NCT04197934

Brief Summary

This phase I trial studies the side effects and best dose of WSD0922-FU for the treatment of glioblastoma, anaplastic astrocytoma, or non-small cell lung cancer that has spread to the central nervous system (central nervous system metastases). WSD0922-FU is a targeted treatment which blocks the EGFR protein - a strategy that has led to a lot of benefit in patients with many different cancers. WSD0922-FU may also be able to get into cancers in the brain and spinal cord and help patients with brain and spinal cord cancers. Funding Source - FDA OOPD

Trial Health

75
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
56

participants targeted

Target at P50-P75 for phase_1

Timeline
58mo left

Started Dec 2019

Longer than P75 for phase_1

Geographic Reach
1 country

3 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress57%
Dec 2019Feb 2031

First Submitted

Initial submission to the registry

November 25, 2019

Completed
18 days until next milestone

First Posted

Study publicly available on registry

December 13, 2019

Completed
7 days until next milestone

Study Start

First participant enrolled

December 20, 2019

Completed
2.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 30, 2022

Completed
8.3 years until next milestone

Study Completion

Last participant's last visit for all outcomes

February 6, 2031

Expected
Last Updated

March 30, 2026

Status Verified

March 1, 2026

Enrollment Period

2.9 years

First QC Date

November 25, 2019

Last Update Submit

March 26, 2026

Conditions

Outcome Measures

Primary Outcomes (1)

  • Recommended phase 2 dose

    The RP2D is either the maximum tolerated dose (MTD) or the highest dose tested (in the case that none of the doses are deemed higher than the MTD), whichever is higher.

    Up to 28 days

Secondary Outcomes (4)

  • Incidence of adverse events

    Up to 4-6 weeks after study completion

  • Overall response rate

    Up to 5 years

  • Duration of response (DOR)

    From the first occurrence of a PR (or better) and progression, assessed up to 5 years

  • Progression Free Survival (PFS)

    From study entry to disease progression, assessed up to 5 years

Other Outcomes (28)

  • Pharmacokinetic (PK) analysis (Dose escalation cohort) - AUCs

    Day 1 Cycle 1 (each cycle is 28 days)

  • Pharmacokinetic (PK) analysis (Dose escalation cohort) - AUCs

    D15 Cycle 1 (each cycle is 28 days)

  • Pharmacokinetic (PK) analysis (Dose escalation cohort) - AUCs

    D1 Cycle 2 (each cycle is 28 days)

  • +25 more other outcomes

Study Arms (4)

Dose escalation (WSD0922-FU)

EXPERIMENTAL

Patients receive WSD0922-FU PO QD or BID on days 1-28. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. Patients also undergo CT, MRI, and blood sample collection on study. Patients with NSCLC with LM also undergo collection of CSF samples on study.

Procedure: Biospecimen Collection - blood samplesProcedure: Biospecimen Collection - CSF samplesProcedure: Computed TomographyDrug: EGFR/EGFRvIII Inhibitor WSD0922-FUProcedure: Magnetic Resonance Imaging

Dose expansion Cohort I (WSD0922-FU)

EXPERIMENTAL

Patients with GBM/AA receive WSD0922-FU PO on days 1 and 4 of cycle 0. Patients then receive WSD0922-FU PO BID on days 1-28 of subsequent cycles. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. Patients also undergo MRI during screening and on study, as well as blood sample collection on study. Patients may undergo additional optional blood sample collection on study.

Procedure: Biospecimen Collection - blood samplesDrug: EGFR/EGFRvIII Inhibitor WSD0922-FUProcedure: Magnetic Resonance Imaging

Dose expansion Cohort II (WSD0922-FU, surgery)

EXPERIMENTAL

Patients with BTP receive a single dose of WSD0922-FU prior to surgery. Patients then undergo surgical resection of brain tumor. After surgery, patients receive WSD0922-FU PO BID on days 1-28. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. Patients also undergo MRI during screening and on study, as well as blood sample collection on study.

Procedure: Biospecimen Collection - blood samplesDrug: EGFR/EGFRvIII Inhibitor WSD0922-FUProcedure: Magnetic Resonance ImagingProcedure: Therapeutic Conventional Surgery

Dose expansion Cohort III (WSD0922-FU)

EXPERIMENTAL

Patients with NSCLC receive WSD0922-FU PO BID on days 1-28 of each cycle. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. Patients also undergo MRI and CT during screening and on study, as well as blood sample collection on study. Patients with LM also undergo collection of CSF samples on study. Patients may also undergo optional blood sample collection on study.

