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CompletedNCT03748953Updated Mar 21, 2025Results posted

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

A Phase 3 interventional study of Ixazomib in Multiple Myeloma, sponsored by Millennium Pharmaceuticals, Inc.. Completed at 10 sites in China. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2025-03-21.

Sponsored by Millennium Pharmaceuticals, Inc. (part of Takeda) · Phase 3, Interventional, and Treatment

Phase
Phase 3
Study type
Interventional
Enrollment
37
Allocation
Not applicable
Ages
18 Years and older
Sex
All
01

Study summary

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

Read the detailed description

The drug being tested in this study is called ixazomib. Ixazomib is being tested to slow disease progression and improve overall survival in Chinese participants who have newly diagnosed multiple myeloma (NDMM) who have had a major positive response to initial therapy and have not undergone stem cell transplantation (SCT). This study will look at the effect of ixazomib has on the length of time that participants are free of disease progression and their overall survival. After the implementation of Amendment 8, participants who received placebo-matching capsules before unblinding and have not yet experienced disease progression will cross over to receive ixazomib.

The study will enroll approximately 37 patients. Participants will be assigned to a single treatment group

  • Ixazomib

All participants will be asked to take one capsule on Days 1, 8, and 15 of every 28-day cycle, for up to approximately 24 months (equivalent to 26 cycles [if no cycle delays], to the nearest complete cycle) or until documented progressive disease (PD) or intolerable toxicity, whichever occurs first.

This multi-center trial will be conducted in China. The overall time to participate in this study is until a total of approximately up to 60 months. Participants will make multiple visits to the clinic, and every 4 weeks until the next line of therapy begins for a follow-up assessment.

02

Conditions studied

  • Multiple Myeloma

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Keywords

  • Drug Therapy
03

In context

Multiple Myeloma

3,760 studies on the registry are indexed under Multiple Myeloma; 769 are open to participants now.

This study's enrollment of 37 is close to the median of 41 across 3,005 interventional studies indexed under Multiple Myeloma.

Browse Multiple Myeloma studies →

Lead sponsor

Millennium Pharmaceuticals, Inc. is the lead sponsor of 178 studies on the registry; none are open to participants now.

Of its 74 completed or terminated interventional studies of FDA-regulated products, 73 (99%) have results posted.

First submitted after Millennium Pharmaceuticals, Inc. became part of Takeda.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  1. Adult male or female participants aged 18 years or older with a confirmed diagnosis of symptomatic NDMM according to standard criteria.
  2. Has completed 6 to 12 months (±2 weeks) of initial therapy, during which the participant was treated to best response, defined as the best response maintained for 2 cycles after the M-protein nadir is reached.
  3. Has documented major response (partial response [PR], very good partial response [VGPR], complete response [CR]) according to the international myeloma working group (IMWG) uniform response criteria, version 2011, after this initial therapy.
  4. Female participants who:

    Are for at least 1 year before the screening visit, OR Are surgically sterile, OR If they are of , agree to practice 2 effective methods of , at the same time, from the time of signing the through 90 days after the last dose of study drug, or Agree to practice true abstinence, when this is in line with the preferred and usual lifestyle of the participant. (Periodic abstinence [e.g., calendar, ovulation,symptothermal, postovulation methods] and withdrawal are not acceptable methods of contraception.)

    Male participants, even if surgically sterilized (i.e., status postvasectomy), who:

    Agree to practice effective barrier contraception during the entire study Treatment period and through 90 days after the last dose of study drug, or Agree to practice true abstinence, when this is in line with the preferred and usual lifestyle of the participant. (Periodic abstinence [e.g., calendar, ovulation, symptothermal, postovulation methods for the female partner] and withdrawal are not acceptable methods of contraception.)

  5. Voluntary written consent must be given before performance of any study-related procedure not part of standard medical care, with the understanding that consent may be withdrawn by the participant at any time without prejudice to future medical care.
  6. Has availability of complete documentation for:

    1. Details of initial disease state, initial therapy, and response
    2. Cytogenetic assessment at diagnosis (cytogenetic assessment performed after diagnosis must be approved by a Takeda project clinician or designee)
    3. International Staging System (ISS) staging at diagnosis (requiring beta 2-microglobulin and serum albumin results).
  7. Has (ECOG) Performance Status of 0 to 2.
  8. Suitable venous access for the study-required blood sampling and consent for the specific amounts that will be taken.
  9. Participant is willing and able to adhere to the study visit schedule and other protocol requirements including blood sampling and bone marrow aspiration.
  10. Participants must meet the following clinical laboratory criteria at study entry:
  1. (ANC) ≥1,000/mm\^3 without growth factor support and count ≥ 75,000/mm\^3. Platelet transfusions to help participants meet eligibility criteria are not allowed within 3 days before enrollment.
  1. Total ≤1.5*the (ULN). 3. and aspartate aminotransferase ≤3*ULN. 4. Calculated ≥30 mL/min (using the Cockroft-Gault equation).

Exclusion criteria

Exclusion Criteria:

  1. Has multiple myeloma that after, or was not responsive to, initial therapy.
  2. Had prior stem-cell transplantation (SCT).
  3. Has radiotherapy within 14 days before enrollment.
  4. Had been diagnosed or treated for another within 5 years before enrollment or previously diagnosed with another malignancy with evidence of residual disease. Participants with nonmelanoma skin cancer or carcinoma in situ of any type are not excluded if they have undergone complete resection.
  5. Female participants who are lactating and breastfeeding or have a positive serum pregnancy test during the Screening period.
  6. Has major surgery within 14 days before enrollment.
  7. Has central nervous system involvement.
  8. Infection requiring intravenous (IV) antibiotic therapy or other serious infection within 14 days before enrollment.
  9. Has diagnosis of Waldenstrom's macroglobulinemia, polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy, and skin changes (POEMS) syndrome, plasma cell leukemia, primary amyloidosis, myelodysplastic syndrome, or myeloproliferative syndrome.
  10. Evidence of current uncontrolled cardiovascular conditions, including uncontrolled hypertension, uncontrolled cardiac arrhythmias, uncontrolled congestive heart failure, unstable angina, or myocardial infarction within the past 6 months.
  11. Systemic treatment with strong cytochrome P450 () inducers (rifampin, rifapentine, rifabutin, carbamazepine, phenytoin, phenobarbital) or use of St. John's wort within 14 days before enrollment.
  12. Ongoing or active infection, known positive, active or C infection.
  13. Has comorbid systemic illnesses or other severe concurrent disease that, in the judgment of the investigator, would make the participant inappropriate for entry into this study or interfere significantly with the proper assessment of safety and toxicity of the prescribed regimens (e.g., peripheral neuropathy (PN) that is Grade 1 with pain or Grade 2 or higher of any cause).
  14. Psychiatric illness/social situation that would limit compliance with study requirements.
  15. Known allergy to any of the study medications, their analogues, or in the various formulations of any agent.
  16. Inability to swallow oral medication, inability or unwillingness to comply with the drug administration requirements, or gastrointestinal (GI) procedure that could interfere with the oral absorption or tolerance of treatment.
  17. Treatment with any products within 30 days before enrollment.
Terms in these criteria, in plain language(18)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Postmenopausal

Periods have stopped for good: usually no period for 12 months in a row without another medical reason, sometimes confirmed by a blood test (FSH).

Childbearing potential

Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).

Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.

Contraception

Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.

Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.

Informed consent

Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.

You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.

ECOG performance status

A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.

0
Fully active, no restrictions.
1
Limited in strenuous activity, but walking and able to do light work, such as housework or office work.
2
Walking and able to look after yourself, but unable to work; up and about more than half of waking hours.
3
Able to look after yourself only in part; in bed or a chair more than half of waking hours.
4
Unable to look after yourself at all; in bed or a chair all day.

Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.

Absolute neutrophil count (ANC)

The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.

It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.

Platelets

Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.

Bilirubin

A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.

Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.

ULN (upper limit of normal)

The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.

“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.

