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Epigenetic Modulator

Bomedemstat for Essential Thrombocythemia

Phase 3

Recruiting

Research Sponsored by Merck Sharp & Dohme LLC

Eligibility Criteria Checklist

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

Must have

Has an inadequate or loss of response to their most recent prior ET therapy, requiring a change of cytoreductive therapy

Has a diagnosis of ET per WHO 2016 diagnostic criteria for myeloproliferative neoplasms

Must not have

Human immunodeficiency virus (HIV)-infected participants with a history of Kaposi's sarcoma and/or Multicentric Castleman's Disease

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 180 weeks

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

This trial is testing a new drug called bomedemstat to see if it is better than the current best available treatment for patients with essential thrombocythemia who do not respond well to or

Who is the study for?

This trial is for individuals with essential thrombocythemia who haven't responded well to or can't tolerate hydroxyurea. They should have a life expectancy over one year, agree to contraception if they can father children, and not be pregnant or breastfeeding if female. A bone marrow fibrosis score of Grade 0 or 1 is required, along with certain blood cell count levels.

What is being tested?

The study tests bomedemstat against the best available treatments like Busulfan and Ruxolitinib in patients with essential thrombocythemia. It aims to see if bomedemstat leads to better long-term control of blood cell counts compared to other options.

What are the potential side effects?

Potential side effects may include those common to cancer therapies such as fatigue, digestive issues, changes in blood counts leading to increased infection risk or bleeding problems, liver function changes, and possible allergic reactions.

Eligibility Criteria

Inclusion Criteria

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

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My last hormone therapy didn’t work, needing a new treatment.

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I have been diagnosed with essential thrombocythemia according to WHO standards.

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My bone marrow fibrosis is low grade.

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My white blood cell count is high enough for treatment.

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I have not responded well or had a bad reaction to hydroxyurea.

Exclusion Criteria

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

Select...

I am HIV positive with a history of Kaposi's sarcoma or Castleman's Disease.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 180 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 180 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 180 weeks for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Durable Clinicohematologic Response (DCHR) Rate

Secondary study objectives

Change From Baseline in Myelofibrosis Symptom Assessment Form (MFSAF) v4.0 Individual Fatigue Symptom Item Score

Change From Baseline in Patient-reported Outcomes Measurement Information System (PROMIS) Fatigue SF-7a Total Fatigue Score

Change From Baseline in Total Symptom Score as Measured on the MFSAF v4.0

+8 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%

Muscular weakness

18%

Decreased appetite

18%

Dyspnoea

18%

Palpitations

18%

Fatigue

18%

Hyperuricaemia

18%

Hyponatraemia

18%

Cough

18%

Fall

18%

Asthenia

18%

Abdominal pain

18%

Alopecia

18%

Lymphopenia

18%

Blood uric acid increased

18%

Pruritus

18%

Insomnia

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

Cellulitis

Pneumonitis

Dysphagia

Vomiting

Skin ulcer

Confusional state

Drooling

Urinary incontinence

Hypotension

Gout

Headache

Blood magnesium decreased

Chest pain

Wound secretion

Blood bilirubin increased

Blood thyroid stimulating hormone increased

Hypophosphataemia

Petechiae

Hyperglycaemia

Non-cardiac chest pain

Stress cardiomyopathy

Lacrimation increased

Myalgia

Blood albumin decreased

Sinus tachycardia

Sepsis

Musculoskeletal chest pain

Blood lactate dehydrogenase increased

Aortic arteriosclerosis

Abdominal pain upper

Blood creatinine increased

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

Abdominal distension

Bone pain

Pyrexia

Hyperactive pharyngeal reflex

Heart rate irregular

Cachexia

Nail disorder

Stomatitis

Neuralgia

Rash maculo-papular

Pallor

Pleural effusion

Flatulence

Haematoma

Neutropenia

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.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Experimental Treatment

Active Control

Group I: BomedemstatExperimental Treatment1 Intervention

Participants will begin treatment at a dose of 50 mg of bomedemstat daily. Dosage will be adjusted either up or down within specified time parameters for each participant to the dose that provides sufficient exposure to safely inhibit thrombopoiesis to decrease platelet counts to the target range. All participants will be treated daily for up to 52 weeks, and are eligible for an extended treatment phase up to 152 weeks.

Group II: Best Available TherapyActive Control4 Interventions

Each participant will receive either anagrelide, busulfan, interferon alfa/pegylated interferon alfa, or ruxolitinib as determined by investigator. All participants will be treated per respective approved product labels for up to 52 weeks. Participants receiving BAT for 52 weeks who stop responding to BAT are eligible to switch to bomedemstat and receive this for up to 152 weeks at the investigators discretion.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Bomedemstat

2021

Completed Phase 2

~250

0 / 6Eligibility criteria met