Bomedemstat for Blood Disorders

Recruiting at 22 trial locations

Overseen By Toll Free Number

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Merck Sharp & Dohme LLC

Must be taking: Bomedemstat

Disqualifiers: Other study, Noncompliance, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

400 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests bomedemstat, a new potential drug, for individuals with certain blood disorders like essential thrombocythemia (ET) and polycythemia vera (PV). The main goal is to assess the treatment's long-term safety and effectiveness. Participants must have been in a previous bomedemstat study for at least six months, shown benefit from the treatment, and experienced no major side effects. Those managing their condition well with bomedemstat and able to take pills at home might be suitable for this trial. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but it mentions that participants should not have received prohibited concomitant medications. It's best to discuss your current medications with the trial team to see if they are allowed.

Is there any evidence suggesting that bomedemstat is likely to be safe for humans?

Research has shown that bomedemstat is generally safe for patients. In earlier studies, no serious safety issues or deaths were linked to bomedemstat, even for patients who used it for up to 979 days. After 24 weeks, 86% of patients remained in the study, indicating good tolerance. Additionally, the U.S. Food and Drug Administration has given special recognition to bomedemstat, highlighting its potential safety and effectiveness for certain blood disorders. ^{1 2 3 4 5}

Why are researchers excited about this study treatment for blood disorders?

Bomedemstat is unique because it targets an enzyme called LSD1 (lysine-specific demethylase 1), which plays a crucial role in the development and progression of certain blood disorders. Unlike traditional treatments that often focus on controlling symptoms or slowing disease progression, Bomedemstat works directly by inhibiting this enzyme, potentially altering the disease's underlying mechanisms. Researchers are excited about Bomedemstat because it offers a novel approach with the promise of more targeted and effective treatment, potentially improving outcomes for patients over the long term.

What evidence suggests that bomedemstat might be an effective treatment for blood disorders?

Research has shown that bomedemstat effectively treats blood disorders such as essential thrombocythemia (ET) and polycythemia vera (PV). Studies have found that bomedemstat helps manage blood cell levels in many patients with ET. Early results also suggest that combining bomedemstat with ruxolitinib improves symptoms and reduces spleen size. Additionally, the U.S. Food and Drug Administration has given bomedemstat special recognition for its potential benefits in treating these conditions. These findings strongly support bomedemstat's effectiveness for these blood disorders. ^{3 5 6 7 8}

Who Is on the Research Team?

Medical Director

Principal Investigator

Merck Sharp and Dohme LLC

Are You a Good Fit for This Trial?

This trial is for people with certain blood disorders like Polycythemia, Essential Thrombocythemia, or Myelofibrosis who have been part of previous bomedemstat studies. They must be able to take oral medication and have shown benefits from at least 6 months of prior treatment with bomedemstat.

Inclusion Criteria

I can take pills and follow home dosing instructions.

I am part of a bomedemstat study by Imago Biosciences or MSD, approved for MK-3543-017.

I've been on bomedemstat for 6+ months, tolerated it well, and it's working for me.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Transition

Participants transition from a prior study to the extension study to continue receiving bomedemstat

Up to 10 years

Follow-up

Participants are monitored for safety and efficacy of long-term bomedemstat treatment

Up to 10 years

What Are the Treatments Tested in This Trial?

Interventions

• Bomedemstat

Trial Overview

The study tests the long-term safety and effectiveness of a drug called Bomedemstat in patients who've previously responded well to it. It's an extension study without hypothesis testing, meaning they're not trying to prove a specific theory but rather gather more data.

How Is the Trial Designed?

Group I: Bomedemstat Experimental Treatment 1 Intervention

Participants will receive oral capsules of bomedemstat once daily for up to 10 years, with the starting dose as the same dose that the participant was on at the time of transition from the feeder study.

Who Is Running the Clinical Trial?

Merck Sharp & Dohme LLC

Lead Sponsor

Trials

4,096

Recruited

5,232,000+

Chirfi Guindo

Merck Sharp & Dohme LLC

Chief Marketing Officer since 2022

Degree in Engineering from Ecole Centrale de Paris, MBA from New York University Stern School of Business

Robert M. Davis

Merck Sharp & Dohme LLC

Chief Executive Officer since 2021

JD from Northwestern University Pritzker School of Law, MBA from Northwestern University Kellogg Graduate School of Management, Bachelor's in Finance from Miami University

Published Research Related to This Trial

Nine LSD1 inhibitors have progressed to clinical trials for treating hematologic malignancies, showing promise as either standalone or combination therapies.

Innovative design strategies, such as the unique binding mode of quinazoline derivatives, are paving the way for the development of reversible LSD1 inhibitors, which may help minimize side effects in patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Recent advances of LSD1/KDM1A inhibitors for disease therapy. Zhang, C., Wang, Z., Shi, Y., et al.[2023]

Lysine-specific demethylase 1 (LSD1) is crucial for regulating cell differentiation and proliferation, and various inhibitors have been developed to target its activity in cancer treatment.

