Multiple MyelomaPhase 1/2NCT03492138
Ixazomib, ONC201, and Dexamethasone in Relapsed/Refractory Multiple Myeloma
A Phase 1/2 study testing ONC201, Ixazomib and Dexamethasone in people with Multiple Myeloma, run by Ajai Chari at 1 site in United States.
Trial summary
ONC201 is a novel dopamine receptor D2 antagonist that is able to activate the integrated stress response pathway. It is active against multiple myeloma cells in vitro, both as a single agent and in combination with corticosteroids and proteasome inhibitors. 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, Ixazomib and Dexamethasone
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteUnited States
- Study dates
- 26 Mar 2018to 14 Jan 2020
- Phase
- Phase 1/2
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Signed Written Informed Consent: Voluntary written consent must be given before performance of any study-related procedure not part of standard medical care
- Target Population
- Symptomatic MM having progressed on 2 prior therapies including proteasome inhibitor (i.e. bortezomib, carfilzomib ixazomib), immunomodulatory drug (i.e., thalidomide, lenalidomide, pomalidomide), and daratumumab or other CD38 targeting monoclonal antibody. Proteasome inhibitor refractory patients are eligible. Subjects must not be candidates for treatment regimens known to provide clinical benefit to be eligible for this study.
- Male or female patients 18 years or older
- Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2.
- Patients must have measurable disease defined by at least 1 of the following 3 measurements:
- Serum M-protein > 0.5 g/dL ii. Urine M-protein > 200 mg/24 hours iii. Serum free light chain assay: involved free light chain level >10 mg/dL (> 100 mg/L) provided the serum free light chain ratio is abnormal
Who cannot join
- Medical History and Concurrent Diseases
- Peripheral neuropathy > Grade 2 or >Grade 1 with pain on clinical examination during the Screening period.
- Significant cardiac disease as determined by the investigator including:
- Known or suspected cardiac amyloidosis ii. Congestive heart failure of Class III or IV of the NYHA classification iii. Uncontrolled angina, hypertension or arrhythmia iv. Myocardial infarction in the past 6 months v. Any uncontrolled or severe cardiovascular disease vi. QTc > 470 milliseconds (msec) on a 12-lead ECG obtained during the Screening period. If a machine reading is above this value, the ECG should be reviewed by a qualified reader and confirmed on a subsequent ECG.
- Known active hepatitis B (defined as most recent serum PCR or hepatitis B surface antigen positive) or active hepatitis C (note, hepatitis C in sustained virologic response defined as negative RNA PCR at least 12 weeks after any therapy is permitted).
- Any medical conditions that, in the investigator's opinion, would impose excessive risk to the subject, e.g., any uncontrolled disease, such as pulmonary disease, infection, seizure disorder, uncontrolled hyperglycemia.
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- Any altered mental status or any psychiatric condition that would interfere with the understanding of the informed consent or limit compliance with study requirements.
- Prior or concurrent malignancy, except for the following: i. Adequately treated basal cell or squamous cell skin cancer ii. Cervical carcinoma in situ iii. Adequately treated Stage I or II cancer from which the subject is currently in complete remission.
- Or any other cancer from which the subject has been disease-free for ≥ 3 years g. Diarrhea > Grade 1, based on the NCI CTCAE grading, in the absence of antidiarrheals.
- Known GI disease or GI procedure that could interfere with the oral absorption or tolerance of study drug, including difficulty swallowing.
- Males or females of childbearing potential who do not agree to practice 2 effective methods of contraception, at the same time through 90 days after the last dose of study drug j. Females who are pregnant or breastfeeding. k. Diagnosis of Waldenstrom's macroglobulinemia, POEMS (polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy, and skin changes) syndrome, plasma cell leukemia, myeloproliferative syndrome, or primary amyloidosis (with the exception of patients whose amyloidosis has been documented as a complication of MM, who will be evaluated on a case-by-case basis for trial participation).
- Infection requiring systemic antibiotic therapy or other serious infection within 14 days before study drug administration.
- Parkinson's disease 4) Physical and Laboratory Test Findings
- Corrected serum calcium ≥ 14 mg/dl within 2 weeks of enrollment (despite appropriate measure such a short course of steroids, bisphosphonates, hydration, and calcitonin).
- Absolute neutrophil count < 1000 cells/mm3. No growth factors allowed within 1 week of enrollment.
- Platelets < 75,000 cell/mm3 (75 x 109/L). Qualifying laboratory value must occur at most recent measurement before enrollment and must be no more than 14 days before enrollment. No transfusions are allowed within 72 hours prior to study drug administration.
- Hemoglobin < 8 g/dL. Qualifying laboratory value must occur at most recent measurement before enrollment and must be no more than 14 days before enrollment. No transfusions are allowed within 72 hours before qualifying laboratory value e. Serum bilirubin ≥ 1.5 x ULN, patients with Gilbert's syndrome and a total bilirubin of < 3 times ULN are permitted) f. AST or ALT ≥ 3 x ULN. g. CrCl < 30 ml/min/1.73m2. Creatinine clearance is estimated by the CKD-EPI formula. (Calculator available at https://www.kidney.org/professionals/kdoqi/gfr_calculator.)
- Prior Therapy or Surgery
- Major surgery or radiation therapy within 14 days before study drug administration.
- Kyphoplasty or vertebroplasty within 1 week of enrollment.
- Administration of chemotherapy, biological, immunotherapy, or investigational agent (therapeutic or diagnostic) within 3 weeks before enrollment (14 days for non-myelosuppressive therapy). Subjects should be 6 weeks from last dose of nitrosourea, nitrogen mustards or monoclonal antibody, 12 weeks from autologous SCT, and 16 weeks from allogeneic SCT.
- Corticosteroids use exceeding a cumulative dose of 160 mg of dexamethasone during screening.
- If prior allogeneic stem cell transplant, history of moderate to severe chronic graft versus host disease (GVHD).
- Treatment with plasmapheresis within 4 weeks before enrollment.
- NSAIDs, IV contrast, aminoglycosides, or other potentially nephrotoxic drugs within 2 weeks of enrollment.
- Current use of a non-standard dialysis membrane.
- Systemic treatment with strong inhibitors or inducers of CYP450 system should not be used on study including but not limited to fluvoxamine, enoxacin, ciprofloxacin, clarithromycin, telithromycin, itraconazole, voriconazole, ketoconazole, nefazodone, posaconazole, rifampin, rifapentine, rifabutin, carbamazepine, phenytoin, phenobarbital, buproprion, fluoxetine, paroxetine, ticlopidine, or St. John's wort within 14 days before the first dose of study treatment. Ixazomib has significant drug-drug interactions with strong CYP3A inducers. No drug-drug interactions with the CYP450 screen have been found with ONC201, but the analysis of these studies is not complete, so during the study use of inhibitors or inducers of CYP450 system is excluded.
- Failure to have fully recovered (i.e., Grade 1 toxicity or less, with the exception of alopecia) from clinically significant effects of prior chemotherapy regardless of interval since last treatment.
- Allergies and Adverse Drug Reaction Known hypersensitivity to bortezomib, ixazomib, dexamethasone, ONC201
Trial locations and contacts
1 site in 1 country.
United States1 site
Icahn School of Medicine at Mount Sinai
New York, New York, 10029
About this study
Official title: A Phase I/II Study of the Addition of Ixazomib to ONC201 and Dexamethasone in Relapsed and/or Refractory Multiple Myeloma
ONC201 is a novel dopamine receptor D2 antagonist that is able to activate the integrated stress response pathway. It is active against multiple myeloma cells in vitro, both as a single agent and in combination with corticosteroids and proteasome inhibitors. In order to document superiority over the combination compared to the individual agents of ixazomib and ONC201 in a single arm study, there will initially be a run-in period of weekly ONC201 625 mg with dexamethasone 40 mg such that if there is progression of disease (25% increase) after 4 weeks or less than a minimal response (25% reduction) after 8 weeks then ixazomib will be added. Dexamethasone is dose-reduced to 20 mg at the same schedule for subjects ≥ 75 years old. If patients do achieve single-agent responses with ONC201 (minimal response or better), they will continue with weekly ONC201 and dexamethasone until progression, with response assessments after each 28-day cycle. Patients who have previously been treated on another clinical trial with weekly ONC201 625mg with dexamethasone with progression while receiving treatment do not need to complete the run-in phase of the study.
