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

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. Identifier: NCT04617002
Expanded Access Status : Available
First Posted : November 5, 2020
Last Update Posted : December 3, 2020
Information provided by (Responsible Party):
Oncoceutics, Inc.

Brief Summary:
This is an intermediate-size expanded access protocol to provide ONC201 to patients with H3 K27M-mutant and/or midline gliomas who cannot access ONC201 through clinical trials.

Condition or disease Intervention/treatment
Glioma H3 K27M Drug: ONC201

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Study Type : Expanded Access
Expanded Access Type : Intermediate-size Population
  See clinical trials of the intervention/treatment in this expanded access record.
Official Title: Intermediate-size Expanded Access to ONC201 for Patients With H3 K27M-mutant and/or Midline Gliomas

Intervention Details:
  • Drug: ONC201
    ONC201 is an oral, small molecule selective antagonist of DRD2

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   3 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All

Inclusion Criteria:

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

    Arm A

    1. Central nervous system tumor that is positive for the H3 K27M mutation (performed in a laboratory with CLIA or equivalent certification);
    2. Central nervous system tumor involving the thalamus, hypothalamus, basal ganglia, brainstem, cerebellum, cerebellar peduncle, midline cortex, corpus callosum, pineal region, optic tract, or optic chiasm.

      Arm B

    3. 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.
  2. Arm A: Patient must have unequivocal radiographic evidence of progressive disease on as defined by RANO criteria or have documented recurrent glioma on diagnostic biopsy.

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

  3. Arm A: Patient must be at least 90 days from completion of radiotherapy. Arm B: Patient must be at least 14 days from completion of radiotherapy.
  4. Patient must be at least 3 years of age.
  5. Patient must weigh at least 10kg.
  6. 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 for bevacizumab), 4 weeks (or 5 half-lives, whichever is shorter) from other anti-tumor therapies, and 1 week from devices used to treat cancer.
  7. Brain MRI within 21 days prior to start of study drug.
  8. 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 tumor
    5. Serum creatinine ≤ 1.5 X ULN (OR creatinine clearance ≥ 60 mL/min/1.73 m2)
  9. 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.
  10. Ability to understand a written informed consent document, and the willingness to sign it. Assent will be obtained when appropriate based on the subjects age.

Exclusion Criteria:

  1. Qualifies for participation in an ongoing ONC201 or ONC206 clinical trial.
  2. Previously or currently participating in an ONC201 clinical trial.
  3. Current or planned participation in a study of an investigational agent (including ONC206) or using an investigational device.
  4. Evidence of leptomeningeal disease or CSF dissemination of disease.
  5. Any known systemic infection that, in the opinion of the investigator, could compromise the safety of the patient, while taking ONC201.
  6. Prolongation of QT/QTcF interval (QTc interval >480 milliseconds) using Frederica's QT correction formula on two ECGs separated by at least 48 hours.
  7. A history of Torsades de Pointes or heart failure, hypokalemia, or family history of prolonged QT Syndrome
  8. Concomitant use of medication(s) known to prolong the QT/QTc interval

Information from the National Library of Medicine

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 identifier (NCT number): NCT04617002

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Contact: Clinical Operations Oncoceutics 1-844-ONCORXS

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United States, District of Columbia
Children's National Medical Center Available
Washington, District of Columbia, United States, 20010
Contact: Lindsay Kilburn, MD, MS    202-476-3643   
United States, Florida
Miami Cancer Institute Available
Miami, Florida, United States, 33176
Contact: Yazmin Odia, MD, MS    786-596-2000   
Principal Investigator: Yazmin Odia, MD, MS         
United States, Georgia
Children's Healthcare of Atlanta, Emory University School of Medicine Available
Atlanta, Georgia, United States, 30322
Contact: Dolly Aguilera, MD    404-785-1112   
United States, Hawaii
Kapi'olani Medical Center for Women and Children Available
Honolulu, Hawaii, United States, 96826
Contact: Andrea Siu, MPH    808-535-7169   
United States, Michigan
University of Michigan Available
Ann Arbor, Michigan, United States, 48109
Contact: Carl Koschmann, MD, MS    734-763-0592   
United States, Missouri
Washington University School of Medicine Available
Saint Louis, Missouri, United States, 63110
Contact: Andrew Cluster, MD    314-454-6018   
United States, New York
New York University Available
New York, New York, United States, 10016
Contact: Sharon Gardner, MD    646-929-7870   
Contact: Anna Yaffe    212-263-9945   
Principal Investigator: Sharon Gardner, MD         
University of Rochester Available
Rochester, New York, United States, 14627
Contact: David Korones, MD    585-275-2981   
Contact: Jennifer Serventi, PA    585-275-5863   
United States, Texas
MD Anderson Cancer Center Available
Houston, Texas, United States, 77030
Contact: Soumen Khatua, MD    713-792-3280   
Contact: Rebecca Harrison, MD    713-792-1263   
Sponsors and Collaborators
Oncoceutics, Inc.
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Responsible Party: Oncoceutics, Inc. Identifier: NCT04617002    
Other Study ID Numbers: ONC028
First Posted: November 5, 2020    Key Record Dates
Last Update Posted: December 3, 2020
Last Verified: December 2020
Additional relevant MeSH terms:
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Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue