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Multiple MyelomaPhase 1NCT05651932

A Study of an MMSET Inhibitor in Patients With Relapsed and Refractory Multiple Myeloma

A Phase 1 study testing Cohort A1 & A2: KTX-1001, Cohort B1 & B2: KTX-1001+Mezigdomide and Cohort C1 & C2: KTX-1001 + Carfilzomib (KYPROLIS®) in people with Multiple Myeloma and Myeloma, run by K36 Therapeutics, Inc. at 29 sites in 4 countries.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial summary

A Phase I study to evaluate the safety of a novel, orally available, selective, and potent small molecule inhibitor of the histone lysine methyl transferase MMSET (also known as NSD2/WHSC1) to prevent the dimethylation of H3K36 in adult patients with relapsed or refractory multiple myeloma (RRMM). Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Multiple Myeloma, Myeloma, Myeloma Multiple
What is being tested?
Cohort A1 & A2: KTX-1001, Cohort B1 & B2: KTX-1001+Mezigdomide and Cohort C1 & C2: KTX-1001 + Carfilzomib (KYPROLIS®)
Could I receive a placebo?
No, everyone receives an active treatment
How many people
165 participants
Where
29 sitesin 4 countries
Study dates
22 Feb 2023to 30 Jun 2028
Phase
Phase 1
Are there results?
Not yet: the study is under way

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

  • ≥ 18 years of age
  • ECOG score ≤ 1
  • Multiple myeloma (as per IMWG)
  • Prior therapy for MM: Participants must have received at least 1 and up to 3 prior lines of therapy as defined by IMWG, and the following drug classes: PI, IMiD, and anti-CD38 antibody. For mezigdomide combination Cohorts B1 and B2, participants must have received at least 2 prior lines of therapy
  • Participants must have a confirmed diagnosis of progressive MM (per IMWG), t(4;14) confirmed by fluorescence in situ hybridization (FISH) testing performed in a centralized Clinical Laboratory Improvement Amendments (CLIA) accredited laboratory via fresh tumor biopsy.
  • Measurable disease, including at least 1 of the following criteria:
Show all 12Show fewer
  • Serum M protein ≥ 0.50 g/dL (by SPEP)
  • Serum IgA ≥ 0.50 g/dL (IgA myeloma patients)
  • Urine M protein ≥ 200 mg/24 h (by UPEP)
  • sFLC involved light chain ≥ 10 mg/dL (100 mg/L) (patients with abnormal sFLC ratio)
  • Bone marrow plasma cells ≥ 30% (if only criterion for measurability)
  • Agreement to enroll into the REMS program (Cohort D- pomalidomide cohort only)

Who cannot join

  • Treatment with the following therapies in the specified time period prior to first dose:
  • Patients in Cohorts B1 and B2 must not have received prior mezigdomide treatment
  • Carfilzomib in the immediate last prior line of therapy for patients enrolled in Cohorts C1 and C2
  • Pomalidomide in the immediate last prior line of therapy for patients enrolled in cohort D
  • Radiation, chemotherapy, immunotherapy, or any other anticancer therapy ≤ 2 weeks
  • Cellular therapies ≤ 8 weeks
Show all 17Show fewer
  • Autologous transplant < 100 days
  • Allogenic transplant ≤ 6 months, or > 6 months with active GVHD
  • Major surgery ≤ 4 weeks
  • Current plasma cell leukemia, POEMS (polyneuropathy, organomegaly, endocrinopathy, and skin changes) syndrome, solitary bone lesion or bone lesions as the only evidence for plasma cell dyscrasia, myelodysplastic syndrome or a myeloproliferative neoplasm or light chain amyloidosis
  • Active CNS disease: participants with previously treated stable CNS disease are eligible, except for Cohorts B1 and B2 for which known CNS myeloma involvement is completely excluded.
  • Inadequate bone marrow function
  • Inadequate renal, hepatic, pulmonary, and cardiac function
  • Active, ongoing, or uncontrolled systemic viral, bacterial, or fungal infection. Permitted prophylactic medications, antimicrobials or antiretroviral therapies defined in protocol.
  • Use of acid reducing agents and strong inhibitors or inducers of CYP3A4 within 7 days or 5 half-lives (whichever is longer) prior to first dose
  • Strong CYP1A2 inhibitors for patients receiving pomalidomide (Cohort D)
  • Active malignancy not related to myeloma requiring therapy within < 2 years prior to enrollment, or not in complete remission, with exceptions defined in protocol.

Trial locations and contacts

29 sites in 4 countries. Contact the study team or a site near you to ask about taking part.

Soo Bang, MHSA contact

1-347-342-7199

sbang@k36tx.com

Latasha Nisbett, MS contact

1-617-347-5787

lnisbett@k36tx.com

29 sites in 4 countries

United States19 sites, 19 recruiting

  • University of Michigan

    Ann Arbor, Michigan, 48109

    Cancer Answer Line, 800-865-1125, canceranswerline@med.umich.edu

    Matthew Pianko, MD

    Not yet recruiting

  • The Winship Cancer Institute of Emory University

    Atlanta, Georgia, 30322

    Geraldine Onwuzor, 404-778-5144, chika.geraldine.onwuzor@emory.edu

    Sagar Lonial, MD, FACP

    Recruiting

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114

    Andrew Yee, MD, 617-724-4000

    Andrew Yee, MD

    Recruiting

  • Dana-Farber Cancer Institute

    Boston, Massachusetts, 02215

    Yuxin Liu, MD, yuxin_liu@dfci.harvard.edu

    Yuxin Liu, MD

    Recruiting

  • Medical University of South Carolina (MUSC) - Hollings Cancer Center

    Charleston, South Carolina, 29425

    Shata Salzer, 843-792-1463, salzers@musc.edu

    Anthony Dominick, DO

    Recruiting

  • Atrium Health, Levine Cancer Institute

    Charlotte, North Carolina, 28204

    Missy Leahy, melissa.leahy@advocatehealth.org

    Cindy Varga, MD

    Recruiting

  • Cleveland Clinic - Taussig Cancer Institute

    Cleveland, Ohio, 44195

    Cancer Answer Line, 216-444-7923, CancerAnswer@ccf.org

    Louis Williams, MD

    Recruiting

  • University of Texas Southwestern Harold C. Simmons Comprehensive Cancer Center

    Dallas, Texas, 75235

    Aimaz Afrough, MD, 833-722-6237, aimaz.afrough@utsouthwestern.edu

    Aimaz Afrough, MD

    Recruiting

  • Duke University Hospital

    Durham, North Carolina, 27705

    Jen Tichon, 919-660-7262, jennifer.tichon@duke.edu

    Cristina Gasparetto, MD

    Recruiting

  • Hackensack University Medical Center

    Hackensack, New Jersey, 07601

    Palka Anand, 551-996-3040, Palka.Anand@hmhn.org

    David S Siegel, MD, PhD

    Recruiting

  • Oncology Consultants

    Houston, Texas, 77030

    Dr. Julio A. Peguero, MD, 713-600-0900, Jpeguero@oncologyconsultants.com

    Julio A Peguero, MD

    Recruiting

  • Mayo Clinic Hospital - Florida

    Jacksonville, Florida, 32224

    Ahsan Rasheed, 904-953-2000, rasheed.ahsan@mayo.edu

    Vivek Roy, MD

    Recruiting

  • University of Miami Don Soffer Clinical Research Center

    Miami, Florida, 33136

    Michelle Armogan, 305-243-7479, mda182@miami.edu

    Dickran Kazandjian, MD

    Not yet recruiting

  • Tennessee Oncology

    Nashville, Tennessee, 37203

    Austin Windsor, 629-278-3554, awindsor@tnonc.com

    Jesus Berdeja, MD

    Recruiting

  • Memorial Sloan-Kettering Cancer Center

    New York, New York, 10065

    Mary Silverberg, silverb1@mskcc.org

    Sharon Kim, kims13@mskcc.org

    Recruiting

  • University of Pennsylvania

    Philadelphia, Pennsylvania, 19104

    Sara Whittington, sara.whittington@pennmedicine.upenn.edu

    Ed Stadtmauer, MD

    Recruiting

  • Mayo Clinic - Transplant Center - Rochester

    Rochester, Minnesota, 55905

    Thomas Nelson, 855-776-0015, nelson.thomas2@mayo.edu

    David Dingli, MD, PhD

    Recruiting

  • UCSF Medical Center - Hematology and Blood and Marrow Transplant Clinic

    San Francisco, California, 94143

    Phu Lam, 415-858-7994, phu.lam@ucsf.edu

    Alfred Chung, MD

    Recruiting

  • Northwest Medical Specialties, PLLC

    Tacoma, Washington, 98405

    Sara Sandefur, 253-380-5299, ssandefur@p1trials.com

    Jorge Chaves, MD

    Recruiting

Other trials in United States

France4 sites, 4 recruiting

  • Centre Hospitalier Universitaire de Nantes (CHU de Nantes) - Hotel-Dieu

    Nantes

    Cyrille Touzeau, MD, 33 (0) 2 40 08 32 71, cyrille.touzeau@chu-nantes.fr

    Cyrille Touzeau, MD

    Recruiting

  • Centre Hospitalier Universitaire de Poitiers (CHU de Poitiers)

    Poitiers

    Romane Giraud, +33 5 16 60 43 06, Romane.giraud@chu-poitiers.fr

    Xavier Leleu, MD

    Recruiting

  • Institut Universitaire du Cancer de Toulouse - Oncopole

    Toulouse

    Pierre Bories, MD

    Recruiting

  • Universitaire de Lille

    Villeneuve-d'Ascq, France

    Salomon Manier, MD, 03 59 50 75 69, Salomon.manier@chru-lille.fr

    Salomon Manier, MD

    Recruiting

Other trials in France

Spain4 sites, 4 recruiting

  • Hospital ClÃ-nic de Barcelona

    Barcelona

    Julio Vidal, +34 93 227 5484, contratoshemato@recerca.clinic.cat

    Laura Rosinol, MD, PhD

    Recruiting

  • Hospital Universitario 12 de Octubre

    Madrid

    Joaquín Martínez López, MD, +34 91 779 2877, jmarti01@med.ucm.es

    Lucía Sánchez Pérez, (+34) 91 779 28 09, luciasanchez12imas@gmail.com

    Recruiting

  • Clínica Universidad de Navarra

    Pamplona, Navarre, 31008

    Ana Valcarcel, avalcarcelg@unav.es

    Paula Rodriguez, MD, PhD

    Recruiting

  • Instituto de Investigacion Biomedica de Salamanca (IBSAL)

    Salamanca

    Carmen Arias, +34 92 329 1100 ext. 56094, ensayosclinicos@ibsal.es

    Maria-Victoria Mateos Manteca, MD, PhD

    Recruiting

Other trials in Spain

Canada2 sites, 2 recruiting

  • Cross Cancer Institute

    Edmonton, Alberta, T6G IZ2

    Sandhu Iwindeep, 780-432-8221, irwindee@ualberta.ca

    Sandhu Irwindeep, MD

    Not yet recruiting

  • University Health Network (UHN) - Princess Margaret Cancer Centre (Princess Margaret Hospital)

    Toronto, Ontario, M5G 2C4

    Saima Dean, saima.dean@uhnresearch.ca

    Suzanne Trudel, MSc, MD

    Recruiting

Other trials in Canada

About this study

Official title: A Phase 1 Study of KTX-1001, an Oral, First-In-Class, Selective, and Potent MMSET Catalytic Inhibitor That Suppresses H3K36me2 in Patients With Relapsed and Refractory Multiple Myeloma

A Phase I study to evaluate the safety of a novel, orally available, selective, and potent small molecule inhibitor of the histone lysine methyl transferase MMSET (also known as NSD2/WHSC1) to prevent the dimethylation of H3K36 in adult patients with relapsed or refractory multiple myeloma (RRMM).

Read the detailed description

This is a Phase I, open-label, dose-escalation and expansion study in adult patients with RRMM.

In the dose escalation phase (Part A), patients will be evaluated for DLTs during Cycle 1 (28 days). The KTX-1001 MTD, RP2D, and schedule will be determined.

In the dose expansion phase (Part B), patients with t(4;14) will receive KTX-1001 at the RP2D alone and in combination with investigational therapy Mezigdomide or SOC therapy (dexamethasone, carfilzomib or pomalidomide) to further define safety and tolerability and provide preliminary efficacy information.

Being tested
Cohort A1 & A2: KTX-1001, Cohort B1 & B2: KTX-1001+Mezigdomide, Cohort C1 & C2: KTX-1001 + Carfilzomib (KYPROLIS®), Cohort D: KTX-1001+ pomalidomide (Pomalyst, Imnovid)
Keywords
NSD2, MMSET, WHSC1, T4;14, T(4;14), translocation, myeloma, RRMM
Medical subject headings
Multiple Myeloma, Neoplasms, Plasma Cell

What participants receive

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

  • Cohort A1 & A2: KTX-1001Drug

    Also called KTX-1001, Dexamethasone

    KTX-1001: Orally for 28 days each cycle until progression. Dexamethasone: Orally once weekly

  • Cohort B1 & B2: KTX-1001+MezigdomideDrug

    Also called Mezigdomide

    Drug: KTX-1001: Orally for 28 days each cycle until progression Drug: Dexamethasone: Orally once weekly Drug: Mezigdomide Dexamethasone: Orally once weekly

  • Cohort C1 & C2: KTX-1001 + Carfilzomib (KYPROLIS®)Drug

    Also called Carfilzomib (KYPROLIS®), Dexamethasone

    Drug: KTX-1001: Orally for 28 days each cycle until progression Drug: Dexamethasone: Orally once weekly Drug: Carfilzomib (KYPROLIS®): IV, once weekly for 3 weeks in each 28-day cycle

  • Cohort D: KTX-1001+ pomalidomide (Pomalyst, Imnovid)Drug

    Also called Pomalidomide (Pomalyst, Imnovid), Dexamethasone

    Drug: KTX-1001: Orally for 28 days each cycle until progression Drug: Dexamethasone: Orally once a week Drug: Pomalidomide (Pomalyst, Imnovid): Orally, for 21 days in each 28-day cycle

Study groups in NCT05651932
Study groupRoleWhat they receive
Cohort A (Single agent): KTX-1001 + dexamethasoneExperimentalCohort A1 (single agent): KTX-1001 at RP2D1 + dex Cohort A2 (single agent): KTX-1001 at RP2D2 + dex
Receives: Drug: Cohort A1 & A2: KTX-1001
Cohort B (Mezigdomide): KTX-1001 + Mezigdomide + dexExperimentalCohort B1 (Mezigdomide): KTX-1001 at RP2D1 + Mezigdomide + dex Cohort B2 (Mezigdomide):: KTX-1001 at RP2D2 + Mezigdomide + dex
Receives: Drug: Cohort B1 & B2: KTX-1001+Mezigdomide
Cohort C (carfilzomib/KYPROLIS®): KTX-1001 + carfilzomib + dexExperimentalCohort C1 (carfilzomib/KYPROLIS®): KTX-1001 at RP2D1 + carfilzomib + dex Cohort C2 (carfilzomib/KYPROLIS®): KTX-1001 at RP2D2 + carfilzomib + dex
Receives: Drug: Cohort C1 & C2: KTX-1001 + Carfilzomib (KYPROLIS®)
Cohort D (pomalidomide): KTX-1001 + pomalidomide + dexExperimentalCohort D (pomalidomide): KTX-1001 at RP2D2 + pomalidomide + dex
Receives: Drug: Cohort D: KTX-1001+ pomalidomide (Pomalyst, Imnovid)

What is being measured

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

  1. Dose Escalation: Determination of Recommended Phase 2 Dose (RP2D) and/or Maximum Tolerated Dose (MTD) Dose Expansion: Provide preliminary efficacy data on the antitumor effects of KTX-1001 in combination with other anti-myeloma therapy

    Incidence of dose-limiting toxicity (DLTs), treatment-emergent adverse events (TEAEs), treatment-related AEs, and clinically significant changes in laboratory test results

    Measured: Cycle 1 (28 days)

Secondary outcomes (3)

  1. Disease Specific Response to KTX-1001± Combination Therapy

    Objective Response Rate (ORR) Duration of Response (DOR) Progression Free Survival (PFS) Minimal Residual Disease (MRD)

    Per IMWG Consensus Criteria for Response and Minimal Residual Disease Assessment in Multiple Myeloma

    Measured: Duration of Study

  2. Pharmacokinetics & Pharmacodynamics KTX-1001± Combination Therapy

    Maximum plasma concentration (Cmax) of KTX-1001 Time to achieve Cmax (tmax) for KTX-1001 Area under the plasma concentration-time curve (AUC) for KTX-1001

    Measured: Duration of Study

  3. Safety profile of KTX-1001± Combination Therapy

    Frequency and severity of TEAEs, treatment-related AEs, and clinically significant changes in laboratory test results

    Measured: Duration of Study

Timeline

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

  1. First posted15 Dec 2022
  2. Study start22 Feb 2023
  3. Primary completion31 Dec 2027 (estimated)
  4. Study completion30 Jun 2028 (estimated)

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
Non-randomised
Intervention model
Single group
Masking
None (open label)
Enrolment
165 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05651932
Sponsor's protocol ID
KTX-MMSET-001
Other IDs
EUCTR No: 2022-500801-41-00
Status last verified
Aug 2026
First submitted
1 Dec 2022
Last updated
22 Oct 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Undecided

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Cohort A1 & A2: KTX-1001, Cohort B1 & B2: KTX-1001+Mezigdomide and Cohort C1 & C2: KTX-1001 + Carfilzomib (KYPROLIS®) in people with Multiple Myeloma and Myeloma, run by K36 Therapeutics, Inc. at 29 sites in 4 countries.

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 12 inclusion and 17 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: looking for participants now, as of the sponsor's last update on 22 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 29 sites in United States, France, Spain, Canada. 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?

This study is still under way. Results are not available in the source record yet.

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

Contact Soo Bang, MHSA (1-347-342-7199, sbang@k36tx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Dose Escalation: Determination of Recommended Phase 2 Dose (RP2D) and/or Maximum Tolerated Dose (MTD) Dose Expansion: Provide preliminary efficacy data on the antitumor effects of KTX-1001 in combination with other anti-myeloma therapy (time frame: Cycle 1 (28 days)). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05651932.

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 22 Oct 2026.

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