At the time of progression, they will proceed to the 3 drug combination phase of the study. It is at the point of 3 drug initiation, that below phase I DLT principles or phase II disease control rate considerations apply.
Read the detailed description
Phase I Study Design
- Phase I will follow a 3+3 dose escalation design to determine the RPTD of ixazomib in combination with ONC201 and Dexamethasone.
The dose escalation rules for the phase I portion of the study are as follows, escalating in cohorts of 3 patients per dose level including a de-escalation option in case of early toxicity. Dose limiting toxicity (DLT) is defined as any grade 3 or higher toxicity seen during the first 28 day cycle of the triplet regimen (ONC201, ixazomib, and dexamethasone). Of note, grade 3 and higher absolute lymphopenia and hematologic toxicities without clinical sequelae (eg. neutropenia without fever/infection, anemia without symptoms, thrombocytopenia without bleeding) and also responsive to growth factors/transfusions will not be considered DLTs. AST or ALT values of ≥ 3x ULN AND with serum total bilirubin level of > 2x ULN or international normalized ratio (INR) > 1.5 without signs of cholestasis and with no other clear alternative reason to explain the observed liver-related laboratory abnormalities is also considered a DLT. Three patients will be treated at the current dose level. If at least 2 patients are observed to have a DLT, the prior dose level is defined as the RPTD unless only 3 patients have been treated at that level, in which case it is the tentative RPTD. If 0 of the 3 patients are observed to have DLT, the dose level is escalated one dose level for the next cohort of 3 patients, and the process continues as above. If exactly 1 of the 3 patients treated show DLT, 3 additional patients are treated at the current dose level. If none of these additional 3 patients show DLT, the dose level is escalated for the next cohort of 3 patients, and the process continues as above; otherwise, the prior dose level is defined as the RPTD (unless only 3 patients have been treated at that level, in which case it is the tentative RPTD). A tentative RPTD becomes final when a total of 6 patients are treated with less than 2 showing DLT.
If, unexpectedly, at least 2 patients are observed to have DLT at the initial dose level 0, then the next dose level evaluated will be the lowest dose level, -2, and further dose escalations to dose level -1 will proceed as outlined above from that dose level. If there is no RPTD declared, there will be no expansion to the phase II portion of the study. The phase I portion of the study will include between 9 and 12 patients depending upon the number of dose levels evaluated.
Phase II Study Design Phase II will follow a Simon's Optimal two-stage design to indicate proof of concept regarding the PFS rate of ixazomib in combination with ONC201 and Dexamethasone at the RPTD established in phase I. Once the RPTD for combination therapy has been established, an additional 24 patients will be enrolled for a total of 30 evaluable patients (24 + 6 from phase I at RPTD).
In considering a Simon's Optimal two-stage design, the plan is to look at each patient at 2 months after initiating study treatment (3 drug combination) and classify each patient as responder or non-responder at the 2 month time point. A responder is a patient who has stable disease or better (not progressed) since initiating study treatment; a non-responder is someone whose disease has progressed according to IMWG criteria. The population of relapsed and refractory MM patients in this study will likely all have progressed on standard therapy; therefore the null hypothesis being tested is that the disease control rate at 2 months is 5%. As per IMWG criteria, if patients show progression after 1 cycle of triplet therapy, they will continue on therapy and a repeat assessment will be performed after cycle 2 in order to confirm progression of disease.
Stage 1:
- Enroll 9 patients including the 6 patients being evaluated from the phase I portion of the study
- Evaluate patients at 2 months post initiation of treatment
- If 0 have responded (progression-free), i.e. if all 9 have progressed, then stop trial for futility and conclude that the proportion who will be progression-free at 2 months is less than 5%.
- If 1 or more have responded (progression-free) at 2 months, then proceed to Stage 2.
