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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 belowSelect...
My last hormone therapy didn’t work, needing a new treatment.
Select...
I have been diagnosed with essential thrombocythemia according to WHO standards.
Select...
My bone marrow fibrosis is low grade.
Select...
My white blood cell count is high enough for treatment.
Select...
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
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 moreSide effects data
From 2022 Phase 1 & 2 trial • 90 Patients • NCT0313618555%
Thrombocytopenia
36%
Contusion
27%
Arthralgia
27%
Dysgeusia
27%
Back pain
27%
Nausea
27%
Anaemia
27%
Depression
27%
Constipation
27%
Oedema peripheral
27%
Hypocalcaemia
18%
Activated partial thromboplastin time prolonged
18%
Muscular weakness
18%
Palpitations
18%
Dyspnoea
18%
Hyperuricaemia
18%
Fatigue
18%
Hyponatraemia
18%
Cough
18%
Fall
18%
Lymphopenia
18%
Blood uric acid increased
18%
Pruritus
18%
Insomnia
18%
Dizziness
18%
Abdominal pain
18%
Diarrhoea
18%
Alopecia
18%
Pain in extremity
18%
Asthenia
18%
Decreased appetite
18%
Hypertension
18%
International normalised ratio increased
18%
Dry mouth
18%
Urine abnormality
9%
Drooling
9%
Dysphagia
9%
Skin ulcer
9%
Confusional state
9%
Hypotension
9%
Headache
9%
Neck pain
9%
Blood bilirubin increased
9%
Blood magnesium decreased
9%
Chest pain
9%
Wound secretion
9%
Petechiae
9%
Hyperglycaemia
9%
Non-cardiac chest pain
9%
Cellulitis
9%
Stress cardiomyopathy
9%
Myalgia
9%
Balance disorder
9%
Sinus tachycardia
9%
Sepsis
9%
Musculoskeletal chest pain
9%
Blood lactate dehydrogenase increased
9%
Eye oedema
9%
Blood creatinine increased
9%
Splenic infarction
9%
Epistaxis
9%
Vomiting
9%
Catathrenia
9%
Traumatic haematoma
9%
Leukopenia
9%
Rash
9%
Hypokalaemia
9%
Breast pain
9%
Pelvic pain
9%
Anal pruritus
9%
Myocardial ischaemia
9%
Hypophosphataemia
9%
Mouth haemorrhage
9%
Blood albumin decreased
9%
Urinary incontinence
9%
Vulvovaginal pruritus
9%
Pneumonitis
9%
Pallor
9%
Disturbance in attention
9%
Hyperhidrosis
9%
Eczema
9%
Abdominal distension
9%
Bone pain
9%
Hyperactive pharyngeal reflex
9%
Heart rate irregular
9%
Cachexia
9%
Nail disorder
9%
Stomatitis
9%
Neuralgia
9%
Pyrexia
9%
Abdominal pain upper
9%
Rash maculo-papular
9%
Haematoma
9%
Neutropenia
9%
Early satiety
9%
Calcium ionised decreased
9%
Gout
9%
Dry skin
9%
Pleural effusion
9%
Lacrimation increased
9%
Flatulence
9%
Aortic arteriosclerosis
9%
Flank pain
9%
Blood alkaline phosphatase increased
9%
Blood thyroid stimulating hormone increased
9%
Abdominal wall haematoma
9%
Hypervolaemia
100%
80%
60%
40%
20%
0%
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
Find a Location
Who is running the clinical trial?
Merck Sharp & Dohme LLCLead Sponsor
4,032 Previous Clinical Trials
5,189,501 Total Patients Enrolled
Medical DirectorStudy DirectorMerck Sharpe & Dohme LLC
2,905 Previous Clinical Trials
8,091,169 Total Patients Enrolled