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Strategic business decision
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This study will include participants with relapsed/refractory (R/R) Multiple Myeloma (MM). MM is a type of cancer of the blood. This study will also include participants with relapsed/refractory (R/R) Diffuse Large B-Cell Lymphoma (DLBCL).
DLBCL is also a type of cancer of the blood. They are referred to as 'relapsed' when the disease has come back after treatment and 'refractory' when treatment no longer works. The study has 2 main parts, called phase 1 and phase 1b. The main objective of both parts will be to evaluate the safety and tolerability of the study drug, called EZM0414.
The main objective of phase 1b will also be to determine the effectiveness of EZM0414. During phase 1 six dose levels will be tested to obtain the most tolerated dose. Participants will receive study drug at the assigned dose level every 28 days. During phase 1b participants will receive study drug at the maximum tolerated dose in 28-day cycles.
The first part of the study will be a Phase 1 dose-escalation designed to evaluate the safety, tolerability, and pharmacokinetics (PK) of EZM0414 in subjects with R/R MM and R/R DLBCL.
Six dose levels starting at 100 mg, then 200 mg, 300 mg, 400 mg, 600 mg, and 900 mg as well as an optional step-down dose of 75 mg (if needed) will be tested. The second part of the study is the Phase 1b dose expansion at the MTD designed to evaluate safety and efficacy in subjects with R/R DLBCL and R/R MM with or without select genetic translocation.
Dose expansion will enroll subjects in 3 cohorts: Cohort 1 for R/R MM subjects with t(4;14), Cohort 2 for R/R MM subjects without t(4;14), and Cohort 3 for subjects with R/R DLBCL.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Open-label EZM0414 | Experimental | Participants will receive EZM0414 in continuous 28-day cycles. EZM0414 will be administered orally once daily (QD) without food. Participants who receive EZM0414 at Maximum tolerated dose (MTD) and do not have Dose limiting toxicities (DLT) in the dose escalation part of the study will be rolled over to a cohort of this dose expansion part. Cohort 1 for R/R MM Participants. Cohort 2 for R/R MM Participants. Cohort 3 for Participants with R/R DLBCL. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| EZM0414 | Drug | Immediate-release film-coated tablets: Six dose levels starting at 100 mg, and then 200 mg, 300 mg, 400 mg, 600 mg, and 900 mg as well as an optional step-down dose level of 75 mg (if needed) |
| Measure | Description | Time Frame |
|---|---|---|
| Part 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (TESAEs) | An adverse event was any untoward medical occurrence in a participant or clinical investigation participant administered a medicinal product and which does not necessarily had a causal relationship with this treatment. A serious adverse event was any untoward medical occurrence that at any dose, resulted in death, was life-threatening, required in-subject hospitalization or prolongation of hospitalization, resulted in persistent or significant disability or incapacity, or resulted in a congenital abnormality or birth defect, was an important medical event. A TEAE was an AE that started or worsened in severity on or after the date of the first dose of the study treatment through 30 days after the end of treatment. | From first dose of study treatment (Day 1) up to 30 days after the last dose of study treatment (Day 57), up to approximately 87 days |
