A study of pelabresib and ruxolitinib compared to placebo and ruxolitinib in adult patients with myelofibrosis who have not received prior treatment with a Janus kinase inhibitor.

Status Authorised, recruitment pending · 9 EU/EEA countries · 79 sites · Protocol CDAK539A12303

Overview

Sponsor-declared trial summary

Phase Therapeutic confirmatory (Phase III)

Status Authorised, recruitment pending

Participants planned 461

Countries 9

Sites 79

Myelofibrosis

Primary objective in participants with TSS ≥25: To determine the efficacy of pelabresib + ruxolitinib compared with placebo +ruxolitinib with regard to spleen volume reduction and symptom improvement at Week 24 Primary objective in participants with TSS ≥ 15 (i.e., in the overall population): To determine the efficacy…

Key facts

Sponsor: Novartis Pharma AG
Participant type: Patients
Age range: 18-64 years, 65+ years
Gender: Male and Female
Therapeutic area: Diseases [C] - Neoplasms [C04]
Decision date (initial): 2026-06-01
Transition trial: No
Low-intervention: No
Rare-disease indication: Yes
Vulnerable population: No

External identifiers

EU CT number: 2025-523555-66-00
WHO UTN: U1111-1335-2469

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Pharmacokinetic, Safety

Primary objective in participants with TSS ≥25: To determine the efficacy of pelabresib +
ruxolitinib compared with placebo +ruxolitinib with regard to spleen volume reduction and symptom improvement at Week 24

Primary objective in participants with TSS ≥ 15 (i.e., in the overall population): To determine the efficacy of pelabresib + ruxolitinib compared with placebo + ruxolitinib with regard to spleen volume reduction and symptom improvement at Week 24

Secondary objectives 12

To determine the efficacy of pelabresib + ruxolitinib compared with placebo + ruxolitinib with regard to spleen volume
To determine the efficacy of pelabresib + ruxolitinib compared with placebo + ruxolitinib with regard to symptoms
To determine the efficacy of pelabresib + ruxolitinib compared with placebo + ruxolitinib with regard to spleen volume reduction and symptom improvement over time (dual response)
To determine the effect of pelabresib + ruxolitinib compared with placebo + ruxolitinib on hemoglobin and anemia
To evaluate overall survival (OS) in participants treated with pelabresib + ruxolitinib compared with placebo + ruxolitinib
To evaluate progression-free survival (PFS) in participants treated with pelabresib + ruxolitinib compared with placebo + ruxolitinib
To evaluate leukemia-free survival (LFS) in participants treated with pelabresib + ruxolitinib compared with placebo + ruxolitinib
To evaluate leukemic transformation in participants treated with pelabresib + ruxolitinib compared with placebo + ruxolitinib
To determine the safety and tolerability of pelabresib + ruxolitinib compared with placebo + ruxolitinib
To characterize the pharmacokinetics (PK) of pelabresib in combination with ruxolitinib
To characterize the PK profile of pelabresib in combination with ruxolitinib in participants enrolled in China and Japan
To assess the effect of pelabresib + ruxolitinib compared with placebo + ruxolitinib on patient reported fatigue and quality of life (QoL) over time

Conditions and MedDRA coding

Myelofibrosis

Version	Level	Code	Term	System organ class
21.0	LLT	10074689	Post polycythemia vera myelofibrosis	10029104
21.0	LLT	10074690	Post essential thrombocythemia myelofibrosis	10029104
20.0	PT	10077161	Primary myelofibrosis	100000004864
20.0	PT	10028537	Myelofibrosis	100000004864