ALT and AST (liver enzymes)

Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.

Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.

Creatinine clearance (CrCl)

How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.

Relapsed and refractory

Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.

Cancer (malignancy)

Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.

Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.

CYP3A (CYP3A4) inhibitors and inducers

CYP3A is a group of enzymes, mainly CYP3A4, in the liver and gut that break down many medicines. Some medicines, supplements and foods (grapefruit, St John’s wort) slow them down or speed them up, which changes how much of a study drug stays in the body.

Studies list them so they can be avoided or switched; bring a list of everything you take, including supplements.

HIV

The human immunodeficiency virus, which weakens the immune system.

Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.

Hepatitis B tests

Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.

Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.

Excipients

The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.

Investigational drug or device

A drug or device being studied in a clinical trial.

It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.

05

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
37 participants (actual)

Study arms

  • Experimental
    Ixazomib

    Participants received ixazomib 3 milligrams (mg), capsules, orally, once on Days 1, 8, and 15 for Cycles 1 through 4, during which if the participants tolerated the initial dose, the dose was escalated to ixazomib 4 mg, capsules, orally, once on Days 1, 8, and 15 for Cycles 5 through 26, or until documented PD or intolerable toxicity, whichever occurred first (cycle length=28 days).

    Drug: Ixazomib

Interventions

06

What researchers measure

Primary outcomes

  1. Number of Participants Categorized According to Performance Status (PS) Based on Eastern Cooperative Oncology Group (ECOG) PS

    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. Only those categories with non-zero values were reported.

    Time frame: Month 25

  2. Percentage of Participants Receiving Ixazomib With Treatment-emergent Adverse Events (TEAEs)

    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.

    Time frame: Up to 58.4 months

  3. Percentage of Participants Receiving Ixazomib With Treatment-emergent Serious Adverse Events (SAEs)

    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 disability or incapacity, was a congenital abnormality or birth defect, or is an important medical event. Percentages are rounded off to the nearest whole number.

    Time frame: Up to 58.4 months

  4. Number of Participants Receiving Ixazomib With Clinically Significant Changes in Safety Laboratory Values

    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.

    Time frame: Up to 58.4 months

Secondary outcomes

  1. Progression-Free Survival (PFS)

    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 development.

    Time frame: From the first dose of study drug to every 4 weeks until PD or death from any cause (up to 58.4 months)

  2. Overall Survival (OS)

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

    Time frame: 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)

  3. Percentage of Participants Who Achieved or Maintained Best Response Before PD or up to Subsequent Therapy

    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 tissue plasmacytomas and \<5% plasma cells in bone marrow. Percentages are rounded off to the nearest whole number.

    Time frame: Up to 58.4 months

  4. Duration of Complete Response (CR)

    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.

    Time frame: Up to 58.4 months

  5. Time to Progression (TTP)

    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.

    Time frame: Up to 58.4 months

  6. Time to Next-Line Therapy (TTNT)

    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.

    Time frame: Up to 58.4 months

  7. Percentage of Participants With A New Primary Malignancy

    Time frame: Up to 58.4 months

  8. 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)

    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.

    Time frame: Baseline, Cycle 26 (cycle length=28 days)

  9. Correlation Between Frailty Status and PFS

    Participant's frailty status is classified as fit, unfit, or frail on the basis of 4 components: age, the Charlson comorbidity scoring system without age weighting, the Katz index of independence in activities of daily living, and the Lawton instrumental activities of daily living scale. The sum of the 4 frailty scores equals the total frailty score. A total frailty score of 0 corresponds to a frailty status of fit; a total score of 1, to unfit; and a total score of 2 or more, to frail. PFS is defined as the time from the date of first dose of study drug to the first occurrence of PD as evaluated by the investigator.

    Time frame: Up to 58.4 months

  10. Correlation Between Frailty Status and OS

    Participant's frailty status is classified as fit, unfit, or frail on the basis of 4 components: age, the Charlson comorbidity scoring system without age weighting, the Katz index of independence in activities of daily living, and the Lawton instrumental activities of daily living scale. The sum of the 4 frailty scores equals the total frailty score. A total frailty score of 0 corresponds to a frailty status of fit; a total score of 1, to unfit; and a total score of 2 or more, to frail. OS was measured as the time from the date of first dose of study drug to the date of death.

