient using up to four FDA approved drugs. In special circumstances, Investigational new drug (IND) study agents may be used.

Experimental: Diffuse midline HGG (Stratum B)
Children and young adults with diffuse midline high grade gliomas receive an individualized treatment plan. Each treatment is different and depends on what the Specialized Tumor Board recommends depending on the molecular profile of the patient's tumor.
Other: Specialized tumor board recommendation
Based on the molecular profile, the specialized tumor board will determine an individualized treatment recommendation for each patient using up to four FDA approved drugs. In special circumstances, Investigational new drug (IND) study agents may be used.




Primary Outcome Measures :
  1. 12 month Progression Free Survival (PFS) for Stratum A [ Time Frame: From date of diagnosis until progression or up to 12 months ]
  2. 12 month Overall Survival (OS) for Stratum B [ Time Frame: From date of diagnosis until death or up to 12 months ]

Secondary Outcome Measures :
  1. Adverse Events will be Assessed by CTCAE v5.0 [ Time Frame: From beginning of enrollment up to 30 days post end of treatment. ]
    Toxicity will be described by reporting Adverse Events (AE). Events will be assessed according to the NCI CTCAE v5.0. All study-related and unrelated Adverse Events (AE) will be collected and reported, together with their maximum intensity among all recorded respective AE.

  2. Serious Adverse Events will be Assessed by CTCAE v5.0 [ Time Frame: From beginning of enrollment up to 30 days post end of treatment. ]
    Toxicity will be described by reporting Serious Adverse Events (SAE). Events will be assessed according to the NCI CTCAE v5.0. All study-related and unrelated Serious Adverse Events (SAE) will be collected and reported, together with their maximum intensity among all recorded respective SAE.



Information from the National Library of Medicine

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Ages Eligible for Study:   up to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Patients with newly diagnosed HGG (including midline HGG but excluding DIPG), who undergo tissue collection as part of standard of care. HGG is defined as either WHO grade III or IV, or testing positive for H3K27M mutation. Patients with disseminated disease are not eligible, and MRI of the spine must be performed if disseminated disease is suspected by the treating physician. Primary spinal cord tumors are eligible.
  • Enrollment within 3 weeks of the start of radiation therapy.
  • Start of radiation therapy within 6 weeks from initial tissue diagnosis.
  • Age ≤ 21 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
  • Adequate tissue for molecular profiling (see Section 8 of the protocol for full details)
  • The effects of the current treatment paradigm on the developing human fetus are unknown. For this reason, females of child-bearing potential and males 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. Males 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 to understand a written informed consent document, and the willingness to sign it.

Exclusion Criteria:

  • Patients who are currently enrolled on another therapeutic clinical trial. Individual cases should be discussed with the study chair.
  • Patients who are currently taking any anti-cancer directed therapy. Steroids are not considered anti-cancer therapy. The use of temozolomide during radiation therapy is allowed at standard dosing (maximum 75 to 90 mg/m^2 daily for a total of 42 days). Any other schedule(s) need to be discussed with the study chair.
  • 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 (as clinically indicated).
  • Patients with inability to return for follow-up visits or obtain follow-up studies required to assess toxicity to therapy. Telemedicine vists are acceptable.

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): NCT03739372


Contacts
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Contact: Sabine Mueller, MD, PhD, MAS 877-827-3222 sabine.mueller@ucsf.edu
Contact: Aubrie Drechsler, BA (415) 502-1600 aubrie.drechsler@ucsf.edu

Locations
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United States, California
University of California, San Francisco Recruiting
San Francisco, California, United States, 94158
Contact: Sabine Mueller, MD, PhD, MAS    415-476-3831    sabine.mueller@ucsf.edu   
Contact: Aubrie Drechsler, BA    415-502-1600    aubrie.drechsler@ucsf.edu   
Principal Investigator: Sabine Mueller, MD, PhD, MAS         
Sponsors and Collaborators
University of California, San Francisco
Pacific Pediatric Neuro-Oncology Consortium
Investigators
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Principal Investigator: Sabine Mueller, MD, PhD, MAS University of California, San Francisco

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Responsible Party: Sabine Mueller, MD, PhD, Associate Adjunct Professor, University of California, San Francisco
ClinicalTrials.gov Identifier: NCT03739372     History of Changes
Other Study ID Numbers: 170827
PNOC008 ( Other Identifier: Pacific Pediatric Neuro-Oncology Consortium (PNOC) )
First Posted: November 12, 2018    Key Record Dates
Last Update Posted: July 11, 2019
Last Verified: July 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: Yes
Device Product Not Approved or Cleared by U.S. FDA: Yes
Product Manufactured in and Exported from the U.S.: No
Keywords provided by Sabine Mueller, MD, PhD, University of California, San Francisco:
High-grade glioma
whole exome sequencing
WES
genomics
RNA sequencing
Additional relevant MeSH terms:
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Glioblastoma
Glioma
Astrocytoma
Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Neoplasms
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue