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GlioblastomaNCT07674095

Assessment of a Large US EHR Database as Meeting FDA Fit-for-Purpose Requirements to Provide External Controls for Study BPM31510 in Participants With Glioblastoma

An observational study of Glioblastoma, run by BPGbio at 1 site in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

Glioblastoma is a rare, aggressive brain tumor that leads to death within 2 years of diagnosis in more than half of patients. There are currently 123 studies ongoing that are testing treatments for glioblastoma. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Glioblastoma
How many people
150 participants
Where
1 siteUnited States
Study dates
1 Mar 2021to 1 Sep 2026
Phase
Observational
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Study population: The primary data source for this study will be the Truveta EHR database with over 150 million patients treated at 31 U.S.-based healthcare systems encompassing >20,000 clinics and >900 hospitals. The Truveta population is representative of the US national population in its distribution of age, gender, and race. Representativeness is evaluated by measuring geographic, racial, ethnic, sex, and age diversity against the US Census in population datasheets. At the state level, benchmark representativeness is based on data coverage for at least 10% of the state's population of covered lives. This benchmark is set by the Center for Medicare and Medicaid Services (CMS) under their qualified entity framework. As of March 2025, Truveta meets this benchmark in 26 states.

Cohort 1 inclusion Criteria:

  1. Between 01-01-2021 and 06-30-2025, an ICD-10-CM code C71.X (malignant brain neoplasm) and/or SNOMED CT 276826005 (malignant glioma of brain)
  2. Between 01-01-2021 and 06-30-2025, current procedural terminology (CPT) codes indicating surgery with either a biopsy or excision of a brain tumor
  3. Patients must be adults at least 18 years of age at time of surgery
  4. CPT codes indicating submission of surgical sample for pathology for IDH testing
  5. SNOMED CT codes for "glioblastoma" after IDH testing
  6. Note that while a diagnosis can be made based on IDH from a biopsy or resection sample, resection is required for study inclusion.
  7. A post-resection CPT code for brain MRI
  8. At least one visit meeting all eligibility criteria between 15- and 50-days post-resection.

Cohort 1 exclusion Criteria:

  1. History of malignant neoplasm of the brain >12 months before index
  2. Patients with prior chemotherapy, radiotherapy, immunotherapy or targeted treatment agents (including anti-angiogenic drugs) less than 12 months before and/or 14 days after resection.

Cohort 2 inclusion Criteria:

Patients must meet all eligibility criteria for Cohort 1 and the following:

  1. Brain MRI scan post-resection available for evaluation
  2. Karnofsky performance score of at least 60
  3. Laboratory values within the following specified ranges:
  4. Absolute neutrophil count of at least 1.5 k/microliter (μL)
  5. Platelet count of at least 100 k/μL
  6. Hemoglobin level of at least 9 gram/deciliter (dL)
  7. Serum creatinine level of at most 1.8 milligram(mg)/dL or creatinine clearance of more than 50 milliliter/min
  8. Bilirubin level of at least 1.5 mg/dL
  9. ALT level of at most 3.0 × upper limit of normal (ULN)
  10. AST of at most 3.0 × ULN
  11. PT of at most 1.5 × ULN
  12. INR of at most 1.5 × ULN
  13. PTT of at most 1.5 × ULN

Cohort 2 exclusion Criteria:

