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PlasmacytomaPhase 1NCT03031730

Testing the Addition of KRT-232 (AMG 232) to Usual Chemotherapy for Relapsed Multiple Myeloma

A Phase 1 study testing Biospecimen Collection, Bone Marrow Aspiration and Biopsy and Carfilzomib in people with Plasmacytoma and Recurrent Multiple Myeloma, run by National Cancer Institute (NCI) at 7 sites in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

This phase I trial studies the side effects and best dose of MDM2 Inhibitor KRT-232 when given together with carfilzomib, lenalidomide, and dexamethasone in treating patient with multiple myeloma that has come back (relapsed) or has not responded to previous treatment (refractory). Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Biospecimen Collection, Bone Marrow Aspiration and Biopsy and Carfilzomib
Could I receive a placebo?
No, everyone receives an active treatment
How many people
35 participants
Where
7 sitesUnited States
Study dates
14 Jun 2018to 24 Jul 2024
Phase
Phase 1
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Subjects must have histologically confirmed diagnosis of multiple myeloma
  • Subjects must have measurable disease, as defined by at least one of the following:
  • Serum monoclonal protein M-protein level >= 0.5 g/dL
  • Urinary M-protein excretion of >= 200 mg over a 24-hour period
  • Involved free light chain level >= 10 mg/dL, along with an abnormal free light chain ratio
  • Subjects must have disease that has relapsed and/or refractory after their most recent therapy, with progressive disease (PD) being defined as an increase of 25% from the lowest response value in any one or more of the following:
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  • Serum M-component protein (the absolute increase must be >= 0.5 g/dL) and/or
  • Urine M-component protein (the absolute increase must be >= 200 mg/24 hours) and/or
  • Only in subjects without a measurable serum and urine M protein level: the difference between involved and uninvolved free light chain (FLC) levels (absolute increase) must be > 10 mg/dL
  • Definite development of new bone lesions or soft tissue plasmacytomas or definite increase in the size of existing bone lesions or soft tissue plasmacytomas
  • Development of hypercalcemia (corrected serum calcium > 11.5 mg/dL) that can be attributed solely to the plasma cell proliferative disorder
  • Subjects with one to three lines of therapy for their disease with a line of therapy defined as one or more cycles of a planned treatment program; using this definition, treatment with induction therapy, followed by high dose chemotherapy and autologous stem cell transplantation, and finally by maintenance therapy, would constitute one line, provided that multiple myeloma did not meet criteria for progression at any time during this period
  • Subjects must have completed their most recent drug therapy directed at multiple myeloma in the following timeframes:
  • Antitumor therapy (chemotherapy, antibody therapy, molecular targeted therapy, retinoid therapy, hormonal therapy, or investigational agent) at least 21 days prior to cycle 1 day 1 (C1D1) KRT-232 (AMG 232) + KRd
  • Corticosteroids at least 3 weeks prior to starting KRT-232 (AMG 232) + KRd, except for a dose equivalent to dexamethasone of =< 4 mg/day
  • Autologous stem cell transplantation at least 12 weeks prior to starting KRT-232 (AMG 232) + KRd
  • Allogeneic stem cell transplantation at least 24 weeks prior to starting KRT-232 (AMG 232) + KRd, and these subjects must also NOT have moderate to severe active acute or chronic graft versus host disease (GVHD)
  • Subjects must be >= 18 years old. Because no dosing or adverse event data are currently available on the use of KRT-232 (AMG 232) in combination with KRd, subjects < 18 years of age are excluded from this study
  • Subjects must have Eastern Cooperative Oncology Group (ECOG) performance status =< 2 (Karnofsky >= 60%)
  • Absolute neutrophil count (ANC) >= 1,000/mcL without growth factors within 2 week of initiation of treatment
  • Platelets >= 50,000 cells/mm^3 if marrow plasmacytosis < 50% OR platelet count >= 30,000 cells/mm^3 if marrow plasmacytosis >= 50%
  • Hemoglobin >= 8 g/dL within 2 weeks of the initiation of treatment
  • Total bilirubin < 1.5 x institutional upper limit of normal (ULN) (< 2.0 x ULN for subjects with documented Gilbert's syndrome or < 3.0 x ULN for subjects for whom the indirect bilirubin level suggests an extrahepatic source of elevation)
  • Aspartate aminotransferase (AST) (serum glutamic-oxaloacetic transaminase [SGOT])/alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase [SGPT]) =< 2.5 x ULN
  • Alkaline phosphatase < 2.0 x ULN (if liver or bone disease are present, < 3.0 x ULN)
  • Creatinine clearance (estimated glomerular filtration rate [eGFR]) >= 50 mL/min/1.73 m^2
  • Prothrombin time (PT) or partial thromboplastin time (PTT) < 1.5 x upper limit of normal (ULN), OR international normalized ratio (INR) < 1.5
  • Subjects who have received radiation therapy targeting > 10% of the bone marrow space must have completed this at least 2 weeks prior to starting therapy with KRT-232 (AMG 232) + KRd
  • Subjects must be able and willing to provide bone marrow biopsies/aspirates and buccal swab as requested by the protocol
  • Subjects must be willing to undergo myeloma genotyping for TP53 mutation, insertion, or deletion at screening
  • Subjects must have an estimated life expectancy of at least 3 months
  • The effects of KRT-232 (AMG 232) on the developing human fetus are unknown; for this reason and because lenalidomide is known to be teratogenic, women of child-bearing potential must commit to either abstaining continuously from heterosexual sexual intercourse or agree to use 2 forms of adequate contraception or continuously abstain from the time of informed consent for the duration of study participation through 5 weeks (women) after receiving the last dose of KRT-232 (AMG 232), lenalidomide, or carfilzomib; should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately; men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of study participation, and 3 months after completion of KRT-232 (AMG 232) administration; this includes one highly effective form of contraception (e.g. intrauterine device [IUD], hormonal [birth control pills, injections, hormonal patches, vaginal rings or implants] or partner's vasectomy) and one additional effective contraceptive method (e.g. male latex or synthetic condom, diaphragm, or cervical cap)
  • Subjects must be able to swallow medication
  • Ability to understand and the willingness to sign a written informed consent document