Stage 2:
- Enroll an additional 21 patients
- Evaluate patients at 2 months post initiation of treatment If 4 or more of the 30 patients have responded (progression-free) at 2 months then the null hypothesis is rejected and further investigation of this treatment combination is warranted.
- Conditions
- Multiple Myeloma
- Being tested
- ONC201, Ixazomib, Dexamethasone
- Keywords
- Multiple Myeloma, Relapsed and Refractory
- Medical subject headings
- Multiple Myeloma, Recurrence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ONC201Drug
625mg
IxazomibDrug
Dose to be determined in Phase 1 of study
DexamethasoneDrug
40mg
| Study group | Role | What they receive |
|---|---|---|
| Participants with relapsed/refractory multiple myeloma | Experimental | ONC201, ixazomib, and dexamethasone in relapsed/refractory multiple myeloma. Run-in phase of ONC201 and dexamethasone weekly until progression at 4 weeks, lack of response at 8 weeks, or progression followed by the addition of weekly ixazomib. Receives: Drug: ONC201; Drug: Ixazomib; Drug: Dexamethasone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Recommended phase II dose (RPTD)
of triplet therapy (ixazomib + ONC201+ dexamethasone) following a 3+3 escalation design
Measured: 9 Months
Secondary outcomes (4)
Disease control rate
2 month disease control rate defined as the proportion of patients that have stable or better disease after two months of treatment initiation according to IMWG criteria: Stable disease, partial remission, or complete remission
Measured: 2 months
Progression free survival (PFS)
Median Progression free survival for the phase I and II patients treated at the recommended phase II dose. PFS is defined as the duration of time from start of treatment to the first occurrence of disease progression or death on study from any cause, whichever occurs earlier.
Measured: 6 months
Duration of response (DOR)
Median duration of response for the phase I and II patients treated at the recommended phase II dose. DOR is defined as the time from first evidence of PR or better to confirmation of disease progression.
Measured: 6 months
Clinical benefit rate (CBR)
Clinical benefit rate for the phase I and II patients treated at the recommended phase II dose level. CBR is the combination of the ORR and minimal response (MR) i.e. this includes sCR, CR, VGPR, PR, and MR.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Apr 2018
- Study start26 Mar 2018
- Primary completion14 Jan 2020
- Study completion14 Jan 2020
Why it stopped: Low enrollment
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Multiple MyelomaStudies of the same condition, open and under way.
- 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
- Not applicable
- Intervention model
- Single group
- Model description
- Single arm, open-label, standard 3+3 phase 1 with a Simon 2-stage design followed by stage 2
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ajai Chari
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Ajai Chari, Icahn School of Medicine at Mount Sinai
- Investigators
- Ajai Chari, MD, Principal investigator, Icahn School of Medicine at Mount Sinai
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03492138
- Sponsor's protocol ID
- GCO 17-2680
- Status last verified
- May 2020
- First submitted
- 22 Mar 2018
- Last updated
- 29 May 2020
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided: Data and safety will be monitored using an internal Data and Safety Monitoring Plan (DSMP) led by the PI. Other members involved with the review will include sub-Investigators, the myeloma research program manager, and clinical research coordinators. All of these individuals have experience in the routine clinical care as well clinical research and monitoring of subjects with multiple myeloma. A statistician will also be involved with the review.
Study at a glance
From the registry record, last updated by the sponsor on 29 May 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II Study of the Addition of Ixazomib to ONC201 and Dexamethasone in Relapsed and/or Refractory Multiple Myeloma
- ClinicalTrials.gov ID
- NCT03492138
- Acronym / sponsor ID
- GCO 17-2680
- Conditions
- Multiple Myeloma
- Keywords
- Multiple Myeloma, Relapsed and Refractory
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Low enrollment
- Enrolment
- 5 participants (actual)
- Lead sponsor
- Ajai ChariUniversity, hospital or other
- Study start
- 26 Mar 2018 (actual)
- Primary completion
- 14 Jan 2020 (actual)
- Study completion
- 14 Jan 2020 (actual)
- First posted
- 10 Apr 2018 (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.
