What side effects are associated with this type of intervention?

Common treatments for Primary Myelofibrosis include ruxolitinib, fedratinib, and hydroxyurea. Ruxolitinib can cause anemia, thrombocytopenia, and increased risk of infections. Fedratinib may lead to gastrointestinal symptoms, anemia, and liver enzyme abnormalities. Hydroxyurea is associated with myelosuppression, gastrointestinal disturbances, and skin ulcers. For LSD1 inhibitors like Bomedemstat, specific side effects are not detailed, but they generally aim to manage symptoms and improve quality of life with a focus on long-term safety and efficacy.

What prior FDA approvals exist?

The FDA has approved several treatments for Primary Myelofibrosis, including ruxolitinib, a JAK1/JAK2 inhibitor, and fedratinib, another JAK2 inhibitor. These drugs target the JAK-STAT pathway, which is often dysregulated in myelofibrosis. While Bomedemstat (an LSD1 inhibitor) is being studied for its potential in treating myelofibrosis, there are no current FDA-approved LSD1 inhibitors for this condition. The focus of existing approved treatments is primarily on JAK inhibition to manage symptoms and modify disease progression.

What other types of research exist?

Promising novel alternatives to Bomedemstat for Primary Myelofibrosis patients in 2024 include the combination of ruxolitinib and panobinostat, which has shown safety and tolerability in a phase I trial. Other potential treatments could involve new JAK inhibitors or combination therapies that are currently under investigation in clinical trials.

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Epigenetic Modulator

Bomedemstat for Myeloproliferative Disorders

Phase 2

Waitlist Available

Research Sponsored by Imago BioSciences,Inc.

Eligibility Criteria Checklist

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

Must have

Be older than 18 years old

Must not have

Females who are pregnant or breastfeeding or plan to become pregnant or breastfeed during the study

Current use of a prohibited medication (e.g., romiplostim)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to approximately 3 years

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing the safety and effectiveness of bomedemstat, a daily oral medication, in patients with a blood disorder called MPN. The drug works by controlling blood cell production.

Who is the study for?

This trial is for patients with myeloproliferative disorders who have already completed a treatment period in a previous bomedemstat study. They must be assessed by the investigator as suitable for continued dosing and agree to use contraception. Excluded are those on certain medications, unable to consent or comply, pregnant or breastfeeding women, participants in other trials, and anyone non-compliant in past studies.

What is being tested?

The trial tests the long-term safety and effectiveness of an oral drug called bomedemstat (MK-3543) for people with blood disorders like primary myelofibrosis and essential thrombocythemia. It's an extension study for those who've been part of prior related research.

What are the potential side effects?

While specific side effects aren't listed here, typical concerns may include digestive issues, fatigue, liver function changes, bleeding problems due to platelet count alterations. Participants' experiences will vary.

Eligibility Criteria

Exclusion Criteria

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

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I am not pregnant, breastfeeding, nor planning to during the study.

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I am not taking any medications that are not allowed in the study.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to approximately 3 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to approximately 3 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to approximately 3 years for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Essential Thrombocythemia (ET) Participants Only: Change from Baseline in Platelet Counts

Myelofibrosis (MF) Participants Only: Change from Baseline in Spleen Volume

Number of Participants who Experience a Serious Adverse Event (SAE)

+3 more

Other study objectives

Change from Baseline in Complete Blood Count (CBC)

