Multiple MyelomaPhase 1/2NCT02863991
Oral ONC201 in Relapsed/Refractory Multiple Myeloma
A Phase 1/2 study testing ONC201 and Dexamethasone in people with Multiple Myeloma, run by Jazz Pharmaceuticals at 2 sites in United States.
Trial summary
This was a Phase 1/2 open-label study of ONC201 administered orally once every week in combination with dexamethasone in adults with relapsed/refractory multiple myeloma. The primary objective of this study was to evaluate the antitumor efficacy of ONC201. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Multiple Myeloma
- What is being tested?
- ONC201 and Dexamethasone
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 17 participants
- Where
- 2 sitesUnited States
- Study dates
- 19 Apr 2017to 16 Dec 2019
- Phase
- Phase 1/2
- 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
- A patient had to meet all of the following criteria to be eligible to participate in the study:
- Must have been refractory to, or not a candidate for, established therapy known to provide clinical benefit for their malignancy.
- Had measurable disease M protein component in serum (at least 0.5 g/dL) and/or urine (if present) (≥0.2 g excreted in a 24 hour collection sample), or serum free light chain level ≥10 mg/dL, provided the serum free light chain ratio was abnormal.
- Was able to swallow and retain oral medication.
- Had all previous therapies for cancer, including radiotherapy, major surgery and investigational therapies discontinued for ≥14 days (≥28 days for mitomycin C or nitrosoureas) before study entry, and had all acute effects of any prior therapy resolved to baseline severity or Grade ≤1 Common Terminology Criteria for Adverse Events (CTCAE v4.03), except alopecia or parameters defined in this eligibility list.
- Were aged ≥18 years.
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- Had an Eastern Cooperative Oncology Group (ECOG) performance status of ≤1.
- Had 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)
- Platelets: ≥75,000/mm3 without platelet transfusion ≤3 days prior to C1D1
- 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)
- Aspartate aminotransferase (AST) (SGOT)/alanine aminotransferase (ALT) (SGPT): ≤2 X ULN; ≤ 5 X ULN if liver dysfunction was felt to be secondary to tumor burden
- Serum creatinine: ≤1.5 X ULN (OR creatinine clearance ≥30 mL/min/1.73 m2)
- Serum or urine pregnancy test (for females of childbearing potential) negative ≤7days of starting treatment
- Had the ability to understand and the willingness to sign a written informed consent document and comply with the study scheduled visits, treatment plans, laboratory tests and other procedures.
- Female patients must have been surgically sterile or be postmenopausal, or must have agreed to use effective contraception during the period of the trial and for at least 90 days after completion of treatment. Male patients must have been surgically sterile or must have agreed 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 was based on the judgment of the principal investigator or a designated associate.
Who cannot join
- A potential patient who met any of the following criteria was ineligible to participate in the study:
- Had active inflammatory gastrointestinal disease, chronic diarrhea (unless related to underlying malignancy or prior related treatment) or history of abdominal fistula, gastrointestinal perforation, peptic ulcer disease, or intra-abdominal abscess within 6 months prior to study enrollment. Gastroesophageal reflux disease under treatment with proton pump inhibitors was allowed.
- Was pregnant or breast feeding.
- Was undergoing current active treatment in another clinical study.
- Had active bacterial, fungal or viral infection including hepatitis B (HBV), hepatitis C (HCV)
- Had known human immunodeficiency virus (HIV) or acquired immunodeficiency syndrome (AIDS)-related illness that was not well controlled.
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- Had active or prior plasma cell leukemia (defined as either 20% of peripheral white blood cell count [WBC] comprised of plasma/CD138+ cells or an absolute count of 2x10^9/L).
- Had solitary bone or solitary extramedullary plasmacytoma as the only evidence of plasma cell dyscrasia.
- Had serum calcium (corrected for albumin) ≥12 mg/dL
- Had 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.
- Had other severe acute or chronic medical or psychiatric condition or laboratory abnormality that may have increased the risk associated with study participation or study drug administration, or may have interfered with the interpretation of study results, or in the judgment of the investigator would have made the patient inappropriate for entry into the study.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
The Mount Sinai Medical Center
New York, New York, 10029-6574
Fox Chase Cancer Center
Philadelphia, Pennsylvania, 19111
About this study
This was a Phase 1/2 open-label study of ONC201 administered orally once every week in combination with dexamethasone in adults with relapsed/refractory multiple myeloma. The primary objective of this study was to evaluate the antitumor efficacy of ONC201.