Procedure: Biospecimen Collection - blood samplesProcedure: Biospecimen Collection - CSF samplesProcedure: Computed TomographyDrug: EGFR/EGFRvIII Inhibitor WSD0922-FUProcedure: Magnetic Resonance Imaging

Interventions

Undergo collection of blood samples

Also known as: Biological Sample Collection, Biospecimen Collected, Biospecimen Collection, Specimen Collection
Dose escalation (WSD0922-FU)Dose expansion Cohort I (WSD0922-FU)Dose expansion Cohort II (WSD0922-FU, surgery)Dose expansion Cohort III (WSD0922-FU)

Undergo collection of CSF samples

Also known as: Biological Sample Collection, Biospecimen Collected, Biospecimen Collection, Specimen Collection
Dose escalation (WSD0922-FU)Dose expansion Cohort III (WSD0922-FU)

Undergo CT

Also known as: CAT, CAT Scan, Computed Axial Tomography, Computerized Axial Tomography, Computerized axial tomography (procedure), Computerized Tomography, CT, CT Scan, tomography
Dose escalation (WSD0922-FU)Dose expansion Cohort III (WSD0922-FU)

Given PO

Also known as: BBB Penetrable EGFR/EGFRvIII Inhibitor WSD0922-FU, EGFR Mutant Inhibitor WSD0922-FU, WSD 0922-FU, WSD-0922-FU, WSD0922-FU
Dose escalation (WSD0922-FU)Dose expansion Cohort I (WSD0922-FU)Dose expansion Cohort II (WSD0922-FU, surgery)Dose expansion Cohort III (WSD0922-FU)

Undergo MRI

Also known as: Magnetic Resonance, Magnetic resonance imaging (procedure), Magnetic Resonance Imaging Scan, Medical Imaging, Magnetic Resonance / Nuclear Magnetic Resonance, MR, MR Imaging, MRI, MRI Scan, NMR Imaging, NMRI, Nuclear Magnetic Resonance Imaging
Dose escalation (WSD0922-FU)Dose expansion Cohort I (WSD0922-FU)Dose expansion Cohort II (WSD0922-FU, surgery)Dose expansion Cohort III (WSD0922-FU)

Undergo surgical resection

Dose expansion Cohort II (WSD0922-FU, surgery)

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Histolopathological and/or molecular confirmation of either glioblastoma, IDH wildtype (GBM), (as defined by either the 2016 or 2021 World Health Organization \[WHO\] classifications) anaplastic astrocytoma, IDH wildtype (AA) (as defined by the 2016 WHO classification) or non-small cell lung cancer (NSCLC)
  • EGFR Status:
  • GBM/AA must either EGFR amplification and/or any activating EGFR mutation (e.g. A289T, EGFRvIII , etc.)
  • NSCLC must have a confirmed activating EGFR mutation (e.g. Del19, L858R, EGFRvIII, G719A, L861Q, T790M, C797S, etc.)
  • Glioblastoma, IDH wildtype/Anaplastic astrocytoma, IDH wildtype (GBM/AA) Cohort:
  • Diagnosis: Histological or molecular confirmation of either glioblastoma, IDH wildtype (GBM) (as defined by either the 2016 or 2021 WHO classifications) or anaplastic astrocytoma, IDH wildtype (AA) (as defined by the 2016 WHO classification)
  • EGFR status: GBM/AA must have EGFRvIII mutation
  • Brain Tumor Penetration (BTP) Cohort:
  • Diagnosis: Histopathological or molecular confirmation of either glioblastoma, IDH wildtype (GBM) (as defined by either the 2016 or 2021 WHO classifications) or anaplastic astrocytoma, IDH wildtype (AA) (as defined by the 2016 WHO classification)
  • EGFR status: GBM/AA must have been previously demonstrated to have either EGFR amplification and/or any activating EGFR mutation based on any prior resection
  • Non-Small Cell Lung Cancer (NSCLC) cohort:
  • Diagnosis: Histological confirmation of non-small cell lung cancer (NSCLC)
  • EGFR status: NSCLC must have confirmed activating EGFR mutation. Following protocol amendment 7, NSCLC must have EGFR C797S mutation.
  • Previous treatments:
  • Patients with GBM/AA must have been previously treated with radiation and temozolomide
  • +45 more criteria

You may not qualify if:

  • Any of the following because this study involves an investigational agent whose genotoxic, mutagenic and teratogenic effects on the developing fetus and newborn are unknown:
  • Pregnant persons
  • Nursing persons
  • Persons of childbearing potential who are unwilling to employ adequate contraception
  • Any of the following prior therapies:
  • Any cytotoxic chemotherapy or other anticancer drugs for the treatment of advanced NSCLC from a previous treatment regimen =\< 14 days prior to registration
  • In patients with NSCLC, treatment with an EGFR TKI (e.g., erlotinib, gefitinib, afatinib or osimertinib) must be discontinued prior to registration. Additionally, prior EGFR TKI therapy must be discontinued within 8 days or 5 half-lives, whichever is longer, prior to study therapy initiation. If sufficient wash-out time has not occurred due to schedule or PK properties, an alternative appropriate wash-out time based on known duration and time to reversibility of drug related adverse events could be agreed upon by the Investigator and Wayshine)
  • Radiation therapy to the brain =\< 12 weeks prior to registration
  • Patients with GBM/AA must not have received (i) nitrosoureas within 42 days of registration, (ii) any chemotherapy or experimental therapy within 28 days or 5 half-lives, whichever is longer, prior to registration
  • Patients with GBM/AA must not have received prior anti-EGFR or EGFRvIII therapies (erlotinib, gefitinib, afatinib, osimertinib, ABT-414, ABBV-221, AMG-595, AMG-596 etc.)
  • Patients with GBM/AA who have been treated with bevacizumab within the last four months are not eligible
  • Received prior systemic biologic therapy (CAR-T, anti-PD-1 / anti-PD-L1, anti-CTLA-4, etc.) within 28 days prior to registration.
  • Co-morbid systemic illnesses or other severe concurrent disease which, in the judgment of the investigator, would make the patient inappropriate for entry into this study or interfere significantly with the proper assessment of safety and toxicity of the prescribed regimens including uncontrolled hypertension and active bleeding diatheses, which in the investigator's opinion makes it undesirable for the patient to participate in the trial or which would jeopardize compliance with the protocol. Screening for chronic conditions is not required
  • Subjects who are human immunodeficiency virus (HIV), hepatitis virus B (HBV), and/or hepatitis virus C (HCV) positive
  • Uncontrolled inter-current illness including, but not limited to:
  • +18 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (3)

Mayo Clinic in Arizona

Scottsdale, Arizona, 85259, United States

Location

Mayo Clinic in Florida

Jacksonville, Florida, 32224-9980, United States

Location

Mayo Clinic in Rochester

Rochester, Minnesota, 55905, United States

Location

Related Links

MeSH Terms

Conditions

AstrocytomaGlioblastomaCarcinoma, Non-Small-Cell LungLung Neoplasms

Interventions

Specimen HandlingMagnetic Resonance Spectroscopy

Condition Hierarchy (Ancestors)

GliomaNeoplasms, NeuroepithelialNeuroectodermal TumorsNeoplasms, Germ Cell and EmbryonalNeoplasms by Histologic TypeNeoplasmsNeoplasms, Glandular and EpithelialNeoplasms, Nerve TissueCarcinoma, BronchogenicBronchial NeoplasmsRespiratory Tract NeoplasmsThoracic NeoplasmsNeoplasms by SiteLung DiseasesRespiratory Tract Diseases

Intervention Hierarchy (Ancestors)

Clinical Laboratory TechniquesDiagnostic Techniques and ProceduresDiagnosisInvestigative TechniquesSpectrum AnalysisChemistry Techniques, Analytical

Study Officials

  • Sani H. Kizilbash, M.D., M.P.H.

    Mayo Clinic in Rochester

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 25, 2019

First Posted

December 13, 2019

Study Start

December 20, 2019

Primary Completion

October 30, 2022

Study Completion (Estimated)

February 6, 2031

Last Updated

March 30, 2026

Record last verified: 2026-03

Locations