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

What's the purpose of this trial?

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).

This trial is currently open and accepting patients.

What will happen during the trial?

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 translocation t(4;14) or a GOF mutation in MMSET (eg, E1099K) will be enrolled. Patients will receive KTX-1001 at the RP2D to further define safety and tolerability and provide preliminary efficacy information.

You may be able to join this trial if you:

The following criteria is a partial list of reasons why patients may be eligible to participate in this clinical trial. Further evaluation with a medical professional is required.

Key Inclusion Criteria:

  • ≥ 18 years of age
  • ECOG score ≤ 2
  • Relapsed or refractory multiple myeloma (as per IMWG)
    • ≥ 3 prior lines of therapy, including a PI, an IMiD, and an anti-CD38 antibody
    • 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, or GOF mutation in MMSET confirmed by local sequencing test (Part B dose expansion cohorts only)
  • 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)
    • ≥ 1 extramedullary lesion ≥ 1 cm in size and able to be followed by imaging assessments (Part A dose escalation cohorts only)
    • Bone marrow plasma cells ≥ 10% (Part A dose escalation cohorts only)

Key Exclusion Criteria:

  • Treatment with the following therapies in the specified time period prior to first dose:
    • 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
  • History of or 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
  • Active malignancy not related to myeloma requiring therapy within < 3 years prior to enrollment, or not in complete remission, with exceptions defined in protocol.

Additional Trial Information

Phase 1

Enrollment: 60 patients (estimated)

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Trial Locations

All Trial Locations

View all clinical trial locations sorted by state.


Mayo Clinic (Arizona)

Phoenix, AZ

Open and Accepting



Mayo Clinic (Jacksonville)

Jacksonville, FL

Open and Accepting


Winship Cancer Institute Emory University

Atlanta, GA

Open and Accepting


University of Kansas Cancer Center (Westwood)

Westwood, KS

Open and Accepting


Massachusetts General Hospital

Boston, MA

Open and Accepting


Mayo Clinic (Rochester)

Rochester, MN

Open and Accepting

New Jersey

New York

Memorial Sloan Kettering Cancer Center

New York, NY

Open and Accepting

North Carolina

Duke Cancer Center Duke University Medical Center

Durham, NC

Open and Accepting



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