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Testing a new drug for patients with advanced 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

Phase 1 Interventional K36 Therapeutics, Inc. · NCT05651932

This study is testing a new drug called KTX-1001 to see if it is safe and effective for adults with advanced multiple myeloma who have not responded to other treatments.

Quick facts

PhasePhase 1
Study typeInterventional
Enrollment125 (estimated)
Ages18 Years and up
SexAll
SponsorK36 Therapeutics, Inc. Industry-sponsored
Drugs / interventionschemotherapy, immunotherapy, Radiation
Locations22 sites (San Francisco, California and 21 other locations)
Trial IDNCT05651932 on ClinicalTrials.gov

What this trial studies

This Phase I study evaluates the safety and tolerability of KTX-1001, a selective small molecule inhibitor targeting the MMSET enzyme, in adults with relapsed or refractory multiple myeloma. The trial consists of a dose escalation phase to determine the maximum tolerated dose and a dose expansion phase focusing on patients with the t(4;14) genetic alteration. Participants will receive KTX-1001 alone or in combination with standard of care therapies to assess its efficacy and safety profile.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 and older with relapsed or refractory multiple myeloma who have undergone at least three prior lines of therapy.

Not a fit: Patients who have not been previously treated for multiple myeloma or those with early-stage disease may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with difficult-to-treat multiple myeloma.

How similar studies have performed: While this approach is novel, similar studies targeting specific genetic alterations in multiple myeloma have shown promise in the past.

Eligibility criteria

Show full inclusion / exclusion criteria 
Key Inclusion Criteria for Dose-Expansion:

* ≥ 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)

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
* 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
* 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.

Where this trial is running

San Francisco, California and 21 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Multiple MyelomaMyelomaMyeloma MultipleNSD2MMSETWHSC1T414
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.