    Time frame: Up to 58.4 months

  11. Plasma Concentration of Ixazomib

    Plasma concentrations of the complete hydrolysis product of ixazomib citrate (ixazomib) were measured using a validated liquid chromatography-tandem mass spectrometry (LC/MS/MS) assay.

    Time frame: Cycle 1 (1 and 4 hours post-dose Day 1, Days 8 and 15 pre-dose); Cycle 2 and 5 (Days 1 and 8 pre-dose) and Cycles 3, 4, 6 to 10 (Day 1 pre-dose) (cycle length=28 days)

  12. Time to Resolution of Peripheral Neuropathy (PN) Events

    PN is defined as the event in the high-level term of peripheral neuropathies not elsewhere classified (NEC) according to the medical dictionary for regulatory activities (MedDRA). A PN event was considered as resolved if its final outcome was resolved with no subsequent PN event of the same preferred term occurring on the resolution date or the day before and after. Time to resolution was defined as the time from the initial onset date (inclusive) to the resolution date for resolved events.

    Time frame: Up to 58.4 months

  13. Time to Improvement of PN Events

    PN is defined as the event in the high-level term of peripheral neuropathies NEC according to the MedDRA. A PN event is considered to be improved if the event improves from the maximum grade; that is, all the grades recorded after the maximum grade are less than the maximum grade. Time to improvement is defined as the time from the initial onset date (inclusive) of the maximum grade to the first onset date that the toxicity grade is below the maximum grade with no higher grade thereafter, or the resolution date, whichever occurs first.

    Time frame: Up to 58.4 months

07

Results

Posted Mar 21, 2025

Participant flow

A total of 37 participants (31 enrolled in this study and 6 rolled over from study C16021\[NCT02312258\]) with a diagnosis of newly diagnosed multiple myeloma (NDMM) not treated with stem cell transplantation (SCT) took part in the study at investigative sites in China from 24 January 2019 to 06 December 2023.

Treatment Prior to Amendment 08
Participant flow — Treatment Prior to Amendment 08
MilestoneIxazomibPlacebo
Started64
Placebo safety population04
Completed60
Not completed04
Withdrew: Discontinued to switch to ixazomib02
Withdrew: Reason not specified02
Treatment Post Amendment 08
Participant flow — Treatment Post Amendment 08
MilestoneIxazomibPlacebo
Started350
Efficacy population330
Ixazomib safety population350
Completed00
Not completed350
Withdrew: Withdrawal by subject180
Withdrew: Reason not specified160
Withdrew: Lost to follow-up10

Outcome measures

PrimaryNumber of Participants Categorized According to Performance Status (PS) Based on Eastern Cooperative Oncology Group (ECOG) PS

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. Only those categories with non-zero values were reported.

Time frame:
Month 25
Reported as:
Count of participants · Participants
Number of Participants Categorized According to Performance Status (PS) Based on Eastern Cooperative Oncology Group (ECOG) PS
ParticipantsIxazomib
ECOG Score 034
ECOG Score 11
PrimaryPercentage of Participants Receiving Ixazomib With Treatment-emergent Adverse Events (TEAEs)

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.

Time frame:
Up to 58.4 months
Reported as:
Number · percentage of participants
Percentage of Participants Receiving Ixazomib With Treatment-emergent Adverse Events (TEAEs)
percentage of participantsIxazomib
Percentage of Participants Receiving Ixazomib With Treatment-emergent Adverse Events (TEAEs)97
PrimaryPercentage of Participants Receiving Ixazomib With Treatment-emergent Serious Adverse Events (SAEs)

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 disability or incapacity, was a congenital abnormality or birth defect, or is an important medical event. Percentages are rounded off to the nearest whole number.

Time frame:
Up to 58.4 months
Reported as:
Number · percentage of participants
Percentage of Participants Receiving Ixazomib With Treatment-emergent Serious Adverse Events (SAEs)
percentage of participantsIxazomib
Percentage of Participants Receiving Ixazomib With Treatment-emergent Serious Adverse Events (SAEs)31
PrimaryNumber of Participants Receiving Ixazomib With Clinically Significant Changes in Safety Laboratory Values

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.

Time frame:
Up to 58.4 months
Reported as:
Count of participants · Participants
Number of Participants Receiving Ixazomib With Clinically Significant Changes in Safety Laboratory Values
ParticipantsIxazomib
Number of Participants Receiving Ixazomib With Clinically Significant Changes in Safety Laboratory Values0
SecondaryProgression-Free Survival (PFS)

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 development.

Time frame:
From the first dose of study drug to every 4 weeks until PD or death from any cause (up to 58.4 months)
Reported as:
Median · months
Progression-Free Survival (PFS)
monthsIxazomib
Progression-Free Survival (PFS)21.3 (9.20 to NA)
SecondaryOverall Survival (OS)

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

Time frame:
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)
Reported as:
Median · months
Overall Survival (OS)
monthsIxazomib
Overall Survival (OS)NA (NA to NA)
SecondaryPercentage of Participants Who Achieved or Maintained Best Response Before PD or up to Subsequent Therapy

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 tissue plasmacytomas and \<5% plasma cells in bone marrow. Percentages are rounded off to the nearest whole number.

Time frame:
Up to 58.4 months
Reported as:
Number · percentage of participants
Percentage of Participants Who Achieved or Maintained Best Response Before PD or up to Subsequent Therapy
percentage of participantsIxazomib
PR3
VGPR12
CR76
SecondaryDuration of Complete Response (CR)

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.

Time frame:
Up to 58.4 months
Reported as:
Median · months
Duration of Complete Response (CR)
monthsIxazomib
Duration of Complete Response (CR)NA (12.88 to NA)
SecondaryTime to Progression (TTP)

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.

Time frame:
Up to 58.4 months
Reported as:
Median · months
Time to Progression (TTP)
monthsIxazomib
Time to Progression (TTP)21.3 (9.20 to NA)
SecondaryTime to Next-Line Therapy (TTNT)

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.

Time frame:
Up to 58.4 months
Reported as:
Median · months
Time to Next-Line Therapy (TTNT)
monthsIxazomib
Time to Next-Line Therapy (TTNT)NA (24.48 to NA)
SecondaryPercentage of Participants With A New Primary Malignancy
Time frame:
Up to 58.4 months
Reported as:
Number · percentage of participants
Percentage of Participants With A New Primary Malignancy
percentage of participantsIxazomib
Percentage of Participants With A New Primary Malignancy0
SecondaryChange 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)

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.

Time frame:
Baseline, Cycle 26 (cycle length=28 days)
Reported as:
Mean · score on a scale
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)
score on a scaleIxazomib
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)-5.3 ± 15.49
SecondaryCorrelation Between Frailty Status and PFS

Participant's frailty status is classified as fit, unfit, or frail on the basis of 4 components: age, the Charlson comorbidity scoring system without age weighting, the Katz index of independence in activities of daily living, and the Lawton instrumental activities of daily living scale. The sum of the 4 frailty scores equals the total frailty score. A total frailty score of 0 corresponds to a frailty status of fit; a total score of 1, to unfit; and a total score of 2 or more, to frail. PFS is defined as the time from the date of first dose of study drug to the first occurrence of PD as evaluated by the investigator.

Time frame:
Up to 58.4 months
Reported as:
Median · months
Correlation Between Frailty Status and PFS
monthsIxazomib
Frailty Status: FitNA (12.68 to NA)
Frailty Status: Unfit20.9 (0.89 to NA)
Frailty Status: Frail6.5 (6.18 to NA)
SecondaryCorrelation Between Frailty Status and OS

Participant's frailty status is classified as fit, unfit, or frail on the basis of 4 components: age, the Charlson comorbidity scoring system without age weighting, the Katz index of independence in activities of daily living, and the Lawton instrumental activities of daily living scale. The sum of the 4 frailty scores equals the total frailty score. A total frailty score of 0 corresponds to a frailty status of fit; a total score of 1, to unfit; and a total score of 2 or more, to frail. OS was measured as the time from the date of first dose of study drug to the date of death.