ONC201 is a novel dopamine receptor D2 antagonist that is able to activate the integrated stress response pathway. It is active against multiple myeloma cells in vitro, both as a single agent and in combination with corticosteroids and proteasome inhibitors. In order to document superiority over the combination compared to the individual agents of ixazomib and ONC201 in a single arm study, there will initially be a run-in period of weekly ONC201 625 mg with dexamethasone 40 mg such that if there is progression of disease (25% increase) after 4 weeks or less than a minimal response (25% reduction) after 8 weeks then ixazomib will be added. Dexamethasone is dose-reduced to 20 mg at the same schedule for subjects ≥ 75 years old. If patients do achieve single-agent responses with ONC201 (minimal response or better), they will continue with weekly ONC201 and dexamethasone until progression, with response assessments after each 28-day cycle. Patients who have previously been treated on another clinical trial with weekly ONC201 625mg with dexamethasone with progression while receiving treatment do not need to complete the run-in phase of the study.
At the time of progression, they will proceed to the 3 drug combination phase of the study. It is at the point of 3 drug initiation, that below phase I DLT principles or phase II disease control rate considerations apply.
Detailed description
Phase I Study Design
- Phase I will follow a 3+3 dose escalation design to determine the RPTD of ixazomib in combination with ONC201 and Dexamethasone.
The dose escalation rules for the phase I portion of the study are as follows, escalating in cohorts of 3 patients per dose level including a de-escalation option in case of early toxicity. Dose limiting toxicity (DLT) is defined as any grade 3 or higher toxicity seen during the first 28 day cycle of the triplet regimen (ONC201, ixazomib, and dexamethasone). Of note, grade 3 and higher absolute lymphopenia and hematologic toxicities without clinical sequelae (eg. neutropenia without fever/infection, anemia without symptoms, thrombocytopenia without bleeding) and also responsive to growth factors/transfusions will not be considered DLTs. AST or ALT values of ≥ 3x ULN AND with serum total bilirubin level of > 2x ULN or international normalized ratio (INR) > 1.5 without signs of cholestasis and with no other clear alternative reason to explain the observed liver-related laboratory abnormalities is also considered a DLT. Three patients will be treated at the current dose level. If at least 2 patients are observed to have a DLT, the prior dose level is defined as the RPTD unless only 3 patients have been treated at that level, in which case it is the tentative RPTD. If 0 of the 3 patients are observed to have DLT, the dose level is escalated one dose level for the next cohort of 3 patients, and the process continues as above. If exactly 1 of the 3 patients treated show DLT, 3 additional patients are treated at the current dose level. If none of these additional 3 patients show DLT, the dose level is escalated for the next cohort of 3 patients, and the process continues as above; otherwise, the prior dose level is defined as the RPTD (unless only 3 patients have been treated at that level, in which case it is the tentative RPTD). A tentative RPTD becomes final when a total of 6 patients are treated with less than 2 showing DLT.
If, unexpectedly, at least 2 patients are observed to have DLT at the initial dose level 0, then the next dose level evaluated will be the lowest dose level, -2, and further dose escalations to dose level -1 will proceed as outlined above from that dose level. If there is no RPTD declared, there will be no expansion to the phase II portion of the study. The phase I portion of the study will include between 9 and 12 patients depending upon the number of dose levels evaluated.
Phase II Study Design Phase II will follow a Simon's Optimal two-stage design to indicate proof of concept regarding the PFS rate of ixazomib in combination with ONC201 and Dexamethasone at the RPTD established in phase I. Once the RPTD for combination therapy has been established, an additional 24 patients will be enrolled for a total of 30 evaluable patients (24 + 6 from phase I at RPTD).
In considering a Simon's Optimal two-stage design, the plan is to look at each patient at 2 months after initiating study treatment (3 drug combination) and classify each patient as responder or non-responder at the 2 month time point. A responder is a patient who has stable disease or better (not progressed) since initiating study treatment; a non-responder is someone whose disease has progressed according to IMWG criteria. The population of relapsed and refractory MM patients in this study will likely all have progressed on standard therapy; therefore the null hypothesis being tested is that the disease control rate at 2 months is 5%. As per IMWG criteria, if patients show progression after 1 cycle of triplet therapy, they will continue on therapy and a repeat assessment will be performed after cycle 2 in order to confirm progression of disease.