Among the LSD1 inhibitors studied, iadademstat (ORY-1001) was found to be the most potent, while many commonly used tool compounds showed low activity and selectivity, indicating that results from these compounds should be interpreted cautiously.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Comprehensive in Vitro Characterization of the LSD1 Small Molecule Inhibitor Class in Oncology. Sacilotto, N., Dessanti, P., Lufino, MMP., et al.[2022]

Iadademstat, a selective LSD1 inhibitor, was found to be safe and well-tolerated in a phase I trial involving 27 patients with relapsed/refractory acute myeloid leukemia (R/R AML), with a recommended dose of 140 µg/m2/d established for further treatment.

The treatment showed promising signs of efficacy, including reductions in blood and bone marrow blast percentages and one complete remission, particularly in patients with MLL/KMT2A-rearranged AML, indicating its potential as a therapeutic option.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

First-in-Human Phase I Study of Iadademstat (ORY-1001): A First-in-Class Lysine-Specific Histone Demethylase 1A Inhibitor, in Relapsed or Refractory Acute Myeloid Leukemia. Salamero, O., Montesinos, P., Willekens, C., et al.[2021]

A new series of derivatives of the LSD1 inhibitor S2157 were developed to improve safety and efficacy, particularly by reducing hERG channel inhibition and enhancing microsomal stability.

The most promising derivative, S1427, demonstrated strong LSD1 inhibitory activity while maintaining favorable safety profiles, making it a potential candidate for cancer treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Design and Synthesis of Tranylcypromine-Derived LSD1 Inhibitors with Improved hERG and Microsomal Stability Profiles. Koda, Y., Sato, S., Yamamoto, H., et al.[2023]

Viscosalactone B has been identified as a novel and selective inhibitor of lysine-specific demethylase 1 (LSD1), showing potent antiproliferative effects against various prostate cancer cell lines with IC50 values ranging from 0.72 to 3.86 μM.

In vivo studies demonstrated that viscosalactone B effectively inhibits tumor growth without causing significant cytotoxicity to major organs in nude mice, suggesting its potential as a safe treatment option for prostate cancer.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Viscosalactone B, a natural LSD1 inhibitor, inhibits proliferation in vitro and in vivo against prostate cancer cells. Yan, G., Zhang, H., Li, Y., et al.[2023]

Citations

1.

clinicaltrials.gov

clinicaltrials.gov/study/NCT06351631

NCT06351631 | A Study to Evaluate Safety and Efficacy of ...

The study will include participants who are safely tolerating bomedemstat, receiving clinical benefit from its use in estimation of the investigator, and have ...

2.

ashpublications.org

ashpublications.org/blood/article/142/Supplement%201/621/499277/Phase-2-Study-to-Assess-the-Safety-and-Efficacy-of

Phase 2 Study to Assess the Safety and Efficacy of ...

These early results suggest that the addition of bomedemstat to a ruxolitinib regimen is well tolerated, improves splenomegaly and symptom scores, and ...

3.

merck.com

merck.com/news/merck-announces-phase-3-trial-initiation-for-bomedemstat-an-investigational-candidate-for-the-treatment-of-certain-patients-with-essential-thrombocythemia/

Merck Announces Phase 3 Trial Initiation for Bomedemstat, ...

Bomedemstat has U.S. Food and Drug Administration Orphan Drug and Fast Track Designations for the treatment of ET and myelofibrosis (MF), Orphan ...

4.

sciencedirect.com

sciencedirect.com/science/article/abs/pii/S000649712305351X

Bomedemstat (IMG-7289), an LSD1 Inhibitor, Manages the ...

This study demonstrates that treatment with bomedemstat results in a consistent hematologic response in the majority of patients with ET.

5.

cancer.gov

cancer.gov/research/participate/clinical-trials-search/v?id=NCI-2024-01821

A Study of Bomedemstat (IMG-7289/MK-3543) Compared ...

The primary study hypothesis is that bomedemstat is superior to the best available therapy with respect to durable clinicohematologic response (DCHR).

6.

clinicaltrials.gov

clinicaltrials.gov/study/NCT06351631

NCT06351631 | A Study to Evaluate Safety and Efficacy of ...

The study will include participants who are safely tolerating bomedemstat, receiving clinical benefit from its use in estimation of the investigator, and have ...

7.

ashpublications.org

ashpublications.org/blood/article/142/Supplement%201/747/502285/Bomedemstat-IMG-7289-an-LSD1-Inhibitor-Manages-the

Bomedemstat (IMG-7289), an LSD1 Inhibitor, Manages the ...

There have been no safety signals or deaths related to drug in patients treated for up to 979 days. At Wk 24, 86% patients remained on study and ...

8.

clinicaltrials.gov

clinicaltrials.gov/study/NCT05223920

An Extension Study of Bomedemstat (IMG-7289/MK-3543) ...

This is a multi-center, open-label extension study to assess the long-term safety and efficacy of bomedemstat administered orally once daily in participants ...