A Study of an MMSET Inhibitor in Patients With Relapsed and Refractory Multiple Myeloma
18 Years and older, Male and Female
KTX-MMSET-001 (primary)
NCI-2023-01697
EUCTR No: 2022-500801-41-00
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).
Objectives
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.
Eligibility
- = 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)
Treatment Sites in Georgia
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