NCT05651932

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

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 125 (estimated)
• Sponsor: K36 Therapeutics, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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).

Detailed description

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 t(4;14) will receive KTX-1001 at the RP2D alone and in combination with investigational therapy Mezigdomide or SOC therapy (dexamethasone, carfilzomib or pomalidomide) to further define safety and tolerability and provide preliminary efficacy information.

Conditions

• Multiple Myeloma
• Myeloma
• Myeloma Multiple

Interventions

Type	Name	Description
DRUG	Cohort A1 & A2: KTX-1001	KTX-1001: Orally for 28 days each cycle until progression. Dexamethasone: Orally once weekly
DRUG	Cohort B1 & B2: KTX-1001+Mezigdomide	Drug: KTX-1001: Orally for 28 days each cycle until progression Drug: Dexamethasone: Orally once weekly Drug: Mezigdomide Dexamethasone: Orally once weekly
DRUG	Cohort C1 & C2: KTX-1001 + Carfilzomib (KYPROLIS®)	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	Cohort D: KTX-1001+ pomalidomide (Pomalyst, Imnovid)	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

Timeline

• Start date: 2023-02-22
• Primary completion: 2027-12-31
• Completion: 2028-06-30
• First posted: 2022-12-15
• Last updated: 2026-02-10

Locations

22 sites across 4 countries: United States, Canada, France, Spain

Regulatory

• FDA-regulated drug study