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

• Sponsor: Chimerix (Industry)
• Overall Status: No longer available
• CT.gov ID: NCT05392374
• Collaborator: (none)
• Locations: 10

Study Details

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.

Condition or Disease	Intervention/Treatment	Phase
Glioma	Drug: ONC201

Study Design

• Study Type: Expanded Access
• Official Title: Expanded Access Use of ONC201 in a Patient With Diffuse Intrinsic Pontine Gliomas

Outcome Measures

Primary Outcome Measures

Eligibility Criteria

Criteria

• Ages Eligible for Study: 6 Months to 18 Years
• Sexes Eligible for Study: All

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:

6. Absolute neutrophil count ≥1,000/mm3 without growth factor use ≤ 7 days prior to treatment (cycle 1 day 1, C1D1)

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1. Hemoglobin>8.0 mg/dL without red blood cell transfusion ≤ 3 days prior to C1D1

2. Total serum bilirubin<1.5 X upper limit of normal (ULN)

3. AST (SGOT)/ALT (SGPT)≤2 X ULN;; ≤ 5 X ULN if there is liver involvement secondary to tumor

4. Serum creatinine≤ 1.5 X ULN (OR creatinine clearance ≥ 60 mL/min/1.73 m2)

5. Ability to understand and the willingness to sign a written informed consent document by the patient or their legal guardian.

6. 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.

7. 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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	University of California San Francisco	San Francisco	California	United States	94143
2	Children's Hospital Colorado	Aurora	Colorado	United States	80045
3	Children's National Medical Center	Washington	District of Columbia	United States	20010
4	Children's Healthcare of Atlanta	Atlanta	Georgia	United States	30342
5	University of Michigan	Ann Arbor	Michigan	United States	48109
6	University of Nebraska Medical Center	Omaha	Nebraska	United States	68198
7	Albany Medical Center	Albany	New York	United States	12208
8	NYU Langone Health	New York	New York	United States	10016
9	Stephen Hassenfeld Children's Center for Cancer and Blood Disorders (NYU)	New York	New York	United States	10016
10	University of Rochester Medical Center	Rochester	New York	United States	14642

Sponsors and Collaborators

• Chimerix

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Chimerix
• ClinicalTrials.gov Identifier: NCT05392374
• Other Study ID Numbers: ONC016
• First Posted: May 26, 2022
• Last Update Posted: May 26, 2022
• Last Verified: May 1, 2022

• Additional relevant MeSH terms: Glioma; Diffuse Intrinsic Pontine Glioma; TIC10 compound

Study Results

No Results Posted as of May 26, 2022