Trials · Topics · Multiple Myeloma
Multiple Myeloma clinical trials
65 Multiple Myeloma trials (Malignant Hematology) summarized for fellows and board review, newest first within each disease. Each summary covers design, results, toxicity and what changed in practice.
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MM (60)
- ENDURANCE trial: lenalidomide maintenance duration N Engl J Med 2026In standard-risk NDMM patients not undergoing up-front ASCT, indefinite-duration lenalidomide maintenance did not prolong OS versus a fixed 2-year course. PFS...
- EXCALIBER-RRMM Lancet Oncol 2026IberDd significantly improved MRD-negative CR vs DVd in 1-2 prior line RRMM, with high but manageable hematologic toxicity. PFS, the second dual primary endpoint, is...
- Felzartamab + Rd (China) Lancet Haematol 2026Felzartamab + Rd significantly prolonged PFS vs Rd in Chinese patients with RRMM, supporting it as a potential new treatment option in this population.
- IsKia Nat Med 2026Isa-KRd increased MRD negativity rates compared with KRd in TE NDMM, with a higher proportion of patients achieving MRD negativity at 10⁻⁵ after consolidation...
- MAIA trial (final OS) Leukemia 2026With more than 7 years of follow-up, D-Rd established a new median OS benchmark of 7.5 years in transplant-ineligible NDMM, a >26-month absolute median OS gain over...
- MajesTEC-9 trial NEJM 2026In relapsed/refractory multiple myeloma after 1-3 prior lines (anti-CD38 and lenalidomide exposed), teclistamab monotherapy significantly improved PFS and OS vs...
- MonumenTAL-3 trial NEJM 2026Both talquetamab-daratumumab combinations (with or without pomalidomide) produced large PFS gains (HR 0.28 and 0.33), deep MRD-negative responses (~46-52% vs 16%)...
- SeaLAND trial (ALLG MM23) Br J Haematol 2026Adding low-dose selinexor to lenalidomide maintenance after ASCT did not improve PFS or significantly increase CR rate, and substantially increased toxicity while...
- Second Primary Malignancy Risk in Multiple Myeloma Cohort Front Oncol 2026MM patients have a distinct second primary malignancy pattern, with hematologic SPMs carrying an approximately 3-fold increased risk. Selected solid tumors (melanoma...
- SUCCESSOR-2 trial Lancet 2026Mezigdomide-carfilzomib-dexamethasone more than doubled median PFS versus Kd (18.0 vs 8.3mo; HR 0.48) as early as first relapse in a heavily...
- AURIGA Blood 2025Daratumumab added to lenalidomide maintenance significantly improved MRD-negative conversion in post-ASCT NDMM patients with residual MRD-positive disease, with an...
- CANOVA JCO 2025CANOVA did not meet its primary PFS endpoint, but venetoclax-dexamethasone demonstrated numerically longer PFS and OS, markedly higher response rates, and MRD...
- CEPHEUS Nat Med 2025D-VRd significantly improved MRD-negativity rates and response depth compared with VRd alone in transplant-ineligible or transplant-deferred NDMM, with a clinically...
- MIDAS NEJM 2025In MRD-negative TE NDMM patients after quadruplet Isa-KRd induction, adding ASCT did not improve MRD-negativity over continued Isa-KRd consolidation, suggesting...
- TECLISTAMAB + DARATUMUMAB NEJM 2025Teclistamab + daratumumab demonstrated dramatically superior PFS, response depth, and MRD negativity rates compared with standard daratumumab-based doublet/triplet...
- BENEFIT Nat Med 2024Addition of bortezomib to an isatuximab + Rd backbone doubled MRD-negativity rates at 18 months in transplant-ineligible NDMM, demonstrating that quadruplet Isa-VRd...
- DREAMM-7 NEJM 2024BVd demonstrated a clinically meaningful PFS benefit over DVd in RRMM, with a median PFS of 36.6 months among the longest reported in a randomized RRMM trial. The...
- DREAMM-8 NEJM 2024BPd significantly improved PFS over PVd in RRMM patients with prior lenalidomide exposure, with deeper responses and avoidance of bortezomib-related neuropathy. This...
- IMROZ NEJM 2024Isatuximab added to VRd (Isa-VRd) significantly prolonged PFS with a 40% reduction in risk of progression or death, and achieved substantially higher CR and...
- LINKER-MM1 JCO 2024Linvoseltamab 200 mg achieved a 70.9% ORR with an exceptional 49.6% ≥CR rate and a median DOR of 29.4 months in RRMM - among the highest ≥CR rates reported for a...
- PERSEUS NEJM 2024D-VRd significantly improved 4-year PFS (84% vs 68%, HR 0.42) and sustained MRD negativity rates more than doubling those of VRd in transplant-eligible NDMM...
- CARTITUDE-2 (Cohort C) Blood 2023Cilta-cel produced an ORR of 60% in heavily pretreated patients largely refractory to prior non-cellular anti-BCMA therapy, supporting activity even after prior...
- CARTITUDE-4 NEJM 2023Cilta-cel produced a 74% reduction in risk of progression or death (HR 0.26) compared to standard salvage regimens in lenalidomide-exposed relapsed/refractory myeloma...
- KarMMa-3 NEJM 2023Ide-cel significantly improved PFS (HR 0.49, mPFS 13.3 vs 4.4 months) and response depth vs standard triplet regimens in 2-4 line triple-class-exposed RRMM...
- MagnetisMM-3 Nat Med 2023Elranatamab achieved a 61% ORR with 35% ≥CR and durable responses (DOR/PFS/OS medians not reached at 14.7 months) in BCMA-na ve RRMM, with all CRS events grade 1-2...
- DETERMINATION NEJM 2022Early ASCT significantly prolonged PFS by 21 months (HR 0.53) compared with VRd + indefinite lenalidomide maintenance in transplant-eligible NDMM, reaffirming upfront...
- MajesTEC-1 NEJM 2022Teclistamab achieved a 63% ORR with 39% ≥CR in triple-class-exposed RRMM - the first off-the-shelf BCMA-directed bispecific antibody to show efficacy in this setting...
- MASTER Trial JCO 2022Dara-KRd with autologous HCT and MRD response-adapted consolidation produced high rates of MRD negativity in newly diagnosed multiple myeloma. For patients with 0 or...
- MonumenTAL-1 NEJM 2022Talquetamab achieved 64-70% ORR at the two recommended doses in heavily pretreated RRMM, with a novel toxicity profile of skin/nail effects and dysgeusia reflecting...
- CARTITUDE-1 Lancet 2021Cilta-cel achieved an unprecedented 97.9% ORR with 67% ≥CR and 93% MRD-negativity in heavily pretreated triple-class-exposed RRMM, establishing the highest response...
- FORTE trial Blood 2021ASCT retains an important consolidation role in young transplant-eligible newly diagnosed MM, with longer PFS than KRd12 alone. KRd induction yields deeper response...
- IKEMA Lancet 2021Isatuximab added to Kd (Isa-Kd) significantly improved PFS (HR 0.53) and response depth including MRD-negativity in 1-3 line RRMM without prior anti-CD38 or...
- KarMMa NEJM 2021Ide-cel achieved a 73% ORR including 33% complete responses in triple-class-exposed RRMM with a manageable CRS profile, marking the first CAR-T cell therapy approval...
- BOSTON Lancet 2020Selinexor + once-weekly bortezomib + dexamethasone (XVd) significantly improved PFS and response rates compared with twice-weekly Vd in 1-3 line RRMM, with notably...
- EMN02/HO95 Lancet Haematol 2020Upfront ASCT significantly improved both PFS and OS vs VMP in transplant-eligible NDMM in the novel-agent era, confirming ASCT as standard of care. VRd consolidation...
- Endurance Trial Lancet Oncol 2020KRd did not improve PFS vs VRd in newly diagnosed MM without high-risk features and carried a distinct toxicity profile (less neuropathy but more...
- GRIFFIN trial Blood 2020Adding daratumumab to RVd improved depth of response (sCR and MRD-negativity) in transplant-eligible NDMM, with responses deepening over continued therapy and a...
- POLLUX Trial Leukemia 2020In relapsed/refractory multiple myeloma, daratumumab added to lenalidomide and dexamethasone resulted in significantly improved PFS compared with Rd alone, with deep...
- CASSIOPEIA Lancet 2019D-VTd induction and consolidation around ASCT significantly improved sCR rate at Day 100 post-transplant and 18-month PFS compared to VTd, with deeper MRD negativity...
- ICARIA-MM Lancet 2019Isatuximab added to pomalidomide + dexamethasone (Isa-Pd) significantly improved PFS (HR 0.596) and OS (HR 0.687) in 2+ prior line RRMM refractory to prior...
- MAIA NEJM 2019Daratumumab added to Rd (DRd) significantly prolonged PFS and OS versus Rd alone in transplant-ineligible NDMM, with markedly higher sCR and MRD-negativity rates...
- MYELOMA XI Lancet Oncol 2019Continuous lenalidomide maintenance significantly improved PFS (HR 0.44) and, at extended follow-up, demonstrated an OS benefit (HR 0.78) in both transplant-eligible...
- OPTIMISMM Trial Lancet Oncol 2019PVd significantly improved progression-free survival over Vd in lenalidomide-exposed relapsed/refractory multiple myeloma, supporting the addition of pomalidomide in...
- STaMINA JCO 2019Neither tandem ASCT nor VRd consolidation improved PFS or OS vs single ASCT followed directly by lenalidomide maintenance. Single ASCT + lenalidomide maintenance...
- STORM NEJM 2019Selinexor + low-dose dexamethasone achieved a 26% ORR in penta-refractory myeloma - a population with no approved treatment options and historically near-zero...
- ALCYONE NEJM 2018Daratumumab added to VMP significantly improved PFS, response depth, and MRD-negativity in transplant-ineligible NDMM, with a PFS hazard ratio of 0.50. The trial...
- FIRST Trial Final Analysis Blood 2018In transplant-ineligible newly diagnosed multiple myeloma, continuous lenalidomide plus dexamethasone significantly improved PFS compared with fixed-duration Rd and...
- IFM 2009 NEJM 2017Upfront ASCT after VRd induction significantly prolonged PFS (50 vs 36 months, HR 0.65) and deepened responses (CR and MRD negativity) compared to VRd alone without...
- Meta-Analysis - Len maintenance post Auto-HCT 2017Lenalidomide maintenance after autologous HCT conferred a significant overall survival benefit and confirmed the PFS benefit versus placebo/observation. Despite an...
- CASTOR Trial NEJM 2016In relapsed/refractory multiple myeloma, daratumumab added to bortezomib and dexamethasone significantly prolonged PFS compared with Vd alone.
- ENDEAVOR Lancet Oncol 2016Carfilzomib 56 mg/m² + dexamethasone (Kd56) significantly improved PFS compared with bortezomib + dexamethasone (Vd) in 1-3 line RRMM, doubling median PFS from 9.4 to...
- SWOG S0777 Trial Lancet 2016Triplet RVD induction significantly improved progression-free and overall survival vs doublet Rd in newly diagnosed MM not slated for immediate transplant...
- TOURMALINE-MM1 NEJM 2016All-oral ixazomib added to Rd (IRd) significantly improved PFS (HR 0.74) in RRMM, providing the first all-oral proteasome-inhibitor-based triplet for myeloma and...
- ASPIRE NEJM 2015Carfilzomib added to Rd (KRd) significantly improved PFS by 8.7 months and demonstrated OS benefit in 1-3 line RRMM, establishing KRd as an effective triplet regimen...
- ELOQUENT-2 NEJM 2015Elotuzumab added to Rd (ERd) significantly improved PFS (HR 0.70) and demonstrated a 4-year OS benefit (HR 0.78) in relapsed myeloma, establishing elotuzumab as the...
- CALGB 100104 NEJM 2012Lenalidomide maintenance post-ASCT significantly improved TTP and OS in NDMM, with a long-term OS benefit of ~1 year. The SPM risk (~7.8%) requires informed-consent...
- EVOLUTION Trial Blood 2012All bortezomib-based regimens were highly active and well tolerated in previously untreated multiple myeloma. No formal statistical comparison between the four arms...
- IFM 2005-02 NEJM 2012Lenalidomide consolidation + maintenance significantly doubled PFS vs placebo post-ASCT in NDMM (41 vs 23 months), with no OS benefit at primary analysis due to...
- MGUS risk stratification Blood 2005Three readily measurable factors (M-protein ≥1.5 g/dL, non-IgG isotype, abnormal FLC ratio) stratify MGUS into low, low-intermediate, high-intermediate, and high-risk...
- SUMMIT trial NEJM 2003Single-agent bortezomib produced clinically meaningful and durable responses in heavily pretreated relapsed/refractory multiple myeloma. Addition of dexamethasone...
Amyloidosis (3)
- ANDROMEDA: Final Survival Analysis Blood 2026Adding subcutaneous daratumumab to VCd produced deeper and faster hematologic responses, improved organ-function recovery, and translated into statistically...
- CARES trials (anselamimab) JCO 2026Anselamimab added to standard plasma cell-directed therapy did not meet its primary composite endpoint of mortality and cardiovascular hospitalization in the overall...
- ANDROMEDA NEJM 2021Daratumumab SC added to VCd tripled the hematologic CR rate (53% vs 18%) and significantly improved cardiac and renal organ responses compared to VCd alone in newly...
SMM (2)
- AQUILA NEJM 2024Daratumumab SC significantly reduced the risk of progression from high-risk SMM to active myeloma compared with active monitoring at 5 years (63% vs 41%), supporting...
- E3A06 JCO 2020Lenalidomide significantly delayed progression from high-risk SMM to active myeloma and demonstrated an OS benefit at 6.5-year follow-up, establishing the first phase...