Stage 1:
- Enroll 9 patients including the 6 patients being evaluated from the phase I portion of the study
- Evaluate patients at 2 months post initiation of treatment
- If 0 have responded (progression-free), i.e. if all 9 have progressed, then stop trial for futility and conclude that the proportion who will be progression-free at 2 months is less than 5%.
- If 1 or more have responded (progression-free) at 2 months, then proceed to Stage 2.
Stage 2:
- Enroll an additional 21 patients
- Evaluate patients at 2 months post initiation of treatment If 4 or more of the 30 patients have responded (progression-free) at 2 months then the null hypothesis is rejected and further investigation of this treatment combination is warranted.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Single arm, open-label, standard 3+3 phase 1 with a Simon 2-stage design followed by stage 2
| Arm | Type | Receives |
|---|---|---|
| Participants with relapsed/refractory multiple myeloma | Experimental | Drug: ONC201, Drug: Ixazomib, Drug: DexamethasoneONC201, ixazomib, and dexamethasone in relapsed/refractory multiple myeloma. Run-in phase of ONC201 and dexamethasone weekly until progression at 4 weeks, lack of response at 8 weeks, or progression followed by the addition of weekly ixazomib. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Signed Written Informed Consent: Voluntary written consent must be given before performance of any study-related procedure not part of standard medical care
- Target Population
Key exclusion criteria (29 listed)
- Medical History and Concurrent Diseases
- Or any other cancer from which the subject has been disease-free for ≥ 3 years g. Diarrhea > Grade 1, based on the NCI CTCAE grading, in the absence of antidiarrheals.
- Known GI disease or GI procedure that could interfere with the oral absorption or tolerance of study drug, including difficulty swallowing.
- Males or females of childbearing potential who do not agree to practice 2 effective methods of contraception, at the same time through 90 days after the last dose of study drug j. Females who are pregnant or breastfeeding. k. Diagnosis of Waldenstrom's macroglobulinemia, POEMS (polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy, and skin changes) syndrome, plasma cell leukemia, myeloproliferative syndrome, or primary amyloidosis (with the exception of patients whose amyloidosis has been documented as a complication of MM, who will be evaluated on a case-by-case basis for trial participation).
- Infection requiring systemic antibiotic therapy or other serious infection within 14 days before study drug administration.
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 |
|---|---|---|
| ONC201625mg | Drug | Participants with relapsed/refractory multiple myeloma |
| IxazomibDose to be determined in Phase 1 of study | Drug | Participants with relapsed/refractory multiple myeloma |
| Dexamethasone40mg | Drug | Participants with relapsed/refractory multiple myeloma |
Primary outcome
Recommended phase II dose (RPTD)
Time frame: 9 Months
of triplet therapy (ixazomib + ONC201+ dexamethasone) following a 3+3 escalation design
4 secondary outcomes
Disease control rate
Time frame: 2 months
Progression free survival (PFS)
Time frame: 6 months
Duration of response (DOR)
Time frame: 6 months
Clinical benefit rate (CBR)
Time frame: 6 months
Results and safety
The study was stopped early (reason given: Low enrollment). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 May 2020.
- Individual participant dataSharing planned: Undecided. Data and safety will be monitored using an internal Data and Safety Monitoring Plan (DSMP) led by the PI. Other members involved with the review will include sub-Investigators, the myeloma research program manager, and clinical research coordinators. All of these individuals have experience in the routine clinical care as well clinical research and monitoring of subjects with multiple myeloma. A statistician will also be involved with the review.
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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.
Ajai Chari, MD principal investigator
Icahn School of Medicine at Mount Sinai
1 site 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, Ixazomib and Dexamethasone in people with Multiple Myeloma, run by Ajai Chari at 1 site 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 7 inclusion and 29 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 29 May 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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?
The study was stopped early (reason given: Low enrollment). 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.
What does the primary outcome measure?
Recommended phase II dose (RPTD) (time frame: 9 Months). 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 29 May 2020.