Note: This study was completed by predecessor company, Oncoceutics, Inc.
Read the detailed description
In Phase 1 of the study, patients were to receive 375 or 625 mg ONC201 once every week in combination with dexamethasone using a 3+3 dose escalation design to evaluate up to 625 mg ONC201 weekly with 20 mg dexamethasone. In Phase 2 of the study, patients were to receive 625 mg ONC201 once every week. Dexamethasone was to be administered at a dose determined in Phase 1.
A treatment cycle was defined as 3 weeks. The dose-limiting toxicity window was defined as the first 3 weeks of treatment (i.e., 1 cycle). Patients may have continued treatment with ONC201 until disease progression, occurrence of an unacceptable adverse event, intercurrent illness or changes in the patient's condition rendered the patient unacceptable to continue, patient decision to withdraw from the study, or discontinuation of the study by the Sponsor.
Assessments of tumor response were conducted using the International Myeloma Working Group response criteria. Safety was assessed through the reporting of adverse events, measurement of vital signs, electrocardiograms, and clinical laboratory results.
Before the study was terminated, a total of 17 patients were enrolled and treated with ONC201: 2 patients received 375 mg ONC201 and 15 patients received 625 mg ONC201.
- Conditions
- Multiple Myeloma
- Being tested
- ONC201, Dexamethasone
- Medical subject headings
- Multiple Myeloma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ONC201Drug
375 mg or 625 mg ONC201
DexamethasoneDrug
20 mg dexamethasone
| Study group | Role | What they receive |
|---|---|---|
| 375 mg ONC201 | Experimental | Patients received 375 mg ONC201 once every week in combination with dexamethasone. Receives: Drug: ONC201; Drug: Dexamethasone |
| 625 mg ONC201 | Experimental | Patients received 625 mg ONC201 once every week in combination with dexamethasone. Receives: Drug: ONC201; Drug: Dexamethasone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Response of Participants at Last On-study Visit (End of Treatment/Follow-up)
Assessments of response were made using the International Myeloma Working Group (IMWG) response criteria and were assessed by magnetic resonance imaging (MRI), computed tomography (CT), or positron emission tomography (PET)/CT scans (when applicable). Per IMWG response criteria, objective response could be defined as follows: complete response (CR), disappearance of any soft tissue plasmacytomas; partial response (PR), a >50% reduction in size of soft tissue plasmacytomas; progressive disease (PD), definite development of new or a definite increase of size of existing soft tissue plasmacytomas; and stable disease (SD), not meeting criteria for CR, PR, or PD.
Measured: The response assessment data reported was conducted at the last on-study visit (end of treatment/follow-up), up to a maximum of 7 months following treatment initiation.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Aug 2016 (estimated)
- Study start19 Apr 2017
- Primary completion17 Sep 2019
- Study completion16 Dec 2019
Why it stopped: This study was terminated due to a change in corporate priorities. The decision to terminate the study was not based on any safety concerns.
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.
- Other trials for Multiple MyelomaStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 17 participants (actual)
Sponsor and investigators
- Lead sponsor
- Jazz Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Oncoceutics, Inc.
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02863991
- Sponsor's protocol ID
- ONC007
- Other IDs
- R01FD005402
- Status last verified
- Jun 2024
- First submitted
- 4 Aug 2016
- Last updated
- 3 Jul 2024
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Oral ONC201 in Relapsed/Refractory Multiple Myeloma
- ClinicalTrials.gov ID
- NCT02863991
- Acronym / sponsor ID
- ONC007
- Conditions
- Multiple Myeloma
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: This study was terminated due to a change in corporate priorities. The decision to terminate the study was not based on any safety concerns.
- Enrolment
- 17 participants (actual)
- Lead sponsor
- Jazz PharmaceuticalsIndustry (pharma, biotech, devices)
- Collaborators
- Oncoceutics, Inc.
- Study start
- 19 Apr 2017 (actual)
- Primary completion
- 17 Sep 2019 (actual)
- Study completion
- 16 Dec 2019 (actual)
- First posted
- 11 Aug 2016 (estimated)
- Results
- Posted 3 Jul 2024
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 1/2 open-label study of ONC201 administered orally once every week in combination with dexamethasone in adults with relapsed/refractory multiple myeloma. The primary objective of this study was to evaluate the antitumor efficacy of ONC201.
Note: This study was completed by predecessor company, Oncoceutics, Inc.