  1. No resection was performed
  2. One-year pre-index history of spontaneous or tumor-related intracranial hemorrhage
  3. One-year pre-index history of diffuse leptomeningeal disease
  4. Patients on dexamethasone daily dosing more than 2 mg at index
  5. Three-month pre-index history of cardiovascular disease (myocardial infarction, coronary artery disease, arrhythmia, unstable angina pectoris, cerebrovascular accident)
  6. Six-month pre-index history of acquired coagulopathies (hemorrhagic disorder due to circulating anticoagulants or acquired coagulation factor deficiency)
  7. Six-month pre-index history of clinically significant bleeding
  8. Known predisposition for bleeding such as von Willebrand's disease or other such condition(s) (i.e., hereditary deficiency of factor VIII, IX and XI)
  9. Six-month pre-index history of significant psychiatric illness
  10. Six-month pre-index history of major infection
  11. Prior malignancy except for non-melanoma skin cancer and carcinoma in situ of the cervix or bladder
  12. Receiving any of the following medications at the index date:
  13. Any anticoagulant, including low-molecular weight heparin and warfarin within two months pre-index
  14. Any digitalis alkaloid (digoxin, digitoxin, lanatoside C) within two months pre-index
  15. Antiangiogenic drugs (e.g., bevacizumab) two weeks before or two weeks after index
  16. Theophylline two weeks before or two weeks after index
  17. Patients with a record of pregnancy, pregnancy termination, or hospital admissions for birth/delivery, or a positive laboratory test indicating pregnancy up to 9 months before index date
  18. Known to be positive for the human immunodeficiency virus
  19. Patients not receiving radiation therapy in the year after diagnosis.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • BPGbio

    Waltham, Massachusetts, 02451

Other trials in United States

About this study

Official title: A Retrospective Observational Cohort Study to Assess the Degree to Which the Truveta Dataset Meets FDA Fit-for-Purpose Requirements for Label-Enabling Regulatory Use for BPM31510 in Patients With Newly Diagnosed Glioblastoma

Glioblastoma is a rare, aggressive brain tumor that leads to death within 2 years of diagnosis in more than half of patients. There are currently 123 studies ongoing that are testing treatments for glioblastoma. BPGbio has developed BPM31510 and is executing an ongoing single-arm Phase 2 study (NCT04752813) targeting 50 participants.

Because glioblastoma is a rare and often fatal disease with multiple drugs in development, a randomized placebo-controlled trial is unlikely to be feasible. Therefore, BPGbio is exploring the possible use of untreated patients from the Truveta database to create a control group. The purpose of this study is to explore whether the Truveta database in this indication meets FDA criteria as fit-for-purpose for regulatory use.

Read the detailed description

This is a retrospective, observational cohort study to assess the degree to which the Truveta dataset meets the United States (US) Food and Drug Administration (FDA) fit-for-purpose requirements for label-enabling regulatory use as an external/synthetic control arm for the single-arm Phase 2 trial BPM31510IV-11 for the study of BPM31510 in subjects with newly diagnosed glioblastoma.

Glioblastoma is a rare disease with approximately 11,000 newly diagnosed cases in the US annually. The application of standard clinical trial eligibility criteria plus the requirement of no prior treatment (radiotherapy, chemotherapy, immunotherapy or targeted agents) in an environment of multiple ongoing Phase 2 and Phase 3 trials in this indication will make recruitment for a randomized, double-blind placebo-controlled trial infeasible. This is likely a core contributor to the lack of new glioblastoma treatments since the approval of temozolomide for newly diagnosed glioblastoma in 2005. Recently, the FDA approved dordaviprone for second-line treatment of diffuse midline glioma with an H3 K27M mutation based on a series of single arm studies with a total of 50 treated participants.

The addition of an external/synthetic control arm to the single-arm, non-randomized, open-label Phase 2 trial (BPM31510IV-11) will provide adequate and well-controlled data demonstrating substantial evidence of effectiveness to serve as the primary basis for approval for BPM31510.

The scientific integrity of this study is dependent on demonstrating that the proposed real-world Truveta database meets FDA requirements for real-world data for label-enabling regulatory submission.

Conditions
Glioblastoma
Keywords
Glioblastoma, External control, Fit-for-purpose, Feasibility, Observational, Retrospective, Electronic Health Records (EHR), Regulatory Approval
Medical subject headings
Glioblastoma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Study groups in NCT07674095
Study groupRoleWhat they receive
Glioblastoma CohortCohort 1 will consist of adults (at least 18 years old) newly diagnosed with glioblastoma, not yet treated with radiation, chemotherapy, immunotherapy or targeted therapy in the 14 days after resection, in the Truveta database between 01-01-2021 and 06-30-2025.
Glioblastoma BPM31510 trial-eligible CohortCohort 2 will consist of a subset of Cohort 1 who meet the eligibility criteria specified in of the Phase 2 BPM31510 trial for which this group potentially will serve as an external comparator arm.

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of Truveta participants with glioblastoma

    The number of Truveta participants eligible for each cohort will be determined:

    • Cohort 1: The number of adults in Truveta with newly diagnosed glioblastoma between 01 Jan 2021 and 30 Jun 2025 based on the below criteria:
    • International Classification of Diseases, Tenth Revision, clinical modification (ICD 10-CM) and/or Systematized Nomenclature of Medicine - Clinical Terms (SNOMED CT) code for malignant brain neoplasm and/or malignant glioma of brain
    • Resection/biopsy with histology submission/Isocitrate Dehydrogenase (IDH) test
    • Post-IDH test SNOMED CT code specific to glioblastoma
    • Post-resection magnetic resonance imaging (MRI)
    • Not yet treated with chemotherapy/radiation/immunotherapy by 14 days after resection.
    • Cohort 2: The number of adults in Truveta who are eligible for Cohort 1 who also meet the eligibility criteria of the Phase 2 BPM31510 trial (for which they might serve as comparison group).

    Measured: 01 Jan 2021 to 30 Jun 2025

  2. Completeness and quality of baseline and follow-up data in Truveta

    This study will characterize baseline variables on the index date for Cohorts 1 and 2 separately in terms of:

    • Distribution of each value
    • Missing values
    • Values with incorrect units
    • Values outside a medically plausible range The index date is defined as the date on which a participant meets all eligibility criteria between 15 and 50 days after resection between 01 Jan 2021 to 30 Jun 2025. For participants who have more than one healthcare visit in which they meet all eligibility criteria, one eligible date will be randomly selected as the index date.

    Measured: 01 Jan 2021 to 30 Jun 2025

  3. Quality of baseline data assessment across subgroups

    If a meaningful amount of baseline data is missing, incorrect or medically implausible ( more than 10%), a test for bias comparing those with and without missing data across the below subgroups will be performed for Cohort 1 and Cohort 2 separately:

    • Age
    • Sex
    • US region
    • Insurance type
    • Index year

    Measured: At the index visit date with a lookback period of 12 months. All participants must have at least 12 months of EHR data before the index date.

Secondary outcomes (1)

  1. Healthcare resource utilization at baseline

    Healthcare resource utilization will be characterized for Cohort 1 and Cohort 2 separately in the 12 months before index based on the following:

    • Outpatient visits
    • Top 20 outpatient diagnoses
    • Hospital admissions
    • Top 20 admission diagnoses
    • Top 20 drugs used

    Measured: 12 months before index.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted29 Jun 2026
  2. Study start1 Mar 2021
  3. Primary completion1 Jul 2026 (estimated)
  4. Study completion1 Sep 2026 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Observational
Observational model
Cohort
Time perspective
Retrospective
Enrolment
150 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07674095
Sponsor's protocol ID
BPM31510IV-11b
Status last verified
Apr 2026
First submitted
11 Jun 2026
Last updated
1 Jul 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No: No IPD will be shared as this is proprietary to Truveta

Publications

  • Thakkar JP, Dolecek TA, Horbinski C, Ostrom QT, Lightner DD, Barnholtz-Sloan JS, Villano JL. Epidemiologic and molecular prognostic review of glioblastoma. Cancer Epidemiol Biomarkers Prev. 2014 Oct;23(10):1985-96. doi: 10.1158/1055-9965.EPI-14-0275. Epub 2014 Jul 22.

    PubMed 25053711

Questions about this trial

What is this clinical trial studying?

An observational study of Glioblastoma, run by BPGbio at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 1 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Truveta participants with glioblastoma (time frame: 01 Jan 2021 to 30 Jun 2025). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT07674095, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Jul 2026.

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