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

Study Purpose

This is an intermediate-size expanded access protocol to provide ONC201 (dordaviprone) to patients with H3 K27M-mutant and/or midline gliomas who cannot access ONC201 (dordaviprone) through clinical trials.

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

Unknown
Study Type

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.


An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.


Searching Both is inclusive of interventional and observational studies.

Expanded Access
Eligible Ages 0 Years and Over
Gender All
More Inclusion & Exclusion Criteria

Inclusion Criteria:

1. Patient meets one or more of the criteria below: Arm A: Closed to further enrollment. Arm B: 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. Arm C: Patients with primary spinal glioma that is positive for the H3 K27M mutation (performed in a laboratory with CLIA or equivalent certification). Primary spinal glioma must be documented in radiology reporting. OR Patients with diffuse glioma that is positive for the H3 K27M mutation (performed in a laboratory with CLIA or equivalent certification) AND radiographic evidence of leptomeningeal disease. Leptomeningeal disease must be documented in radiology reporting. Arm D: Closed to further enrollment. Arm E: Patients with H3 K27M-mutant glioma or midline glioma of unknown H3 K27M mutational status who received ONC201 and/or ONC206 from an alternative (non-Chimerix) source prior to 31 December 2023 as evidenced by documentation (such as pharmacy receipts). Other examples may be confirmed by medical monitor. Detection of H3 K27M mutation should be performed in a CLIA-certified or equivalent laboratory. Arm F: Patients with H3 K27M-mutant diffuse glioma or midline glioma with unknown H3 K27M mutational status who have completed frontline radiotherapy prior to 31 December 2023. Detection of H3 K27M mutation should be performed in a CLIA-certified or equivalent laboratory. 2. Disease Status: Arm B: Patient is not required to have radiographic or clinical evidence of progressive disease. Arm C: Patient must have progressive disease as defined by RANO criteria or have documented recurrent glioma on diagnostic biopsy. Arm E: Not Applicable. Arm F: Patient must have progressive disease as defined by RANO criteria or have documented recurrent glioma on diagnostic biopsy. 3. Prior Radiotherapy: Arm B: Patient must be at least 14 days from completion of radiotherapy. Arm C: Patient must be at least 90 days from completion of radiotherapy and at least 14 days from reirradiation if applicable. Arm E: Not applicable. Arm F: Patient must be at least 90 days from completion of frontline radiotherapy and at least 14 days from reirradiation if applicable. 4. (Not applicable; criterion removed in Version 5). 5. Patient must weigh at least 10kg. 6. Washouts: Arm B, C & F: 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 (no washout required 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. Arm E: No washouts are required for ONC021 and ONC206. All other anti-cancer agents need to be discontinued prior to enrollment with the exception of bevacizumab. 7. Arm B, C & F: MRI of patient's glioma obtained within 28 days prior to the start of study drug. Arm E: MRI will be obtained within 8 weeks prior to enrollment. 8. 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]) 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 ≥60mL/min/1.73m2) Arm E: Patients with organ and marrow function values outside the defined criteria must be approved by medical monitor. 9. 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. 10. Ability to understand a written informed consent document, and the willingness to sign it. Assent will be obtained when appropriate based on the patient's age.

Exclusion Criteria:

1. Qualifies for participation in an ongoing ONC201 or ONC206 clinical trial. 2. Arm B, C & F: Previously or current enrollment in an ONC201 clinical study (including open-label and blinded studies) or expanded access protocol or previous exposure to ONC201 from any other source for treatment of CNS tumor. Arm E: Previous or current enrollment in an ONC201 clinical study (including open-label and blinded studies) or expanded access protocol for the treatment of CNS tumor. 3. Arms B, C, E & F: Current or planned participation in a study of an investigational agent (including ONC206) or using an investigational device. 4. (Not applicable; criterion removed in Version 4). 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 Fridericia's QT correction formula on two ECGs separated by at least 2 days. 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.

Trial Details

Trial ID:

This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.

NCT04617002
Phase

Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.

Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

Chimerix
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

N/A
Principal Investigator Affiliation N/A
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

Industry
Overall Status Available
Countries United States
Conditions

The disease, disorder, syndrome, illness, or injury that is being studied.

