Overview
Sponsor-declared trial summary
Phase
Therapeutic confirmatory (Phase III)
Status
Ongoing, recruiting
Participants planned
384
Countries
10
Sites
44
Essential Thrombocythemia patients who have an inadequate response to or are intolerant of hydroxyurea
To compare bomedemstat to best available therapy with respect to durable DCHR.
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] - Cardiovascular Diseases [C14]
- Trial duration
- 15 Apr 2024 → ongoing
- Decision date (initial)
- 2024-03-28
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- No
- Vulnerable population
- Yes
- Funding sources
- Merck Sharp & Dohme LLC
External identifiers
- EU CT number
- 2023-504865-21-00
- WHO UTN
- U1111-1290-2942
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Therapy, Safety, Pharmacokinetic, Pharmacogenomic, Pharmacogenetic, Efficacy
To compare bomedemstat to best available therapy with respect to durable DCHR.
Secondary objectives 9
- To compare bomedemstat to best available therapy with respect to change in fatigue score based on MFSAF v4.0.
- To compare bomedemstat to best available therapy with respect to change in total fatigue score based on PROMIS Fatigue SF-7a.
- To compare bomedemstat to best available therapy with respect to change in total symptom score based on MFSAF v4.0.
- To evaluate DOCHR for both treatment arms.
- To evaluate DOHR for both treatment arms
- To evaluate the incidence of thrombotic events for both treatment arms.
- To evaluate the incidence of major hemorrhagic events for both treatment arms
- To evaluate the incidence of disease progression for both treatment arms
- To evaluate the safety and tolerability of bomedemstat
Conditions and MedDRA coding
Essential Thrombocythemia patients who have an inadequate response to or are intolerant of hydroxyurea
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 21.0 | LLT | 10015494 | Essential thrombocythemia | 10029104 |
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 7
- Has a diagnosis of essential thrombocythemia (ET) per World Health Organization (WHO) 2016 diagnostic criteria for myeloproliferative neoplasms. (confirmed by a central pathologist).
- 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
- Has a history of inadequate response to or intolerance of hydroxyurea based on modified European LeukemiaNet (ELN) criteria for hydroxyurea resistance or intolerance.
- Has an inadequate or loss of response to their most recent prior ET therapy, requiring a change of cytoreductive therapy
- Has a platelet count > 450 × 10^9/L (450k /μL) assessed up to 72 hours before first dose of study intervention
- Has an absolute neutrophil count (ANC) ≥0.75 × 10^9/L assessed up to 72 hours before first dose of study intervention
- Participants may have received up to 3 prior ET-directed cytoreductive agents including hydroxyurea
Exclusion criteria 5
- Known immediate or delayed hypersensitivity reaction or idiosyncrasy to drugs chemically related to bomedemstat or lysine demethylase or monoamine oxidase inhibitor (LSDi or MAOi) that contraindicates participation
- History of any illness/impairment of gastrointestinal (GI) function that might interfere with drug absorption (eg, chronic diarrhea or history of gastric bypass surgical procedure), confound the study results or pose an additional risk to the individual by participation in the study
- Evidence at the time of Screening of increased risk of bleeding
- History of a malignancy, unless potentially curative treatment has been completed with no evidence of malignancy for 2 years. Note: The time requirement does not apply to participants who underwent successful definitive resection of basal cell carcinoma of the skin, squamous cell carcinoma of the skin, or carcinoma in situ, excluding carcinoma in situ of the bladder
- Human immunodeficiency virus (HIV)-infected participants with a history of Kaposi’s sarcoma and/or Multicentric Castleman’s Disease
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- Durable Clinicohematologic Response (DCHR) Rate
Secondary endpoints 10
- 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
- Duration of Clinicohematologic Response (DOCHR)
- Duration of Hematologic Remission (DOHR)
- Percentage of Participants with Thrombotic Events
- Percentage of Participants with Major Hemorrhagic Events
- Disease Progression Rate
- Number of Participants with An Adverse Event (AE)
- Number of Participants Discontinuing from Study Therapy Due to an AE
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 4
PRD10818118 · Product
- Active substance
- Bomedemstat
- Substance synonyms
- MK-3543, N-[(2S)-5-{[(1R,2S)-2-(4-FLUOROPHENYL)CYCLOPROPYL]AMINO}-1-(4-METHYLPIPERAZIN-1-YL)-1-OXOPENTAN-2-YL]-4-(1H-1,2,3-TRIAZOL-1-YL)BENZAMIDE, IMG-7289
- Pharmaceutical form
- CAPSULE, HARD
- Route of administration
- ORAL
- Max daily dose
- 175 mg milligram(s)
- Max total dose
- 191625 mg milligram(s)
- Max treatment duration
- 36 Month(s)
- Authorisation status
- Not Authorised
- MA holder
- MERCK & CO. INC.
- Paediatric formulation
- No
- Orphan designation
- No
PRD10818117 · Product
- Active substance
- Bomedemstat
- Substance synonyms
- MK-3543, N-[(2S)-5-{[(1R,2S)-2-(4-FLUOROPHENYL)CYCLOPROPYL]AMINO}-1-(4-METHYLPIPERAZIN-1-YL)-1-OXOPENTAN-2-YL]-4-(1H-1,2,3-TRIAZOL-1-YL)BENZAMIDE, IMG-7289
- Pharmaceutical form
- CAPSULE, HARD
- Route of administration
- ORAL
- Max daily dose
- 175 mg milligram(s)
- Max total dose
- 191625 mg milligram(s)
- Max treatment duration
- 36 Month(s)
- Authorisation status
- Not Authorised
- MA holder
- MERCK & CO. INC.
- Paediatric formulation
- No
- Orphan designation
- No
PRD10818116 · Product
- Active substance
- Bomedemstat
- Substance synonyms
- MK-3543, N-[(2S)-5-{[(1R,2S)-2-(4-FLUOROPHENYL)CYCLOPROPYL]AMINO}-1-(4-METHYLPIPERAZIN-1-YL)-1-OXOPENTAN-2-YL]-4-(1H-1,2,3-TRIAZOL-1-YL)BENZAMIDE, IMG-7289
- Pharmaceutical form
- CAPSULE, HARD
- Route of administration
- ORAL
- Max daily dose
- 175 mg milligram(s)
- Max total dose
- 191625 mg milligram(s)
- Max treatment duration
- 36 Month(s)
- Authorisation status
- Not Authorised
- MA holder
- MERCK & CO. INC.
- Paediatric formulation
- No
- Orphan designation
- No
PRD10914816 · Product
- Active substance
- Bomedemstat
- Substance synonyms
- MK-3543, N-[(2S)-5-{[(1R,2S)-2-(4-FLUOROPHENYL)CYCLOPROPYL]AMINO}-1-(4-METHYLPIPERAZIN-1-YL)-1-OXOPENTAN-2-YL]-4-(1H-1,2,3-TRIAZOL-1-YL)BENZAMIDE, IMG-7289
- Pharmaceutical form
- CAPSULE, HARD
- Route of administration
- ORAL
- Max daily dose
- 175 mg milligram(s)
- Max total dose
- 191625 mg milligram(s)
- Max treatment duration
- 36 Month(s)
- Authorisation status
- Not Authorised
- MA holder
- MERCK & CO. INC.
- Paediatric formulation
- No
- Orphan designation
- No
Comparator 6
SUB05993MIG · Substance
- Active substance
- Busulfan
- Pharmaceutical form
- TABLET
- Route of administration
- ORAL
- Max daily dose
- 4 mg milligram(s)
- Max total dose
- 2016 mg milligram(s)
- Max treatment duration
- 36 Month(s)
- Authorisation status
- Authorised
- ATC code
- - — -
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Anagrelide Hydrochloride Monohydrate
SUB75321 · Substance
- Active substance
- Anagrelide Hydrochloride Monohydrate
- Pharmaceutical form
- CAPSULE
- Route of administration
- ORAL
- Max daily dose
- 10 mg milligram(s)
- Max total dose
- 3650 mg milligram(s)
- Max treatment duration
- 36 Month(s)
- Authorisation status
- Authorised
- ATC code
- - — -
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
SUB32273 · Substance
- Active substance
- Ruxolitinib
- Pharmaceutical form
- TABLET
- Route of administration
- ORAL
- Max daily dose
- 20 mg milligram(s)
- Max total dose
- 21900 mg milligram(s)
- Max treatment duration
- 36 Month(s)
- Authorisation status
- Authorised
- ATC code
- - — -
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
SUB32273 · Substance
- Active substance
- Ruxolitinib
- Pharmaceutical form
- TABLET
- Route of administration
- ORAL
- Max daily dose
- 20 mg milligram(s)
- Max total dose
- 21900 mg milligram(s)
- Max treatment duration
- 36 Month(s)
- Authorisation status
- Authorised
- ATC code
- - — -
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
SUB16452MIG · Substance
- Active substance
- Peginterferon ALFA-2A
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- SUBCUTANEOUS INJECTION
- Max daily dose
- 6.43 µg microgram(s)
- Max total dose
- 7020 µg microgram(s)
- Max treatment duration
- 36 Month(s)
- Authorisation status
- Authorised
- ATC code
- - — -
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
SUB16452MIG · Substance
- Active substance
- Peginterferon ALFA-2A
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- SUBCUTANEOUS INJECTION
- Max daily dose
- 6.43 µg microgram(s)
- Max total dose
- 7020 µg microgram(s)
- Max treatment duration
- 36 Month(s)
- Authorisation status
- Authorised
- ATC code
- - — -
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
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
- Nati Lerman
Public contact point
- Organisation
- Merck Sharp & Dohme LLC
- Contact name
- Nati Lerman
Third parties 6
| Organisation | City, country | Duties |
|---|---|---|
| Hematogenix Laboratory Services LLC ORG-100040020
|
Tinley Park, United States | Laboratory analysis |
| Signant Health Global Solutions Limited ORG-100047290
|
Dublin 2, Ireland | Interactive response technologies (IRT) |
| AG Mednet Inc. ORG-100039869
|
Boston, United States | Code 13 |
| Perceptive Eclinical Limited ORG-100041144
|
Nottingham, United Kingdom | Other |
| Signant Health Global Solutions Limited ORG-100047290
|
Dublin 2, Ireland | E-data capture |
| Parexel International Corp. ORG-100007310
|
Auburndale, United States | Other |
Locations
10 EU/EEA countries · 44 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Belgium | Ongoing, recruitment ended | 3 | 1 |
| France | Ongoing, recruitment ended | 24 | 4 |
| Germany | Ongoing, recruiting | 12 | 3 |
| Hungary | Ongoing, recruitment ended | 12 | 4 |
| Italy | Ongoing, recruiting | 33 | 10 |
| Netherlands | Ongoing, recruitment ended | 12 | 3 |
| Poland | Ongoing, recruitment ended | 6 | 1 |
| Portugal | Ongoing, recruiting | 9 | 3 |
| Spain | Ongoing, recruiting | 38 | 13 |
| Sweden | Ongoing, recruitment ended | 6 | 2 |
| Rest of world
Israel, China, Taiwan, Argentina, Hong Kong, Japan, New Zealand, Australia, Korea, Republic of, United States, Turkey, United Kingdom, Brazil, Colombia
|
— | 229 | — |
Investigational sites
Centre Hospitalier Regional Universitaire De Tours
Hematology and cell therapy, 2 Boulevard Tonnelle, 37000, Tours
Hopital Saint Louis
Pharmacologic and Clinical Investigations, 1 Avenue Claude Vellefaux, 75010, Paris
CHRU De Nancy
Hematology and Intern Medicine, Rue Du Morvan, 54500, Vandoeuvre Les Nancy
Hospices Civils De Lyon
Hematology, 165 Chemin Du Grand Revoyet, 69310, Pierre Benite
Universitaetsklinikum Aachen AöR
Klinik für Hämatologie, Onkologie, Hämostaseologie und Stammzelltransplantation, Pauwelsstrasse 30, 52074, Aachen
Technische Universitat Dresden
Medizinische Klinik und Poliklinik I, Fetscherstrasse 74, Johannstadt-Nord, Dresden
Martin-Luther-Universitaet Halle-Wittenberg
Universitätsklinik und Poliklinik für Innere Medizin IV, Ernst-Grube-Strasse 40, Kroellwitz, Halle (Saale)
Gyor-Moson-Sopron Varmegyei Petz Aladar Egyetemi Oktato Korhaz
II. Belgyógyászat - Haematológia, Vasvari Pal Utca 2-4, 9024, Gyor
Szabolcs-Szatmar-Bereg Varmegyei Oktatokorhaz
Haematológiai osztály, Szent Istvan Utca 68, 4400, Nyiregyhaza
Semmelweis University
Belgyógyászati és Hematológiai Klinika, Szentkiralyi Utca 46, VIII Kerulet, Budapest VIII
University Of Debrecen
Belgyógyászati Klinika, Nagyerdei Korut 98, 4032, Debrecen
Universita' Degli Studi Di Ferrara
U.O. di Ematologia, Via Aldo Moro 8, 44124, Ferrara
Azienda Ospedaliera Ospedale Di Circolo E Fondazione Macchi
S.C. Ematologia Trial Unit, Viale Luigi Borri 57, 21100, Varese
Azienda Ospedaliera Nazionale Ss Antonio E Biagio E C Arrigo Alessandria
SCDU Ematologia, Via Venezia 16, 15121, Alexandria
Azienda Ospedaliero Universitaria Delle Marche
SOD Clinica Ematologica, Via Conca 71, 60126, Ancona
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
SC Ematologia, Via Francesco Sforza 28, 20122, Milan
Istituto Romagnolo Per Lo Studio Dei Tumori Dino Amadori IRST S.r.l.
