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NCT05392374 · CITED IN SOURCE DOCUMENTS

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

An NCI or FDA source cites this study. A citation does not establish that it applies to an individual diagnosis.

Phase
Not reported
Status at capture
NO LONGER AVAILABLE
Registry last update
2022-05-26

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.

Open the original ClinicalTrials.gov record → · Download preserved record

What did the study report?

Outcomes, safety and baseline populations are separate source sections. Quality-of-life measures appear under their original outcome titles.

No posted result sections were captured. Registered plans do not establish that a treatment works.

Who could take part
eligibility Criteria
Inclusion Criteria: 1. Patient with DIPG who failed at least one line of prior therapy such as focal radiation therapy 2. Patient must be \> than 6 months and \< 18 years of age. 3. Confirmed evidence of disease progression from immediately prior therapy or refractory to the immediately prior treatment. 4. Karnofsky/Lansky performance status ≥ 50. 5. 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) \_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_\_ CONFIDENTIAL Page 3 of 45 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) 6. Ability to understand and the willingness to sign a written informed consent document by the patient or their legal guardian. 7. 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. 8. Patient must be able to swallow capsules and retain orally administered medication. Exclusion Criteria: 1. Body weight \>10Kg. 2. Known active bacterial, fungal or viral infection including hepatitis B (HBV), hepatitis C (HCV). 3. Known human immunodeficiency virus (HIV) or acquired immunodeficiency syndrome (AIDS)--related illness. 4. 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). 5. Solitary bone or solitary extramedullary plasmacytoma as the only evidence of plasma cell dyscrasia. 6. 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. 7. 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.
maximum Age
18 Years
minimum Age
6 Months
std Ages
  1. CHILD
  2. ADULT
Treatment arms and interventions
interventions
  1. description
    ONC201 is an oral, small molecule selective antagonist of DRD2
    name
    ONC201
    type
    DRUG
Study designSource null
EnrollmentSource null
Registered outcome plans (not posted results)
Full study description
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.
Source references
references
  1. citation
    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
    type
    DERIVED
Source notices and limitations
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    Preserved source evidence · Not medical advice