Intermediate-size Expanded Access to ONC201 for Patients With H3 K27M-mutant and/or Midline Gliomas

March 14, 2024 updated by: Chimerix
This is an intermediate-size expanded access protocol to provide ONC201 (dordaviprone) to patients with H3 K27M-mutant and/or midline gliomas who cannot access ONC201 (dordaviprone) through clinical trials.

Study Overview

Status

Available

Conditions

Intervention / Treatment

Study Type

Expanded Access

Expanded Access Type

  • Intermediate-size Population

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85006
        • Available
        • BMDACC at Banner University Medical Center Phoenix
        • Contact:
        • Principal Investigator:
          • Na Tosha Gatson, MD
    • California
      • Orange, California, United States, 92868
        • Available
        • Children's Hospital of Orange County
        • Contact:
        • Principal Investigator:
          • Mariko Sato
      • San Diego, California, United States, 92123
        • Available
        • Rady Children's Hospital
        • Contact:
      • Santa Monica, California, United States, 90404
    • Colorado
      • Aurora, Colorado, United States, 80045
    • District of Columbia
      • Washington, District of Columbia, United States, 20010
    • Florida
    • Georgia
      • Athens, Georgia, United States, 30607
        • Available
        • University Cancer & Blood Center
        • Principal Investigator:
          • Petros Nikolinakos, MD
        • Contact:
    • Hawaii
      • Honolulu, Hawaii, United States, 96826
        • Available
        • Kapi'olani Medical Center for Women and Children
        • Contact:
        • Principal Investigator:
          • Kelley Hutchins, DO
    • Illinois
      • Chicago, Illinois, United States, 60661
    • Iowa
      • Iowa City, Iowa, United States, 52242
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • Available
        • University of Michigan
        • Contact:
        • Principal Investigator:
          • Carl Koschmann, MD, MS
    • Minnesota
      • Minneapolis, Minnesota, United States, 55455
        • Available
        • University of Minnesota
        • Contact:
    • Missouri
      • Saint Louis, Missouri, United States, 63110
        • Available
        • Washington University in St. Louis
        • Contact:
        • Contact:
        • Principal Investigator:
          • Andrew Cluster, MD
    • Nebraska
      • Omaha, Nebraska, United States, 68198
        • Available
        • University of Nebraska Medical Center - Adults Only
        • Contact:
    • New Jersey
      • Summit, New Jersey, United States, 07901
    • New York
      • Albany, New York, United States, 12208
        • Available
        • Albany Medical Center
        • Contact:
      • New York, New York, United States, 10016
        • Available
        • New York University Langone - Active, Not Enrolling
        • Contact:
        • Principal Investigator:
          • Sharon Gardner, MD
      • Rochester, New York, United States, 14627
    • Oregon
      • Portland, Oregon, United States, 97225
        • Available
        • Providence Neurological Specialties Clinic
        • Principal Investigator:
          • Prakash Ambady
        • Contact:
    • Texas
      • Dallas, Texas, United States, 75390
        • Available
        • University of Texas Southwestern
        • Contact:
        • Principal Investigator:
          • Daniel Bowers, MD
      • Houston, Texas, United States, 77030
        • Available
        • MD Anderson Cancer Center
        • Contact:
        • Principal Investigator:
          • Nazanin Majd
    • Utah
      • Salt Lake City, Utah, United States, 84112
        • Available
        • Huntsman Cancer Institute
        • Principal Investigator:
          • Joe Mendez
        • Contact:
    • Virginia
      • Norfolk, Virginia, United States, 23507
        • Available
        • Children's Hospital of The King's Daughters
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 second and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

N/A

Description

Inclusion Criteria:

  1. Patient meets one or more of the criteria below:

    Arm A - Closed to further enrollment

    Arm B - Diffuse intrinsic pontine glioma (DIPG), defined as tumors with a pontine epicenter and diffuse involvement of the pons. H3 K27M status does not have to be known or positive for this arm.

    Arm C

    1. Patients with primary spinal glioma that is positive for the H3 K27M mutation (performed in a laboratory with Clinical Laboratory Improvement Amendments [CLIA] or equivalent certification). Primary spinal glioma must be documented in radiology reporting. OR
    2. Patients with diffuse glioma that is positive for the H3 K27M mutation (performed in a laboratory with CLIA or equivalent certification) AND radiographic evidence of leptomeningeal disease. Leptomeningeal disease must be documented in radiology reporting.

    Arm D - Closed to further enrollment.

    Arm E - Patients with H3 K27M-mutant glioma or midline glioma of unknown H3 K27M mutational status who received ONC201 and/or ONC206 from an alternative (non-Chimerix) source prior to 30 November 2023 as evidenced by documentation (such as pharmacy receipts). Other examples may be confirmed by medical monitor. Detection of H3 K27M mutation should be performed in a CLIA-certified or equivalent laboratory.