Oncologia ed Ematologia Clinica e Sperimentale, Via Piero Maroncelli 40, 47014, Meldola
Azienda Ospedaliera Ordine Mauriziano Di Torino
SCDU Ematologia, Via Ferdinando Magellano 1, 10128, Turin
Istituto Oncologico Veneto
UOC Oncoematologia, Via Dei Carpani 16/z, 31033, Castelfranco Veneto
Careggi University Hospital
Ematologia, Largo Giovanni Alessandro Brambilla 3, 50134, Florence
Azienda USL IRCCS Di Reggio Emilia
Ematologia, Viale Risorgimento 80, 42123, Reggio Emilia
Universitair Medisch Centrum Groningen
Hematology, Hanzeplein 1, 9713 GZ, Groningen
Stichting Martini Ziekenhuis
Internal medicine, Van Swietenplein 1, 9728 NT, Groningen
Albert Schweitzer Ziekenhuis
Hematology, Albert Schweitzerplaats 25, 3318 AT, Dordrecht
Instituto Portugues De Oncologia Do Porto Francisco Gentil E.P.E.
Hematology and bone marrow transplantation service, Rua Dr. Antonio Bernardino De Almeida, 4200-072, Porto
CCAB Centro Clinico Academico Braga Associacao
Oncology Service, Lugar De Sete Fontes S Victor, 4710-243, Braga
Unidade Local De Saude De Lisboa Ocidental E.P.E.
Hematology Service, Estrada Forte Do Alto Duque, 1449-005, Lisbon
Complexo Hospitalario Universitario De Santiago
Servicio de hematología, Calle Choupana Da S/n, 15706, Santiago De Compostela
Hospital Costa Del Sol
Servicio de hematología, Terreno Autovia Mediterraneo A-7 S/n, 29603, Marbella
Institut Catala D'oncologia
Servicio de hematología, Avinguda De La Gran Via De L'hospitalet 199-203, 08908, L'hospitalet De Llobregat
Institut Catala D'oncologia
Servicio de hematología, Carretera Canyet S/n, 08916, Badalona
Hospital Universitario Ramon Y Cajal
Servicio de hematología, Carretera Del Colmenar Viejo Km 9 100, Por El Pardo, Madrid
Hospital Clinic De Barcelona
Servicio de hematología, Calle Villarroel 170, 08036, Barcelona
Hospital Universitario Virgen De La Victoria
Servicio de hematología, Calle Del Arroyo Teatinos Sn, 29010, Malaga
Hospital Universitario Dr Peset Aleixandre
Servicio de hematología, Avinguda De Gaspar Aguilar 90, 46017, Valencia
Hospital Universitario 12 De Octubre
Servicio de hematología y hemoterapia, Bloque D, Avenida De Cordoba Sn, Madrid
Hospital General Universitario De Albacete
Servicio de hematología, Calle Hermanos Falco 37, 02006, Albacete
Hospital Del Mar
Hematology, Passeig Maritim De La Barceloneta 25-29, 08003, Barcelona
Hospital Universitario De Salamanca
Servicio de hematología, Paseo De San Vicente 58-182, 37007, Salamanca
Hospital Universitari Vall D Hebron
Servicio de hematología, Edificio Materno-Infantil, Passeig De La Vall D'hebron 119-129, Barcelona
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| Belgium | 2025-03-14 | 2025-03-27 | 2025-07-29 | ||
| France | 2024-06-11 | 2024-09-19 | 2026-05-27 | ||
| Germany | 2024-05-29 | 2024-07-03 | |||
| Hungary | 2024-06-20 | 2024-10-24 | 2025-10-27 | ||
| Italy | 2024-05-02 | 2024-05-27 | |||
| Netherlands | 2024-05-14 | 2024-08-15 | 2025-07-29 | ||
| Poland | 2024-05-13 | 2024-10-15 | 2025-08-26 | ||
| Portugal | 2024-06-19 | 2025-08-05 | |||
| Spain | 2024-04-15 | 2024-04-26 | |||
| Sweden | 2024-06-07 | 2024-09-23 | 2025-08-15 |
Oversight and notifications
Regulatory notifications under CTR Articles 38, 52, 53, 54 and 77
Urgent safety measures 1 · Art. 54 CTR
Urgent safety measure US-51090
- Event date
- 2024-10-11
- Submission date
- 2024-10-11
- In response to
- OTHER
- Member states affected
- Belgium, France, Germany, Hungary, Italy, Portugal, Spain, Sweden, Netherlands, Poland
- Event description
- Global shortage of interferon
- Measures taken
- After the consultation by the Reporting Member State, the Sponsor will implement central sourcing of interferon to ensure treatment continuity for the subjects already assigned to interferon in the clinical study MK-3543-006.
