Clinical trial • Phase III • Haematology

Bomedemstat for Essential thrombocythemia

Phase III trial of Bomedemstat for Essential thrombocythemia.

Overview

Trial Therapeutic Area
Haematology
Trial Disease
Essential thrombocythemia
Trial Stage
Phase III
Drug Modality
Small molecule
Paediatric Trial
Yes

Key dates

Initial CTIS Submission Date
14-06-2024
First CTIS Authorization Date
30-09-2024

Trial design

Randomised, hydroxycarbamide (over-encapsulated) — dose/schedule not specified in the part i/ii data; placebo arms: placebo for hydroxycarbamide and placebo for bomedemstat (no dose/schedule).-controlled Phase III trial across 49 sites in Spain, Germany, Sweden and others.

Randomised
Yes
Comparator
Hydroxycarbamide (over-encapsulated) — dose/schedule not specified in the Part I/II data; Placebo arms: Placebo for hydroxycarbamide and Placebo for Bomedemstat (no dose/schedule).
Target Sample Size
243
Trial Duration For Participant
1080

Eligibility

Recruits 243 paediatric patients.

Vulnerable Population
No vulnerable population selected. Informed consent is obtained from participants; no assent/parental consent procedures are specified in the Part I/II data provided.

Inclusion criteria

  • {"criterion_text":"- 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.\n- 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.\n- Has received no prior cytoreductive treatment for their ET.\n- Human Immunodeficiency Virus (HIV)-infected participants have well controlled HIV on antiretroviral therapy.\n- 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.\n- Participants with history of Hepatitis C Virus (HCV) infection are eligible if HCV viral load is undetectable."}

Exclusion criteria

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

  • {"endpoint_text":"- Durable Clinicohematologic Response (DCHR) Rate.","definition_or_measurement_approach":""}

Secondary endpoints

  • {"endpoint_text":"- Change From Baseline in Myelofibrosis Symptom Assessment Form version 4.0 (MFSAF v4.0) Individual Fatigue Symptom Item Score.","definition_or_measurement_approach":"Change from baseline measured using the MFSAF v4.0 individual fatigue symptom item score."}
  • {"endpoint_text":"- Change From Baseline in Patient-reported Outcomes Measurement Information System (PROMIS) Fatigue SF-7a Total Fatigue Score.","definition_or_measurement_approach":"Change from baseline measured using PROMIS Fatigue SF-7a total fatigue score."}
  • {"endpoint_text":"- Change From Baseline in MFSAF v4.0 Total Symptom Score.","definition_or_measurement_approach":"Change from baseline measured using MFSAF v4.0 total symptom score."}
  • {"endpoint_text":"- Duration of Clinicohematologic Response (DOCHR).","definition_or_measurement_approach":"Duration measure of the clinicohematologic response (DOCHR); specific operational definition not provided in the Part I/II data."}
  • {"endpoint_text":"- Duration of Hematologic Remission (DOHR).","definition_or_measurement_approach":"Duration measure of hematologic remission (DOHR); specific operational definition not provided in the Part I/II data."}
  • {"endpoint_text":"- Number of Participants Who Experience Thrombotic Events.","definition_or_measurement_approach":"Count of participants experiencing thrombotic events."}
  • {"endpoint_text":"- Number of Participants Who Experience Major Hemorrhagic Events.","definition_or_measurement_approach":"Count of participants experiencing major hemorrhagic events."}
  • {"endpoint_text":"- Disease Progression Rate.","definition_or_measurement_approach":"Rate of disease progression; specific criteria for progression not provided in the Part I/II data."}
  • {"endpoint_text":"- Number of Participants Who Experience One or More Adverse Events (AEs)","definition_or_measurement_approach":"Count of participants with one or more adverse events."}
  • {"endpoint_text":"- Number of Participants Who Discontinue Study Intervention Due to an AE.","definition_or_measurement_approach":"Count of participants discontinuing study intervention due to an adverse event."}

Recruitment

Registry Or Advocacy Recruitment
True - recruitment materials include advocacy/patient advocacy documents (e.g., 'L1_Patient advocacy_AUT_DE_for pub', 'K2_Recruitment Doc Advocacy Card_SWE_SV_for pub').
Digital Remote Recruitment
True - digital/remote methods are indicated (e.g., website recruitment document for Poland 'K2_Recruitment Doc Website_POL_PL', and a document 'L1_ICF_Optional_Greenphire adults' indicates use of Greenphire-related processes in Sweden).
Planned Sample Size
243
Recruitment Window Months
55
Consent Approach
Informed consent is obtained from participants; country-specific subject information and informed consent forms are available (documents for ESP, DEU/EN, SWE, HUN, ITA, POL, FRA, etc.). Optional consents are provided in documentation (examples: optional limited screening, optional pregnant partner, genetic consent, FBR consent). No specific assent or parental consent procedures for minors are described in the Part I/II data.

