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NCT03748953

Study of Oral Ixazomib Maintenance Therapy After Initial Therapy in Participants With Newly Diagnosed Multiple Myeloma Not Treated With Stem Cell Transplantation (SCT)

Completed Phase 3 Results posted Last updated 21 March 2025
What this trial tests

Phase 3 trial testing Ixazomib in Multiple Myeloma in 37 participants. Completed in 6 December 2023.

Timeline
24 January 2019
Primary endpoint
6 December 2023
6 December 2023

Quick facts

Lead sponsorMillennium Pharmaceuticals, Inc.
PhasePhase 3
StatusCompleted
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment37
Start date24 January 2019
Primary completion6 December 2023
Estimated completion6 December 2023
Sites10 locations across China

Drugs / interventions tested

Conditions studied

Sponsor

Millennium Pharmaceuticals, Inc. — 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.

Number of Participants Categorized According to Performance Status (PS) Based on Eastern Cooperative Oncology Group (ECOG) PS Primary · Month 25

ECOG PS was used to assess physical health of participants. ECOG PS grade:0= fully active, able to carry on all pre-disease performance without restriction,1= restricted in physically strenuous activity but ambulatory and able to carry out work of a light or sedentary nature 2= ambulatory and capable of all self-care but unable to carry out any work activities. Up and about more than 50% of waking hours, 3= capable of only limited self-care, 4= completely disabled, cannot carry on any selfcare, totally confined to bed or chair confined to bed or chair more than 50% of waking hours and 5= dead.

ECOG Score 0
GroupValue95% CI
Ixazomib34
ECOG Score 1
GroupValue95% CI
Ixazomib1
Percentage of Participants Receiving Ixazomib With Treatment-emergent Adverse Events (TEAEs) Primary · Up to 58.4 months

Adverse events (AEs) were defined as any unfavorable and unintended sign, symptom or disease temporally associated with the use of a medicinal product reported from first dose of study drug through 30 days after the last dose of study drug. A TEAE was defined as an AE that started or worsened after first study drug administration and within 30 days of last dose of study drug. Percentages are rounded off to the nearest whole number.

GroupValue95% CI
Ixazomib97
Percentage of Participants Receiving Ixazomib With Treatment-emergent Serious Adverse Events (SAEs) Primary · Up to 58.4 months

AEs were defined as any unfavorable and unintended sign, symptom or disease temporally associated with the use of a medicinal product reported from first dose of study drug through 30 days after the last dose of study drug. A TEAE was defined as an AE that started or worsened after first study drug administration and within 30 days of last dose of study drug. An SAE was defined as any untoward medical occurrence that at any dose resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disabili

GroupValue95% CI
Ixazomib31
Number of Participants Receiving Ixazomib With Clinically Significant Changes in Safety Laboratory Values Primary · Up to 58.4 months

Clinical laboratory assessments included hematology, serum chemistry, and urinalysis. Any clinically significant changes in the clinical laboratory value over time based on the investigator's interpretation were reported.

GroupValue95% CI
Ixazomib0
Progression-Free Survival (PFS) Secondary · From the first dose of study drug to every 4 weeks until PD or death from any cause (up to 58.4 months)

PFS was defined as the time from the date of first dose of study drug to the first occurrence of PD as evaluated by the investigator or death from any cause, whichever occurred first. PD was defined as ≥25% increase from lowest value in serum M component or urine M-component; difference between involved and uninvolved free light chain (FLC) levels (absolute increase \>10 milligrams per deciliter \[mg/dL\]); bone marrow plasma cell percent ≥10%; new bone lesions or soft tissue plasmacytomas development or definite increase in existing bone lesions/soft tissue plasmacytomas size; hypercalcaemia

GroupValue95% CI
Ixazomib21.39.20 – NA
Overall Survival (OS) Secondary · From the first dose of study drug to every 12 weeks during follow-up after PD or next line therapy or death whichever occurred later (up to 58.4 months)

OS was measured as the time from the date of first dose of study drug to the date of death.

GroupValue95% CI
IxazomibNANA – NA
Percentage of Participants Who Achieved or Maintained Best Response Before PD or up to Subsequent Therapy Secondary · Up to 58.4 months

Response was assessed according to International Myeloma Working Group (IMWG) criteria. Best response includes partial response (PR), very good partial response (VGPR), and complete response (CR). PR as per IMWG criteria is 50% reduction of serum M-protein and reduction in 24-hour urinary M-protein by ≥90% or to less than (\<)200 mg per 24 hours. VGPR is serum and urine M-protein detectable by immunofixation but not on electrophoresis or ≥90% reduction in serum M-protein plus urine M-protein level \<100 mg per 24 hours. CR is negative immunofixation of serum and urine and disappearance of soft

PR
GroupValue95% CI
Ixazomib3
VGPR
GroupValue95% CI
Ixazomib12
CR
GroupValue95% CI
Ixazomib76
Duration of Complete Response (CR) Secondary · Up to 58.4 months

Duration of CR was defined as the time from the date of first dose of study drug or the date of CR to the date of first documentation of PD. CR was defined as negative immunofixation on the serum and urine; soft tissue plasmacytomas disappearance; \<5% plasma cells (PCs) in bone marrow.

GroupValue95% CI
IxazomibNA12.88 – NA
Time to Progression (TTP) Secondary · Up to 58.4 months

TTP was defined as the time from the date of first dose of study drug to the date of first documentation of PD. PD was defined as ≥25% increase from lowest value in serum M component or urine M-component; difference between involved and uninvolved FLC levels (absolute increase \>10 mg/dL); bone marrow plasma cell percent ≥10%; new bone lesions or soft tissue plasmacytomas development or definite increase in existing bone lesions/soft tissue plasmacytomas size; hypercalcaemia development.

GroupValue95% CI
Ixazomib21.39.20 – NA
Time to Next-Line Therapy (TTNT) Secondary · Up to 58.4 months

TTNT was defined as the time from the date of first dose of study drug to the date of the first dose of next-line of antineoplastic therapy.

GroupValue95% CI
IxazomibNA24.48 – NA
Percentage of Participants With A New Primary Malignancy Secondary · Up to 58.4 months
GroupValue95% CI
Ixazomib0
Change From Baseline in the European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire (QLQ)-C30 as Measured by the Global Health Status (GHS) Secondary · Baseline, Cycle 26 (cycle length=28 days)

The EORTC QLQ-C30 is a questionnaire to assess the overall quality of life of cancer patients. The change from baseline in GHS (EORTC QLQ-C30) score is presented. Participant responses to the question "How would you rate your overall health during the past week?" are scored on a 7-point scale (1=very poor to 7=excellent). Using linear transformation, raw scores are standardized, so that scores range from 0 to 100. A higher score indicates a better overall GHS.

GroupValue95% CI
Ixazomib-5.3± 15.49

Adverse events — posted to ClinicalTrials.gov

Time frame: Up to 58.4 months. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Ixazomib
Serious: 11/35 (31%)
Deaths: 1/35
Placebo
Serious: 0/4 (0%)
Deaths: 0/4

Serious adverse events (12 terms)

ReactionSystemIxazomibPlacebo
COVID-19Infections and infestations
Cardiac failure congestiveCardiac disorders
CataractEye disorders
Chest discomfortGeneral disorders
CholelithiasisHepatobiliary disorders
PneumoniaInfections and infestations
Femur fractureInjury, poisoning and procedural complications
Post procedural haemorrhageInjury, poisoning and procedural complications
PeriarthritisMusculoskeletal and connective tissue disorders
Rotator cuff syndromeMusculoskeletal and connective tissue disorders
Cerebral infarctionNervous system disorders
AngioedemaSkin and subcutaneous tissue disorders
Other adverse events (58 terms — click to expand)

ReactionSystemIxazomibPlacebo
Upper respiratory tract infectionInfections and infestations
DiarrhoeaGastrointestinal disorders
VomitingGastrointestinal disorders
COVID-19Infections and infestations
Platelet count decreasedInvestigations
NauseaGastrointestinal disorders
PyrexiaGeneral disorders
NasopharyngitisInfections and infestations
PneumoniaInfections and infestations
Lymphocyte count decreasedInvestigations
White blood cell count decreasedInvestigations
ArthralgiaMusculoskeletal and connective tissue disorders
AnaemiaBlood and lymphatic system disorders
ToothacheGastrointestinal disorders
BronchitisInfections and infestations
Weight decreasedInvestigations
Decreased appetiteMetabolism and nutrition disorders
DizzinessNervous system disorders
HeadacheNervous system disorders
Dermatitis acneiformSkin and subcutaneous tissue disorders
EczemaSkin and subcutaneous tissue disorders
LeukopeniaBlood and lymphatic system disorders
ThrombocytopeniaBlood and lymphatic system disorders
FatigueGeneral disorders
Peripheral swellingGeneral disorders
Aspartate aminotransferase increasedInvestigations
Neutrophil count decreasedInvestigations
HypercholesterolaemiaMetabolism and nutrition disorders
HyperglycaemiaMetabolism and nutrition disorders
HypocalcaemiaMetabolism and nutrition disorders
Back painMusculoskeletal and connective tissue disorders
Neuropathy peripheralNervous system disorders
CoughRespiratory, thoracic and mediastinal disorders
HypertensionVascular disorders
NeutropeniaBlood and lymphatic system disorders
Abdominal distensionGastrointestinal disorders
Abdominal painGastrointestinal disorders
ConstipationGastrointestinal disorders
GastritisGastrointestinal disorders
Mouth ulcerationGastrointestinal disorders

Most-reported serious reactions: COVID-19, Cardiac failure congestive, Cataract, Chest discomfort, Cholelithiasis, Pneumonia, Femur fracture, Post procedural haemorrhage.

Data from ClinicalTrials.gov NCT03748953 adverse events section.

Sponsor's own description

The purpose of this study is to determine the long-term safety and tolerability of ixazomib maintenance therapy.

Publications & conference data

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

  1. Developments in continuous therapy and maintenance treatment approaches for patients with newly diagnosed multiple myeloma.
    Dimopoulos MA, Jakubowiak AJ, McCarthy PL, Orlowski RZ, et al · · 2020 · cited 87× · PMID 32054831 · DOI 10.1038/s41408-020-0273-x
  2. Minimal Residual Disease in Myeloma: Application for Clinical Care and New Drug Registration.
    Anderson KC, Auclair D, Adam SJ, Agarwal A, et al · · 2021 · cited 44× · PMID 34321279 · DOI 10.1158/1078-0432.ccr-21-1059
  3. Boosting Immunity against Multiple Myeloma.
    Lopes R, Ferreira BV, Caetano J, Caetano J, et al · · 2021 · cited 6× · PMID 33799565 · DOI 10.3390/cancers13061221
  4. Targeting proteostasis for cancer therapy: current advances, challenges, and future perspectives.
    Zhang C, Li J, Tang Q, Li L, et al · · 2025 · cited 4× · PMID 41121138 · DOI 10.1186/s12943-025-02472-x

Verify or expand the search:

Other trials of Ixazomib

Trials testing the same drug.

Other recruiting trials for Multiple Myeloma

Currently open trials in the same condition.

Other Millennium Pharmaceuticals, Inc. trials

Trials by the same sponsor.

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Data sources for this page

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/NCT03748953.

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