| Part 1: Number of Participants With Dose-limiting Toxicities (DLTs) | According to National Cancer Institute's Common Terminology Criteria for Adverse Events version 5.0, DLT was defined as any of following AE that occurred in Part 1 during first cycle of study treatment: grade (G)4 neutropenia lasting >5 days; G3 febrile neutropenia; G3 thrombocytopenia with clinically significant bleeding; G4 thrombocytopenia; G4 anemia unexplained by underlying disease; any other non-hematological toxicity ≥3 except: alopecia, G3 nausea/vomiting or diarrhea for <3 days with supportive care, G3 fatigue for <1 week, G3 or higher isolated electrolyte abnormalities for up to 3 days, not clinically complicated, and resolved spontaneously, or responded to conventional medical interventions; G3 or higher amylase/lipase elevation without symptoms of pancreatitis; G3 tumor lysis syndrome for up to 3 days, not clinically complicated, and resolved spontaneously, or responded to conventional medical interventions; and any participant meeting Hy's law criteria. | From first dose of study treatment (Cycle 1 Day 1) up to end of the Cycle 1 (Cycle 1 Day 28), maximum of 28 days |
| Part 2: Objective Response Rate (ORR) | ORR was planned to be assessed in Part 2. ORR was defined as percentage of responders as assessed by Investigator per International Myeloma Working Group (IMWG) 2016 guidelines for MM (complete response [CR], stringent CR [sCR], partial response [PR], very good PR [VGPR]) or Lugano 2014 guidelines for DLBCL (CR+PR). Per IMWG response criteria, CR: negative immunofixation on serum and urine; disappearance of soft tissue plasmacytomas; <5% plasma cells in bone marrow(BM); sCR: CR + normal free light chain (FLC) ratio; absence of BM clonal cells by immunohistochemistry; VGPR: serum and urine M-protein (MP) detected by immunofixation but not on electrophoresis or ≥90% reduction in serum MP + urine MP<100 mg/24 hours (h) or ≥90% decrease in sum of maximal perpendicular diameter (SPD) compared to baseline in soft tissue plasmacytoma; PR: ≥50% reduction of serum MP and reduction in 24 h urinary M-protein by ≥90% or to <200 mg/24 h; ≥50% reduction in size (SPD) of soft tissue plasmacytomas. |
| Measure | Description | Time Frame |
|---|---|---|
| Part 2: Progression-free Survival (PFS) | PFS was planned to be assessed in Part 2. PFS was defined as the time from start of treatment until the first documented progressive disease (PD), as assessed by Investigator per IMWG 2016 guidelines for MM or Lugano 2014 guidelines for DLBCL or death due to any cause, whichever occurred first. Per IMWG response criteria, PD was defined as any 1 or more of the following criteria: a) increase of ≥25% from lowest confirmed value in either serum MP, serum MP increase ≥1 gram/deciliter (g/dL) if the lowest M-component was ≥5 g/dL or urine M-component, b) appearance of new lesion(s), ≥50% increase from nadir in SPD of >1 lesion, or ≥50% increase in the longest diameter of a previous lesion >1 centimeter (cm) in short axis. |
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Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Ipsen Medical Director | Ipsen | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| City of Hope | Duarte | California | 91010 | United States | ||
| Astera Cancer Care |
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The study was planned to have a screening period (28 days), treatment period (Part 1 and Part 2) and safety follow-up period (30 days). Due to early study termination, only results of the Part 1 are presented. A total of 13 participants were enrolled in Part 1 of the study. EZM0414 is also referred to as IPN60210.
This first-in-human, Phase I/Ib, open-label, multi-center study was conducted in participants with relapsed/refractory (R/R) multiple myeloma (MM) and R/R diffuse large B cell lymphoma (DLBCL) at 15 investigational sites for Part 1 (dose escalation). The study was planned to be conducted in 2 parts: Part 1 and Part 2 (dose expansion). The study was terminated prior to completing Part 1 of the study. Part 2 was not started.
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| ID | Title | Description |
|---|---|---|
| FG000 | Part 1: EZM0414 100 mg | Participants with translocation (t)(4;14) R/R MM or non-t(4;14) R/R MM or R/R DLBCL received EZM0414 100 milligrams (mg) tablet orally once daily (QD) in continuous 28-day cycles (except on Days 2 and 3 of Cycle 1) until consent withdrawal, unacceptable toxicity, disease progression or need for treatment prohibited on this study. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jul 6, 2022 | Apr 17, 2025 |
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| Tumour assessments were planned to be performed at Screening (within 28 days before start of study treatment) or Cycle 1 Day 1 (pre-dose) up to end of the study, approximately 23 months |
| Tumour assessments were planned to be performed at Screening (within 28 days before start of study treatment) or Cycle 1 Day 1 (pre-dose) up to end of the study, approximately 23 months |
| Part 2: Disease Control Rate (DCR) | DCR was planned to be assessed in Part 2.DCR was defined as percentage of participants with confirmed CR,sCR,PR,VGPR,minimal response(MR) or stable disease(SD) per IMWG 2016 guidelines for MM/CR,PR, or SD per Lugano 2014 guidelines for DLBCL.Per IMWG response criteria,CR:negative immunofixation on serum and urine;disappearance of soft tissue plasmacytomas;<5% plasma cells in BM; sCR:CR+normal FLC ratio;absence of BM clonal cells by immunohistochemistry; VGPR:serum and urine MP detected by immunofixation but not electrophoresis or ≥90% reduction in serum MP+urine MP<100 mg/24h or ≥90% decrease in SPD in soft tissue plasmacytoma; PR:≥50% reduction of serum MP and reduction in 24h urinary M-protein by ≥90% or to <200mg/24h;≥50% reduction in size(SPD) of soft tissue plasmacytomas. MR:≥25% but ≤49% reduction in serum MP and reduction in 24h urine MP by 50-89%, which exceed 200 mg/24h; ≥50% reduction in size(SPD) of soft tissue plasmacytomas.SD:Not meeting criteria for CR,VGPR,PR,MR, or PD. | Tumour assessments were planned to be performed at Screening (within 28 days before start of study treatment) or Cycle 1 Day 1 (pre-dose) up to end of the study, approximately 23 months |
| Part 2: Duration of Response (DOR) | DOR was planned to be assessed in Part 2. DOR was defined as the time from initial CR or PR to documented progression or death, whichever came first, as assessed by Investigator per IMWG 2016 guidelines for MM or Lugano 2014 guidelines for DLBCL. Per IMWG response criteria, CR: negative immunofixation on serum and urine; disappearance of any soft tissue plasmacytomas; <5% plasma cells in BM. PR: ≥50% reduction of serum MP and reduction in 24 h urinary M-protein by ≥90% or to <200 mg/24 h; ≥50% reduction in the size (SPD) of soft tissue plasmacytomas. PD: any 1 or more of the following criteria: a) increase of ≥25% from lowest confirmed value in either serum MP, serum MP increase ≥1 g/dL if the lowest M-component was ≥5 g/dL or urine M-component, b) appearance of new lesion(s), ≥50% increase from nadir in SPD of >1 lesion, or ≥50% increase in the longest diameter of a previous lesion >1 cm in short axis. | Tumour assessments were planned to be performed at Screening (within 28 days before start of study treatment) or Cycle 1 Day 1 (pre-dose) up to end of the study, approximately 23 months |
| East Brunswick |
| New Jersey |
| 08816 |
| United States |
| Weill Cornell Medicine | New York | New York | 10021 | United States |
| Icahn School of Medicine at Mount Sinai | New York | New York | 10029 | United States |
| Memorial Sloan-Kettering Cancer Center | New York | New York | 10065 | United States |
| Baylor University Medical Center (Texas Oncology) | Dallas | Texas | 75246 | United States |
| MD Anderson Cancer Center | Houston | Texas | 77030 | United States |
| NEXT Virginia | Fairfax | Virginia | 22031 | United States |
| FG001 |
| Part 1: EZM0414 200 mg |
Participants with t(4;14) R/R MM or non-t(4;14) R/R MM or R/R DLBCL received EZM0414 200 mg tablet orally QD in continuous 28-day cycles (except on Days 2 and 3 of Cycle 1) until consent withdrawal, unacceptable toxicity, disease progression or need for treatment prohibited on this study. |
| FG002 | Part 1: EZM0414 300 mg | Participants with t(4;14) R/R MM or non-t(4;14) R/R MM or R/R DLBCL received EZM0414 300 mg tablet orally QD in continuous 28-day cycles (except on Days 2 and 3 of Cycle 1) until consent withdrawal, unacceptable toxicity, disease progression or need for treatment prohibited on this study. |
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| NOT COMPLETED |
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The safety population included all participants who received at least 1 dose of the study treatment in Part 1.
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| ID | Title | Description |
|---|---|---|
| BG000 | Part 1: EZM0414 100 mg | Participants with t(4;14) R/R MM or non-t(4;14) R/R MM or R/R DLBCL received EZM0414 100 mg tablet orally QD in continuous 28-day cycles (except on Days 2 and 3 of Cycle 1) until consent withdrawal, unacceptable toxicity, disease progression or need for treatment prohibited on this study. |
| BG001 | Part 1: EZM0414 200 mg | Participants with t(4;14) R/R MM or non-t(4;14) R/R MM or R/R DLBCL received EZM0414 200 mg tablet orally QD in continuous 28-day cycles (except on Days 2 and 3 of Cycle 1) until consent withdrawal, unacceptable toxicity, disease progression or need for treatment prohibited on this study. |
| BG002 | Part 1: EZM0414 300 mg | Participants with t(4;14) R/R MM or non-t(4;14) R/R MM or R/R DLBCL received EZM0414 300 mg tablet orally QD in continuous 28-day cycles (except on Days 2 and 3 of Cycle 1) until consent withdrawal, unacceptable toxicity, disease progression or need for treatment prohibited on this study. |
| BG003 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants | No |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants | No |
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| Race (NIH/OMB) | Count of Participants | Participants | No |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Part 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (TESAEs) | An adverse event was any untoward medical occurrence in a participant or clinical investigation participant administered a medicinal product and which does not necessarily had a causal relationship with this treatment. A serious adverse event was any untoward medical occurrence that at any dose, resulted in death, was life-threatening, required in-subject hospitalization or prolongation of hospitalization, resulted in persistent or significant disability or incapacity, or resulted in a congenital abnormality or birth defect, was an important medical event. A TEAE was an AE that started or worsened in severity on or after the date of the first dose of the study treatment through 30 days after the end of treatment. | The safety population included all participants who received at least 1 dose of the study treatment in Part 1. The study was terminated prior to completing Part 1 of the study. Part 2 was not started. | Posted | Count of Participants | Participants | No | From first dose of study treatment (Day 1) up to 30 days after the last dose of study treatment (Day 57), up to approximately 87 days |
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| Primary | Part 1: Number of Participants With Dose-limiting Toxicities (DLTs) | According to National Cancer Institute's Common Terminology Criteria for Adverse Events version 5.0, DLT was defined as any of following AE that occurred in Part 1 during first cycle of study treatment: grade (G)4 neutropenia lasting >5 days; G3 febrile neutropenia; G3 thrombocytopenia with clinically significant bleeding; G4 thrombocytopenia; G4 anemia unexplained by underlying disease; any other non-hematological toxicity ≥3 except: alopecia, G3 nausea/vomiting or diarrhea for <3 days with supportive care, G3 fatigue for <1 week, G3 or higher isolated electrolyte abnormalities for up to 3 days, not clinically complicated, and resolved spontaneously, or responded to conventional medical interventions; G3 or higher amylase/lipase elevation without symptoms of pancreatitis; G3 tumor lysis syndrome for up to 3 days, not clinically complicated, and resolved spontaneously, or responded to conventional medical interventions; and any participant meeting Hy's law criteria. | The DLT evaluable population included dose escalation dose level participants in the Safety population who received at least 80% of planned study treatment during Cycle 1. | Posted | Count of Participants | Participants | No | From first dose of study treatment (Cycle 1 Day 1) up to end of the Cycle 1 (Cycle 1 Day 28), maximum of 28 days |
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| Primary | Part 2: Objective Response Rate (ORR) | ORR was planned to be assessed in Part 2. ORR was defined as percentage of responders as assessed by Investigator per International Myeloma Working Group (IMWG) 2016 guidelines for MM (complete response [CR], stringent CR [sCR], partial response [PR], very good PR [VGPR]) or Lugano 2014 guidelines for DLBCL (CR+PR). Per IMWG response criteria, CR: negative immunofixation on serum and urine; disappearance of soft tissue plasmacytomas; <5% plasma cells in bone marrow(BM); sCR: CR + normal free light chain (FLC) ratio; absence of BM clonal cells by immunohistochemistry; VGPR: serum and urine M-protein (MP) detected by immunofixation but not on electrophoresis or ≥90% reduction in serum MP + urine MP<100 mg/24 hours (h) or ≥90% decrease in sum of maximal perpendicular diameter (SPD) compared to baseline in soft tissue plasmacytoma; PR: ≥50% reduction of serum MP and reduction in 24 h urinary M-protein by ≥90% or to <200 mg/24 h; ≥50% reduction in size (SPD) of soft tissue plasmacytomas. | The study was terminated prior to completing Part 1 of the study. Part 2 was not started. | Posted | Tumour assessments were planned to be performed at Screening (within 28 days before start of study treatment) or Cycle 1 Day 1 (pre-dose) up to end of the study, approximately 23 months |