Who cannot join

  • Subjects with myeloma that is relapsed and/or refractory to KRd when used in combination defined as progression of disease while on therapy or within 60 days of completing therapy
  • Subjects must show evidence of wild-type (WT) p53 status on somatic tissue specimens as assessed by deoxyribonucleic acid (DNA) sequencing; note that since patients with relapsed myeloma have a rapidly proliferating disease, patient can be enrolled and begin treatment prior to obtaining the results of this test; patients who are discovered to have a TP53 mutation will be removed from study after cycle one and can continue on carfilzomib, lenalidomide, and dexamethasone (KRd); all enrolled patients will be followed for toxicity
  • Subjects who have not recovered from toxicities from prior anti-tumor therapy, defined as not having resolved to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0 grade 0 or 1, or to levels dictated in the eligibility criteria with the exception of alopecia (grade 2 or 3 toxicities from prior antitumor therapy that are considered irreversible [defined as having been present and stable for > 6 months], such as grade 2 chemotherapy-induced peripheral neuropathy, may be allowed if they are not otherwise described in the exclusion criteria AND there is agreement to allow by both the investigator and sponsor)
  • Subjects who are receiving any other investigational agents
  • Subjects who have undergone major surgery within 28 days of study day 1; vertebroplasty and/or kyphoplasty, which must have been performed at least 1 week prior to starting KRT-232 (AMG 232) + KRd
  • Subjects with known central nervous system involvement of myeloma should be excluded from this clinical trial because of their poor prognosis and because they often develop progressive neurologic dysfunction that would confound the evaluation of neurologic and other adverse events
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  • Subjects with history of allergic reactions attributed to compounds of similar chemical or biologic composition to KRT-232 (AMG 232) or carfilzomib, lenalidomide, or dexamethasone
  • Subjects' medication list such as herbal medicines (e.g., St. John's wort), vitamins, and supplements will be reviewed before starting first dose of KRT-232 (AMG 232) and at each clinic visit; any potential drug interactions will be brought and discussed with the principal investigator; use of any known CYP3A4 substrates with narrow therapeutic window (such as alfentanil, astemizole, cisapride, dihydroergotamine, pimozide, quinidine, sirolimus, or terfanide) within the 14 days prior to receiving the first dose of KRT-232 (AMG 232) is not permitted; other medications (such as fentanyl and oxycodone) may be allowed per investigator's assessment/evaluation
  • Treatment with medications known to cause QTc interval prolongation within 7 days of study day 1 is not permitted unless approved by the sponsor; use of ondansetron is permitted for treatment of nausea and vomiting
  • Current use of warfarin, factor Xa inhibitors and direct thrombin inhibitors; Note: low molecular weight heparin and prophylactic low dose warfarin are permitted; PT/PTT must meet the inclusion criteria; subjects taking warfarin must have their INR followed closely
  • Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements
  • Subjects with myocardial infarction within 6 months of study day 1, symptomatic congestive heart failure (New York Heart Association [NYHA] class III and higher), unstable angina, or cardiac arrhythmia requiring medication are excluded
  • Subjects with gastrointestinal (GI) tract disease causing the inability to take oral medication, malabsorption syndrome, requirement for intravenous alimentation, prior surgical procedures affecting absorption, uncontrolled inflammatory GI disease (e.g., Crohn's disease, ulcerative colitis)
  • Subjects with history of bleeding diathesis
  • Subjects with active infection requiring IV antibiotics within 2 weeks of study enrollment (day 1) are excluded
  • Positive hepatitis B surface antigen (HepBsAg) (indicative of chronic hepatitis B), positive hepatitis total core antibody with negative HBsAG (suggestive of occult hepatitis B), or detectable hepatitis C virus RNA by a polymerase-chain reaction (PCR) assay (indicative of active hepatitis C - screening is generally done by hepatitis C antibody [HepCAb], followed by hepatitis C virus RNA by PCR if HepCAb is positive); subjects with hepatitis B virus suppressed on therapy, and previously treated/eradicated hepatitis C virus are eligible for study
  • Human immunodeficiency virus (HIV)-positive subjects positive for human immunodeficiency virus (HIV) are NOT excluded from this study, but HIV-positive subjects must have:
  • A stable regimen of highly active anti-retroviral therapy (HAART)
  • No requirement for concurrent antibiotics or antifungal agents for the prevention of opportunistic infections
  • CD4 count above 250 cells/mcL and an undetectable HIV viral load on standard PCR-based test
  • Pregnant women are excluded from this study because KRT-232 (AMG 232) is an agent with the potential for teratogenic or abortifacient effects; because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with KRT-232 (AMG 232), breastfeeding should be discontinued if the mother is treated with KRT-232 (AMG 232); these potential risks may also apply to other agents used in this study
  • Women who are lactating/breast feeding or who plan to breastfeed while on study through 1 week after receiving the last dose of study drug
  • Subjects with prior treatment with an MDM2 inhibitor

Trial locations and contacts

7 sites in 1 country.

7 sites in 1 country

United States7 sites
  • UCHealth University of Colorado Hospital

    Aurora, Colorado, 80045

  • University of Texas at Austin

    Austin, Texas, 78712

  • M D Anderson Cancer Center

    Houston, Texas, 77030

  • NYP/Weill Cornell Medical Center

    New York, New York, 10065

  • University of California Davis Comprehensive Cancer Center

    Sacramento, California, 95817

  • Huntsman Cancer Institute/University of Utah

    Salt Lake City, Utah, 84112

  • University of Texas Health Science Center at San Antonio

    San Antonio, Texas, 78229

Other trials in United States

About this study

Official title: A Phase 1 Dose-Escalation and Exploratory Dose Expansion Study of KRT-232 (AMG 232) in Combination With Carfilzomib, Lenalidomide, and Dexamethasone in Relapsed and/or Refractory Myeloma

This phase I trial studies the side effects and best dose of MDM2 Inhibitor KRT-232 when given together with carfilzomib, lenalidomide, and dexamethasone in treating patient with multiple myeloma that has come back (relapsed) or has not responded to previous treatment (refractory). KRT-232 (AMG 232) may stop the growth of cancer cells by blocking a protein called MDM2 that is needed for cell growth. Lenalidomide help shrink or slow the growth of multiple myeloma. Drugs used in chemotherapy, such as carfilzomib and dexamethasone, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving MDM2 Inhibitor KRT-232, lenalidomide, carfilzomib, and dexamethasone together may work better in treating patients with multiple myeloma.

Read the detailed description

PRIMARY OBJECTIVES:

  1. Evaluate safety and tolerability of MDM2 Inhibitor KRT-232 (KRT-232 [AMG 232]) in combination with carfilzomib, lenalidomide, and dexamethasone (KRd). (Part A) II. Determine the maximum tolerated dose (MTD) and recommended phase 2 dose (RP2D) of KRT-232 (AMG 232) in combination with carfilzomib, lenalidomide, and dexamethasone (KRd). (Part A) III. Confirm the safety and tolerability of KRT-232 (AMG 232) in combination with carfilzomib, lenalidomide, and dexamethasone (KRd) at MTD/tentative RP2D in a goal of 10 subjects with relapsed and/or refractory myeloma. (Part B)

SECONDARY OBJECTIVES:

  1. Evaluate pharmacodynamic (PD) effects of KRT-232 (AMG 232) through serum MIC-1 levels. (Part A) II. Assess KRT-232 (AMG 232) exposure-response relationships (PD, toxicity, and efficacy). (Part A) III. Evaluate the overall response rate of KRT-232 (AMG 232) in combination with carfilzomib, lenalidomide, and dexamethasone (KRd) in relapsed/refractory myeloma by International Myeloma Working Group (IMWG) criteria. (Part B) IV. Evaluate PD effects of KRT-232 (AMG 232) through serum MIC-1 levels. (Part B) V. Assess KRT-232 (AMG 232) exposure-response relationships (PD, toxicity, and efficacy). (Part B)

EXPLORATORY OBJECTIVES:

  1. To observe and record anti-tumor activity of KRT-232 (AMG 232) in combination with carfilzomib, lenalidomide, and dexamethasone (KRd) in relapsed/refractory myeloma by International Myeloma Working Group (IMWG) criteria. (Part A) II. Evaluate ribonucleic acid (RNA) expression levels of relevant genes in the TP53 pathway that may predict response to therapy using pre- and post-treatment bone marrow biopsies. (Parts A and B)

OUTLINE: This is a dose-escalation study of MDM2 Inhibitor KRT-232.

Patients receive MDM2 Inhibitor KRT-232 orally (PO) once daily (QD) on days 1-7, carfilzomib intravenously (IV) over 10-30 minutes on days 1-2, 8-9, and 15-16 of cycles 1-12 and on days 1-2 and 15-16 of cycles 13-18, lenalidomide PO on days 1-21, and dexamethasone PO or dexamethasone sodium phosphate IV on days 1, 8, 15, and 22. Cycles repeat every 28 days in the absence of disease progression or unexpected toxicity. Patients undergo echocardiography during screening and bone marrow biopsy and aspiration, and blood sample collection throughout the study.

After completion of study treatment, patients are followed up for 30 days.

Being tested
Biospecimen Collection, Bone Marrow Aspiration and Biopsy, Carfilzomib, Dexamethasone, Dexamethasone Sodium Phosphate
Medical subject headings
Plasmacytoma, Multiple Myeloma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Biospecimen CollectionProcedure or surgery

    Also called Biological Sample Collection, Biospecimen Collected, Specimen Collection

    Undergo blood sample collection

  • Bone Marrow Aspiration and BiopsyProcedure or surgery

    Undergo bone marrow aspiration and biopsy

  • CarfilzomibDrug

    Also called Carfilnat, CFZ, Kyprolis, PR 171

    Given IV

  • DexamethasoneDrug

    Also called Aacidexam, Adexone, Aknichthol Dexa, Alba-Dex

    Given PO

  • Dexamethasone Sodium PhosphateDrug

    Also called Cebedex, Corson, Dalalone, Decaject

    Given IV

  • EchocardiographyProcedure or surgery

    Also called EC

    Undergo echocardiography

  • LenalidomideDrug

    Also called CC 5013, CC-5013, CC5013, CDC 501

    Given PO

  • NavtemadlinDrug

    Also called (3R,5R,6S)-5-(3-Chlorophenyl)-6-(4-chlorophenyl)-3-methyl-1-((1S)-2-methyl-1-(((1-methylethyl)sulfonyl)methyl)propyl)-2-oxo-3-piperidineacetic Acid, AMG 232, AMG-232, KRT 232

    Given PO

Study groups in NCT03031730
Study groupRoleWhat they receive
Treatment (AMG 232, carfilzomib, lenalidomide, dexamethasone)ExperimentalPatients receive MDM2 Inhibitor KRT-232 PO QD on days 1-7, carfilzomib IV over 10-30 minutes on days 1-2, 8-9, and 15-16 of cycles 1-12 and on days 1-2 and 15-16 of cycles 13-18, lenalidomide PO on days 1-21, and dexamethasone PO or dexamethasone sodium phosphate IV on days 1, 8, 15, and 22. Cycles repeat every 28 days in the absence of disease progression or unexpected toxicity. Patients undergo echocardiography during screening and bone marrow biopsy and aspiration, and blood sample collection throughout the study.
Receives: Procedure: Biospecimen Collection; Procedure: Bone Marrow Aspiration and Biopsy; Drug: Carfilzomib; Drug: Dexamethasone; Drug: Dexamethasone Sodium Phosphate; Procedure: Echocardiography; Drug: Lenalidomide; Drug: Navtemadlin

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Evaluate Safety and Tolerability of KRT-232 (AMG 232) in Combination With Carfilzomib, Lenalidomdie, and Dexamethasone (KRd)

    Measured: at least 6 months of treatment, an average of 1 year

  2. Determine Maximum Tolerated Dose (MTD) and Recommended Phase 2 Dose (RP2D) of KRT-232 (AMG 232) in Combination With Carfilzomib, Lenalidomdie, and Dexamethasone (KRd)

    Measured: at least 6 months of treatment, an average of 1 year

Secondary outcomes (2)
  1. Evaluate PD Effects of KRT-232 (AMG 232) Through Serum MIC-1 Levels.

    Measured: 30 days after last dose

  2. Assess KRT-232 (AMG 232) Exposure-response Relationships (PD, Toxicity, and Efficacy).

    Measured: 30 days after last dose

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted26 Jan 2017 (estimated)
  2. Study start14 Jun 2018
  3. Primary completion24 Jul 2024
  4. Study completion24 Jul 2024

Why it stopped: Inadequate accrual rate

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
35 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03031730
Sponsor's protocol ID
NCI-2017-00099
Other IDs
NCI-2017-00099, NCI10076, 10076, 10076, UM1CA186688
Status last verified
Jan 2026
First submitted
25 Jan 2017
Last updated
10 Feb 2026
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Biospecimen Collection, Bone Marrow Aspiration and Biopsy and Carfilzomib in people with Plasmacytoma and Recurrent Multiple Myeloma, run by National Cancer Institute (NCI) at 7 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 34 inclusion and 23 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 10 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 7 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 10 Feb 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Evaluate Safety and Tolerability of KRT-232 (AMG 232) in Combination With Carfilzomib, Lenalidomdie, and Dexamethasone (KRd) (time frame: at least 6 months of treatment, an average of 1 year). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Feb 2026.

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