What side effects are associated with this type of intervention?

Common treatments for Multiple Myeloma, such as bortezomib, dexamethasone, and other proteasome inhibitors, often result in side effects including fatigue, gastrointestinal issues (diarrhea, constipation), peripheral neuropathy, and infections. Combination therapies, like those involving carfilzomib or pomalidomide, can also lead to hypertension, cardiac events, and hematologic toxicities such as anemia, thrombocytopenia, and neutropenia. These side effects vary in severity and frequency, and treatment regimens are often adjusted to manage them effectively.

What prior FDA approvals exist?

The FDA has approved several treatments for Multiple Myeloma that target various pathways. Notably, proteasome inhibitors like bortezomib and carfilzomib, immunomodulatory drugs such as lenalidomide and pomalidomide, and monoclonal antibodies like daratumumab have been approved. These treatments work through different mechanisms, including proteasome inhibition, immune modulation, and targeting specific antigens on myeloma cells. While specific MMSET inhibitors are not yet approved, these existing treatments offer a range of therapeutic options for managing Multiple Myeloma.

What other types of research exist?

Promising novel alternatives to MMSET inhibitors for Multiple Myeloma patients include: 1) Proteasome inhibitors like ixazomib, which has shown efficacy in relapsed disease. 2) Immunomodulatory drugs such as lenalidomide and pomalidomide. 3) Monoclonal antibodies like daratumumab, which can be used alone or in combination with other agents. 4) Combination regimens like daratumumab plus CyBorD (cyclophosphamide, bortezomib, and dexamethasone). These alternatives have demonstrated effectiveness in clinical trials and offer various mechanisms of action to target multiple myeloma.

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Histone Lysine Methyltransferase Inhibitor

MMSET Inhibitor for Multiple Myeloma

Phase 1

Recruiting

Research Sponsored by K36 Therapeutics, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

≥ 18 years of age

ECOG score ≤ 2

Must not have

Radiation, chemotherapy, immunotherapy, or any other anticancer therapy ≤ 2 weeks

Cellular therapies ≤ 8 weeks

Timeline

Screening 3 weeks

Treatment Varies

Follow Up duration of study

Awards & highlights

No Placebo-Only Group

Summary

This trial tests a new pill that blocks an enzyme to help treat adults with a hard-to-treat type of blood cancer. The goal is to see if it is safe and effective.

Who is the study for?

This trial is for adults with relapsed or refractory multiple myeloma who have tried all other treatments without success. They must have certain levels of M protein in their urine or blood, and some may need to show specific genetic changes related to their cancer. People can't join if they've had a recent transplant, major surgery, other active cancers, inadequate organ function, or certain infections.

What is being tested?

The study tests KTX-1001, an oral drug designed to inhibit MMSET activity in patients with multiple myeloma that has come back after treatment or hasn't responded to therapy. It's a Phase I trial focused on safety and how well the body tolerates the drug.

What are the potential side effects?

While not specified here, side effects could include typical reactions seen with cancer therapies such as nausea, fatigue, liver issues (due to metabolism of the drug), potential blood disorders (as it affects bone marrow cells), and possibly unique effects from inhibiting MMSET.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am 18 years old or older.

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I can take care of myself and am up and about more than 50% of my waking hours.

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My multiple myeloma has returned or is not responding to treatment.

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I have had at least 3 treatments including a proteasome inhibitor, an immunomodulatory drug, and an anti-CD38 antibody.

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I have tried all treatments for my condition without success or cannot tolerate them.

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My test results show I have the t(4;14) genetic change or a specific mutation in MMSET.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I haven't had cancer treatment like radiation or chemotherapy in the last 2 weeks.

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I have not had cellular therapy in the last 8 weeks.

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I had a stem cell transplant using my own cells less than 100 days ago.

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I have never had plasma cell leukemia, POEMS syndrome, solitary bone lesion, myelodysplastic syndrome, myeloproliferative neoplasm, or light chain amyloidosis.

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I have an active brain or spinal cord disease.

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My bone marrow is not working properly.

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My kidney, liver, lung, or heart function is not normal.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ cycle 1 (28 days)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~cycle 1 (28 days)

This trial's timeline: 3 weeks for screening, Varies for treatment, and cycle 1 (28 days) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Dose Escalation: Determination of Recommended Phase 2 Dose (RP2D) and/or Maximum Tolerated Dose (MTD)

Secondary study objectives

Disease Specific Response to KTX-1001± Combination Therapy

Pharmacokinetics & Pharmacodynamics KTX-1001± Combination Therapy

Safety profile of KTX-1001± Combination Therapy

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

3Treatment groups

Experimental Treatment

Group I: Cohort D (pomalidomide): KTX-1001 + DEX + pomalidomideExperimental Treatment1 Intervention

Cohort D (pomalidomide): KTX-1001 at RP2D1/2 + DEX + pomalidomide

Group II: Cohort C (carfilzomib): KTX-1001 + DEX + carfilzomibExperimental Treatment1 Intervention

Cohort C1 (carfilzomib): KTX-1001 at RP2D1 + DEX + carfilzomib Cohort C2 (carfilzomib): KTX-1001 at RP2D2 + DEX + carfilzomib

Group III: Cohort A (Single agent): KTX-1001 + dexamethasone (DEX)Experimental Treatment1 Intervention

Cohort A1 (single agent): KTX-1001 at RP2D1 + DEX Cohort A2 (single agent): KTX-1001 at RP2D2 + DEX

0 / 13Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Multiple Myeloma include proteasome inhibitors (e.g., bortezomib), which block the proteasome's ability to degrade proteins, leading to cancer cell death; immunomodulatory drugs (e.g., lenalidomide), which enhance the immune system's ability to attack myeloma cells; and monoclonal antibodies (e.g., daratumumab), which target specific proteins on the surface of myeloma cells to induce immune-mediated destruction. Novel agents like histone deacetylase inhibitors and MMSET inhibitors (which prevent H3K36 dimethylation) target epigenetic modifications to disrupt cancer cell growth and survival. These treatments are crucial as they offer multiple mechanisms to combat the disease, potentially overcoming resistance and improving patient outcomes.

Breast Cancer Resistance to Cyclin-Dependent Kinases 4/6 Inhibitors: Intricacy of the Molecular Mechanisms.Novel direct and indirect cyclin-dependent kinase modulators for the prevention and treatment of human neoplasms.New drugs in multiple myeloma.