Methods

  • Country-specific recruitment arrangements and informed consent procedure documents (K1/K2) available for participating countries (e.g., Spain, Germany, Sweden, Austria, Hungary, Italy, Poland, Denmark, France) as listed in the trial documents.
  • Patient brochures / patient visit guides (K2 recruitment documents) for multiple countries (explicit document titles present in the CTIS documents list).
  • Country-specific website recruitment material (document: K2_Recruitment Doc Website_POL_PL).

Geography

Total Number Of Sites
49
Total Number Of Participants
153

Spain

Earliest CTIS Part Ii Submission Date
30-08-2024
Latest Decision Or Authorization Date
13-04-2026
Processing Time Days
591
Number Of Sites
10
Number Of Participants
30

Sites

Site Name
Hospital Costa Del Sol
Department Name
Oncology
Contact Person Name
Maria Isabel Mata
Contact Person Email
maribelmata500@gmail.com
Site Name
Clinica Universidad De Navarra
Department Name
Oncology
Contact Person Name
Carolina Guillen
Contact Person Email
cguillenrie@unav.es
Site Name
Hospital Clinic De Barcelona
Department Name
Oncology
Contact Person Name
Ana Triguero
Contact Person Email
atriguero@clinic.cat
Site Name
Hospital Universitario De Salamanca
Department Name
Oncology
Contact Person Name
Jesus Maria Hernandez
Contact Person Email
jmhr@usal.es
Site Name
Hospital Universitario Ramon Y Cajal
Department Name
Oncology
Contact Person Name
Jose Valentin Garcia
Contact Person Email
jvalentingg@gmail.com
Site Name
Institut Catala D'oncologia
Department Name
Oncology
Contact Person Name
Carolina Martinez
Site Name
Hospital Universitario 12 De Octubre
Department Name
Oncology
Contact Person Name
Gonzalo Carreño
Site Name
Instituto De Investigacion En Ciencias De La Salud Germans Trias I Pujol
Department Name
Hematology Department
Contact Person Name
Blanca Xicoy Cirici
Contact Person Email
bxicoy@iconco
Site Name
Hospital Universitario Marques De Valdecilla
Department Name
Oncology
Contact Person Name
Miguel Angel Cortes
Contact Person Email
miguelangel.cortes@scsalud.es
Site Name
Hospital Universitari Vall D Hebron
Department Name
Oncology
Contact Person Name
Maria Laura Fox
Contact Person Email
laura.fox@vallhebron.cat

Germany

Earliest CTIS Part Ii Submission Date
30-08-2024
Latest Decision Or Authorization Date
09-04-2026
Processing Time Days
587
Number Of Sites
7
Number Of Participants
18

Sites

Site Name
Universitaetsklinikum Jena KöR
Department Name
Klinik für Innere Medizin II, Abt. Hämatologie und internistische Onkologie
Contact Person Name
Carl Claudius Crodel
Site Name
Dr. Vehling-Kaiser MVZ GmbH
Department Name
N/A - private practice
Contact Person Name
Ursula Vehling-Kaiser
Contact Person Email
studien@vehling-kaiser.de
Site Name
Universitaetsklinikum Essen AöR
Department Name
Klinik für Hämatologie und Stammzelltransplantation
Contact Person Name
Joachim Goethert
Contact Person Email
benjamin.bietsch@uk-essen.de
Site Name
Martin-Luther-Universitaet Halle-Wittenberg
Department Name
Klinik und Poliklinik für Innere Medizin V
Contact Person Name
Haifa Katrin Al-Ali
Contact Person Email
dekanin@medizin.uni-halle.de
Site Name
Universitaetsklinikum Aachen AöR
Department Name
Klinik für Hämatologie, Onkologie, Hämostaseologie und Stammzelltransplantation
Contact Person Name
Steffen Koschmieder
Contact Person Email
skoschmieder@ukaachen.de
Site Name
Medizinische Hochschule Hannover
Department Name
Abteilung für Hämatologie, Hämostaseologie, Onkologie und Stammzelltransplantation
Contact Person Name
Susanne Isfort
Site Name
Johannes Gutenberg University Mainz
Department Name
III. Medizinische Klinik und Poliklinik
Contact Person Name
Daniel Sasca

Sweden

Earliest CTIS Part Ii Submission Date
29-08-2024
Latest Decision Or Authorization Date
08-04-2026
Processing Time Days
587
Number Of Sites
5
Number Of Participants
12