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| Secondary | Part 2: Progression-free Survival (PFS) | PFS was planned to be assessed in Part 2. PFS was defined as the time from start of treatment until the first documented progressive disease (PD), as assessed by Investigator per IMWG 2016 guidelines for MM or Lugano 2014 guidelines for DLBCL or death due to any cause, whichever occurred first. Per IMWG response criteria, PD was defined as any 1 or more of the following criteria: a) increase of ≥25% from lowest confirmed value in either serum MP, serum MP increase ≥1 gram/deciliter (g/dL) if the lowest M-component was ≥5 g/dL or urine M-component, b) appearance of new lesion(s), ≥50% increase from nadir in SPD of >1 lesion, or ≥50% increase in the longest diameter of a previous lesion >1 centimeter (cm) in short axis. | The study was terminated prior to completing Part 1 of the study. Part 2 was not started. | Posted | Tumour assessments were planned to be performed at Screening (within 28 days before start of study treatment) or Cycle 1 Day 1 (pre-dose) up to end of the study, approximately 23 months |
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| Secondary | Part 2: Disease Control Rate (DCR) | DCR was planned to be assessed in Part 2.DCR was defined as percentage of participants with confirmed CR,sCR,PR,VGPR,minimal response(MR) or stable disease(SD) per IMWG 2016 guidelines for MM/CR,PR, or SD per Lugano 2014 guidelines for DLBCL.Per IMWG response criteria,CR:negative immunofixation on serum and urine;disappearance of soft tissue plasmacytomas;<5% plasma cells in BM; sCR:CR+normal FLC ratio;absence of BM clonal cells by immunohistochemistry; VGPR:serum and urine MP detected by immunofixation but not electrophoresis or ≥90% reduction in serum MP+urine MP<100 mg/24h or ≥90% decrease in SPD in soft tissue plasmacytoma; PR:≥50% reduction of serum MP and reduction in 24h urinary M-protein by ≥90% or to <200mg/24h;≥50% reduction in size(SPD) of soft tissue plasmacytomas. MR:≥25% but ≤49% reduction in serum MP and reduction in 24h urine MP by 50-89%, which exceed 200 mg/24h; ≥50% reduction in size(SPD) of soft tissue plasmacytomas.SD:Not meeting criteria for CR,VGPR,PR,MR, or PD. | The study was terminated prior to completing Part 1 of the study. Part 2 was not started. | Posted | Tumour assessments were planned to be performed at Screening (within 28 days before start of study treatment) or Cycle 1 Day 1 (pre-dose) up to end of the study, approximately 23 months |
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| Secondary | Part 2: Duration of Response (DOR) | DOR was planned to be assessed in Part 2. DOR was defined as the time from initial CR or PR to documented progression or death, whichever came first, as assessed by Investigator per IMWG 2016 guidelines for MM or Lugano 2014 guidelines for DLBCL. Per IMWG response criteria, CR: negative immunofixation on serum and urine; disappearance of any soft tissue plasmacytomas; <5% plasma cells in BM. PR: ≥50% reduction of serum MP and reduction in 24 h urinary M-protein by ≥90% or to <200 mg/24 h; ≥50% reduction in the size (SPD) of soft tissue plasmacytomas. PD: any 1 or more of the following criteria: a) increase of ≥25% from lowest confirmed value in either serum MP, serum MP increase ≥1 g/dL if the lowest M-component was ≥5 g/dL or urine M-component, b) appearance of new lesion(s), ≥50% increase from nadir in SPD of >1 lesion, or ≥50% increase in the longest diameter of a previous lesion >1 cm in short axis. | The study was terminated prior to completing Part 1 of the study. Part 2 was not started. | Posted | Tumour assessments were planned to be performed at Screening (within 28 days before start of study treatment) or Cycle 1 Day 1 (pre-dose) up to end of the study, approximately 23 months |
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Adverse events were collected from first dose of study treatment (Day 1) up to 30 days after the last dose of study treatment (Day 57), up to approximately 87 days. All-cause mortality (death) was assessed from first dose of study treatment (Day 1) up to early termination of the study, approximately 23 months.