Time frame:
Up to 58.4 months
Reported as:
Median · months
Correlation Between Frailty Status and OS
monthsIxazomib
Frailty Status: FitNA (NA to NA)
Frailty Status: UnfitNA (NA to NA)
Frailty Status: FrailNA (NA to NA)
SecondaryPlasma Concentration of Ixazomib

Plasma concentrations of the complete hydrolysis product of ixazomib citrate (ixazomib) were measured using a validated liquid chromatography-tandem mass spectrometry (LC/MS/MS) assay.

Time frame:
Cycle 1 (1 and 4 hours post-dose Day 1, Days 8 and 15 pre-dose); Cycle 2 and 5 (Days 1 and 8 pre-dose) and Cycles 3, 4, 6 to 10 (Day 1 pre-dose) (cycle length=28 days)
Reported as:
Geometric mean · nanograms per milliliter (ng/mL)
Plasma Concentration of Ixazomib
nanograms per milliliter (ng/mL)Ixazomib
Cycle 1 Day 1: 1 Hour Post-dose15.3 ± 181
Cycle 1 Day 1: 4 Hours Post-dose16.6 ± 94.4
Cycle 1 Day 8: Pre-dose2.25 ± 51.5
Cycle 1 Day 15: Pre-dose4 ± 53.6
Cycle 2 Day 1: Pre-dose2.92 ± 46.8
Cycle 2 Day 8: Pre-dose4.37 ± 42.5
Cycle 3 Day 1: Pre-dose3.15 ± 41.9
Cycle 4 Day 1: Pre-dose3.16 ± 59.4
Cycle 5 Day 1: Pre-dose3.25 ± 42.1
Cycle 5 Day 8: Pre-dose5.29 ± 36.9
Cycle 6 Day 1: Pre-dose3.58 ± 41.3
Cycle 7 Day 1: Pre-dose4.04 ± 48.5
Cycle 8 Day 1: Pre-dose3.28 ± 64.7
Cycle 9 Day 1: Pre-dose3.7 ± 31.3
Cycle 10 Day 1: Pre-dose2.99 ± 37.6
SecondaryTime to Resolution of Peripheral Neuropathy (PN) Events

PN is defined as the event in the high-level term of peripheral neuropathies not elsewhere classified (NEC) according to the medical dictionary for regulatory activities (MedDRA). A PN event was considered as resolved if its final outcome was resolved with no subsequent PN event of the same preferred term occurring on the resolution date or the day before and after. Time to resolution was defined as the time from the initial onset date (inclusive) to the resolution date for resolved events.

Time frame:
Up to 58.4 months
Reported as:
Median · days
Time to Resolution of Peripheral Neuropathy (PN) Events
daysIxazomib
Time to Resolution of Peripheral Neuropathy (PN) Events24.0 (7.0 to NA)
SecondaryTime to Improvement of PN Events

PN is defined as the event in the high-level term of peripheral neuropathies NEC according to the MedDRA. A PN event is considered to be improved if the event improves from the maximum grade; that is, all the grades recorded after the maximum grade are less than the maximum grade. Time to improvement is defined as the time from the initial onset date (inclusive) of the maximum grade to the first onset date that the toxicity grade is below the maximum grade with no higher grade thereafter, or the resolution date, whichever occurs first.

Time frame:
Up to 58.4 months
Reported as:
Median · days
Time to Improvement of PN Events
daysIxazomib
Time to Improvement of PN Events8.0 (7.0 to NA)

Adverse events

Collected over Up to 58.4 months. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Ixazomib1/35 (2.9%)11/35 (31.4%)33/35 (94.3%)
Placebo0/4 (0%)0/4 (0%)4/4 (100%)
Most frequent serious events
Showing 10 of 12
Most frequent serious events
EventIxazomibPlacebo
COVID-19Infections and infestations2/350/4
Cardiac failure congestiveCardiac disorders1/350/4
CataractEye disorders1/350/4
Chest discomfortGeneral disorders1/350/4
CholelithiasisHepatobiliary disorders1/350/4
PneumoniaInfections and infestations1/350/4
Femur fractureInjury, poisoning and procedural complications1/350/4
Post procedural haemorrhageInjury, poisoning and procedural complications1/350/4
PeriarthritisMusculoskeletal and connective tissue disorders1/350/4
Rotator cuff syndromeMusculoskeletal and connective tissue disorders1/350/4
Most frequent other events
Showing 10 of 58
Most frequent other events
EventIxazomibPlacebo
ArthralgiaMusculoskeletal and connective tissue disorders5/352/4
PruritusSkin and subcutaneous tissue disorders1/352/4
Upper respiratory tract infectionInfections and infestations14/351/4
DiarrhoeaGastrointestinal disorders12/351/4
VomitingGastrointestinal disorders9/350/4
Breath odourGastrointestinal disorders0/351/4
GastritisGastrointestinal disorders2/351/4
Tongue ulcerationGastrointestinal disorders0/351/4
NasopharyngitisInfections and infestations5/351/4
Weight increasedInvestigations2/351/4

Baseline characteristics

Efficacy Population included all participants who received ixazomib treatment for the duration of their study participation. As prespecified in the statistical analysis plan (SAP), the Efficacy Population contained only one arm i.e., Ixazomib (data for the participants who received placebo before Protocol Amendment 8 was not collected per statistical analysis plan), thus data is presented accordingly.

Age, Continuous
Age, Continuous(years)Ixazomib
Mean65.80 ± 7.551
Sex: Female, Male
Sex: Female, Male(Participants)Ixazomib
Female12
Male21
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Ixazomib
Hispanic or Latino0
Not Hispanic or Latino33
Unknown or Not Reported0
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Ixazomib
American Indian or Alaska Native0
Asian33
Native Hawaiian or Other Pacific Islander0
Black or African American0
White0
More than one race0
Unknown or Not Reported0
08

Study locations

10 sites
  • Beijing Chaoyang Hospital Capital Medical University
    Beijing, Beijing 100020, China
  • Peking University Third Hospital
    Beijing, Beijing 100191, China
  • Peking Union Medical College Hospital
    Beijing, Beijing 100730, China
  • Tongji Hospital Tongji Medical College Huazhong University of Science and Technology
    Wuhan, Hubei 430030, China
  • Jiangsu Province Hospital (the First Affiliated Hospital With Nanjing Medical University)
    Nanjing, Jiangsu 210029, China
  • Shengjing Hospital of China Medical University
    Shenyang, Liaoning 110004, China
  • Renji Hospital Shanghai Jiaotong University School of Medicine
    Shanghai, Shanghai 200001, China
  • Shanghai Chang Zheng Hospital
    Shanghai, Shanghai 200003, China
  • Ruijin Hospital Shanghai Jiaotong University School of Medicine
    Shanghai, Shanghai 200025, China
  • 1st Affiliated Hospital of Zhejiang University
    Hangzhou, Zhejiang 310003, China
09

References and documents

Study documents

PDFs hosted by the registry, ClinicalTrials.gov.

Individual participant data

Plan to share: Yes — Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.

Supporting information: Study protocol, Statistical analysis plan, Informed consent form, Clinical study report

Access criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.

Data sharing details ↗ · vivli.org

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Mar 21, 2025, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT03748953
Responsible party
Sponsor
First posted
Nov 21, 2018
Start date
Jan 24, 2019
Primary completion
Dec 6, 2023
Completion
Dec 6, 2023
Results posted
Mar 21, 2025
Last update
Mar 21, 2025

Study contacts

Medical Director
study director · Takeda

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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This study is completed, as verified in Mar 2025. You cannot join it, but the record below documents what was studied.

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Studies of the drug tested here: Ixazomib

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