Recruiting
Phase 1

MMSET Inhibitor

Sponsor:

K36 Therapeutics, Inc.

Code:

NCT05651932

Conditions

Multiple Myeloma

Myeloma

Myeloma Multiple

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

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)

Study Details

Brief 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).

Conditions

Multiple Myeloma

Myeloma

Myeloma Multiple

Study ID

NCT05651932

Start date

Feb 22, 2023

Status verified date

Aug, 2026

Completion date

Jun 30, 2028

Anticipated

Primary completion date

Dec 31, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Key Inclusion Criteria for Dose-Expansion:

  • ≥ 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:

  • 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)

Key Exclusion Criteria for Dose-Expansion:

  • 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
  • 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.

Study Design

Enrollment

165 participants

Anticipated

Allocation

Non randomized

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Cohort A (Single agent): KTX-1001 + dexamethasone

Cohort A1 (single agent): KTX-1001 at RP2D1 + dex Cohort A2 (single agent): KTX-1001 at RP2D2 + dex

experimental: Cohort B (Mezigdomide): KTX-1001 + Mezigdomide + dex

Cohort B1 (Mezigdomide): KTX-1001 at RP2D1 + Mezigdomide + dex Cohort B2 (Mezigdomide):: KTX-1001 at RP2D2 + Mezigdomide + dex

experimental: Cohort C (carfilzomib/KYPROLIS®): KTX-1001 + carfilzomib + dex

Cohort C1 (carfilzomib/KYPROLIS®): KTX-1001 at RP2D1 + carfilzomib + dex Cohort C2 (carfilzomib/KYPROLIS®): KTX-1001 at RP2D2 + carfilzomib + dex

experimental: Cohort D (pomalidomide): KTX-1001 + pomalidomide + dex

Cohort D (pomalidomide): KTX-1001 at RP2D2 + pomalidomide + dex

Interventions

Cohort A1 & A2: KTX-1001

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

Cohort B1 & B2: KTX-1001+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: 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: 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

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) ]

Central Contacts and Locations

Central contacts

Soo Bang, MHSA

1-347-342-7199sbang@k36tx.com

Latasha Nisbett, MS

1-617-347-5787lnisbett@k36tx.com

Locations

UCSF Medical Center - Hematology and Blood and Marrow Transplant Clinic

Recruiting

San Francisco, California, United States, 94143

Contacts

Phu Lam

415-858-7994phu.lam@ucsf.edu

Principal Investigator:

Alfred Chung, MD

Mayo Clinic Hospital - Florida

Recruiting

Jacksonville, Florida, United States, 32224

Contacts

Ahsan Rasheed

904-953-2000rasheed.ahsan@mayo.edu

Principal Investigator:

Vivek Roy, MD

The Winship Cancer Institute of Emory University

Recruiting

Atlanta, Georgia, United States, 30322

Contacts

Geraldine Onwuzor

404-778-5144chika.geraldine.onwuzor@emory.edu

Principal Investigator:

Sagar Lonial, MD, FACP

Massachusetts General Hospital

Recruiting

Boston, Massachusetts, United States, 02114

Contacts

Andrew Yee, MD

617-724-4000

Principal Investigator:

Andrew Yee, MD

Dana-Farber Cancer Institute

Recruiting

Boston, Massachusetts, United States, 02215

Contacts

Yuxin Liu, MD

yuxin_liu@dfci.harvard.edu

Principal Investigator:

Yuxin Liu, MD

Mayo Clinic - Transplant Center - Rochester

Recruiting

Rochester, Minnesota, United States, 55905

Contacts

Thomas Nelson

855-776-0015nelson.thomas2@mayo.edu

Principal Investigator:

David Dingli, MD, PhD

Hackensack University Medical Center

Recruiting

Hackensack, New Jersey, United States, 07601

Contacts

Palka Anand

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

Principal Investigator:

David S Siegel, MD, PhD

Memorial Sloan-Kettering Cancer Center

Recruiting

New York, New York, United States, 10065

Contacts

Mary Silverberg

silverb1@mskcc.org

Sharon Kim

kims13@mskcc.org

Principal Investigator:

Saad Usmani, MD, FACP

Atrium Health, Levine Cancer Institute

Recruiting

Charlotte, North Carolina, United States, 28204

Contacts

Missy Leahy

melissa.leahy@advocatehealth.org

Principal Investigator:

Cindy Varga, MD

Duke University Hospital

Recruiting

Durham, North Carolina, United States, 27705

Contacts

Jen Tichon

919-660-7262jennifer.tichon@duke.edu

Principal Investigator:

Cristina Gasparetto, MD

University of Pennsylvania

Recruiting

Philadelphia, Pennsylvania, United States, 19104

Contacts

Sara Whittington

sara.whittington@pennmedicine.upenn.edu

Principal Investigator:

Ed Stadtmauer, MD

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

Recruiting

Charleston, South Carolina, United States, 29425

Contacts

Shata Salzer

843-792-1463salzers@musc.edu

Principal Investigator:

Anthony Dominick, DO

Tennessee Oncology

Recruiting

Nashville, Tennessee, United States, 37203

Contacts

Austin Windsor

629-278-3554awindsor@tnonc.com

Principal Investigator:

Jesus Berdeja, MD

University of Texas Southwestern Harold C. Simmons Comprehensive Cancer Center

Recruiting

Dallas, Texas, United States, 75235

Contacts

Aimaz Afrough, MD

833-722-6237aimaz.afrough@utsouthwestern.edu

Principal Investigator:

Aimaz Afrough, MD

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

Recruiting

Toronto, Ontario, Canada, M5G 2C4

Contacts

Saima Dean

saima.dean@uhnresearch.ca

Principal Investigator:

Suzanne Trudel, MSc, MD

More Information

Sponsor

K36 Therapeutics, Inc.

Last update posted

Aug 18, 2026

Last verified

Aug, 2026

Keywords

  • NSD2
  • MMSET
  • WHSC1
  • T4;14
  • T(4;14)
  • translocation
  • myeloma
  • RRMM

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by K36 Therapeutics, Inc. on 2026-08-18.