Bomedemstat vs Hydroxyurea for Essential Thrombocythemia

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Merck Sharp & Dohme LLC

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Hemic and Lymphatic Diseases [C15]

Trial duration

15 Nov 2024 → ongoing

Decision date (initial)

2024-10-07

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

Funding sources

Merck Sharp & Dohme LLC

External identifiers

EU CT number

2023-505232-36-00

WHO UTN

U1111-1290-8287

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy, Pharmacogenomic, Pharmacokinetic, Pharmacogenetic, Safety, Efficacy

1-. To compare bomedemstat to hydroxyurea with respect to DCHR.

Secondary objectives 9

1. To compare bomedemstat to hydroxyurea with respect to change in fatigue score based on MFSAF v4.0.
2. To compare bomedemstat to hydroxyurea with respect to change in total fatigue score based on PROMIS Fatigue SF-7a.
3. To compare bomedemstat to hydroxyurea with respect to change in total symptom score based on MFSAF v4.0.
4. To evaluate DOCHR for both treatment arms.
5. To evaluate DOHR for both treatment arms.
6. To evaluate the incidence of thrombotic events for both treatment arms.
7. To evaluate the incidence of major hemorrhagic events for both treatment arms.
8. To evaluate the incidence of disease progression for both treatment arms.
9. To evaluate the safety and tolerability of bomedemstat.

Conditions and MedDRA coding

Essential thrombocythemia

Regulatory references

Scientific advice from competent authorities

European Medicines Agency

Plan to share IPD

Yes

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

1. Diagnosis of Essential Thrombocythemia (ET) based on World Health Organization Criteria for myeloproliferative neoplasms, and in indication for cytoreductive therapy regardless of age or risk status.
2. Has a centrally assessed bone marrow fibrosis score of Grade 0 or Grade 1, as per a modified version of the European Consensus Criteria for Grading Myelofibrosis.
3. Has received no prior cytoreductive treatment for their ET.
4. Human Immunodeficiency Virus (HIV)-infected participants have well controlled HIV on antiretroviral therapy.
5. 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.
6. Participants with history of Hepatitis C Virus (HCV) infection are eligible if HCV viral load is undetectable.

Exclusion criteria 5

1. History of any illness/impairment of gastrointestinal function that might interfere with drug absorption.
2. History of a malignancy, unless potentially curative treatment has been completed with no evidence of malignancy for 2 years.
3. HIV-infected participants with a history of Kaposi’s sarcoma and/or Multicentric Castleman’s Disease.
4. Has an active infection requiring systemic therapy.
5. 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.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Durable Clinicohematologic Response (DCHR) Rate.

Secondary endpoints 10

1. Change From Baseline in Myelofibrosis Symptom Assessment Form version 4.0 (MFSAF v4.0) Individual Fatigue Symptom Item Score.
2. Change From Baseline in Patient-reported Outcomes Measurement Information System (PROMIS) Fatigue SF-7a Total Fatigue Score.
3. Change From Baseline in MFSAF v4.0 Total Symptom Score.
4. Duration of Clinicohematologic Response (DOCHR).
5. Duration of Hematologic Remission (DOHR).
6. Number of Participants Who Experience Thrombotic Events.
7. Number of Participants Who Experience Major Hemorrhagic Events.
8. Disease Progression Rate.
9. Number of Participants Who Experience One or More Adverse Events (AEs)
10. Number of Participants Who Discontinue Study Intervention Due to an AE.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 4

Comparator 1

Placebo 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Merck Sharp & Dohme LLC

Sponsor organisation

Merck Sharp & Dohme LLC

Address

126 East Lincoln Avenue

City

Rahway

Postcode

07065-4607

Country

United States

Scientific contact point

Organisation

Merck Sharp & Dohme LLC

Contact name

Uzor Ogbu

Public contact point

Organisation

Merck Sharp & Dohme LLC

Contact name

Uzor Ogbu

Third parties 5

Locations

9 EU/EEA countries · 49 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Oversight and notifications

Regulatory notifications under CTR Articles 38, 52, 53, 54 and 77

Temporary halts 4 · Art. 38 CTR

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 72 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

5 submissions — initial application plus substantial / non-substantial modifications