A Study of an MMSET Inhibitor in Patients With Relapsed and Refractory Multiple Myeloma
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
2 other identifiers
interventional
125
4 countries
22
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).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_1 multiple-myeloma
Started Feb 2023
Typical duration for phase_1 multiple-myeloma
22 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
December 1, 2022
CompletedFirst Posted
Study publicly available on registry
December 15, 2022
CompletedStudy Start
First participant enrolled
February 22, 2023
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 31, 2027
ExpectedStudy Completion
Last participant's last visit for all outcomes
June 30, 2028
February 10, 2026
January 1, 2026
4.9 years
December 1, 2022
February 6, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (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
Cycle 1 (28 days)
Secondary Outcomes (3)
Disease Specific Response to KTX-1001± Combination Therapy
Duration of Study
Pharmacokinetics & Pharmacodynamics KTX-1001± Combination Therapy
Duration of Study
Safety profile of KTX-1001± Combination Therapy
Duration of Study
Study Arms (4)
Cohort A (Single agent): KTX-1001 + dexamethasone
EXPERIMENTALCohort A1 (single agent): KTX-1001 at RP2D1 + dex Cohort A2 (single agent): KTX-1001 at RP2D2 + dex
Cohort B (Mezigdomide): KTX-1001 + Mezigdomide + dex
EXPERIMENTALCohort B1 (Mezigdomide): KTX-1001 at RP2D1 + Mezigdomide + dex Cohort B2 (Mezigdomide):: KTX-1001 at RP2D2 + Mezigdomide + dex
Cohort C (carfilzomib/KYPROLIS®): KTX-1001 + carfilzomib + dex
EXPERIMENTALCohort C1 (carfilzomib/KYPROLIS®): KTX-1001 at RP2D1 + carfilzomib + dex Cohort C2 (carfilzomib/KYPROLIS®): KTX-1001 at RP2D2 + carfilzomib + dex
Cohort D (pomalidomide): KTX-1001 + pomalidomide + dex
EXPERIMENTALCohort D (pomalidomide): KTX-1001 at RP2D1 or RP2D2 + pomalidomide + dex
Interventions
KTX-1001: Orally for 28 days each cycle until progression. Dexamethasone: Orally once weekly
Drug: KTX-1001: Orally for 28 days each cycle until progression Drug: Dexamethasone: Orally once weekly Drug: Mezigdomide Dexamethasone: Orally once weekly
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
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
Eligibility Criteria
You may qualify if:
- ≥ 18 years of age
- ECOG score ≤ 1
- Multiple myeloma (as per IMWG)
- ≥ 3 prior lines of therapy, including a PI, an IMiD, and an anti-CD38 antibody
- Patients must be refractory to their last prior therapy
- Cohorts A1/A2: Patients must have exhausted available therapeutic options that are expected to provide a meaningful clinical benefit, either through disease relapse, treatment refractory disease, intolerance, or refusal of the therapy
- t(4;14) confirmed by standard of care FISH testing
- 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)
You may not qualify if:
- 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
- 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 14 days or 5 half-lives prior to first dose
- +2 more criteria
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- K36 Therapeutics, Inc.lead
- Bristol-Myers Squibbcollaborator
Study Sites (22)
UCSF Medical Center - Hematology and Blood and Marrow Transplant Clinic
San Francisco, California, 94143, United States
Mayo Clinic Hospital - Florida
Jacksonville, Florida, 32224, United States
The Winship Cancer Institute of Emory University
Atlanta, Georgia, 30322, United States
Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
Dana-Farber Cancer Institute
Boston, Massachusetts, 02215, United States
Mayo Clinic - Transplant Center - Rochester
Rochester, Minnesota, 55905, United States
Hackensack University Medical Center
Hackensack, New Jersey, 07601, United States
Memorial Sloan-Kettering Cancer Center
New York, New York, 10065, United States
Atrium Health, Levine Cancer Institute
Charlotte, North Carolina, 28204, United States
Duke University Hospital
Durham, North Carolina, 27705, United States
University of Pennsylvania
Philadelphia, Pennsylvania, 19104, United States
Tennessee Oncology
Nashville, Tennessee, 37203, United States
University of Texas Southwestern Harold C. Simmons Comprehensive Cancer Center
Dallas, Texas, 75235, United States
University Health Network (UHN) - Princess Margaret Cancer Centre (Princess Margaret Hospital)
Toronto, Ontario, M5G 2C4, Canada
Universitaire de Lille
Villeneuve-d'Ascq, France, France
Centre Hospitalier Universitaire de Nantes (CHU de Nantes) - Hotel-Dieu
Nantes, France
Centre Hospitalier Universitaire de Poitiers (CHU de Poitiers)
Poitiers, France
Institut Universitaire du Cancer de Toulouse - Oncopole
Toulouse, France
Clínica Universidad de Navarra
Pamplona, Navarre, 31008, Spain
Hospital ClÃ-nic de Barcelona
Barcelona, Spain
Hospital Universitario 12 de Octubre
Madrid, Spain
Instituto de Investigacion Biomedica de Salamanca (IBSAL)
Salamanca, Spain
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
December 1, 2022
First Posted
December 15, 2022
Study Start
February 22, 2023
Primary Completion (Estimated)
December 31, 2027
Study Completion (Estimated)
June 30, 2028
Last Updated
February 10, 2026
Record last verified: 2026-01