for people ages 18 years and up (full criteria)
at San Francisco, California and other locations
study started
completion around
Principal Investigator
by Alfred Chung, MD
Headshot of Alfred Chung
Alfred Chung



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).

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


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.


Multiple Myeloma, Myeloma, Myeloma Multiple, NSD2, MMSET, WHSC1, T4;14, T(4;14), translocation, RRMM, Plasma Cell Neoplasms, KTX-1001


You can join if…

Open to people ages 18 years and up

  • ≥ 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)

You CAN'T join if...

  • 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.


  • UCSF Medical Center - Hematology and Blood and Marrow Transplant Clinic accepting new patients
    San Francisco California 94143 United States
  • Mayo Clinic Hospital - Phoenix accepting new patients
    Phoenix Arizona 85259 United States

Lead Scientist at UCSF

  • Alfred Chung, MD
    Dr. Alfred Chung is a hematologist-oncologist who cares for patients with blood cancers, including leukemias, lymphomas and myelomas. He sees patients who are in the hospital for treatment, including those who need a bone marrow transplant and those receiving chemotherapy or cellular therapies to bolster the immune system.


accepting new patients
Start Date
Completion Date
K36 Therapeutics, Inc.
Phase 1 research study
Study Type
Expecting 60 study participants
Last Updated