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 7

Participants have diagnosis of primary myelofibrosis (PMF) or post-polycythemia vera myelofibrosis (post-PV MF) or post-essential thrombocythemia myelofibrosis (post-ET Confidential Novartis Clinical Trial Protocol (Version No. 00) Page 13 of 132 Protocol No. CDAK539A12303 MF) according to the International Consensus Classification (ICC) of Myeloid Neoplasms and Acute Leukemias 2022
DIPSS risk category of intermediate-1, intermediate-2 or high-risk
Spleen volume ≥ 450 cm3 by CT or MRI scan (local read sufficient if no central read available)
Have an average TSS of ≥15 within 7 days prior to randomization, using MFSAF v. 4.0 (at least 4 out of 7 TSS assessments required for average calculation)
Participants with an Eastern Cooperative Oncology Group (ECOG) performance status score of 0, 1, or 2
Blasts <5% in peripheral blood. Assessment of blasts in peripheral blood is mandatory at screening
Platelet count ≥ 100 x 10⁹//L in the absence of growth factors or transfusions for the previous 4 weeks

Exclusion criteria 6

Prior splenectomy at any time or splenic irradiation in the previous 6 months
Prior hematopoietic cell transplant or participant anticipated to receive a hematopoietic cell transplant within 24 weeks from the date of randomization
Blasts ≥ 5% in bone marrow if results available at screening or history of accelerated phase (AP) or leukemic transformation
History of a malignancy (other than MF, PPV-MF or PET-MF) in the past 3 years in need of systemic treatment
Received any approved or investigational agent other than hydroxyurea or anagrelide for the treatment of MF within 14 days of first dose of study treatment or within 5 half-lives of the approved or investigational agent, whichever is longer
Prior treatment with any JAK inhibitor or Bromodomain and extraterminal domain (BET) inhibitor

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

Co-primary endpoints: • Spleen response defined as achieving ≥ 35% reduction in spleen volume (SVR35) from baseline to Week 24 as measured by MRI (or CT scan) and assessed by central radiology read • Absolute change from baseline in total symptom score (TSS) at Week 24 as measured by MFSAF v4.0

Secondary endpoints 12

Spleen response defined as achieving ≥ 35% reduction in spleen volume (SVR35) from baseline to Week 12, Week 36, Week 48 and thereafter, as measured by MRI (or CT scan) • Absolute change from baseline and percentage change from baseline in spleen volume over time • Time to first SVR35 response • Duration of response defined as the time from first SVR35 response to loss of response for any participant who reaches SVR35 at any time
Symptom response defined as achieving≥ 50%reduction in TSS50 from baseline to Week 24 as measured by the MFSAF v4.0 Symptom response defined as achieving≥ 50%reduction in TSS50 from baseline to Week12,36,48 and thereafter as measured by theMFSAFv4.0 Absolute change from baseline and percentage change from baseline in TSS over time Time to first TSS50 response Duration of TSS50 response defined as the time from first TSS50 response to loss of response for any participant who reaches TSS50 at any
Dual response defined as achieving both ≥ 35% reduction in spleen volume (SVR35) and ≥ 50% reduction in total symptom score (TSS50) from baseline to Week 12, 24, 36, 48 and thereafter with SVR measured by MRI (or CT scan) and assessed by central radiology read and TSS measured by MFSAF v4.0
Hemoglobin response defined as a ≥1.5 g/dL average increase in hemoglobin from baseline in any 12-week mean hemoglobin concentration Hemoglobin change from baseline Anemia response (major response, minor response, stable anemia, progressive anemia) as per proposed 2024 IWG-ELN criteria in participants with transfusion dependent anemia and non-transfusion dependent anemia
OS defined as the time from randomization until death from any cause
PFS defined as the time from randomization until documented progression, or death from any cause whichever comes first
LFS defined as the time from randomization until leukemic transformation, or death from any cause whichever comes first
Exposure-adjusted incidence rate (EAIR) of participants with leukemic transformation
Treatment-emergent AEs of all grades and SAEs
Pelabresib plasma concentrations at specific time points
Pelabresib plasma PK parameters (Cmax, Tmax, AUCtau)
Change from baseline over time in fatigue as measured by PROMIS SF v1.0 Fatigue 7a Change from baseline over time in overall QOL and functional scales as measured by the EORTC QLQ-C30

Investigational products

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

Test 2

DAK539

PRD6381262 · Product

Active substance: Pelabresib Monohydrate
Substance synonyms: 2-((4S)-6-(4-chlorophenyl)-1-methyl-4H-benzo[C]isoxazolo[4,5-e]azepin-4-yl)acetamide monohydrate, CPI-232 monohydrate, CPI-0610 monohydrate, 2-((4s)-6-(4-chlorophenyl)-1-methyl-4h-isoxazolo(5,4-d)(2)benzazepin-4-yl)acetamide monohydrate
Pharmaceutical form: TABLET
Route of administration: ORAL
Max daily dose: 175 mg milligram(s)
Max total dose: 175 mg milligram(s)
Max treatment duration: 54 Month(s)
Authorisation status: Not Authorised
MA holder: NOVARTIS PHARMA AG
Paediatric formulation: No
Orphan designation: No

DAK539

PRD6381263 · Product

Active substance: Pelabresib Monohydrate
Substance synonyms: 2-((4S)-6-(4-chlorophenyl)-1-methyl-4H-benzo[C]isoxazolo[4,5-e]azepin-4-yl)acetamide monohydrate, CPI-232 monohydrate, CPI-0610 monohydrate, 2-((4s)-6-(4-chlorophenyl)-1-methyl-4h-isoxazolo(5,4-d)(2)benzazepin-4-yl)acetamide monohydrate
Pharmaceutical form: TABLET
Route of administration: ORAL
Max daily dose: 175 mg milligram(s)
Max total dose: 175 mg milligram(s)
Max treatment duration: 54 Month(s)
Authorisation status: Not Authorised
MA holder: NOVARTIS PHARMA AG
Paediatric formulation: No
Orphan designation: No

Placebo 1

Placebo to DAK539

N/A · Product

Other product name: N/A
Pharmaceutical form: N/A
ATC code: N/A — N/A
Marketing authorisation: N/A
MA holder: N/A
MA country: Iceland
Paediatric formulation: No

Auxiliary 9

Jakavi 20 mg tablets

PRD868097 · Product

Active substance: Ruxolitinib
Substance synonyms: INCB018424, INC-424, INCB-018424
Pharmaceutical form: TABLET
Route of administration: ORAL
Max daily dose: 25 mg milligram(s)
Max total dose: 25 mg milligram(s)
Max treatment duration: 54 Month(s)
Authorisation status: Authorised
ATC code: L01EJ01 — -
Marketing authorisation: EU/1/12/773/010
MA holder: NOVARTIS EUROPHARM LIMITED
MA country: EU
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: Yes
Modification description: The drug product is packaged in a different packaging presentation relative to the commercially available product. The clinical supplies are packaged in HDPE bottles with child resistant closures.

Jakavi 10 mg tablets

PRD2387736 · Product

Active substance: Ruxolitinib
Substance synonyms: INCB018424, INC-424, INCB-018424
Pharmaceutical form: TABLET
Route of administration: ORAL
Max daily dose: 25 mg milligram(s)
Max total dose: 25 mg milligram(s)
Max treatment duration: 54 Month(s)
Authorisation status: Authorised
ATC code: L01EJ01 — -
Marketing authorisation: EU/1/12/773/014
MA holder: NOVARTIS EUROPHARM LIMITED
MA country: EU
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: Yes
Modification description: The drug product is packaged in a different packaging presentation relative to the commercially available product. The clinical supplies are packaged in HDPE bottles with child resistant closures.

Jakavi 5 mg tablets

PRD868100 · Product

Active substance: Ruxolitinib
Substance synonyms: INCB018424, INC-424, INCB-018424
Pharmaceutical form: TABLET
Route of administration: ORAL
Max daily dose: 25 mg milligram(s)
Max total dose: 25 mg milligram(s)
Max treatment duration: 54 Month(s)
Authorisation status: Authorised
ATC code: L01EJ01 — -
Marketing authorisation: EU/1/12/773/004
MA holder: NOVARTIS EUROPHARM LIMITED
MA country: EU
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: Yes
Modification description: The drug product is packaged in a different packaging presentation relative to the commercially available product. The clinical supplies are packaged in HDPE bottles with child resistant closures.

