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A Study of an MMSET Inhibitor in Patients With Relapsed and Refractory Multiple Myeloma

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05651932
Recruitment Status : Recruiting
First Posted : December 15, 2022
Last Update Posted : May 3, 2023
Sponsor:
Information provided by (Responsible Party):
K36 Therapeutics, Inc.

Study Description
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Brief 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).

Condition or disease Intervention/treatment Phase
Multiple Myeloma Myeloma Myeloma Multiple Drug: KTX-1001 Phase 1

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 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.

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 60 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : February 22, 2023
Estimated Primary Completion Date : December 2024
Estimated Study Completion Date : October 2025

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Multiple Myeloma

Arms and Interventions
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Arm Intervention/treatment
Experimental: KTX-1001
KTX-1001 will be administered orally, daily for 28 days.
 Drug: KTX-1001
KTX-1001 will be administered orally, daily for 28 days.


Outcome Measures
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Primary Outcome Measures :
  1. Incidence of dose-limiting toxicity (DLTs) [ Time Frame: Cycle 1 (28 days) ]
    Treatment-emergent adverse events (AEs), treatment-related AEs, and clinically significant changes in laboratory test results will be evaluated


Secondary Outcome Measures :
  1. Maximum plasma concentration (Cmax) of KTX-1001 [ Time Frame: Cycle 1 (28 days) ]
  2. Time to achieve Cmax (tmax) for KTX-1001 [ Time Frame: Cycle 1 (28 days) ]
  3. Area under the plasma concentration-time curve (AUC) for KTX-1001 [ Time Frame: Cycle 1 (28 days) ]
  4. Objective response rate (ORR) for KTX-1001 [ Time Frame: Cycle 1 (28 days) ]
    Per IMWG Consensus Criteria for Response and Minimal Residual Disease Assessment in Multiple Myeloma

  5. Duration of response (DOR) for KTX-1001 [ Time Frame: Cycle 1 (28 days) ]
  6. Progression-free survival (PFS) for KTX-1001 [ Time Frame: Cycle 1 (28 days) ]
  7. Overall survival (OS) for KTX-1001 [ Time Frame: Cycle 1 (28 days) ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  • ≥ 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)

Key Exclusion Criteria:

  • 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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05651932


Locations
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United States, Arizona
Mayo Clinic Recruiting
Scottsdale, Arizona, United States, 85259
Contact: Peter Bergsagel, MD    855-776-0015    bergsagel.leif@mayo.edu   
Contact: Erick Gaxiola Perez    855-776-0015    Gaxiola.Erick@mayo.edu   
United States, California
University of California, San Francisco Recruiting
San Francisco, California, United States, 94143
Contact: Alfred Chung, MD    415-502-4215    alfred.chung@ucsf.edu   
Contact: Benjamin Sun    415-476-9608    Benjamin.Sun@ucsf.edu   
United States, Florida
Mayo Clinic Recruiting
Jacksonville, Florida, United States, 32224
Contact: Vivek Roy, MD    855-776-0015    Roy.Vivek@mayo.edu   
Contact: Justin Guidry    855-776-0015    Guidry.Justin@mayo.edu   
United States, Massachusetts
Massachusetts General Hospital Cancer Center Recruiting
Boston, Massachusetts, United States, 02114
Contact: Andrew Yee, MD    614-724-4000    AYEE1@mgh.harvard.edu   
Contact: Marianela Oser       MOSER2@mgh.harvard.edu   
United States, Minnesota
Mayo Clinic Recruiting
Rochester, Minnesota, United States, 55905
Contact: David Dingli, MD PhD    855-776-0015    dingli.david@mayo.edu   
Contact: Thomas Nelson    855-776-0015    Nelson.Thomas2@mayo.edu   
United States, New Jersey
Hackensack University Medical Center Recruiting
Hackensack, New Jersey, United States, 07601
Contact: David Siegel, MD    551-996-8704    davids.siegel@hmhn.org   
Contact: Xueru Mu    551-996-8170    xueru.mu@hmhn.org   
United States, North Carolina
Duke University Hospital Recruiting
Durham, North Carolina, United States, 27705
Contact: Cristina Gasparetto, MD    919-668-8222    gaspa001@mc.duke.edu   
United States, Tennessee
Sarah Cannon Research Institute at Tennessee Oncology Recruiting
Nashville, Tennessee, United States, 37203
Contact: Jesus Berdeja, MD    615-320-5090    jberdeja@tnonc.com   
Contact: Sarah Ladd    615-524-4133    sarah.ladd@sarahcannon.com   
Sponsors and Collaborators
K36 Therapeutics, Inc.
More Information
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Responsible Party: K36 Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT05651932    
Other Study ID Numbers: KTX-MMSET-001
EUCTR No: 2022-500801-41-00 ( Other Identifier: European Medicines Agency )
First Posted: December 15, 2022    Key Record Dates
Last Update Posted: May 3, 2023
Last Verified: April 2023

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by K36 Therapeutics, Inc.:
NSD2
MMSET
WHSC1
T4;14
 T(4;14)
translocation
myeloma
RRMM
Additional relevant MeSH terms:
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Multiple Myeloma
Neoplasms, Plasma Cell
Neoplasms by Histologic Type
Neoplasms
Hemostatic Disorders
Vascular Diseases
Cardiovascular Diseases
 Paraproteinemias
Blood Protein Disorders
Hematologic Diseases
Hemorrhagic Disorders
Lymphoproliferative Disorders
Immunoproliferative Disorders
Immune System Diseases