GliomaPhase 2NCT03295396
ONC201 in Adults With Recurrent H3 K27M-mutant Glioma
A Phase 2 study testing Dordaviprone (ONC201) in people with Glioma, run by Jazz Pharmaceuticals at 9 sites in United States.
Trial summary
This was a Phase 2, open-label, 2-arm study of dordaviprone (ONC201) in patients with recurrent H3 K27M- mutant glioma. The primary assessment of dordaviprone (ONC201) involved evaluating its anti-tumor activity through the overall response rate according to the Response Assessment in Neuro-Oncology (RANO) criteria… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Glioma
- What is being tested?
- Dordaviprone (ONC201)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 73 participants
- Where
- 9 sitesUnited States
- Study dates
- 30 Oct 2017to 19 Jul 2023
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Had histologically confirmed diagnosis of high-grade glioma (HGG) in any tumor sample and presence of histone H3 K27M mutation detected in a Clinical Laboratory Improvement Amendment (CLIA) certified laboratory by immunohistochemistry or DNA sequencing test on any glioma tumor sample.
- Had unequivocal evidence of progressive disease on contrast-enhanced brain computed tomography (CT) or magnetic resonance imaging (MRI) as defined by Response Assessment in Neuro-Oncology (RANO)-HGG criteria, or have documented recurrent glioma on diagnostic biopsy.
- Had measurable disease by RANO-HGG criteria.
- Patients must have had previous therapy with at least radiotherapy.
- Had no more than two prior episodes of recurrence from radiotherapy and/or chemotherapy. Use of bevacizumab solely for treatment of radiation necrosis, pseudoprogression, or treatment effect was not considered a recurrence.
- Had an interval of at least 90 days from the completion of radiotherapy to the first dose of dordaviprone (ONC201). If patients were within 90 days of radiotherapy, they may have still been eligible if they met one or more of the following criteria.
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- 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, magnetic resonance (MR) spectroscopy, or MR perfusion imaging consistent with true progressive disease, rather than pseudoprogression or radiation necrosis obtained within 28 days of registration.
- From the projected start of scheduled study treatment, the following time periods must have had elapsed: 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 antibodies, or 4 weeks (or 5 half-lives, whichever is shorter) from other anti-tumor therapies.
- All adverse events Grade >1 related to prior therapies (chemotherapy, radiotherapy, and/or surgery) must have been resolved to Grade 1 or baseline, except for alopecia and sensory neuropathy Grade ≤2, or other Grade ≤2 not constituting a safety risk based on investigator's judgment, are acceptable.
- Were male or female aged ≥18 years.
- Had a Karnofsky Performance Status (KPS) ≥60.
- Had adequate organ and marrow function as defined below, all screening labs should be performed within 14 days of treatment initiation:
- leukocytes ≥ 3,000/mcL
- absolute neutrophil count ≥ 1,500/mcL
- platelets ≥ 75,000/mcL
- hemoglobin > 8.0 mg/dL
- total bilirubin ≤ 2.0 × upper limit of normal (ULN)
- aspartate aminotransferase (AST) [SGOT]/alanine aminotransferase (ALT) [SGPT] ≤ 2.5 × ULN
- creatinine ≤ULN OR creatinine clearance ≥60 mL/min/1.73 m2 for patients with creatinine levels above normal.
- Had contrast-enhanced head CT or brain MRI and entire spine MRI within 14 days prior to start of study drug.
- Corticosteroid dose must have been stable or decreasing for at least 3 days prior to the baseline CT or MRI scan.
- Women of childbearing potential (WOCBP) and men must have agreed to use adequate contraception prior to study entry and for the duration of study participation and for 30 days after the last dose of therapy. Highly effective contraceptive measures include: stable use of oral contraceptives such as combined estrogen and progestogen and progestogen only hormonal contraception or other prescription pharmaceutical contraceptives for 2 or more menstrual cycles prior to screening; intrauterine device [IUD]; intrauterine hormone-releasing system (IUS); bilateral tubal ligation; vasectomy and sexual abstinence.
- WOCBP must have had a negative serum or urine pregnancy test within 28 days of initiation of dosing.
- Contraception was not required for men with documented vasectomy.
- Postmenopausal women must have been amenorrheic for at least 12 months in order not to be considered of childbearing potential.
- Pregnancy testing and contraception were not required for women with documented hysterectomy or tubal ligation.
- Had availability of a paraffin-embedded or frozen tumor-tissue block with a minimum of 1 cm2 of tumor surface area, or 20 unstained slides from the tumor tissue specimen if a tumor block cannot be submitted. If a patient has had only a stereotactic biopsy, then 5 unstained slides may be accepted with prior approval from the Sponsor, however all efforts must be made to obtain as close to 20 slides as possible.
- Had the ability to be able to swallow and retain orally administered medication
- Had the ability to understand and the willingness to sign a written informed consent document. Only patients who had capacity to consent were enrolled in the study.
Who cannot join
- Arm B: Had a primary malignant lesion located in the pons or spinal cord.
- Arm B: Had atypical non-astrocytic histologies such as ependymoma, ganglioma and pleomorphic xanthoastrocytoma, or pilocytic astrocytoma and subependymal giant cell astrocytoma (SEGA).
- Arm B: Had prior bevacizumab treatment of >4 doses of >7.5 mg/kg
- Had a history of allergic reactions attributed to compounds of similar chemical or biologic composition to dordaviprone (ONC201) or its excipients.
- Had current or planned participation in a study of an investigational agent or using an investigational device.
- Had presence of diffuse leptomeningeal disease or evidence of cerebrospinal fluid (CSF) dissemination.
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- Had uncontrolled intercurrent illness including, but not limited to, ongoing or active infection or psychiatric illness/social situations that would limit compliance with study requirements.
- Had an active infection requiring systemic therapy.
- Were pregnant and/or breastfeeding women or unable to maintain use of contraception while on study and for 30 days after the last dose of study drug. Dordaviprone (ONC201) is a novel agent with unknown potential for teratogenic or abortifacient effects.
- Had known HIV-positive test on combination antiretroviral therapy.
- Had known history of cardiac arrhythmias including atrial fibrillation, tachyarrhythmias or bradycardia, unless arrhythmia is controlled and after Cardiology has cleared patient to receive dordaviprone (ONC201). Receiving therapeutic agents known to prolong QT interval will be excluded. History of congested heart failure (CHF), or myocardial infarction (MI) or stroke in the last 3 months will be excluded.
- Had active illicit drug use or diagnosis of alcoholism.
- Had tumors with known IDH1 (isocitrate dehydrogenase 1) or known IDH2 mutations as determined by immunohistochemistry for the IDH1 R132H variant or by direct sequencing. IDH1/2-mutant gliomas have a markedly longer overall survival rate compared to those with IDH1/2-wildtype glioma (Parsons et al., 2008; Yan et al., 2009), indicating IDH1/2-mutant gliomas have a distinct natural history.
- Had tumors with known 1p/19q co-deletion.
- Had known additional malignancy that is progressing or requires active treatment within 3 years of start of study drug. Exceptions include basal cell carcinoma of the skin, squamous cell carcinoma of the skin, in situ melanoma, or in situ cervical cancer that has undergone potentially curative therapy.
- Had any surgery (not including minor diagnostic procedures such as lymph node biopsy) within 2 weeks of baseline disease assessments; or not fully recovered from any side effects of previous procedures. An interval of 1 week for stereotactic brain biopsy from the start of study treatment is acceptable.
- Had concomitant use of potent cytochrome P450 (CYP)3A4/5 inhibitors during the treatment phase of the study and within 72 hours prior to starting study drug administration.
- Had concomitant use of potent CYP3A4/5 inducers, which include enzyme inducing antiepileptic drugs (EIAEDs), during the treatment phase of the study and within 2 weeks prior to starting treatment. Concurrent dexamethasone is allowed.
Trial locations and contacts
9 sites in 1 country.
9 sites in 1 country
United States9 sites
University of Michigan
Ann Arbor, Michigan, 48109
Levine Cancer Institute
Charlotte, North Carolina, 28204
MD Anderson Cancer Center
Houston, Texas, 77030
University of Minnesota
Minneapolis, Minnesota, 55455
New York University School of Medicine
New York, New York, 10016
Columbia University
New York, New York, 10032
Hospital of the University of Pennsylvania
Philadelphia, Pennsylvania, 19104
University of California, San Francisco
San Francisco, California, 94143-0112
Stanford Cancer Center
Stanford, California, 94305
About this study
Official title: A Phase II, Open-label Study of ONC201 in Adults With Recurrent High-grade Glioma
This was a Phase 2, open-label, 2-arm study of dordaviprone (ONC201) in patients with recurrent H3 K27M- mutant glioma.
The primary assessment of dordaviprone (ONC201) involved evaluating its anti-tumor activity through the overall response rate according to the Response Assessment in Neuro-Oncology (RANO) criteria for high-grade glioma (HGG).
Read the detailed description
This study included 2 arms:
- Arm A included patients with recurrent H3 K27M-mutant glioma.
- Arm B included patients with recurrent H3 K27M-mutant glioma, but excluded patients with the following:
- Primary malignant lesion located in the pons or spinal cord.
- Atypical non-astrocytic histologies such as ependymoma, ganglioma and pleomorphic xanthoastrocytoma, or pilocytic astrocytoma and subependymal giant cell astrocytoma (SEGA).
- Prior bevacizumab treatment of >4 doses of >7.5 mg/kg
Patients received dordaviprone (ONC201) 625 mg once weekly.
The primary assessment of dordaviprone (ONC201) involved evaluating its anti-tumor activity through the overall response rate according to the Response Assessment in Neuro-Oncology (RANO) criteria for high-grade glioma (HGG). Safety was also assessed, with evaluations including the reporting of adverse events, as well as measurements of vital signs and clinical laboratory results.
This study was terminated by an administrative protocol amendment (17 January 2023). The decision to terminate the study was not related to any safety concerns with dordaviprone (ONC201). Before the study was terminated, a total of 73 patients were enrolled and received at least 1 dose of dordaviprone (ONC201).
- Conditions
- Glioma
- Being tested
- Dordaviprone (ONC201)
- Medical subject headings
- Glioma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Dordaviprone (ONC201)Drug
Also called Dordaviprone
Dordaviprone (ONC201) is a central nervous system (CNS)-penetrant, small-molecule imipridone that acts as a mitochondrial caseinolytic protease P (ClpP) agonist and a dopamine receptor D2 (DRD2) antagonist.
| Study group | Role | What they receive |
|---|---|---|
| Arm A | Experimental | Patients received 625 mg dordaviprone (ONC201) once weekly.
Arm A included patients with recurrent H3 K27M-mutant glioma including those with diffuse intrinsic pontine glioma (DIPG), primary spinal tumors, and some atypical histologies. Receives: Drug: Dordaviprone (ONC201) |
| Arm B | Experimental | Patients received 625 mg dordaviprone (ONC201) once weekly.
Arm B included patients with recurrent H3 K27M-mutant glioma, but excluded patients with the following:
* Primary malignant lesion located in the pons or spinal cord.
* Atypical non-astrocytic histologies such as ependymoma, ganglioma and pleomorphic xanthoastrocytoma, or pilocytic astrocytoma and subependymal giant cell astrocytoma (SEGA).
* Prior bevacizumab treatment of >4 doses of >7.5 mg/kg Receives: Drug: Dordaviprone (ONC201) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Patients With Overall Response
Quantitative thresholds for objective response by Response Assessment in Neuro-Oncology (RANO) criteria for target lesions on radiographic imaging are Complete Response (CR) is the disappearance of all target lesions; Partial Response (PR) requires a ≥50% decrease in the sum of products of perpendicular diameters of target lesions from baseline. Other considerations for RANO response include assessments of non-target lesions, corticosteroids, and performance status. The Overall Response Rate is the proportion of patients who achieve either CR or PR. Tumor assessments were conducted at 8 weeks (±1 week) following the initiation of therapy and every 8 weeks (±1 week) thereafter.
All patients who received at least one dose of dordaviprone (ONC201) were included in the analysis, including patients with diffuse intrinsic pontine glioma (DIPG) or primary spinal tumors, which are historically not assessable for response using RANO-HGG criteria due to anatomical or imaging limitations.
Measured: From first dose of study treatment through study completion, an average of 1 year
Secondary outcomes (1)
Duration of Response (DOR)
DOR is defined as the time from the first documented response to the earliest date of disease progression or death, whichever occurred first. Patients who had not progressed or died at the time of analysis were censored at their last adequate tumor assessment.
Measured: From objective response (complete response or partial response) per RANO to disease progression or death, up to 40.6 months.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Sep 2017
- Study start30 Oct 2017
- Primary completion31 Dec 2022
- Study completion19 Jul 2023
Why it stopped: The Sponsor terminated the study to prioritize enrollment in a randomized Phase 3 trial of ONC201 in an earlier setting. This decision was unrelated to any safety concerns with dordaviprone (ONC201).
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 73 participants (actual)
Sponsor and investigators
- Lead sponsor
- Jazz Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Oncoceutics, Inc.
- Responsible party
- Sponsor
- Investigators
- Allen Mellemed, MD, Study director, Chimerix, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03295396
- Sponsor's protocol ID
- ONC013
- Status last verified
- Jul 2025
- First submitted
- 1 Aug 2017
- Last updated
- 28 Jul 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- 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 28 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II, Open-label Study of ONC201 in Adults With Recurrent High-grade Glioma
- ClinicalTrials.gov ID
- NCT03295396
- Acronym / sponsor ID
- ONC013
- Conditions
- Glioma
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The Sponsor terminated the study to prioritize enrollment in a randomized Phase 3 trial of ONC201 in an earlier setting. This decision was unrelated to any safety concerns with dordaviprone (ONC201).
- Enrolment
- 73 participants (actual)
- Lead sponsor
- Jazz PharmaceuticalsIndustry (pharma, biotech, devices)
- Collaborators
- Oncoceutics, Inc.
- Study start
- 30 Oct 2017 (actual)
- Primary completion
- 31 Dec 2022 (actual)
- Study completion
- 19 Jul 2023 (actual)
- First posted
- 27 Sep 2017 (actual)
- Results
- Posted 28 Jul 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This was a Phase 2, open-label, 2-arm study of dordaviprone (ONC201) in patients with recurrent H3 K27M- mutant glioma.
The primary assessment of dordaviprone (ONC201) involved evaluating its anti-tumor activity through the overall response rate according to the Response Assessment in Neuro-Oncology (RANO) criteria for high-grade glioma (HGG).
Detailed description
This study included 2 arms:
- Arm A included patients with recurrent H3 K27M-mutant glioma.
- Arm B included patients with recurrent H3 K27M-mutant glioma, but excluded patients with the following:
- Primary malignant lesion located in the pons or spinal cord.
- Atypical non-astrocytic histologies such as ependymoma, ganglioma and pleomorphic xanthoastrocytoma, or pilocytic astrocytoma and subependymal giant cell astrocytoma (SEGA).
- Prior bevacizumab treatment of >4 doses of >7.5 mg/kg
Patients received dordaviprone (ONC201) 625 mg once weekly.
The primary assessment of dordaviprone (ONC201) involved evaluating its anti-tumor activity through the overall response rate according to the Response Assessment in Neuro-Oncology (RANO) criteria for high-grade glioma (HGG). Safety was also assessed, with evaluations including the reporting of adverse events, as well as measurements of vital signs and clinical laboratory results.
This study was terminated by an administrative protocol amendment (17 January 2023). The decision to terminate the study was not related to any safety concerns with dordaviprone (ONC201). Before the study was terminated, a total of 73 patients were enrolled and received at least 1 dose of dordaviprone (ONC201).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Arm A | Experimental | Drug: Dordaviprone (ONC201)Patients received 625 mg dordaviprone (ONC201) once weekly. Arm A included patients with recurrent H3 K27M-mutant glioma including those with diffuse intrinsic pontine glioma (DIPG), primary spinal tumors, and some atypical histologies. |
| Arm B | Experimental | Drug: Dordaviprone (ONC201)Patients received 625 mg dordaviprone (ONC201) once weekly. Arm B included patients with recurrent H3 K27M-mutant glioma, but excluded patients with the following: * Primary malignant lesion located in the pons or spinal cord. * Atypical non-astrocytic histologies such as ependymoma, ganglioma and pleomorphic xanthoastrocytoma, or pilocytic astrocytoma and subependymal giant cell astrocytoma (SEGA). * Prior bevacizumab treatment of >4 doses of >7.5 mg/kg |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (31 listed)
- Had histologically confirmed diagnosis of high-grade glioma (HGG) in any tumor sample and presence of histone H3 K27M mutation detected in a Clinical Laboratory Improvement Amendment (CLIA) certified laboratory by immunohistochemistry or DNA sequencing test on any glioma tumor sample.
- Had unequivocal evidence of progressive disease on contrast-enhanced brain computed tomography (CT) or magnetic resonance imaging (MRI) as defined by Response Assessment in Neuro-Oncology (RANO)-HGG criteria, or have documented recurrent glioma on diagnostic biopsy.
- Had measurable disease by RANO-HGG criteria.
- Patients must have had previous therapy with at least radiotherapy.
- Had no more than two prior episodes of recurrence from radiotherapy and/or chemotherapy. Use of bevacizumab solely for treatment of radiation necrosis, pseudoprogression, or treatment effect was not considered a recurrence.
Key exclusion criteria (18 listed)
- Arm B: Had a primary malignant lesion located in the pons or spinal cord.
- Arm B: Had atypical non-astrocytic histologies such as ependymoma, ganglioma and pleomorphic xanthoastrocytoma, or pilocytic astrocytoma and subependymal giant cell astrocytoma (SEGA).
- Arm B: Had prior bevacizumab treatment of >4 doses of >7.5 mg/kg
- Had a history of allergic reactions attributed to compounds of similar chemical or biologic composition to dordaviprone (ONC201) or its excipients.
- Had current or planned participation in a study of an investigational agent or using an investigational device.
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 |
|---|---|---|
| Dordaviprone (ONC201)Also: DordaviproneDordaviprone (ONC201) is a central nervous system (CNS)-penetrant, small-molecule imipridone that acts as a mitochondrial caseinolytic protease P (ClpP) agonist and a dopamine receptor D2 (DRD2) antagonist. | Drug | Arm A, Arm B |
Primary outcome
Number of Patients With Overall Response
Time frame: From first dose of study treatment through study completion, an average of 1 year
Quantitative thresholds for objective response by Response Assessment in Neuro-Oncology (RANO) criteria for target lesions on radiographic imaging are Complete Response (CR) is the disappearance of all target lesions; Partial Response (PR) requires a ≥50% decrease in the sum of products of perpendicular diameters of target lesions from baseline. Other considerations for RANO response include assessments of non-target lesions, corticosteroids, and performance status. The Overall Response Rate is the proportion of patients who achieve either CR or PR. Tumor assessments were conducted at 8 weeks (±1 week) following the initiation of therapy and every 8 weeks (±1 week) thereafter. All patients who received at least one dose of dordaviprone (ONC201) were included in the analysis, including patients with diffuse intrinsic pontine glioma (DIPG) or primary spinal tumors, which are historically not assessable for response using RANO-HGG criteria due to anatomical or imaging limitations.
1 secondary outcome
Duration of Response (DOR)
Time frame: From objective response (complete response or partial response) per RANO to disease progression or death, up to 40.6 months.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Jul 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- 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.
Allen Mellemed, MD study director
Chimerix, Inc.
9 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Dordaviprone (ONC201) in people with Glioma, run by Jazz Pharmaceuticals at 9 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 31 inclusion and 18 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: stopped early, as of the sponsor's last update on 28 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 28 Jul 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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.
What does the primary outcome measure?
Number of Patients With Overall Response (time frame: From first dose of study treatment through study completion, an average of 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03295396, 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 28 Jul 2025.