Expanded Access Use of ONC201 in a Patient With Diffuse Intrinsic Pontine Gliomas

NCT05392374 | Unknown

• 1 other identifier: ONC016
• Study Type: expanded_access
• Target: N/A
• Locations: 1 country
• Sites: 10

Brief Summary

This is an intermediate-size expanded access protocol to provide ONC201 to patients with diffuse intrinsic pontine gliomas who cannot access ONC201 through clinical trials.

Conditions

Glioma

Interventions

ONC201 DRUG

ONC201 is an oral, small molecule selective antagonist of DRD2

Eligibility Criteria

• Age: 6 Months - 18 Years
• Sex: all
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Patient with DIPG who failed at least one line of prior therapy such as focal radiation therapy
• Patient must be \> than 6 months and \< 18 years of age.
• Confirmed evidence of disease progression from immediately prior therapy or refractory to the immediately prior treatment.
• Karnofsky/Lansky performance status ≥ 50.
• Adequate organ and marrow function as defined below:
• Absolute neutrophil count ≥1,000/mm3 without growth factor use ≤ 7 days prior to treatment (cycle 1 day 1, C1D1)
• \_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_ CONFIDENTIAL Page 3 of 45
• Hemoglobin\>8.0 mg/dL without red blood cell transfusion ≤ 3 days prior to C1D1
• Total serum bilirubin\<1.5 X upper limit of normal (ULN)
• AST (SGOT)/ALT (SGPT)≤2 X ULN;; ≤ 5 X ULN if there is liver involvement secondary to tumor
• Serum creatinine≤ 1.5 X ULN (OR creatinine clearance ≥ 60 mL/min/1.73 m2)
• Ability to understand and the willingness to sign a written informed consent document by the patient or their legal guardian.
• If patient is of child--bearing age, female patients must be surgically sterile or be postmenopausal, or must agree to use effective contraception during the period of the trial and for at least 90 days after completion of treatment. Male patients must be surgically sterile or must agree to use effective contraception during the period of the trial 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 or a designated associate.
• Patient must be able to swallow capsules and retain orally administered medication.

You may not qualify if:

• Body weight \>10Kg.
• Known active bacterial, fungal or viral infection including hepatitis B (HBV), hepatitis C (HCV).
• Known human immunodeficiency virus (HIV) or acquired immunodeficiency syndrome (AIDS)--related illness.
• Active or prior plasma cell leukemia (defined as either 20% of peripheral WBC comprised of plasma/CD138+ cells or an absolute count of 2 x 10\^9/L).
• Solitary bone or solitary extramedullary plasmacytoma as the only evidence of plasma cell dyscrasia.
• Any of the following in the previous 6 months: myocardial infarction, severe/unstable angina, coronary/peripheral artery bypass graft, symptomatic congestive heart failure, cerebrovascular accident, transient ischemic attack or symptomatic pulmonary embolism.
• Other severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation or study drug administration, or may interfere with the interpretation of study results, or in the judgment of the investigator would make the patient inappropriate for entry into the study.

Sponsors & Collaborators

• Jazz Pharmaceuticals: lead

Study Sites (10)

University of California San Francisco

San Francisco, California, 94143, United States

Location

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States

Location

Children's Healthcare of Atlanta

Atlanta, Georgia, 30342, United States

Location

University of Michigan

Ann Arbor, Michigan, 48109, United States

Location

University of Nebraska Medical Center

Omaha, Nebraska, 68198, United States

Location

Albany Medical Center

Albany, New York, 12208, United States

Location

NYU Langone Health

New York, New York, 10016, United States

Location

Stephen Hassenfeld Children's Center for Cancer and Blood Disorders (NYU)

New York, New York, 10016, United States

Location

University of Rochester Medical Center

Rochester, New York, 14642, United States

Location

Related Publications (1)

• Arrillaga-Romany I, Gardner SL, Odia Y, Aguilera D, Allen JE, Batchelor T, Butowski N, Chen C, Cloughesy T, Cluster A, de Groot J, Dixit KS, Graber JJ, Haggiagi AM, Harrison RA, Kheradpour A, Kilburn LB, Kurz SC, Lu G, MacDonald TJ, Mehta M, Melemed AS, Nghiemphu PL, Ramage SC, Shonka N, Sumrall A, Tarapore RS, Taylor L, Umemura Y, Wen PY. ONC201 (Dordaviprone) in Recurrent H3 K27M-Mutant Diffuse Midline Glioma. J Clin Oncol. 2024 May 1;42(13):1542-1552. doi: 10.1200/JCO.23.01134. Epub 2024 Feb 9.

  PMID: 38335473 DERIVED

MeSH Terms

Conditions

Glioma

Interventions

TIC10 compound

Condition Hierarchy (Ancestors)

Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue

Study Design

• Study Type: expanded access
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 1, 2021
• First Posted: May 26, 2022
• Last Updated: May 26, 2022

Record last verified: 2022-05

Locations

US (10)