Sites

Site Name
Uppsala University Hospital
Department Name
VO Hematologi
Contact Person Name
Stina Söderlund
Contact Person Email
stina.soderlund@akademiska.se
Site Name
Sahlgrenska University Hospital-Vaestra Goetalandsregionen
Department Name
Department of hematology and coagulation
Contact Person Name
Lovisa Wennström
Contact Person Email
lovisa.vennstrom@vgregion.se
Site Name
Karolinska University Hospital
Department Name
Hematologi
Contact Person Name
Anna Ravn Landtblom
Site Name
Region Oerebro Laen
Department Name
VO Medicin
Contact Person Name
Erik Ahlstrand
Site Name
Region Skane Skanes Universitetssjukhus
Department Name
Hematologiska kliniken
Contact Person Name
Stefan Scheding
Contact Person Email
Stefan.Scheding@skane.se

Austria

Earliest CTIS Part Ii Submission Date
05-09-2024
Latest Decision Or Authorization Date
09-04-2026
Processing Time Days
581
Number Of Sites
1
Number Of Participants
6

Sites

Site Name
Ordensklinikum Linz GmbH
Department Name
Hämatologie und Onkologie
Contact Person Name
Veronika Buxhofer-Ausch

Hungary

Earliest CTIS Part Ii Submission Date
26-07-2024
Latest Decision Or Authorization Date
10-04-2026
Processing Time Days
623
Number Of Sites
5
Number Of Participants
15

Sites

Site Name
University Of Debrecen
Department Name
Belgyógyászati Klinika (Hematológia)
Contact Person Name
Árpád Illés
Contact Person Email
illes.arpad@med.unideb.hu
Site Name
Somogy Varmegyei Kaposi Mor Oktato Korhaz
Department Name
Hematológiai Osztály
Contact Person Name
Miklós Egyed
Contact Person Email
dregyedmiklos@yahoo.com
Site Name
Szabolcs-Szatmar-Bereg Varmegyei Oktatokorhaz
Department Name
Hematológia Osztály
Contact Person Name
László Rejtő
Contact Person Email
dr.rejto.laszlo@szszbmk.hu
Site Name
University Of Szeged
Department Name
Belgyógyászati Klinika
Contact Person Name
Szabolcs Modok
Contact Person Email
moszadokbolcs@gmail.com
Site Name
Gyor-Moson-Sopron Varmegyei Petz Aladar Egyetemi Oktato Korhaz
Department Name
Hematológia
Contact Person Name
Zsolt Lázár
Contact Person Email
lazarzsolt@petz.gyor.hu

Italy

Earliest CTIS Part Ii Submission Date
27-08-2024
Latest Decision Or Authorization Date
10-04-2026
Processing Time Days
591
Number Of Sites
7
Number Of Participants
18

Sites

Site Name
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
Department Name
SC Ematologia
Contact Person Name
Daniele Cattaneo
Site Name
Azienda Ospedaliero-Universitaria Ss Antonio E Biagio E Cesare Arrigo
Department Name
SCDU Ematologia
Contact Person Name
Monia Marchetti
Contact Person Email
monia.marchetti@ospedale.al.it
Site Name
Azienda Ospedaliero-Universitaria Di Bologna IRCCS Istituto Di Ricerca E Di Cura A Carattere Scientifico
Department Name
UO Ematologia
Contact Person Name
Francesca Palandri
Contact Person Email
francesca.palandri@unibo.it
Site Name
Careggi University Hospital
Department Name
Ematologia
Contact Person Name
Alessandro Maria Vannucchi
Contact Person Email
a.vannucchi@unifi.it
Site Name
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Department Name
Ematologia
Contact Person Name
Valerio De Stefano
Contact Person Email
valerio.destefano@unicatt.it
Site Name
Azienda Ospedaliera Ospedale Di Circolo E Fondazione Macchi
Department Name
S.C. Ematologia Trial Unit
Contact Person Name
Marco Brociner
Site Name
Fondazione IRCCS Policlinico San Matteo
Department Name
SC Ematologia I
Contact Person Name
Elisa Rumi
Contact Person Email
elisa.rumi@unipv.it

Poland

Earliest CTIS Part Ii Submission Date
29-08-2024
Latest Decision Or Authorization Date
09-04-2026
Processing Time Days
588
Number Of Sites
3
Number Of Participants
12

