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 to patients with H3 K27M-mutant and/or midline gliomas who cannot access ONC201 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 2 Years and Over
Gender All
More Inclusion & Exclusion Criteria

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. 3. 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. 3. 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. 4. Patient must be at least 2 years of age. 5. Patient must weigh at least 10kg. 6. 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. 7. Brain MRI within 21 days prior to start of study drug. 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 ≥ 60 mL/min/1.73 m2) 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 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

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.

Rady Children's Hospital, San Diego, California

Status

Available

Address

Rady Children's Hospital

San Diego, California, 92123

Site Contact

Megan Paul, MD

mrpaul@rchsd.org

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

Akanksha.Sharma@providence.org

1-919-806-1074

Children's Hospital of Colorado, Aurora, Colorado

Status

Available

Address

Children's Hospital of Colorado

Aurora, Colorado, 80045

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

LKilburn@childrensnational.org

202-476-3643

Miami Cancer Institute, Miami, Florida

Status

Available

Address

Miami Cancer Institute

Miami, Florida, 33176

Site Contact

Yazmin Odia, MD, MS

DG-MCIResearchNurses@baptisthealth.net

786-596-2000

Atlanta, Georgia

Status

Available

Address

ON HOLD: (currently not accepting new patients) Children's Healthcare of Atlanta, Emory University School of Medicine

Atlanta, Georgia, 30322

Site Contact

Dolly Aguilera, MD

AflacDevTReferral@choa.org

404-785-1112

Honolulu, Hawaii

Status

Available

Address

Kapi'olani Medical Center for Women and Children

Honolulu, Hawaii, 96826

Site Contact

Andrea Siu, MPH

Andrea.siu@hawaiipacifichealth.org

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

awaanders@luriechildrens.org

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

david-dickens@uiowa.edu

1-919-806-1074

University of Michigan, Ann Arbor, Michigan

Status

Available

Address

University of Michigan

Ann Arbor, Michigan, 48109

Site Contact

Misty Gravelin

gravelim@med.umich.edu

734-763-0592

University of Minnesota, Minneapolis, Minnesota

Status

Available

Address

University of Minnesota

Minneapolis, Minnesota, 55455

Site Contact

Clark Chen, MD, PhD

ccchen@umn.edu

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

acluster@wustl.edu

1-919-806-1074

Summit, New Jersey

Status

Available

Address

Overlook Medical Center/ Atlantic Health System

Summit, New Jersey, 07901

Site Contact

Jeanna Johnson

Jeanna.Johnson@atlantichealth.org

973-829-4869

Albany Medical Center, Albany, New York

Status

Available

Address

Albany Medical Center

Albany, New York, 12208

Site Contact

Lauren Weintraub, MD

weintrl@amc.edu

1-919-806-1074

New York University, New York, New York

Status

Available

Address

New York University

New York, New York, 10016

Site Contact

Sharon Gardner, MD

Sharon.Gardner@nyumc.org

646-929-7870

University of Rochester, Rochester, New York

Status

Available

Address

University of Rochester

Rochester, New York, 14627

Site Contact

David Korones, MD

David_korones@urmc.rochester.edu

585-275-2981

University of Texas Southwestern, Dallas, Texas

Status

Available

Address

University of Texas Southwestern

Dallas, Texas, 75390

Site Contact

Alison Patterson

alison.patterson@childrens.com

214-456-2726

Norfolk, Virginia

Status

Available

Address

Children's Hospital of The King's Daughters

Norfolk, Virginia, 23507

Site Contact

Melissa Mark, MD

Melissa.Mark@CHKD.ORG

1-919-806-1074

The content provided by NBTS on clinical trials is for informational purposes only and is not a substitute for medical consultation with your healthcare provider. We do not recommend or endorse any specific study and you are advised to discuss the information shown with your healthcare provider. While we believe the information presented on this website to be accurate at the time of writing, we do not guarantee that its contents are correct, complete, or applicable to any particular individual situation. We strongly encourage individuals to seek out appropriate medical advice and treatment from their physicians. We cannot guarantee the availability of any clinical trial listed and will not be responsible if you are considered ineligible to participate in a given clinical trial. We are also not liable for any injury arising as a result of participation. For a full description of terms please refer to: Terms, Conditions & Privacy