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NCT01734928: OPTIMISMM

Safety and Efficacy of Pomalidomide, Bortezomib and Low-dose Dexamethasone in Subjects With Relapsed or Refractory Multiple Myeloma

Completed Phase 3 Results posted Last updated 6 June 2023
What this trial tests

Phase 3 trial testing Pomalidomide in Multiple Myeloma in 559 participants. Completed in 13 May 2022.

Timeline
7 January 2013
Primary endpoint
9 May 2022
13 May 2022

Quick facts

Lead sponsorCelgene
PhasePhase 3
StatusCompleted
Study typeINTERVENTIONAL
Allocationrandomized
Designparallel
Maskingnone
Primary purposetreatment
Enrollment559
Start date7 January 2013
Primary completion9 May 2022
Estimated completion13 May 2022
Sites321 locations across Italy, Finland, Japan, Ireland, Poland, Denmark, Netherlands, Russia

Drugs / interventions tested

Conditions studied

Sponsor

Celgene — full company profile →

Who can join

18 and older, any sex, with Multiple Myeloma. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Progression Free Survival by Independent Response Adjudication Committee (IRAC) Primary · From randomization to progressive disease or death during the IRAC assessment period, up to approximately 42 months

Progression free survival (PFS) will be calculated as the time between the randomization and progressive disease (PD) or death. Progressive Disease is defined as an Increase of ≥ 25% from nadir in: * Serum M-component and/or (the absolute increase must be ≥ 0.5 g/dL)g * Urine M-component and/or (the absolute increase must be ≥ 200 mg/24 hours) * In patients without measurable serum and urine M-protein levels the difference between involved and uninvolved FLC levels, the absolute increase must be \> 100 mg/dL. * Bone marrow plasma cell percentage, the absolute % must be ≥ 10%h * Definite deve

GroupValue95% CI
Treatment 1: POM+BTZ+LD-DEX11.209.66 – 13.73
Treatment 2: BTZ+LD-DEX7.105.88 – 8.48
Overall Survival (OS) Secondary · From randomization to date of death, up to approximately 65 months

Overall survival (OS) is calculated as the time from randomization to death from any cause.

GroupValue95% CI
Treatment 1: POM+BTZ+LD-DEX35.5828.55 – 41.20
Treatment 2: BTZ+LD-DEX31.6126.05 – 37.16
Overall Response Rate by Independent Response Adjudication Committee (IRAC) Secondary · From randomization to progressive disease or death during the IRAC assessment period, up to approximately 42 months

The ORR together with the relative proportions in each response category (ie, stringent CR \[sCR\], CR, very good PR \[VGPR\], PR, SD, and PD) by treatment using the IMWG criteria will be examined. Complete Response: Negative immunofixation on the serum and urine and disappearance of any soft tissue plasmacytomas and ≤ 5% plasma cells in bone marrow SCR: CR+ Normal FLC ratio and Absence of clonal cells in bone marrow by immunohistochemistry or immunofluorescence VGPR: Serum and urine M-protein detectable by immunofixation but not on electrophoresis or 90% or greater reduction in serum M-pro

Stringent complete response
GroupValue95% CI
Treatment 1: POM+BTZ+LD-DEX9
Treatment 2: BTZ+LD-DEX2
Complete Reponse
GroupValue95% CI
Treatment 1: POM+BTZ+LD-DEX35
Treatment 2: BTZ+LD-DEX9
Very Good Partial Response
GroupValue95% CI
Treatment 1: POM+BTZ+LD-DEX104
Treatment 2: BTZ+LD-DEX40
Partial Response
GroupValue95% CI
Treatment 1: POM+BTZ+LD-DEX83
Treatment 2: BTZ+LD-DEX88
Stable Disease
GroupValue95% CI
Treatment 1: POM+BTZ+LD-DEX32
Treatment 2: BTZ+LD-DEX106
Progressive Disease
GroupValue95% CI
Treatment 1: POM+BTZ+LD-DEX11
Treatment 2: BTZ+LD-DEX16
Not Evaluable
GroupValue95% CI
Treatment 1: POM+BTZ+LD-DEX7
Treatment 2: BTZ+LD-DEX17
Duration of Response by Independent Response Adjudication Committee (IRAC) Secondary · From randomization to progressive disease or death during the IRAC assessment period, up to approximately 42 months

Duration of myeloma response is defined as the duration from the time when the IMWG response criteria are first met for sCR or CR or VGPR or PR until the first date the response criteria are met for PD or until the subject died from any cause, whichever occurs first. Complete Response: Negative immunofixation on the serum and urine and disappearance of any soft tissue plasmacytomas and ≤ 5% plasma cells in bone marrow SCR: CR+ Normal FLC ratio and Absence of clonal cells in bone marrow by immunohistochemistry or immunofluorescence VGPR: Serum and urine M-protein detectable by immunofixation

GroupValue95% CI
Treatment 1: POM+BTZ+LD-DEX13.7010.94 – 18.10
Treatment 2: BTZ+LD-DEX10.948.11 – 14.78
Number of Participants With Grade 3-4 Treatment Emergent Adverse Events (TEAE) Secondary · From first dose to 28 days after the last dose (up to approximately 44 months

Treatment-emergent adverse events (TEAEs) are defined as any AE occurring or worsening on or after the first dose date of the study treatment and within 28 days after the last dose date.

GroupValue95% CI
Treatment 1: POM+BTZ+LD-DEX259
Treatment 2: BTZ+LD-DEX194
Number of Participants With Grade 5 Treatment Emergent Adverse Events (TEAE) Secondary · From first dose to 28 days after the last dose (up to approximately 44 months

Treatment-emergent adverse events (TEAEs) are defined as any AE occurring or worsening on or after the first dose date of the study treatment and within 28 days after the last dose date.

GroupValue95% CI
Treatment 1: POM+BTZ+LD-DEX29
Treatment 2: BTZ+LD-DEX12

Adverse events — posted to ClinicalTrials.gov

Time frame: AEs and SAEs: From first treatment to 28 days after last dose, up to approximately 44 months on average of 10 months. All-Cause mortality: from randomization to end of the study, approximately 65 months.. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Treatment 1: POM + BTZ + LD-DEX
Serious: 177/278 (64%)
Deaths: 197/281
Treatment 2: BTZ + LD-DEX
Serious: 119/270 (44%)
Deaths: 193/278

Serious adverse events (277 terms)

ReactionSystemTreatment 1: POM + BTZ + L…Treatment 2: BTZ + LD-DEX
PneumoniaInfections and infestations
PyrexiaGeneral disorders
InfluenzaInfections and infestations
Lower respiratory tract infectionInfections and infestations
Atrial fibrillationCardiac disorders
General physical health deteriorationGeneral disorders
Pulmonary embolismRespiratory, thoracic and mediastinal disorders
Acute kidney injuryRenal and urinary disorders
DiarrhoeaGastrointestinal disorders
Septic shockInfections and infestations
Basal cell carcinomaNeoplasms benign, malignant and unspecified (incl cysts and polyps)
SyncopeNervous system disorders
AnaemiaBlood and lymphatic system disorders
Febrile neutropeniaBlood and lymphatic system disorders
Respiratory tract infectionInfections and infestations
SepsisInfections and infestations
DeathGeneral disorders
Clostridium difficile colitisInfections and infestations
Squamous cell carcinoma of skinNeoplasms benign, malignant and unspecified (incl cysts and polyps)
DyspnoeaRespiratory, thoracic and mediastinal disorders
Deep vein thrombosisVascular disorders
Hyperviscosity syndromeBlood and lymphatic system disorders
ThrombocytopeniaBlood and lymphatic system disorders
Cardiac failureCardiac disorders
Cardiac failure congestiveCardiac disorders
Other adverse events (70 terms — click to expand)

ReactionSystemTreatment 1: POM + BTZ + L…Treatment 2: BTZ + LD-DEX
NeutropeniaBlood and lymphatic system disorders
Peripheral sensory neuropathyNervous system disorders
ThrombocytopeniaBlood and lymphatic system disorders
FatigueGeneral disorders
ConstipationGastrointestinal disorders
DiarrhoeaGastrointestinal disorders
Oedema peripheralGeneral disorders
AnaemiaBlood and lymphatic system disorders
PyrexiaGeneral disorders
Upper respiratory tract infectionInfections and infestations
CoughRespiratory, thoracic and mediastinal disorders
DyspnoeaRespiratory, thoracic and mediastinal disorders
Back painMusculoskeletal and connective tissue disorders
NauseaGastrointestinal disorders
InsomniaPsychiatric disorders
AstheniaGeneral disorders
DizzinessNervous system disorders
HypokalaemiaMetabolism and nutrition disorders
BronchitisInfections and infestations
HyperglycaemiaMetabolism and nutrition disorders
Pain in extremityMusculoskeletal and connective tissue disorders
Muscular weaknessMusculoskeletal and connective tissue disorders
ArthralgiaMusculoskeletal and connective tissue disorders
LeukopeniaBlood and lymphatic system disorders
Urinary tract infectionInfections and infestations
Viral upper respiratory tract infectionInfections and infestations
VomitingGastrointestinal disorders
Accidental overdoseInjury, poisoning and procedural complications
HeadacheNervous system disorders
TremorNervous system disorders
RashSkin and subcutaneous tissue disorders
Abdominal painGastrointestinal disorders
Muscle spasmsMusculoskeletal and connective tissue disorders
Decreased appetiteMetabolism and nutrition disorders
InfluenzaInfections and infestations
Atrial fibrillationCardiac disorders
ConjunctivitisInfections and infestations
PneumoniaInfections and infestations
HypotensionVascular disorders
DyspepsiaGastrointestinal disorders

Most-reported serious reactions: Pneumonia, Pyrexia, Influenza, Lower respiratory tract infection, Atrial fibrillation, General physical health deterioration, Pulmonary embolism, Acute kidney injury.

Data from ClinicalTrials.gov NCT01734928 adverse events section.

Sponsor's own description

The purpose of this study is to compare the efficacy of the combination of pomalidomide, bortezomib and low dose dexamethasone to the combination of bortezomib and low dose dexamethasone in participants with relapsed/refractory multiple myeloma. This study will also assess how safe the combination of pomalidomide, bortezomib and low dose dexamethasone is compared to the combination of bortezomib and low dose dexamethasone.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Pomalidomide, bortezomib, and dexamethasone for patients with relapsed or refractory multiple myeloma previously treated with lenalidomide (OPTIMISMM): a randomised, open-label, phase 3 trial.
    Richardson PG, Oriol A, Beksac M, Liberati AM, et al · · 2019 · cited 300× · PMID 31097405 · DOI 10.1016/s1470-2045(19)30152-4
  2. Pomalidomide: the new immunomodulatory agent for the treatment of multiple myeloma.
    Chanan-Khan AA, Swaika A, Paulus A, Kumar SK, et al · · 2013 · cited 94× · PMID 24013664 · DOI 10.1038/bcj.2013.38
  3. Evolving paradigms in the treatment of relapsed/refractory multiple myeloma: increased options and increased complexity.
    Cornell RF, Kassim AA. · · 2016 · cited 67× · PMID 26726946 · DOI 10.1038/bmt.2015.307
  4. Emerging agents and regimens for multiple myeloma.
    Yang Y, Li Y, Gu H, Dong M, et al · · 2020 · cited 57× · PMID 33168044 · DOI 10.1186/s13045-020-00980-5
  5. Pomalidomide, bortezomib, and dexamethasone for multiple myeloma previously treated with lenalidomide (OPTIMISMM): outcomes by prior treatment at first relapse.
    Dimopoulos M, Weisel K, Moreau P, Anderson LD, et al · · 2021 · cited 49× · PMID 32895455 · DOI 10.1038/s41375-020-01021-3
  6. Pomalidomide, bortezomib, and dexamethasone for patients with relapsed lenalidomide-refractory multiple myeloma.
    Paludo J, Mikhael JR, LaPlant BR, Halvorson AE, et al · · 2017 · cited 48× · PMID 28684537 · DOI 10.1182/blood-2017-05-782961
  7. Relapsed/Refractory Multiple Myeloma in 2020/2021 and Beyond.
    Podar K, Leleu X. · · 2021 · cited 42× · PMID 34680303 · DOI 10.3390/cancers13205154
  8. Novel agents for multiple myeloma to overcome resistance in phase III clinical trials.
    Orlowski RZ. · · 2013 · cited 33× · PMID 24135408 · DOI 10.1053/j.seminoncol.2013.07.007

Verify or expand the search:

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Trials testing the same drug.

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Trials by the same sponsor.

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Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT01734928.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing