GliomaNCT05392374
Expanded Access Use of ONC201 in a Patient With Diffuse Intrinsic Pontine Gliomas
A study testing ONC201 in people with Glioma, run by Jazz Pharmaceuticals at 10 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 6 months to 18 years
- Conditions the study is for
- Glioma
- What is being tested?
- ONC201
- Type of treatment
- Pill or oral medicine
- How many people
- Not given
- Where
- 10 sitesUnited States
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 months to 18 years
- Child, Adult
Who may be able to join
- Patient with DIPG who failed at least one line of prior therapy such as focal radiation therapy
- Patient must be > than 6 months and < 18 years of age.
- Confirmed evidence of disease progression from immediately prior therapy or refractory to the immediately prior treatment.
- Karnofsky/Lansky performance status ≥ 50.
- Adequate organ and marrow function as defined below:
- Absolute neutrophil count ≥1,000/mm3 without growth factor use ≤ 7 days prior to treatment (cycle 1 day 1, C1D1)
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- Hemoglobin>8.0 mg/dL without red blood cell transfusion ≤ 3 days prior to C1D1
- Total serum bilirubin<1.5 X upper limit of normal (ULN)
- AST (SGOT)/ALT (SGPT)≤2 X ULN;; ≤ 5 X ULN if there is liver involvement secondary to tumor
- Serum creatinine≤ 1.5 X ULN (OR creatinine clearance ≥ 60 mL/min/1.73 m2)
- Ability to understand and the willingness to sign a written informed consent document by the patient or their legal guardian.
- 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.
- Patient must be able to swallow capsules and retain orally administered medication.
Who cannot join
- Body weight >10Kg.
- Known active bacterial, fungal or viral infection including hepatitis B (HBV), hepatitis C (HCV).
- Known human immunodeficiency virus (HIV) or acquired immunodeficiency syndrome (AIDS)--related illness.
- 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).
- Solitary bone or solitary extramedullary plasmacytoma as the only evidence of plasma cell dyscrasia.
- 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.
- 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.
Trial locations and contacts
10 sites in 1 country.
10 sites in 1 country
United States10 sites
Albany Medical Center
Albany, New York, 12208
University of Michigan
Ann Arbor, Michigan, 48109
Children's Healthcare of Atlanta
Atlanta, Georgia, 30342
Children's Hospital Colorado
Aurora, Colorado, 80045
NYU Langone Health
New York, New York, 10016
Stephen Hassenfeld Children's Center for Cancer and Blood Disorders (NYU)
New York, New York, 10016
University of Nebraska Medical Center
Omaha, Nebraska, 68198
University of Rochester Medical Center
Rochester, New York, 14642
University of California San Francisco
San Francisco, California, 94143
Children's National Medical Center
Washington D.C., District of Columbia, 20010
About this study
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.
- Conditions
- Glioma
- Being tested
- ONC201
- Medical subject headings
- Glioma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ONC201Drug
ONC201 is an oral, small molecule selective antagonist of DRD2
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 May 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for GliomaStudies of the same condition, open and under way.
- More trials run by Jazz PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Jazz Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05392374
- Sponsor's protocol ID
- ONC016
- Status last verified
- May 2022
- First submitted
- 1 Dec 2021
- Last updated
- 26 May 2022
- Results posted
- No
Publications
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.
PubMed 38335473
Study at a glance
From the registry record, last updated by the sponsor on 26 May 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Expanded Access Use of ONC201 in a Patient With Diffuse Intrinsic Pontine Gliomas
- ClinicalTrials.gov ID
- NCT05392374
- Acronym / sponsor ID
- ONC016
- Conditions
- Glioma
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- Jazz PharmaceuticalsIndustry (pharma, biotech, devices)
- First posted
- 26 May 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
Who may qualify
- Ages
- 6 months to 18 years
- Age groups
- Child, Adult
Key inclusion criteria (14 listed)
- Patient with DIPG who failed at least one line of prior therapy such as focal radiation therapy
- Patient must be > than 6 months and < 18 years of age.
- Confirmed evidence of disease progression from immediately prior therapy or refractory to the immediately prior treatment.
- Karnofsky/Lansky performance status ≥ 50.
- Adequate organ and marrow function as defined below:
Key exclusion criteria (7 listed)
- Body weight >10Kg.
- Known active bacterial, fungal or viral infection including hepatitis B (HBV), hepatitis C (HCV).
- Known human immunodeficiency virus (HIV) or acquired immunodeficiency syndrome (AIDS)--related illness.
- 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).
- Solitary bone or solitary extramedullary plasmacytoma as the only evidence of plasma cell dyscrasia.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ONC201ONC201 is an oral, small molecule selective antagonist of DRD2 | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 May 2022.
- Derived from this study: PubMed 38335473Arrillaga-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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
10 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing ONC201 in people with Glioma, run by Jazz Pharmaceuticals at 10 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 14 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 26 May 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05392374, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 May 2022.