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 121 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Protocol_2023-504865-21_SM25_for pub | 09R |
| Protocol (for publication) | D4_Copyright Statement_SM13_for pub | 04DEC2024 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements and IC Procedure_BEL_EN_for pub | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements and IC Procedure_DEU_EN_for pub | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements and IC Procedure_ESP_ES_for pub | 21SEP2023R |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements and IC Procedure_FRA_FR_SM25-RFI005_for pub | 15APR2026R |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements and IC Procedure_HUN_EN_for pub | 03JUN2024 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements and IC Procedure_ITA_EN_for pub | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements and IC Procedure_NLD_EN_for pub | v6-0 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements and IC Procedure_POL_PL_SM25_for pub | 2.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements and IC Procedure_PRT_EN_for pub | 2-0 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements and IC Procedure_SWE_SV_for pub | 1.0 |
| Recruitment arrangements (for publication) | K2_Recruitment Doc Advertisement_NLD_NL_for pub | 2 |
| Recruitment arrangements (for publication) | K2_Recruitment Doc Brochure_DEU_DE_for pub | v0-00-1 |
| Recruitment arrangements (for publication) | K2_Recruitment Doc Master Tissue Brochure_BEL_EN_for pub | 00-1 |
| Recruitment arrangements (for publication) | K2_Recruitment Doc Master Tissue Brochure_BEL_FR_for pub | 00-1 |
| Recruitment arrangements (for publication) | K2_Recruitment Doc Master Tissue Brochure_BEL_NL_for pub | 00-1 |
| Recruitment arrangements (for publication) | K2_Recruitment Doc Master Tissue Brochure_FRA_FR_for pub | 00.1 |
| Recruitment arrangements (for publication) | K2_Recruitment Doc Patient Brochure_BEL_EN_for pub | 00-1 |
| Recruitment arrangements (for publication) | K2_Recruitment Doc Patient Brochure_BEL_FR_for pub | 00-1 |
| Recruitment arrangements (for publication) | K2_Recruitment Doc Patient Brochure_BEL_NL_for pub | 00-1 |
| Recruitment arrangements (for publication) | K2_Recruitment Doc Patient Brochure_FRA_FR_for pub | 00.1 |
| Recruitment arrangements (for publication) | K2_Recruitment Doc Patient Brochure_HUN_HU_for pub | 0.00.1 |
| Recruitment arrangements (for publication) | K2_Recruitment Doc Patient Brochure_NLD_NL_for pub | 2-0 |
| Recruitment arrangements (for publication) | K2_Recruitment Doc Patient Visit Guide_BEL_EN_for pub | 00.1 |
| Recruitment arrangements (for publication) | K2_Recruitment Doc Patient Visit Guide_BEL_FR_for pub | 00.1 |
| Recruitment arrangements (for publication) | K2_Recruitment Doc Patient Visit Guide_BEL_NL_for pub | 00.1 |
| Recruitment arrangements (for publication) | K2_Recruitment Doc Website_POL_PL_SM25_for pub | 19NOV2025 |
| Subject information and informed consent form (for publication) | L1_ICF_FBR consent_2023-504865-21_ESP_ES_SM25_for pub | 02 |
| Subject information and informed consent form (for publication) | L1_ICF_FBR consent_BEL_EN_SM25_for pub | 0.02 |
| Subject information and informed consent form (for publication) | L1_ICF_FBR consent_BEL_FR_SM25_for pub | 0.02 |
| Subject information and informed consent form (for publication) | L1_ICF_FBR consent_BEL_NL_SM25_for pub | 0.02 |
| Subject information and informed consent form (for publication) | L1_ICF_FBR consent_DEU_DE_SM25_for pub | 0.02 |
| Subject information and informed consent form (for publication) | L1_ICF_FBR consent_FRA_FR_SM25_for pub | 0.01R |
| Subject information and informed consent form (for publication) | L1_ICF_FBR consent_HUN_HU_SM25_for pub | 0.02 |
| Subject information and informed consent form (for publication) | L1_ICF_FBR consent_ITA_IT_for pub | 01 |
| Subject information and informed consent form (for publication) | L1_ICF_FBR consent_NLD_NL_SM25_for pub | 0.02 |
| Subject information and informed consent form (for publication) | L1_ICF_FBR consent_POL_PL_SM25_for pub | 02 |
| Subject information and informed consent form (for publication) | L1_ICF_FBR consent_SWE_SV_SM25_for pub | 0.02 |
| Subject information and informed consent form (for publication) | L1_ICF_FBR data privacy_ITA_IT_for pub | 03JUN2024 |
| Subject information and informed consent form (for publication) | L1_ICF_Genetic consent_HUN_HU_for pub | 0.01 |
| Subject information and informed consent form (for publication) | L1_ICF_Genetic consent_PRT_PT_for pub | AM01v1.00 |
| Subject information and informed consent form (for publication) | L1_ICF_Main addendum rejoining trial_BEL_EN_SM13_for pub | 00 |
| Subject information and informed consent form (for publication) | L1_ICF_Main addendum rejoining trial_BEL_FR_SM13_for pub | 00 |
| Subject information and informed consent form (for publication) | L1_ICF_Main addendum rejoining trial_BEL_NL_SM13_for pub | 00 |
| Subject information and informed consent form (for publication) | L1_ICF_Main addendum rejoining trial_DEU_DE_SM13_for pub | 0.00 |
| Subject information and informed consent form (for publication) | L1_ICF_Main addendum rejoining trial_ESP_ES_SM13_for pub | 00 |
| Subject information and informed consent form (for publication) | L1_ICF_Main addendum rejoining trial_FRA_FR_SM13_for pub | 0.00 |
| Subject information and informed consent form (for publication) | L1_ICF_Main addendum rejoining trial_HUN_HU_SM13_for pub | 0.00 |
| Subject information and informed consent form (for publication) | L1_ICF_Main addendum rejoining trial_ITA_IT_SM25_for pub | 00 |
| Subject information and informed consent form (for publication) | L1_ICF_Main addendum rejoining trial_NLD_NL_SM13_for pub | 0.00 |
| Subject information and informed consent form (for publication) | L1_ICF_Main addendum rejoining trial_POL_PL_SM13_for pub | 00 |
| Subject information and informed consent form (for publication) | L1_ICF_Main addendum rejoining trial_PRT_PT_SM13_for pub | 00 |
| Subject information and informed consent form (for publication) | L1_ICF_Main addendum rejoining trial_SWE_SV_SM13_for pub | 00 |
| Subject information and informed consent form (for publication) | L1_ICF_Main addendum_FRA_FR_SM15_for pub | AM02v2.00 |
| Subject information and informed consent form (for publication) | L1_ICF_Main addendum_FRA_FR_SM25_for pub | AM02v2.01R |
| Subject information and informed consent form (for publication) | L1_ICF_Main consent_BEL_EN_SM25_for pub | AM03v3.02R |
| Subject information and informed consent form (for publication) | L1_ICF_Main consent_BEL_FR_SM25_for pub | AM03v3.02R |
| Subject information and informed consent form (for publication) | L1_ICF_Main consent_BEL_NL_SM25_for pub | AM03v3.02R |
| Subject information and informed consent form (for publication) | L1_ICF_Main consent_DEU_DE_SM25-RFI003_for pub | AM02v2-02R |
| Subject information and informed consent form (for publication) | L1_ICF_Main consent_ESP_ES_SM25_for pub | AM02v2.02R |
| Subject information and informed consent form (for publication) | L1_ICF_Main consent_FRA_FR_SM25_for pub | AM02v2.01R |
| Subject information and informed consent form (for publication) | L1_ICF_Main consent_HUN_HU_SM25_for pub | AM02v2.01R |
| Subject information and informed consent form (for publication) | L1_ICF_Main consent_ITA_IT_SM25_for pub | AM02v2.02 |
| Subject information and informed consent form (for publication) | L1_ICF_Main consent_NLD_NL_SM25_for pub | AM02v2.01R |
| Subject information and informed consent form (for publication) | L1_ICF_Main consent_POL_PL_SM25_for pub | AM02v2.02R |
