Tests KTX-1001 alone and combined with other drugs in relapsed myeloma

Trial details

Who runs it

K36 Therapeutics, Inc.

Trial code

NCT05651932EU 2022-500801-41-00

Record updated

22 October 2026

Original description

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).Read full original descriptionShow less

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

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).

Inclusion: ≥ 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).

Exclusion: 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; 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; 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; 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; 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; Inadequate bone marrow function.

ClinicalTrials.gov NCT05651932 · EU CTIS 2022-500801-41-00