Glioma, H3 K27M

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

Phoenix, Arizona

Status

Available

Address

BMDACC at Banner University Medical Center Phoenix

Phoenix, Arizona, 85006

Site Contact

Madelyn Cooper

[email protected]

1-919-806-1074

Los Angeles, California

Status

Available

Address

Kaiser Permanente Los Angeles Medical Center

Los Angeles, California, 90027

Site Contact

Hema V Buddha

[email protected]

1-919-806-1074

Children's Hospital of Orange County, Orange, California

Status

Available

Address

Children's Hospital of Orange County

Orange, California, 92868

Site Contact

Mariko Sato

[email protected]

1-919-806-1074

Rady Children's Hospital, San Diego, California

Status

Available

Address

Rady Children's Hospital

San Diego, California, 92123

Site Contact

Megan Paul, MD

[email protected]

1-919-806-1074

Providence Saint John's Health Center, Santa Monica, California

Status

Available

Address

Providence Saint John's Health Center

Santa Monica, California, 90404

Site Contact

Akanksha Sharma, MD

[email protected]

1-919-806-1074

Children's Hospital of Colorado, Aurora, Colorado

Status

Available

Address

Children's Hospital of Colorado

Aurora, Colorado, 80045

Site Contact

Ashley Mettetal

[email protected]

720-777-5305

Children's National Medical Center, Washington, District of Columbia

Status

Available

Address

Children's National Medical Center

Washington, District of Columbia, 20010

Site Contact

Lindsay Kilburn, MD, MS

[email protected]

202-476-3643

Miami Cancer Institute, Miami, Florida

Status

Available

Address

Miami Cancer Institute

Miami, Florida, 33176

Site Contact

Yazmin Odia, MD, MS

[email protected]

786-596-2000

University Cancer & Blood Center, Athens, Georgia

Status

Available

Address

University Cancer & Blood Center

Athens, Georgia, 30607

Site Contact

Nikki Pope

[email protected]

1-919-806-1074

Honolulu, Hawaii

Status

Available

Address

Kapi'olani Medical Center for Women and Children

Honolulu, Hawaii, 96826

Site Contact

Andrea Siu, MPH

[email protected]

808-535-7169

Lurie Children's Hospital, Chicago, Illinois

Status

Available

Address

Lurie Children's Hospital

Chicago, Illinois, 60661

Site Contact

Angela Waanders, MD, MPH

[email protected]

1-919-806-1074

University of Iowa Hospitals and Clinics, Iowa City, Iowa

Status

Available

Address

University of Iowa Hospitals and Clinics

Iowa City, Iowa, 52242

Site Contact

David Dickens, MD

[email protected]

1-919-806-1074

University of Michigan, Ann Arbor, Michigan

Status

Available

Address

University of Michigan

Ann Arbor, Michigan, 48109

Site Contact

Misty Gravelin

[email protected]

734-763-0592

University of Minnesota, Minneapolis, Minnesota

Status

Available

Address

University of Minnesota

Minneapolis, Minnesota, 55455

Site Contact

Clark Chen, MD, PhD

[email protected]

1-919-806-1074

Washington University in St. Louis, Saint Louis, Missouri

Status

Available

Address

Washington University in St. Louis

Saint Louis, Missouri, 63110

Site Contact

Andrew Cluster, MD

[email protected]

1-919-806-1074

Omaha, Nebraska

Status

Available

Address

University of Nebraska Medical Center - Adults Only

Omaha, Nebraska, 68198

Site Contact

Nicole Shonka

[email protected]

1-919-806-1074

Summit, New Jersey

Status

Available

Address

Overlook Medical Center/ Atlantic Health System

Summit, New Jersey, 07901

Albany Medical Center, Albany, New York

Status

Available

Address

Albany Medical Center

Albany, New York, 12208

Site Contact

Lauren Weintraub, MD

[email protected]

1-919-806-1074

New York, New York

Status

Available

Address

New York University Langone - Active, Enrolling

New York, New York, 10016

Site Contact

Anna Yaffe

[email protected]

1-919-806-1074

University of Rochester, Rochester, New York

Status

Available

Address

University of Rochester

Rochester, New York, 14627

Site Contact

David Korones, MD

[email protected]

585-275-2981

Portland, Oregon

Status

Available

Address

Providence Neurological Specialties Clinic

Portland, Oregon, 97225

Site Contact

Sara Guedry

[email protected]

1-919-806-1074

University of Texas Southwestern, Dallas, Texas

Status

Available

Address

University of Texas Southwestern

Dallas, Texas, 75390

Site Contact

Alison Patterson

[email protected]

214-456-2726

MD Anderson Cancer Center, Houston, Texas

Status

Available

Address

MD Anderson Cancer Center

Houston, Texas, 77030

Site Contact

Teresa Hannah

[email protected]

1-919-806-1074

Huntsman Cancer Institute, Salt Lake City, Utah

Status

Available

Address

Huntsman Cancer Institute

Salt Lake City, Utah, 84112

Site Contact

Yuri Kida

[email protected]

1-919-806-1074

Norfolk, Virginia

Status

Available

Address

Children's Hospital of The King's Daughters

Norfolk, Virginia, 23507

Site Contact

Melissa Mark, MD

[email protected]

1-919-806-1074

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