Detailed description
In Phase 1 of the study, patients were to receive 375 or 625 mg ONC201 once every week in combination with dexamethasone using a 3+3 dose escalation design to evaluate up to 625 mg ONC201 weekly with 20 mg dexamethasone. In Phase 2 of the study, patients were to receive 625 mg ONC201 once every week. Dexamethasone was to be administered at a dose determined in Phase 1.
A treatment cycle was defined as 3 weeks. The dose-limiting toxicity window was defined as the first 3 weeks of treatment (i.e., 1 cycle). Patients may have continued treatment with ONC201 until disease progression, occurrence of an unacceptable adverse event, intercurrent illness or changes in the patient's condition rendered the patient unacceptable to continue, patient decision to withdraw from the study, or discontinuation of the study by the Sponsor.
Assessments of tumor response were conducted using the International Myeloma Working Group response criteria. Safety was assessed through the reporting of adverse events, measurement of vital signs, electrocardiograms, and clinical laboratory results.
Before the study was terminated, a total of 17 patients were enrolled and treated with ONC201: 2 patients received 375 mg ONC201 and 15 patients received 625 mg ONC201.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 375 mg ONC201 | Experimental | Drug: ONC201, Drug: DexamethasonePatients received 375 mg ONC201 once every week in combination with dexamethasone. |
| 625 mg ONC201 | Experimental | Drug: ONC201, Drug: DexamethasonePatients received 625 mg ONC201 once every week in combination with dexamethasone. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (17 listed)
- A patient had to meet all of the following criteria to be eligible to participate in the study:
- Must have been refractory to, or not a candidate for, established therapy known to provide clinical benefit for their malignancy.
- Had measurable disease M protein component in serum (at least 0.5 g/dL) and/or urine (if present) (≥0.2 g excreted in a 24 hour collection sample), or serum free light chain level ≥10 mg/dL, provided the serum free light chain ratio was abnormal.
- Was able to swallow and retain oral medication.
- Had all previous therapies for cancer, including radiotherapy, major surgery and investigational therapies discontinued for ≥14 days (≥28 days for mitomycin C or nitrosoureas) before study entry, and had all acute effects of any prior therapy resolved to baseline severity or Grade ≤1 Common Terminology Criteria for Adverse Events (CTCAE v4.03), except alopecia or parameters defined in this eligibility list.
Key exclusion criteria (11 listed)
- A potential patient who met any of the following criteria was ineligible to participate in the study:
- Had active inflammatory gastrointestinal disease, chronic diarrhea (unless related to underlying malignancy or prior related treatment) or history of abdominal fistula, gastrointestinal perforation, peptic ulcer disease, or intra-abdominal abscess within 6 months prior to study enrollment. Gastroesophageal reflux disease under treatment with proton pump inhibitors was allowed.
- Was pregnant or breast feeding.
- Was undergoing current active treatment in another clinical study.
- Had active bacterial, fungal or viral infection including hepatitis B (HBV), hepatitis C (HCV)
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 |
|---|---|---|
| ONC201375 mg or 625 mg ONC201 | Drug | 375 mg ONC201, 625 mg ONC201 |
| Dexamethasone20 mg dexamethasone | Drug | 375 mg ONC201, 625 mg ONC201 |
Primary outcome
Response of Participants at Last On-study Visit (End of Treatment/Follow-up)
Time frame: The response assessment data reported was conducted at the last on-study visit (end of treatment/follow-up), up to a maximum of 7 months following treatment initiation.
Assessments of response were made using the International Myeloma Working Group (IMWG) response criteria and were assessed by magnetic resonance imaging (MRI), computed tomography (CT), or positron emission tomography (PET)/CT scans (when applicable). Per IMWG response criteria, objective response could be defined as follows: complete response (CR), disappearance of any soft tissue plasmacytomas; partial response (PR), a >50% reduction in size of soft tissue plasmacytomas; progressive disease (PD), definite development of new or a definite increase of size of existing soft tissue plasmacytomas; and stable disease (SD), not meeting criteria for CR, PR, or PD.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Jul 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing ONC201 and Dexamethasone in people with Multiple Myeloma, run by Jazz Pharmaceuticals at 2 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 17 inclusion and 11 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 3 Jul 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 3 Jul 2024). 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?
Response of Participants at Last On-study Visit (End of Treatment/Follow-up) (time frame: The response assessment data reported was conducted at the last on-study visit (end of treatment/follow-up), up to a maximum of 7 months following treatment initiation.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 3 Jul 2024.