| Subject information and informed consent form (for publication) | L1_ICF_Main consent_PRT_PT_SM25_for pub | AM02v2.02 |
| Subject information and informed consent form (for publication) | L1_ICF_Main consent_SWE_SV_SM25_for pub | AM02v2.02 |
| Subject information and informed consent form (for publication) | L1_ICF_Main data privacy_ITA_IT_SM25_for pub | 19NOV2025 |
| Subject information and informed consent form (for publication) | L1_ICF_Main Informed consent_ITA_IT_SM25_for pub | AM02v2-01 |
| Subject information and informed consent form (for publication) | L1_ICF_Optional screening consent_BEL_NL_for pub | AM01v1.00R |
| Subject information and informed consent form (for publication) | L1_ICF_Optional_add crossborder_DEU_DE_for pub | 0-00R |
| Subject information and informed consent form (for publication) | L1_ICF_Optional_addendum_POL_PL_for pub | 1.00R |
| Subject information and informed consent form (for publication) | L1_ICF_Optional_ClinCard_BEL_EN_SM13_for pub | 01 |
| Subject information and informed consent form (for publication) | L1_ICF_Optional_ClinCard_BEL_FR_SM13_for pub | 01 |
| Subject information and informed consent form (for publication) | L1_ICF_Optional_ClinCard_BEL_NL_SM13_for pub | 01 |
| Subject information and informed consent form (for publication) | L1_ICF_Optional_data privacy_prescreening_ITA_IT_SM25_for pub | 19NOV25 |
| Subject information and informed consent form (for publication) | L1_ICF_Optional_DILI sample_ITA_IT_SM25_for pub | 19NOV2025 |
| Subject information and informed consent form (for publication) | L1_ICF_Optional_Greenphire adult_SWE_SV_for pub | 00 |
| Subject information and informed consent form (for publication) | L1_ICF_Optional_Greenphire adults_PRT_PT_SM13_for pub | 00 |
| Subject information and informed consent form (for publication) | L1_ICF_Optional_pregnancy follow-up_PRT_PT_for pub | 00 |
| Subject information and informed consent form (for publication) | L1_ICF_Optional_pregnant partner_BEL_EN_for pub | 0.00 |
| Subject information and informed consent form (for publication) | L1_ICF_Optional_pregnant partner_BEL_FR_for pub | 0.00 |
| Subject information and informed consent form (for publication) | L1_ICF_Optional_pregnant partner_BEL_NL_for pub | 0.00 |
| Subject information and informed consent form (for publication) | L1_ICF_Optional_pregnant partner_ESP_ES_for pub | 0.0 |
| Subject information and informed consent form (for publication) | L1_ICF_Optional_pregnant partner_HUN_HU_for pub | v0-00R |
| Subject information and informed consent form (for publication) | L1_ICF_Optional_prescreening_DEU_DE_for pub | 1.00R |
| Subject information and informed consent form (for publication) | L1_ICF_Optional_prescreening_FRA_FR_for pub | AM01v1.00R |
| Subject information and informed consent form (for publication) | L1_ICF_Optional_prescreening_ITA_IT_SM25_for pub | AM01v1.00 |
| Subject information and informed consent form (for publication) | L1_ICF_Optional_prescreening_NLD_NL_for pub | AM01_v1.00 |
| Subject information and informed consent form (for publication) | L1_ICF_Optional_prescreening_PRT_PT_for pub | AM01v1.00 |
| Subject information and informed consent form (for publication) | L1_ICF_Optional_prescreening_SWE_SV_for pub | AM01v1.00 |
| Subject information and informed consent form (for publication) | L1_ICF_Optional_screening consent_BEL_EN_for pub | AM01v1.00R |
| Subject information and informed consent form (for publication) | L1_ICF_Optional_screening consent_BEL_FR_for pub | AM01v1.00R |
| Subject information and informed consent form (for publication) | L1_ICF_Optional_screening consent_BEL_NL_for pub | AM01v1.00R |
| Subject information and informed consent form (for publication) | L1_ICF_Optional_screening consent_HUN_HU_for pub | AM01v1.00R |
| Subject information and informed consent form (for publication) | L1_ICF_Optional_screening genetic consent_HUN_HU_for pub | 0.01 |