    Arm F - Patients with H3 K27M-mutant diffuse glioma or midline glioma with unknown H3 K27M mutational status who have completed frontline radiotherapy at least 6 weeks prior to 30 November 2023. Detection of H3 K27M mutation should be performed in a CLIA-certified or equivalent laboratory

  2. Disease Status

    Arm B: Patient is not required to have radiographic or clinical evidence of progressive disease.

    Arm C: Patient must have progressive disease as defined by Response Assessment in Neuro-Oncology (RANO) criteria or have documented recurrent glioma on diagnostic biopsy.

    Arm E: Not Applicable

    Arm F: Patient must have progressive disease as defined by RANO criteria or have documented recurrent glioma on diagnostic biopsy.

  3. Prior Radiotherapy

    Arm B: Patient must be at least 14 days from completion of radiotherapy.

    Arm C: Patient must be at least 90 days from completion of radiotherapy.

    Arm E: Not applicable.

    Arm F: Patient must be at least 90 days from completion of frontline radiotherapy and at least 14 days from reirradiation if applicable.

  4. Patient must weigh at least 10kg.

  5. Washouts

    Arm B, C & F: From the projected start of scheduled study treatment, the following time periods must have elapsed from prior anti-cancer treatments: 5 half-lives from any investigational agent, 4 weeks from cytotoxic therapy (except 23 days for temozolomide and 6 weeks from nitrosoureas), 6 weeks from anti-cancer antibodies (except 21 days forbevacizumab), 4 weeks (or 5 half-lives, whichever is shorter) from other anti-tumor therapies, and 1 week from devices used to treat cancer.

    Arm E: No washouts are required for ONC021 and ONC206. All other anti-cancer agents need to be discontinued prior to enrollment with the exception of bevacizumab.

  6. Magnetic resonance imaging (MRI)

    Arms B, C & F: Magnetic resonance imaging (MRI)of patient's glioma within 28 days prior to start of study drug.

    Arm E: MRI will be obtained within 8 weeks prior to enrollment

  7. Adequate organ and marrow function as defined below:

    1. Absolute neutrophil count≥1,000/mm3 without growth factor use ≤7 days prior to treatment (Cycle 1 Day 1 [C1D1])
    2. Hemoglobin ≥8.0 mg/dL without red blood cell transfusion ≤3 days prior to C1D1
    3. Total serum bilirubin≤ 1.5 X upper limit of normal (ULN)
    4. AST (SGOT)/ALT (SGPT)≤2 X ULN; ≤5 X ULN if there is liver involvement secondary to tumore. Serum creatinine ≤1.5 X ULN (OR creatinine clearance ≥60mL/min/1.73m2)
    5. Arm E: Patients with organ and marrow function values outside the defined criteria must be approved by medical monitor
  8. For patients post pubertal: Female patients must agree to use effective contraception while taking ONC201 and for at least 90 days after completion of treatment. Male patients must be surgically sterile or must agree to use effective contraception while taking ONC201 and for at least 90 days after completion of treatment. The decision of effective contraception will be based on the judgment of the principal investigator.
  9. Ability to understand a written informed consent document, and the willingness to sign it. Assent will be obtained when appropriate based on the patient age.

Exclusion Criteria:

  1. Qualifies for participation in an ongoing ONC201 or ONC206 clinical trial.

  2. Arms B, C & F: Previously or current enrollment in an ONC201 clinical trial (including open-label and blinded studies) or expanded access protocol or previous exposure to ONC201 from any source.

    Arm E: Previous or current enrollment in an ONC201 clinical study (including open-label and blinded studies) or expanded access protocol for the treatment of CNS tumor

  3. Arms B, C, E and F: Current or planned participation in a study of an investigational agent (including ONC206) or using an investigational device.
  4. Any known systemic infection that, in the opinion of the investigator, could compromise the safety of the patient, while taking ONC201.
  5. Prolongation of QT/QTcF interval (QTc interval >480 milliseconds) using Fridericia's QT correction formula on two ECGs separated by at least 2 days.
  6. A history of Torsades de Pointes or heart failure, hypokalemia, or family history of prolonged QT Syndrome
  7. Concomitant use of medication(s) known to prolong the QT/QTc interval.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Chimerix

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

First Submitted

October 29, 2020

First Submitted That Met QC Criteria

October 29, 2020

First Posted (Actual)

November 5, 2020

Study Record Updates

Last Update Posted (Actual)

March 15, 2024

Last Update Submitted That Met QC Criteria

March 14, 2024

Last Verified

March 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ONC028

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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