Clinical trial NCT05392374: Expanded Access Use of ONC201 in a Patient With Diffuse Intrinsic Pontine Gliomas
Official title: Expanded Access Use of ONC201 in a Patient With Diffuse Intrinsic Pontine Gliomas
Study type: EXPANDED_ACCESS
Overall status: NO_LONGER_AVAILABLE
Conditions: Glioma
Lead sponsor: Chimerix
Sponsor class: INDUSTRY
First posted: 2022-05-26
Minimum age (years): 6
Maximum age (years): 18
Standard ages: CHILD
ADULT

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.


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)

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   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.

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: San Francisco
country: United States
facility: University of California San Francisco
state: California
city: Aurora
country: United States
facility: Children's Hospital Colorado
state: Colorado
city: Washington
country: United States
facility: Children's National Medical Center
state: District of Columbia
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta
state: Georgia
city: Ann Arbor
country: United States
facility: University of Michigan
state: Michigan
city: Omaha
country: United States
facility: University of Nebraska Medical Center
state: Nebraska
city: Albany
country: United States
facility: Albany Medical Center
state: New York
city: New York
country: United States
facility: NYU Langone Health
state: New York
city: New York
country: United States
facility: Stephen Hassenfeld Children's Center for Cancer and Blood Disorders (NYU)
state: New York
city: Rochester
country: United States
facility: University of Rochester Medical Center
state: New York
Study officials:
References: