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

• Sponsor: Chimerix (Industry)
• Overall Status: Available
• CT.gov ID: NCT04617002
• Collaborator: (none)
• Locations: 22

Study Details

Study Description

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	Phase
Glioma; H3 K27M	Drug: ONC201

Study Design

• Study Type: Expanded Access
• Official Title: Intermediate-size Expanded Access to ONC201 for Patients With H3 K27M-mutant and/or Midline Gliomas

Outcome Measures

Primary Outcome Measures

Eligibility Criteria

Criteria

• Ages Eligible for Study: 2 Years and Older
• 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

1. 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.

1. 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.

2. Patient must be at least 2 years of age.

3. Patient must weigh at least 10kg.

4. 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.

5. Brain MRI within 21 days prior to start of study drug.

6. Adequate organ and marrow function as defined below:

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

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

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

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

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

12. 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.

13. 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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Children's Hospital of Orange County	Orange	California	United States	92868
2	Rady Children's Hospital	San Diego	California	United States	92123
3	Providence Saint John's Health Center	Santa Monica	California	United States	90404
4	Providence Saint John's Health Center	Santa Monica	California	United States	90404
5	Children's Hospital of Colorado	Aurora	Colorado	United States	80045
6	Children's National Medical Center	Washington	District of Columbia	United States	20010
7	Miami Cancer Institute	Miami	Florida	United States	33176
8	Children's Healthcare of Atlanta, Emory University School of Medicine	Atlanta	Georgia	United States	30322
9	Kapi'olani Medical Center for Women and Children	Honolulu	Hawaii	United States	96826
10	Lurie Children's Hospital	Chicago	Illinois	United States	60661
11	University of Iowa Hospitals and Clinics	Iowa City	Iowa	United States	52242
12	University of Michigan	Ann Arbor	Michigan	United States	48109
13	University of Minnesota	Minneapolis	Minnesota	United States	55455
14	Washington University in St. Louis	Saint Louis	Missouri	United States	63110
15	University of Nebraska Medical Center - Adults Only	Omaha	Nebraska	United States	68198
16	Overlook Medical Center/ Atlantic Health System	Summit	New Jersey	United States	07901
17	Albany Medical Center	Albany	New York	United States	12208
18	New York University	New York	New York	United States	10016
19	University of Rochester	Rochester	New York	United States	14627
20	University of Texas Southwestern	Dallas	Texas	United States	75390
21	MD Anderson Cancer Center	Houston	Texas	United States	77030
22	Children's Hospital of The King's Daughters	Norfolk	Virginia	United States	23507

Sponsors and Collaborators

• Chimerix

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Chimerix
• ClinicalTrials.gov Identifier: NCT04617002
• Other Study ID Numbers: ONC028
• First Posted: Nov 5, 2020
• Last Update Posted: Jun 16, 2022
• Last Verified: Mar 1, 2022

• Additional relevant MeSH terms: Glioma; TIC10 compound

Study Results

No Results Posted as of Jun 16, 2022