NCT04764942

Brief Summary

This phase I/II trial identifies the best dose and side effects of selinexor, and how well it works when given in combination with pomalidomide and dexamethasone with or without carfilzomib in treating patients with multiple myeloma that has come back (relapsed) and does not respond to treatment with proteasome inhibitors and immunomodulatory drugs (refractory). Selinexor is an oral agent that blocks a protein called Exportin 1 (XPO1 or CRM1) that is abundant in a wide variety of cancers, including multiple myeloma. Carfilzomib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Pomalidomide may stop the growth of blood vessels, stimulate the immune system, and kill cancer cells. Anti-inflammatory drugs, such as dexamethasone may lower the body's immune response and are used with other drugs in the treatment of some types of cancer. The addition of selinexor may allow better control of relapsed refractory multiple myeloma than is possible with pomalidomide and dexamethasone with or without carfilzomib.

Trial Health

75
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
39

participants targeted

Target at P50-P75 for phase_1

Timeline
20mo left

Started May 2021

Longer than P75 for phase_1

Geographic Reach
1 country

1 active site

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress76%
May 2021Dec 2027

First Submitted

Initial submission to the registry

February 17, 2021

Completed
4 days until next milestone

First Posted

Study publicly available on registry

February 21, 2021

Completed
2 months until next milestone

Study Start

First participant enrolled

May 6, 2021

Completed
5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 6, 2026

Completed
1.6 years until next milestone

Study Completion

Last participant's last visit for all outcomes

December 17, 2027

Expected
Last Updated

February 25, 2026

Status Verified

February 1, 2026

Enrollment Period

5 years

First QC Date

February 17, 2021

Last Update Submit

February 23, 2026

Conditions

Outcome Measures

Primary Outcomes (2)

  • Maximum tolerated dose of selinexor in combination with carfilzomib, pomalidomide and dexamethasone (Phase I)

    A standard 3+3 phase I design will be utilized. Three patients will be treated at each dose level and observed for a minimum of four weeks (i.e. one full cycle) before new patients are treated. Doses will not be escalated in any individual patient.

    Up to 30 days after completion of treatment

  • Proportion of patients who achieve a confirmed response with selinexor in combination with pomalidomide and dexamethasone (Phase 2)

    A confirmed response is defined as an stringent complete response (sCR), complete response (CR), very good \[partial response (VGPR), or partial response (PR) noted as the objective status on two consecutive evaluations. The proportion of successes will be estimated by the number of successes divided by the total number of evaluable patients. Ninety-five percent confidence intervals for the true success proportion will be calculated according to the approach of Duffy and Santner.

    Up to 3 years

Secondary Outcomes (5)

  • Duration of response

    Up to 3 years

  • Clinical benefit rate

    Up to 3 years

  • Overall survival

    From registration to death due to any cause, assessed up to 3 years

  • Progression-free survival

    From registration to the earliest date of documentation of disease progression, relapse, or death due to any cause, assessed up to 3 years

  • Incidence of adverse events

    Up to 3 years

Other Outcomes (4)

  • Relationship between risk groups

    Up to 3 years

  • Minimal residual disease (MRD)

    Up to 3 years

  • Overall health-related quality of life

    Up to 18 cycles (each cycle is 28 days)

  • +1 more other outcomes

Study Arms (2)

Arm A (selinexor, dexamethasone, carfilzomib, carfilzomib)

EXPERIMENTAL

Patients receive selinexor PO and dexamethasone PO on days 1, 8 15, and 22, carfilzomib IV on days 1, 8, and 15, and pomalidomide PO on days 1-21. Treatment repeats every 28 days for up to 18 cycles in the absence of disease progression or unacceptable toxicity. Patients also undergo PET/CT or CT and bone marrow biopsy and aspiration during screening and on the trial. Patients may optionally undergo blood sample collection during screening and on the trial.

Drug: CarfilzomibDrug: DexamethasoneDrug: PomalidomideOther: Quality-of-Life AssessmentOther: Questionnaire AdministrationDrug: SelinexorProcedure: X-Ray ImagingProcedure: Computed TomographyProcedure: Positron Emission TomographyProcedure: Bone Marrow BiopsyProcedure: Bone Marrow AspirationProcedure: Biospecimen Collection

Arm B (selinexor, dexamethasone, pomalidomide)

EXPERIMENTAL

Patients receive selinexor PO and dexamethasone PO on days 1, 8, 15, and 22, and pomalidomide PO on days 1-21. Treatment repeats every 28 days for up to 18 cycles in the absence of disease progression or unacceptable toxicity. Patients also undergo PET/CT or CT and bone marrow biopsy and aspiration during screening and on the trial. Patients may optionally undergo blood sample collection during screening and on the trial.

Drug: DexamethasoneDrug: PomalidomideOther: Quality-of-Life AssessmentOther: Questionnaire AdministrationDrug: SelinexorProcedure: X-Ray ImagingProcedure: Computed TomographyProcedure: Positron Emission TomographyProcedure: Bone Marrow BiopsyProcedure: Bone Marrow AspirationProcedure: Biospecimen Collection

Interventions

Given IV

Also known as: Carfilnat, CFZ, Kyprolis, PR-171
Arm A (selinexor, dexamethasone, carfilzomib, carfilzomib)

Given PO

Also known as: Aacidexam, Adexone, Aknichthol Dexa, Alba-Dex, Alin, Alin Depot, Alin Oftalmico, Amplidermis, Anemul mono, Auricularum, Auxiloson, Baycadron, Baycuten, Baycuten N, Cortidexason, Cortisumman, Decacort, Decadrol, Decadron, Decadron DP, Decalix, Decameth, Decasone R.p., Dectancyl, Dekacort, Deltafluorene, Deronil, Desamethasone, Desameton, Dexa-Mamallet, Dexa-Rhinosan, Dexa-Scheroson, Dexa-sine, Dexacortal, Dexacortin, Dexafarma, Dexafluorene, Dexalocal, Dexamecortin, Dexameth, Dexamethasone Intensol, Dexamethasonum, Dexamonozon, Dexapos, Dexinoral, Dexone, Dinormon, Dxevo, Fluorodelta, Fortecortin, Gammacorten, Hemady, Hexadecadrol, Hexadrol, Lokalison-F, Loverine, Methylfluorprednisolone, Millicorten, Mymethasone, Orgadrone, Spersadex, TaperDex, Visumetazone, ZoDex
Arm A (selinexor, dexamethasone, carfilzomib, carfilzomib)Arm B (selinexor, dexamethasone, pomalidomide)

Given PO

Also known as: 4-Aminothalidomide, Actimid, CC-4047, Imnovid, Pomalyst
Arm A (selinexor, dexamethasone, carfilzomib, carfilzomib)Arm B (selinexor, dexamethasone, pomalidomide)

Ancillary studies

Also known as: Quality of Life Assessment
Arm A (selinexor, dexamethasone, carfilzomib, carfilzomib)Arm B (selinexor, dexamethasone, pomalidomide)

Ancillary studies

Arm A (selinexor, dexamethasone, carfilzomib, carfilzomib)Arm B (selinexor, dexamethasone, pomalidomide)

Given PO

Also known as: ATG-010, CRM1 Nuclear Export Inhibitor KPT-330, KPT-330, Selective Inhibitor of Nuclear Export KPT-330, SINE KPT-330, Xpovio
Arm A (selinexor, dexamethasone, carfilzomib, carfilzomib)Arm B (selinexor, dexamethasone, pomalidomide)
X-Ray ImagingPROCEDURE

Undergo x-ray imaging

Also known as: Conventional X-Ray, Diagnostic Radiology, Medical Imaging, X-Ray, Plain film radiographs, Radiographic Imaging, Static X-Ray, X-Ray
Arm A (selinexor, dexamethasone, carfilzomib, carfilzomib)Arm B (selinexor, dexamethasone, pomalidomide)

Undergo PET/CT

Also known as: CAT, CAT Scan, Computed Axial Tomography, Computerized Axial Tomography, CT, CT Scan
Arm A (selinexor, dexamethasone, carfilzomib, carfilzomib)Arm B (selinexor, dexamethasone, pomalidomide)

Undergo PET/CT

Also known as: Medical Imaging, Positron Emission Tomography, PET, PET Scan, Positron Emission Tomography Scan, Positron-Emission Tomography, Proton magnetic resonance spectroscopic imaging
Arm A (selinexor, dexamethasone, carfilzomib, carfilzomib)Arm B (selinexor, dexamethasone, pomalidomide)

Undergo bone marrow biopsy and aspiration

Also known as: Biopsy of Bone Marrow, Biopsy, Bone Marrow
Arm A (selinexor, dexamethasone, carfilzomib, carfilzomib)Arm B (selinexor, dexamethasone, pomalidomide)

Undergo bone marrow biopsy and aspiration

Arm A (selinexor, dexamethasone, carfilzomib, carfilzomib)Arm B (selinexor, dexamethasone, pomalidomide)

Undergo blood sample collection

Also known as: Biological Sample Collection, Biospecimen Collected, Specimen Collection, Blood Sample Collection
Arm A (selinexor, dexamethasone, carfilzomib, carfilzomib)Arm B (selinexor, dexamethasone, pomalidomide)

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Age \>= 18 years
  • Diagnosis of RRMM with progressive disease at study entry as per the International Myeloma Working Group (IMWG) uniform criteria
  • Measurable disease by IMWG criteria as defined by at least one of the following:
  • Serum M-protein \>= 0.5 g/dL
  • Urine M-protein \>= 200 mg in a 24-hour collection
  • Serum free light chain level \>= 10 mg/dL provided the free light chain ratio is abnormal
  • For patients with extramedullary measurable disease (EMD) by CT or MRI or the CT portion of the PET/CT: Must have at least one lesion that has a single diameter of \>= 2 cm. Skin lesions can be used if the area is \>= 2cm in at least one diameter and measured with a ruler
  • Bone marrow plasma cells \>= 30%
  • Patients with IgA myeloma in whom serum protein electrophoresis is deemed unreliable, due to co-migration of normal serum proteins with the para protein in the beta region, may be considered eligible as long as total serum IgA level is elevated above normal range
  • Prior treatment:
  • Arm A: At least one of the following must be true: (1) Subjects must have been previously treated with at least 3 prior lines of therapy, including a proteasome inhibitor and an immunomodulatory drug (IMiD) (2) Subjects who are refractory to carfilzomib and/or pomalidomide may enroll in Arm A using the quadruplet regimen, SKPd, provided carfilzomib, pomalidomide and dexamethasone (KPd) triplet is not the most recent line of prior therapy and that they have been previously treated with at least 3 prior lines of therapy, including a proteasome inhibitor and an IMiD. Carfilzomib/Pomalidomide refractory status is defined by the IMWG criteria: disease that is nonresponsive (stable disease \[SD\] or progressive disease \[PD\]) while on therapy, or progresses within 60 days of last therapy in patients who have achieved minimal response (MR) or better at some point previously before then progressing in their disease course.
  • Arm B: Subjects must have progressive disease and been exposed to up to 2 prior lines of therapy, including a proteasome inhibitor and lenalidomide
  • Provide written informed consent
  • Willing to return to enrolling institution for follow-up (during the active monitoring phase of the study) and ability to adhere with the study visit schedule and other protocol procedures
  • Willingness to provide mandatory tissue specimens for correlative research
  • +16 more criteria

You may not qualify if:

  • History of myocardial infarction =\< 6 months prior to pre-registration, or congestive heart failure requiring use of ongoing maintenance therapy for life threatening ventricular arrhythmias. Unstable angina within 4 months prior to randomization, New York Heart Association (NYHA) class III or IV heart failure, left ventricular ejection fraction (LVEF) \< 40%, uncontrolled angina, corrected QT (QTc) interval \>= 470 msec, History of severe coronary artery disease, severe uncontrolled ventricular arrhythmias including uncontrolled chronic atrial fibrillation/atrial flutter, history of torsades de pointe, sick sinus syndrome, or electrocardiographic evidence of acute ischemia or grade 3 conduction system abnormalities unless subject has a pacemaker
  • Failure to recover from acute, reversible effects of prior therapy regardless of interval since last treatment.
  • EXCEPTION: Grade 1 peripheral (sensory) neuropathy that has been stable for at least 1 month since completion of prior treatment
  • Uncontrolled intercurrent non-cardiac illness including, but not limited to:
  • Ongoing or active infection. Uncontrolled infection requiring parenteral antibiotics, antivirals, or antifungals =\< 14 days prior to registration; patients with controlled infection or on prophylactic antibiotics are permitted in the study
  • Psychiatric illness/social situations
  • Dyspnea at rest due to complications of advanced malignancy or other disease that requires continuous oxygen therapy
  • Any other conditions that would limit compliance with study requirements
  • Patients known to be human immunodeficiency virus (HIV) positive and/or currently receiving antiretroviral therapy
  • Currently receiving any other investigational agent which would be considered as a treatment for RRMM
  • Non investigational radiation, chemotherapy, or immunotherapy or any other anticancer therapy =\< 14 days or five half-lives, whichever is shorter prior to registration. Note: (localized radiation to a single site =\< 7 days prior to registration is allowed)
  • Participation in an investigational anti-cancer study =\< 21 days or five half-lives whichever is shorter prior to registration
  • Major Surgery =\< 21 days prior to registration
  • Co-morbid systemic illnesses or other severe concurrent disease which, in the judgment of the investigator, would make the patient inappropriate for entry into this study or interfere significantly with the proper assessment of safety and toxicity of the prescribed regimens
  • Other active malignancy =\< 5 years prior to registration. EXCEPTIONS: Non-melanotic skin cancer or carcinoma-in-situ of the cervix that has undergone potentially curative therapy. NOTE: If there is a history of prior malignancy, they must not be receiving other specific treatment for their cancer
  • +22 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Mayo Clinic in Rochester

Rochester, Minnesota, 55905, United States

Location

Related Links

MeSH Terms

Conditions

Multiple Myeloma

Interventions

carfilzomibDexamethasoneCalcium Dobesilateauricularumdexamethasone acetatedexamethasone 21-phosphatepomalidomideselinexorX-RaysPhantoms, ImagingMagnetic Resonance SpectroscopyBiopsySpecimen Handling

Condition Hierarchy (Ancestors)

Neoplasms, Plasma CellNeoplasms by Histologic TypeNeoplasmsHemostatic DisordersVascular DiseasesCardiovascular DiseasesParaproteinemiasBlood Protein DisordersHematologic DiseasesHemic and Lymphatic DiseasesHemorrhagic DisordersLymphoproliferative DisordersImmunoproliferative DisordersImmune System Diseases

Intervention Hierarchy (Ancestors)

PregnadienetriolsPregnadienesPregnanesSteroidsFused-Ring CompoundsPolycyclic CompoundsSteroids, FluorinatedBenzenesulfonatesBenzene DerivativesHydrocarbons, AromaticHydrocarbons, CyclicHydrocarbonsOrganic ChemicalsArylsulfonatesArylsulfonic AcidsSulfonic AcidsSulfur AcidsSulfur CompoundsElectromagnetic RadiationElectromagnetic PhenomenaMagnetic PhenomenaPhysical PhenomenaRadiationRadiation, IonizingEquipment and SuppliesSpectrum AnalysisChemistry Techniques, AnalyticalInvestigative TechniquesCytodiagnosisCytological TechniquesClinical Laboratory TechniquesDiagnostic Techniques and ProceduresDiagnosisDiagnostic Techniques, SurgicalSurgical Procedures, Operative

Study Officials

  • Prashant Kapoor, M.D.

    Mayo Clinic in Rochester

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 17, 2021

First Posted

February 21, 2021

Study Start

May 6, 2021

Primary Completion

May 6, 2026

Study Completion (Estimated)

December 17, 2027

Last Updated

February 25, 2026

Record last verified: 2026-02

Locations