Change from Baseline in Spleen Size

Change from Baseline in the Mutant (Variant) Allele Burden

+7 more

Side effects data

From 2022 Phase 1 & 2 trial • 90 Patients • NCT03136185

55%

Thrombocytopenia

36%

Contusion

27%

Dysgeusia

27%

Oedema peripheral

27%

Arthralgia

27%

Back pain

27%

Depression

27%

Nausea

27%

Anaemia

27%

Constipation

27%

Hypocalcaemia

18%

Dyspnoea

18%

Fatigue

18%

Palpitations

18%

Decreased appetite

18%

Muscular weakness

18%

Hyperuricaemia

18%

Hyponatraemia

18%

Cough

18%

Fall

18%

Asthenia

18%

Abdominal pain

18%

Alopecia

18%

Lymphopenia

18%

Blood uric acid increased

18%

Insomnia

18%

Pruritus

18%

Dizziness

18%

Diarrhoea

18%

Pain in extremity

18%

Hypertension

18%

International normalised ratio increased

18%

Activated partial thromboplastin time prolonged

18%

Dry mouth

18%

Urine abnormality

Pneumonitis

Urinary incontinence

Skin ulcer

Cellulitis

Hypotension

Gout

Headache

Wound secretion

Blood bilirubin increased

Blood magnesium decreased

Blood thyroid stimulating hormone increased

Hypophosphataemia

Petechiae

Hyperglycaemia

Non-cardiac chest pain

Abdominal distension

Stress cardiomyopathy

Lacrimation increased

Myalgia

Blood albumin decreased

Sinus tachycardia

Sepsis

Musculoskeletal chest pain

Blood lactate dehydrogenase increased

Aortic arteriosclerosis

Confusional state

Abdominal pain upper

Blood creatinine increased

Bone pain

Epistaxis

Balance disorder

Eye oedema

Dry skin

Splenic infarction

Catathrenia

Traumatic haematoma

Leukopenia

Rash

Blood alkaline phosphatase increased

Hypokalaemia

Breast pain

Pelvic pain

Anal pruritus

Myocardial ischaemia

Neck pain

Flank pain

Disturbance in attention

Hyperhidrosis

Eczema

Pyrexia

Dysphagia

Hyperactive pharyngeal reflex

Rash maculo-papular

Heart rate irregular

Cachexia

Nail disorder

Stomatitis

Neuralgia

Pleural effusion

Flatulence

Haematoma

Neutropenia

Pallor

Chest pain

Drooling

Vomiting

Early satiety

Calcium ionised decreased

Mouth haemorrhage

Vulvovaginal pruritus

Abdominal wall haematoma

Hypervolaemia

100%

80%

60%

40%

20%

Study treatment Arm

Ph 2b PPV-MF: Bomedemstat 0.6 mg/kg/d

Ph 1/2a PMF: Bomedemstat 0.25 mg/kg/d

Ph 1/2a PPV-MF: Bomedemstat 0.25 mg/kg/d

Ph 1/2a PET-MF: Bomedemstat 0.25 mg/kg/d

Ph 2b PMF: Bomedemstat 0.5 mg/kg/d

Ph 2b PPV-MF: Bomedemstat 0.5 mg/kg/d

Ph 2b PET-MF: Bomedemstat 0.5 mg/kg/d

Ph 2b PMF: Bomedemstat 0.6 mg/kg/d

Ph 2b PET-MF: Bomedemstat 0.6 mg/kg/d

Awards & Highlights

No Placebo-Only Group

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

Trial Design

1Treatment groups

Experimental Treatment

Group I: BomedemstatExperimental Treatment1 Intervention

Participants will receive bomedemstat daily for 169 days with additional treatment continuing in participants deriving clinical benefit.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Bomedemstat

2021

Completed Phase 2

~250

0 / 2Eligibility 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 Primary Myelofibrosis (PMF) include JAK inhibitors and LSD1 inhibitors. JAK inhibitors, such as ruxolitinib and fedratinib, work by inhibiting the overactive Janus kinase pathway, thereby reducing symptoms like splenomegaly and improving quality of life. LSD1 inhibitors, like bomedemstat, target the enzyme lysine-specific demethylase 1 (LSD1), which plays a role in epigenetic regulation. By inhibiting LSD1, these drugs can potentially alter gene expression involved in the proliferation and survival of malignant cells. These mechanisms are important for PMF patients as they offer targeted therapeutic options that can effectively manage symptoms and potentially modify disease progression.

Advances in myelofibrosis: a clinical case approach.Targeting myeloproliferative neoplasms with JAK inhibitors.