The safety population included all participants who received at least 1 dose of the study treatment in Part 1. The study was terminated prior to completing Part 1 of the study. Part 2 was not started.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Part 1: EZM0414 100 mg | Participants with t(4;14) R/R MM or non-t(4;14) R/R MM or R/R DLBCL received EZM0414 100 mg tablet orally QD in continuous 28-day cycles (except on Days 2 and 3 of Cycle 1) until consent withdrawal, unacceptable toxicity, disease progression or need for treatment prohibited on this study. | 0 | 4 | 1 | 4 | 3 | 4 |
| EG001 | Part 1: EZM0414 200 mg | Participants with t(4;14) R/R MM or non-t(4;14) R/R MM or R/R DLBCL received EZM0414 200 mg tablet orally QD in continuous 28-day cycles (except on Days 2 and 3 of Cycle 1) until consent withdrawal, unacceptable toxicity, disease progression or need for treatment prohibited on this study. | 0 | 3 | 0 | 3 | 3 | 3 |
| EG002 | Part 1: EZM0414 300 mg | Participants with t(4;14) R/R MM or non-t(4;14) R/R MM or R/R DLBCL received EZM0414 300 mg tablet orally QD in continuous 28-day cycles (except on Days 2 and 3 of Cycle 1) until consent withdrawal, unacceptable toxicity, disease progression or need for treatment prohibited on this study. | 0 | 6 | 1 | 6 | 5 | 6 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal infection | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
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| Urosepsis | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
| |
| Small intestinal perforation | Gastrointestinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Platelet count decreased | Investigations | MedDRA 24.1 | Systematic Assessment |
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| Lymphocyte count decreased | Investigations | MedDRA 24.1 | Systematic Assessment |
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| Neutrophil count decreased | Investigations | MedDRA 24.1 | Systematic Assessment |
| |
| Amylase increased | Investigations | MedDRA 24.1 | Systematic Assessment |
| |
| Electrocardiogram QT prolonged | Investigations | MedDRA 24.1 | Systematic Assessment |
| |
| International normalised ratio increased | Investigations | MedDRA 24.1 | Systematic Assessment |
| |
| Protein total increased | Investigations | MedDRA 24.1 | Systematic Assessment |
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| White blood cell count decreased | Investigations | MedDRA 24.1 | Systematic Assessment |
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| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Anaemia | Blood and lymphatic system disorders | MedDRA 24.1 | Systematic Assessment |
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| Lymphadenopathy | Blood and lymphatic system disorders | MedDRA 24.1 | Systematic Assessment |
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| Fatigue | General disorders | MedDRA 24.1 | Systematic Assessment |
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| Face oedema | General disorders | MedDRA 24.1 | Systematic Assessment |
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| Gait disturbance | General disorders | MedDRA 24.1 | Systematic Assessment |
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| Non-cardiac chest pain | General disorders | MedDRA 24.1 | Systematic Assessment |
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| Oedema peripheral | General disorders | MedDRA 24.1 | Systematic Assessment |
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| Pyrexia | General disorders | MedDRA 24.1 | Systematic Assessment |
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| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 24.1 | Systematic Assessment |
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| Bone pain | Musculoskeletal and connective tissue disorders | MedDRA 24.1 | Systematic Assessment |
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| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 24.1 | Systematic Assessment |
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| Musculoskeletal chest pain | Musculoskeletal and connective tissue disorders | MedDRA 24.1 | Systematic Assessment |
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| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 24.1 | Systematic Assessment |
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| Diarrhoea | Gastrointestinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Abdominal pain upper | Gastrointestinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Constipation | Gastrointestinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Dry mouth | Gastrointestinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Nausea | Gastrointestinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Upper respiratory tract infection | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
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| Urinary tract infection | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
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| Otitis externa | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA 24.1 | Systematic Assessment |
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| Dyskinesia | Nervous system disorders | MedDRA 24.1 | Systematic Assessment |
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| Hypotension | Vascular disorders | MedDRA 24.1 | Systematic Assessment |
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| Palpitations | Cardiac disorders | MedDRA 24.1 | Systematic Assessment |
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| Sinus tachycardia | Cardiac disorders | MedDRA 24.1 | Systematic Assessment |
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| Hypomagnesaemia | Metabolism and nutrition disorders | MedDRA 24.1 | Systematic Assessment |
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| Hyperphosphataemia | Metabolism and nutrition disorders | MedDRA 24.1 | Systematic Assessment |
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| Hyperuricaemia | Metabolism and nutrition disorders | MedDRA 24.1 | Systematic Assessment |
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| Hypoalbuminaemia | Metabolism and nutrition disorders | MedDRA 24.1 | Systematic Assessment |
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| Hyponatraemia | Metabolism and nutrition disorders | MedDRA 24.1 | Systematic Assessment |
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| Anxiety | Psychiatric disorders | MedDRA 24.1 | Systematic Assessment |
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| Confusional state | Psychiatric disorders | MedDRA 24.1 | Systematic Assessment |
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| Dermatitis | Skin and subcutaneous tissue disorders | MedDRA 24.1 | Systematic Assessment |
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| Rash maculo-papular | Skin and subcutaneous tissue disorders | MedDRA 24.1 | Systematic Assessment |
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| Eye pain | Eye disorders | MedDRA 24.1 | Systematic Assessment |
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| Fall | Injury, poisoning and procedural complications | MedDRA 24.1 | Systematic Assessment |
| |
| Tumour pain | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 24.1 | Systematic Assessment |
|
This study was terminated prior to completing Part 1 due to business reasons. Study termination was not due to safety concerns.
