Individualized Treatment Plan in Children and Young Adults With Relapsed Medulloblastoma (PNOC027)
|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: NCT05057702|
Recruitment Status : Not yet recruiting
First Posted : September 27, 2021
Last Update Posted : November 9, 2021
|Condition or disease||Intervention/treatment||Phase|
|Medulloblastoma Medulloblastoma, Childhood Medulloblastoma Recurrent||Other: Specialized Tumor Board Treatment Plan||Not Applicable|
This is a single arm multi-center pilot trial within the Pacific Pediatric Neuro-Oncology Consortium (PNOC). Relapsed participants will receive an individualized treatment recommendation including up to four FDA-approved drugs based on the results of real-time high-throughput drug screening, whole exome sequencing (WES), and RNA sequencing.
I. To determine the feasibility of using the results of real-time in vitro drug screening, whole exome sequencing, and RNA sequencing of participant-derived specimens to guide treatment recommendations by a specialized tumor board, in a clinically-actionable timeframe, for children and young adults with recurrent medulloblastoma.
I. To determine the safety and describe the toxicity of treating children and young adults with relapsed medulloblastoma according to a specialized tumor board that makes treatment recommendations based on real-time drug screening and genomic sequencing.
I. To estimate the objective response rate, progression free survival at 6 months (PFS-6) and overall survival (OS) of relapsed medulloblastoma in children and young adults treated with an individualized treatment regimen.
II. To assess Quality of Life (QOL) measures in participants with relapsed medulloblastoma treated with an individualized regimen.
III. To archive tumor and normal DNA from each participant along with serial blood draw following therapies as biospecimens for later studies to determine whether circulating tumor DNA (ctDNA) sequences in the participant's blood serve as biomarkers of tumor burden, response to therapy, or development of drug resistance.
Participants may continue treatment as tolerated for up to two years or until disease progression. Participants will be followed until progression, death, or up to 5 years from start of therapy.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||10 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Pilot Trial of Real Time Drug Screening and Genomic Testing to Determine an Individualized Treatment Plan in Children and Young Adults With Relapsed Medulloblastoma|
|Estimated Study Start Date :||November 30, 2021|
|Estimated Primary Completion Date :||December 31, 2024|
|Estimated Study Completion Date :||January 31, 2027|
Experimental: Individualized Treatment Recommendation
Participants will receive an individualized treatment recommendation including a combination of up to four FDA-approved drugs within 21 business days of tissue acquisition using the results of real-time high-throughput drug screening, whole exome sequencing (WES), and RNA sequencing.
Other: Specialized Tumor Board Treatment Plan
Specialized Treatment Plan of up to four FDA approved drugs based on participant's screening results will be assigned by PNOC specialized tumor board
- Median Time from tissue collection to issued treatment plan from the specialized tumor board [ Time Frame: Up to 21 days ]Time to tissue collection will be used to determine the feasibility of using the results of real-time in vitro drug screening, WES and RNAseq of participant-derived specimens to guide treatment recommendations by a specialized tumor board, in a clinically-actionable timeframe, for children and young adults with recurrent medulloblastoma. Participants are expected to receive treatment plan within 21 business days
- Proportion of participants with Adverse Events [ Time Frame: Up to 2 years ]Proportion of participants with grade 3 or higher toxicities classified according to the NCI Common Terminology Criteria for Adverse Events (CTCAE) version 5.0. Adverse events and clinically significant laboratory abnormalities (meeting Grade 3 or higher criteria will be summarized by maximum grade and relationship to study drug(s).
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): NCT05057702
|Contact: Aubrie Drechsler||415-502-1600||PNOC027@ucsf.edu|
|United States, California|
|Rady Children's Hospital|
|San Diego, California, United States, 92123|
|Principal Investigator: John Crawford, MD, MS|
|Sub-Investigator: Jennifer Elster, MD|
|University of California, San Francisco|
|San Francisco, California, United States, 94143|
|Contact: Aubrie Drechsler 415-502-1600 PNOC027@ucsf.edu|
|Principal Investigator: Sabine Mueller, MD, PhD|
|Sub-Investigator: Alyssa Reddy, MD|
|United States, Missouri|
|St. Louis Children's Hospital|
|Saint Louis, Missouri, United States, 63110|
|Sub-Investigator: Mohamed Abdelbaki, MD|
|Principal Investigator: Josh Rubin, MD, PhD|
|Principal Investigator:||Sabine Mueller, MD, PhD, MAS||University of California, San Francisco|
|Study Chair:||Joshua Rubin, MD, PhD||Washington University School of Medicine|
|Study Chair:||Robert Wechsler-Reya, PhD||Sanford Burnham Prebs Medical Discovery Institute|
|Study Chair:||Margaret Shatara, MD||Washington University School of Medicine|
|Study Chair:||Megan Paul, MD||Rady Children's Hospital|