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

NCT03134131 | Unknown

• 1 other identifier: ONC018
• Study Type: expanded_access
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

The objective of this expanded access program is to provide ONC201 to eligible patients with previously-treated glioma that exhibits the H3 K27M mutation and/or that is located in the midline region of the brain.

Conditions

Glioma

Interventions

ONC201 DRUG

ONC201 is an oral, small molecule selective antagonist of DRD2 that induces tumor cell death.

Eligibility Criteria

• Age: 3 Years+
• Sex: all
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Patient must have one type of diagnosis below:
• A glioma that is positive for the H3 K27M mutation (performed in a laboratory with CLIA certification);
• A grade III or IV glioma involving the thalamus, hypothalamus, brainstem, cerebellum, midbrain, or spinal cord;
• Diffuse intrinsic pontine glioma (DIPG), defined as tumors with a pontine epicenter and diffuse involvement of the pons. These patients are eligible with or without a tissue biopsy.
• Unequivocal evidence of progressive disease on as defined by RANO criteria or have documented recurrent glioma on diagnostic biopsy.
• Patient must have had previous therapy that includes radiotherapy.
• Interval of at least 90 days from the completion of radiotherapy to the first dose of ONC201. If patients are within 90 days of radiotherapy, they may still be eligible if they meet one or more of the following criteria.
• Progressive tumor is outside the original high-dose radiotherapy target volume as determined by the treating investigator, or
• Histologic confirmation of tumor through biopsy or resection, or
• Nuclear medicine imaging, MR spectroscopy, or MR perfusion imaging consistent with true progressive disease, rather than pseudoprogression or radiation necrosis obtained within 28 days of registration.
• Patient must be at least 3 years of age.
• Patient must weigh at least 10kg.
• Patient must be able to swallow and retain orally administered medication. For patients unable to swallow capsules, oral ONC201 will be administered as a liquid formulation in Ora-Sweet.
• 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), or 4 weeks (or 5 half-lives, whichever is shorter) from other anti-tumor therapies.
• Contrast-enhanced head CT or brain MRI within 21 days prior to start of study drug.

You may not qualify if:

• Qualifies for participation in an ongoing ONC201 clinical trial or is already participating in an ONC201 clinical trial.
• Current or planned participation in a study of an investigational agent or using an investigational device.
• Evidence of diffuse leptomeningeal disease or CSF dissemination.
• Any known systemic infection that, in the opinion of the investigator, could compromise the safety of the patient, while taking ONC201.

Sponsors & Collaborators

• Jazz Pharmaceuticals: lead

Study Sites (1)

Oncoceutics

Philadelphia, Pennsylvania, 19104, 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: April 20, 2017
• First Posted: April 28, 2017
• Last Updated: March 14, 2022

Record last verified: 2020-10

Locations

US (1)