| Subject information and informed consent form (for publication) | L1_ICF_Optional_trial at a glance_BEL_EN_for pub | 0.00 |
| Subject information and informed consent form (for publication) | L1_ICF_Optional_trial at a glance_BEL_FR_for pub | 0.00 |
| Subject information and informed consent form (for publication) | L1_ICF_Optional_trial at a glance_BEL_NL_for pub | 0.00 |
| Subject information and informed consent form (for publication) | L1_ICF_Optional_withdrawal_PRT_PT_for pub | 00 |
| Subject information and informed consent form (for publication) | L1_ICF_Optional-Limited Screening consent_ESP_ES_for pub | AM01v1.00R |
| Subject information and informed consent form (for publication) | L1_Patient ID Card_HUN_HU_for pub | 1-0R |
| Summary of Product Characteristics (SmPC) (for publication) | E2_SmPC RSI_ RUXOLITINIB ORAL_Novartis Pharmaceuticals UK_SM25_for pub | 09SEP2025 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_SmPC RSI_BUSULFAN ORAL Aspen Pharma Trading Limited_SM25_for pub | 16JUL2025 |
| Summary of Product Characteristics (SmPC) (for publication) | G2_SmPC Q_ANAGRELIDE_for pub | Generic UK |
| Summary of Product Characteristics (SmPC) (for publication) | G2_SmPC Q_Peginterferon alfa-2a_for pub | Aspire |
| Synopsis of the protocol (for publication) | D1_PPLS_ 2023-504865-21_SM25_for pub | 5.0 |
| Synopsis of the protocol (for publication) | D1_PPLS_2023-504865-21_BEL_DE_SM25_for pub | 5.0 |
| Synopsis of the protocol (for publication) | D1_PPLS_2023-504865-21_BEL_FR_SM25_for pub | 5.0 |
| Synopsis of the protocol (for publication) | D1_PPLS_2023-504865-21_BEL_NL_SM25_for pub | 5.0 |
| Synopsis of the protocol (for publication) | D1_PPLS_2023-504865-21_DEU_DE_SM25_for pub | 5.0 |
| Synopsis of the protocol (for publication) | D1_PPLS_2023-504865-21_ESP_ES_SM25_for pub | 5.0 |
| Synopsis of the protocol (for publication) | D1_PPLS_2023-504865-21_FRA_FR_SM25_for pub | 5.0 |
| Synopsis of the protocol (for publication) | D1_PPLS_2023-504865-21_HUN_HU_SM25_for pub | 5.0 |
| Synopsis of the protocol (for publication) | D1_PPLS_2023-504865-21_ITA_IT_SM25_for pub | 5.0 |
| Synopsis of the protocol (for publication) | D1_PPLS_2023-504865-21_NLD_NL_SM25_for pub | 5.0 |
| Synopsis of the protocol (for publication) | D1_PPLS_2023-504865-21_POL_PL_SM25_for pub | 5.0 |
| Synopsis of the protocol (for publication) | D1_PPLS_2023-504865-21_PRT_PT_SM25_for pub | 5 |
| Synopsis of the protocol (for publication) | D1_PPLS_2023-504865-21_SWE_SV_SM25_for pub | 5.0 |
| Synopsis of the protocol (for publication) | D1_Protocol Scientific Synopsis_HUN_HU_SM25-RFI004_for pub | 09R |
Application history
13 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2023-11-16 | France | Acceptable 2024-03-25
|
2024-03-26 |
| 2 | SUBSTANTIAL MODIFICATION | SM-1 | 2024-04-03 | Acceptable | 2024-05-07 | |
| 3 | SUBSTANTIAL MODIFICATION | SM-4 | 2024-04-03 | Acceptable | 2024-05-08 | |
| 4 | SUBSTANTIAL MODIFICATION | SM-2 | 2024-04-05 | France | Acceptable | 2024-04-26 |
| 5 | SUBSTANTIAL MODIFICATION | SM-3 | 2024-04-05 | Acceptable | 2024-05-09 | |
| 6 | SUBSTANTIAL MODIFICATION | SM-5 | 2024-04-05 | Acceptable | 2024-05-15 | |
| 7 | SUBSTANTIAL MODIFICATION | SM-6 | 2024-06-16 | France | Acceptable 2024-09-13
|
2024-09-13 |
| 8 | SUBSTANTIAL MODIFICATION | SM-7 | 2024-10-22 | France | Acceptable 2025-02-10
|
2025-02-11 |
| 9 | NON SUBSTANTIAL MODIFICATION | NSM-1 | 2025-02-17 | France | Acceptable 2025-02-10
|
2025-02-17 |
| 10 | SUBSTANTIAL MODIFICATION | SM-12 | 2025-02-18 | Acceptable | 2025-02-27 | |
| 11 | SUBSTANTIAL MODIFICATION | SM-13 | 2025-04-03 | France | Acceptable 2025-07-07
|
2025-07-09 |
| 12 | SUBSTANTIAL MODIFICATION | SM-15 | 2025-07-31 | France | Acceptable 2025-10-06
|
2025-10-07 |
| 13 | SUBSTANTIAL MODIFICATION | SM-25 | 2026-01-29 | France | Acceptable 2026-05-11
|
2026-05-12 |