Sites

Site Name
Uniwersytecki Szpital Kliniczny Nr 1 W Lublinie
Department Name
Klinika Hematoonkologii i Transplantacji Szpiku
Contact Person Name
Marek Hus
Contact Person Email
hematoonkologia@usk1.pl
Site Name
Swietokrzyskie Centrum Onkologii Samodzielny Publiczny Zaklad Opieki Zdrowotnej W Kielcach
Department Name
Klinika Hematologii i Transplantacji Szpiku
Contact Person Name
Paweł Steckiewicz
Site Name
Pratia Hematologia Sp. z o.o.
Department Name
Pratia Onkologia Katowice
Contact Person Name
Sebastian Grosicki
Contact Person Email
anna.jakubiec@pratia.com

Denmark

Earliest CTIS Part Ii Submission Date
18-09-2024
Latest Decision Or Authorization Date
07-04-2026
Processing Time Days
566
Number Of Sites
4
Number Of Participants
12

Sites

Site Name
Region Midtjylland
Department Name
Department of Hematology
Contact Person Name
Peter an Kooten Niekerk
Contact Person Email
petevank@rm.dk
Site Name
Rigshospitalet
Department Name
Hematology - CTU
Contact Person Name
Mette Clausen
Contact Person Email
mette.borg.clausen@regionh.dk
Site Name
Region Sjaelland
Department Name
Department of hematology
Contact Person Name
Hans Hasselbalch
Contact Person Email
hkhl@regionsjaelland.dk
Site Name
Odense University Hospital
Department Name
Department of Hematology
Contact Person Name
Mette Brabrand
Contact Person Email
mette.brabrand@rsyd.dk

France

Earliest CTIS Part Ii Submission Date
06-09-2024
Latest Decision Or Authorization Date
07-05-2026
Processing Time Days
608
Number Of Sites
7
Number Of Participants
30

Sites

Site Name
Hopital Saint Louis
Department Name
Centre d'Investigations Cliniques
Contact Person Name
Jean-Jacques Kiladjian
Contact Person Email
jean-jacques.kiladjian@aphp.fr
Site Name
Centre Hospitalier Universitaire De Nice
Department Name
Hematology
Contact Person Name
Michael Loschi
Contact Person Email
loschi.m@chu-nice.fr
Site Name
Centre Hospitalier Universitaire De Bordeaux
Department Name
service de médecine interne et maladies infectieuses
Contact Person Name
Jean-François Viallard
Site Name
Centre Hospitalier Regional Universitaire De Tours
Department Name
Hématologie et Thérapie Cellulaire
Contact Person Name
Antoine Machet
Contact Person Email
a.machet@chu-tours.fr
Site Name
Centre Hospitalier Regional De Marseille
Contact Person Name
Regis Costello
Contact Person Email
regis.costello@ap-hm.fr
Site Name
Hospices Civils De Lyon
Contact Person Name
Vincent Alcazer
Contact Person Email
vincent.alcazer@chu-lyon.fr
Site Name
Centre Hospitalier Universitaire De Toulouse
Contact Person Name
Suzanne Tavitian

Sponsor

Primary sponsor

Full Name
Merck Sharp & Dohme LLC
Organisation Type
Pharmaceutical company
Country Of Registered Address
United States

Contract research organisations

Name
Signant Health Global Solutions Limited
Responsibilities
sponsorDuties codes: 3; contact jitendra.pidathala@signanthealth.com
Name
Hematogenix Laboratory Services LLC
Responsibilities
sponsorDuties codes: 4; contact rbudz@hematogenix.com
Name
Parexel International Corp.
Responsibilities
EUB services (Call center and Medical services); contact +MSDEMU@parexel.com
Name
AG Mednet Inc.
Responsibilities
sponsorDuties codes: 13; contact support@agmednet.com

Third parties

  • {"country":"Ireland","full_name":"Signant Health Global Solutions Limited","duties_or_roles":"sponsorDuties codes: 3","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Hematogenix Laboratory Services LLC","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"United States","full_name":"Parexel International Corp.","duties_or_roles":"EUB services (Call center and Medical services)","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"AG Mednet Inc.","duties_or_roles":"sponsorDuties codes: 13","organisation_type":"Pharmaceutical company"}

Investigational products

Investigational Product Name
MK-3543
Active Substance
Bomedemstat
Modality
Small molecule
Routes Of Administration
ORAL USE
Route
ORAL
Authorisation Status
prodAuthStatus: 1
Maximum Dose
175 mg (maxDailyDoseAmount)
Investigational Product Name
HYDROXYCARBAMIDE
Active Substance
Hydroxycarbamide (hydroxyurea)
Modality
Small molecule
Routes Of Administration
ORAL USE
Route
ORAL
Authorisation Status
prodAuthStatus: 2
Investigational Product Name
Placebo for hydroxycarbamide
Modality
Other
Investigational Product Name
Placebo for Bomedemstat
Modality
Other

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