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Director | Ipsen | see email | clinical.trials@ipsen.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jul 23, 2024 | Apr 17, 2025 | SAP_001.pdf |
| ID | Term |
|---|---|
| D009101 | Multiple Myeloma |
| D016403 | Lymphoma, Large B-Cell, Diffuse |
| ID | Term |
|---|---|
| D054219 | Neoplasms, Plasma Cell |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D020141 | Hemostatic Disorders |
| D014652 | Vascular Diseases |
| D002318 | Cardiovascular Diseases |
| D010265 | Paraproteinemias |
| D001796 | Blood Protein Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D006474 | Hemorrhagic Disorders |
| D008232 | Lymphoproliferative Disorders |
| D007160 | Immunoproliferative Disorders |
| D007154 | Immune System Diseases |
| D016393 | Lymphoma, B-Cell |
| D008228 | Lymphoma, Non-Hodgkin |
| D008223 | Lymphoma |
| D008206 | Lymphatic Diseases |
Not provided
Not provided
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| Title | Measurements |
|---|---|
|
| OG001 | Part 1: EZM0414 200 mg | Participants with t(4;14) R/R MM or non-t(4;14) R/R MM or R/R DLBCL received EZM0414 200 mg tablet orally QD in continuous 28-day cycles (except on Days 2 and 3 of Cycle 1) until consent withdrawal, unacceptable toxicity, disease progression or need for treatment prohibited on this study. |
| OG002 | Part 1: EZM0414 300 mg | Participants with t(4;14) R/R MM or non-t(4;14) R/R MM or R/R DLBCL received EZM0414 300 mg tablet orally QD in continuous 28-day cycles (except on Days 2 and 3 of Cycle 1) until consent withdrawal, unacceptable toxicity, disease progression or need for treatment prohibited on this study. |
|
|
| OG001 |
| Part 2: Cohort 2: R/R MM Without t(4;14) |
Participants with R/R MM without t(4;14) were planned to receive EZM0414 tablet at MTD orally QD in continuous 28-day cycles until consent withdrawal, unacceptable toxicity, disease progression or need for treatment prohibited on this study. |
| OG002 | Part 2: Cohort 3: R/R DLBCL | Participants with R/R DLBCL were planned to receive EZM0414 tablet at MTD orally QD in continuous 28-day cycles until consent withdrawal, unacceptable toxicity, disease progression or need for treatment prohibited on this study. |
|
| OG002 | Part 2: Cohort 3: R/R DLBCL | Participants with R/R DLBCL were planned to receive EZM0414 tablet at MTD orally QD in continuous 28-day cycles until consent withdrawal, unacceptable toxicity, disease progression or need for treatment prohibited on this study. |
|
| Part 2: Cohort 2: R/R MM Without t(4;14) |
Participants with R/R MM without t(4;14) were planned to receive EZM0414 tablet at MTD orally QD in continuous 28-day cycles until consent withdrawal, unacceptable toxicity, disease progression or need for treatment prohibited on this study. |
| OG002 | Part 2: Cohort 3: R/R DLBCL | Participants with R/R DLBCL were planned to receive EZM0414 tablet at MTD orally QD in continuous 28-day cycles until consent withdrawal, unacceptable toxicity, disease progression or need for treatment prohibited on this study. |
|
Participants with R/R MM without t(4;14) were planned to receive EZM0414 tablet at MTD orally QD in continuous 28-day cycles until consent withdrawal, unacceptable toxicity, disease progression or need for treatment prohibited on this study. |
| OG002 | Part 2: Cohort 3: R/R DLBCL | Participants with R/R DLBCL were planned to receive EZM0414 tablet at MTD orally QD in continuous 28-day cycles until consent withdrawal, unacceptable toxicity, disease progression or need for treatment prohibited on this study. |
|