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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06456346




Registration number
NCT06456346
Ethics application status
Date submitted
7/06/2024
Date registered
13/06/2024
Date last updated
15/10/2024

Titles & IDs
Public title
Bomedemstat vs Hydroxyurea for Essential Thrombocythemia (MK-3543-007)
Scientific title
A Phase 3, Randomized, Double-blind, Active-Comparator-Controlled Clinical Study to Evaluate the Efficacy and Safety of Bomedemstat (MK-3543) Versus Hydroxyurea in Cytoreductive Therapy Naïve Essential Thrombocythemia Participants
Secondary ID [1] 0 0
2023-505232-36
Secondary ID [2] 0 0
3543-007
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Essential Thrombocythemia 0 0
Condition category
Condition code
Blood 0 0 0 0
Haematological diseases
Blood 0 0 0 0
Other blood disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Bomedemstat
Treatment: Drugs - Hydroxyurea
Treatment: Drugs - Bomedemstat placebo
Treatment: Drugs - Hydroxyurea placebo

Experimental: Bomedemstat - Participants will receive active bomedemstat and hydroxyurea placebo daily for up to approximately 52 weeks. 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. Participants who complete treatment at Week 52 will be eligible to continue treatment in the extended treatment phase. Unblinding will occur and placebo discontinued once all participants have completed at least 52 weeks of therapy or otherwise discontinued.

Active comparator: Hydroxyurea - Participants will receive active hydroxyurea and bomedemstat placebo daily for up to 52 weeks. 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. Participants who complete treatment at Week 52 will be eligible to continue treatment in the extended treatment phase. Unblinding will occur and placebo discontinued once all participants have completed at least 52 weeks of therapy or otherwise discontinued.


Treatment: Drugs: Bomedemstat
Oral capsule

Treatment: Drugs: Hydroxyurea
Oral capsule

Treatment: Drugs: Bomedemstat placebo
Oral capsule placebo

Treatment: Drugs: Hydroxyurea placebo
Oral capsule placebo
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Durable Clinicohematologic Response (DCHR) Rate
Timepoint [1] 0 0
Up to Week 52
Secondary outcome [1] 0 0
Change From Baseline in Myelofibrosis Symptom Assessment Form version 4.0 (MFSAF v4.0) Individual Fatigue Symptom Item Score
Timepoint [1] 0 0
Baseline and pre-specified timepoints up to Week 52
Secondary outcome [2] 0 0
Change From Baseline in Patient-reported Outcomes Measurement Information System (PROMIS) Fatigue SF-7a Total Fatigue Score
Timepoint [2] 0 0
Baseline and pre-specified timepoints up to Week 52
Secondary outcome [3] 0 0
Change From Baseline in MFSAF v4.0 Total Symptom Score
Timepoint [3] 0 0
Baseline and Week 52
Secondary outcome [4] 0 0
Duration of Hematologic Remission (DOHR)
Timepoint [4] 0 0
Up to Week 52
Secondary outcome [5] 0 0
Number of Participants Who Experience Thrombotic Events
Timepoint [5] 0 0
Up to approximately 52 weeks
Secondary outcome [6] 0 0
Number of Participants Who Experience Major Hemorrhagic Events
Timepoint [6] 0 0
Up to approximately 52 weeks
Secondary outcome [7] 0 0
Disease Progression Rate
Timepoint [7] 0 0
Up to Week 52
Secondary outcome [8] 0 0
Event Free Survival (EFS)
Timepoint [8] 0 0
Up to Week 52
Secondary outcome [9] 0 0
Number of Participants Who Experience One or More Adverse Events (AEs)
Timepoint [9] 0 0
Up to approximately 52 weeks
Secondary outcome [10] 0 0
Number of Participants Who Discontinue Study Intervention Due to an AE
Timepoint [10] 0 0
Up to approximately 52 weeks

Eligibility
Key inclusion criteria
* Diagnosis of Essential Thrombocythemia (ET) based on World Health Organization Criteria for myeloproliferative neoplasms, and an indication for cytoreductive therapy regardless of age or risk status
* Has a bone marrow fibrosis score of Grade 0 or Grade 1, as per a modified version of the European Consensus Criteria for Grading Myelofibrosis
* Has received no prior cytoreductive treatment for their ET
* Human Immunodeficiency Virus (HIV)-infected participants have well controlled HIV on antiretroviral therapy
* Participants who are Hepatitis B surface antigen (HBsAg) positive are eligible if they have received Hepatitis B Virus (HBV) antiviral therapy for at least 4 weeks and have undetectable HBV viral load
* Participants with history of Hepatitis C Virus (HCV) infection are eligible if HCV viral load is undetectable
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* History of any illness/impairment of gastrointestinal function that might interfere with drug absorption
* History of a malignancy, unless potentially curative treatment has been completed with no evidence of malignancy for 2 years
* HIV-infected participants with a history of Kaposi's sarcoma and/or Multicentric Castleman's Disease
* Has an active infection requiring systemic therapy
* Has had a major surgery <4 weeks prior to first dose of study intervention or has not recovered from side effects of major surgery >4 weeks prior to first dose

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
16/07/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
14/05/2029
Actual
Sample size
Target
300
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
SA
Recruitment hospital [1] 0 0
Royal Adelaide Hospital-Haematology Clinical Trials Unit ( Site 0203) - Adelaide
Recruitment postcode(s) [1] 0 0
5000 - Adelaide
Recruitment outside Australia
Country [1] 0 0
Argentina
State/province [1] 0 0
Tucuman
Country [2] 0 0
Chile
State/province [2] 0 0
Biobio
Country [3] 0 0
Chile
State/province [3] 0 0
Coquimbo
Country [4] 0 0
Chile
State/province [4] 0 0
Region M. De Santiago
Country [5] 0 0
Hong Kong
State/province [5] 0 0
Hksar
Country [6] 0 0
Israel
State/province [6] 0 0
Haifa
Country [7] 0 0
Israel
State/province [7] 0 0
Jerusalem
Country [8] 0 0
Israel
State/province [8] 0 0
Ramat Gan
Country [9] 0 0
Israel
State/province [9] 0 0
Zerifin
Country [10] 0 0
Japan
State/province [10] 0 0
Ehime
Country [11] 0 0
Japan
State/province [11] 0 0
Hokkaido
Country [12] 0 0
Japan
State/province [12] 0 0
Hyogo
Country [13] 0 0
Japan
State/province [13] 0 0
Ishikawa
Country [14] 0 0
Japan
State/province [14] 0 0
Mie
Country [15] 0 0
Japan
State/province [15] 0 0
Miyagi
Country [16] 0 0
Japan
State/province [16] 0 0
Osaka
Country [17] 0 0
Japan
State/province [17] 0 0
Tokyo
Country [18] 0 0
Japan
State/province [18] 0 0
Yamanashi
Country [19] 0 0
Japan
State/province [19] 0 0
Fukuoka
Country [20] 0 0
Japan
State/province [20] 0 0
Fukushima
Country [21] 0 0
Japan
State/province [21] 0 0
Miyazaki
Country [22] 0 0
Japan
State/province [22] 0 0
Okayama

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Merck Sharp & Dohme LLC
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
The purpose of this study is to evaluate the efficacy and safety of bomedemstat compared with hydroxyurea in cytoreductive therapy naïve essential thrombocythemia (ET) participants for whom cytoreductive therapy is indicated. Its primary objective is to compare bomedemstat to hydroxyurea with respect to durable clinicohematologic response (DCHR). The primary hypothesis is that bomedemstat is superior to hydroxyurea with respect to DCHR.
Trial website
https://clinicaltrials.gov/study/NCT06456346
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Medical Director
Address 0 0
Merck Sharp & Dohme LLC
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Toll Free Number
Address 0 0
Country 0 0
Phone 0 0
1-888-577-8839
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT06456346