xPedite: A Study to Expedite DIPG and DMG Research
Study Details
Study Description
Brief Summary
This study will gather data from new and existing patients with patient medical records, and patient/family/caregiver reported information to establish a clear natural history of disease suitable to serve as an external, contemporary or historical control arm for future therapeutic development programs of drugs, devices, or biologic interventions in DMG or DIPG.
Detailed Description
xPEDITE is a completely virtual, decentralized, nationwide,real-time, real-world observational study to collect, annotate, standardize, and report the critical data elements of DMG, inclusive of DIPG, in a regulatory-compliant framework. Patients participate by eConsent to the pan-cancer master observational protocol XCELSIOR (NCT03793088). This protocol is a sub-study of XCELSIOR and does not require an additional written consent. Medical records are accessed from institutions directly via eFax or paper fax, online from patient EMR portals, direct from DNA/RNA sequencing and molecular profiling vendors, and via electronic health information exchanges. Medical records are received or converted to electronic/digitized formats (CCDA, FHIR, PDF) and sorted by medical record type (clinic visit, in-patient hospital, out-patient clinic, infusion and out-patient pharmacies, etc.) and made machine-readable to support data annotation, full text searches, and natural language processing (NLP) algorithms to further facilitate feature identification. Data elements are annotated comprehensively and longitudinally from diagnosis to final outcome and include patient level clinical features required to report endpoints in DMG and DIPG clinical trials such as anti-cancer interventions, non-cancer medications, genomics/biomarker results, radiological endpoints, steroid use, vitals, demographics, and locations of care, among others. This study does not require data entry by treating site staff or physicians. Centralized data annotation is completed by xCures remote study staff. Data elements are annotated in a central electronic data capture (EDC) system and coded to Observational Medical Outcomes Partnership (OMOP)-based ontologies (SNOMED, LOINC, ICD-O-3, CTCAE, RxNorm, MedDRA, and others) in one process, permitting standardization of verbatim terms from medical records. Data is collected in a 21 CFR Part 11-compliant EDC system with formal QC/QA process, medical review, and source data verification. Beyond EMR data, raw DICOM images (MRI, CT files) are collected from all sites of care and de-identified. Imaging will be subjected to a blinded central radiological review to assess sizes, and response or progression. For all patients, genomics results (PDFs, variant call files, and raw FASTQ files when available) are collected from commercial and academic sources and centralized. Additionally, patient- and caregiver-reported outcome questionnaires (PROs) are collected to measure the impact of disease and medical care on the patient and family and others living around them to determine the aspects of care that are most important to them. Mobility and neurological assessments will be videoed and submitted at regular intervals to prospectively document changes in clinical status by expert electronic clinician-reported outcomes (ClinROs). Together, these clinical, imaging, biomarker, and assessment data will provide a comprehensive and longitudinal documentation of DMG and DIPG disease course.
Study Design
Outcome Measures
Primary Outcome Measures
- To evaluate progression-free survival rate at 6 Months [6 Months]
To evaluate PFS rate at 6 months of standard of care treatments by mRANO in subjects with DIPG or DMG
Secondary Outcome Measures
- To evaluate Duration of Clinical Benefit (DOCB) [Up to 12 months]
DOCB defined as time from date of first documented objective response (CR or PR) and SD to first documented progression or death due to any cause.
- To evaluate clinical benefit rate Clinical Benefit Rate (CBR) (Complete Response (CR), Partial Response (PR), and Stable Disease (SD) [Up to 12 months]
CBR defined as the proportion of subjects with a BOR of CR or PR or SD.
- To evaluate OS (Overall Survival) [Up to 12 months]
OS, defined as time from first dose of treatment to death due to any cause.
Eligibility Criteria
Criteria
Inclusion Criteria:
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Diagnosis of diffuse midline glioma according to the WHO 2021 Classification of Tumors of the Central Nervous System diagnostic criteria including diffuse intrinsic pontine glioma (DIPG). In the absence of a pathologically confirmed diagnosis, a grade IV glioma involving the thalamus, hypothalamus, brainstem, cerebellum, midbrain, or spinal cord, or with a pontine epicenter and diffuse involvement of the pons.
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Patients with any performance status, comorbidity or disease severity are eligible
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Patients or their legally-authorized representative must be willing and able to provide electronic, informed consent (and assent, if applicable)
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Informed consent obtained for the XCELSIOR longitudinal outcomes registry (NCT03793088).
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Patients must be a resident of or receiving care within the United States or US territories.
Exclusion Criteria:
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Patient or legally-authorized representative is unable to provide informed consent.
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Patient or caregiver is unable to complete the PRO and ClinRO by an electronic platform.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | xCures, Inc. | Los Altos | California | United States | 94022 |
Sponsors and Collaborators
- xCures
- SonALAsense, Inc.
Investigators
- Principal Investigator: Mark Shapiro, PhD, xCures
Study Documents (Full-Text)
None provided.More Information
Additional Information:
Publications
None provided.- XPED-2023