Jakavi 5 mg tablets

PRD868101 · Product

Active substance: Ruxolitinib
Substance synonyms: INCB018424, INC-424, INCB-018424
Pharmaceutical form: TABLET
Route of administration: ORAL
Max daily dose: 25 mg milligram(s)
Max total dose: 25 mg milligram(s)
Max treatment duration: 54 Month(s)
Authorisation status: Authorised
ATC code: L01EJ01 — -
Marketing authorisation: EU/1/12/773/005
MA holder: NOVARTIS EUROPHARM LIMITED
MA country: EU
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: Yes
Modification description: The drug product is packaged in a different packaging presentation relative to the commercially available product. The clinical supplies are packaged in HDPE bottles with child resistant closures.

Jakavi 5 mg tablets

PRD868102 · Product

Active substance: Ruxolitinib
Substance synonyms: INCB018424, INC-424, INCB-018424
Pharmaceutical form: TABLET
Route of administration: ORAL
Max daily dose: 25 mg milligram(s)
Max total dose: 25 mg milligram(s)
Max treatment duration: 54 Month(s)
Authorisation status: Authorised
ATC code: L01EJ01 — -
Marketing authorisation: EU/1/12/773/006
MA holder: NOVARTIS EUROPHARM LIMITED
MA country: EU
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: Yes
Modification description: The drug product is packaged in a different packaging presentation relative to the commercially available product. The clinical supplies are packaged in HDPE bottles with child resistant closures.

Jakavi 15 mg tablets

PRD868095 · Product

Active substance: Ruxolitinib
Substance synonyms: INCB018424, INC-424, INCB-018424
Pharmaceutical form: TABLET
Route of administration: ORAL
Max daily dose: 25 mg milligram(s)
Max total dose: 25 mg milligram(s)
Max treatment duration: 54 Month(s)
Authorisation status: Authorised
ATC code: L01EJ01 — -
Marketing authorisation: EU/1/12/773/008
MA holder: NOVARTIS EUROPHARM LIMITED
MA country: EU
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: Yes
Modification description: The drug product is packaged in a different packaging presentation relative to the commercially available product. The clinical supplies are packaged in HDPE bottles with child resistant closures.

Jakavi 15 mg tablets

PRD868094 · Product

Active substance: Ruxolitinib
Substance synonyms: INCB018424, INC-424, INCB-018424
Pharmaceutical form: TABLET
Route of administration: ORAL
Max daily dose: 25 mg milligram(s)
Max total dose: 25 mg milligram(s)
Max treatment duration: 54 Month(s)
Authorisation status: Authorised
ATC code: L01EJ01 — -
Marketing authorisation: EU/1/12/773/007
MA holder: NOVARTIS EUROPHARM LIMITED
MA country: EU
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: Yes
Modification description: The drug product is packaged in a different packaging presentation relative to the commercially available product. The clinical supplies are packaged in HDPE bottles with child resistant closures.

Jakavi 20 mg tablets

PRD868098 · Product

Active substance: Ruxolitinib
Substance synonyms: INCB018424, INC-424, INCB-018424
Pharmaceutical form: TABLET
Route of administration: ORAL
Max daily dose: 25 mg milligram(s)
Max total dose: 25 mg milligram(s)
Max treatment duration: 54 Month(s)
Authorisation status: Authorised
ATC code: L01EJ01 — -
Marketing authorisation: EU/1/12/773/011
MA holder: NOVARTIS EUROPHARM LIMITED
MA country: EU
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: Yes
Modification description: The drug product is packaged in a different packaging presentation relative to the commercially available product. The clinical supplies are packaged in HDPE bottles with child resistant closures.

Jakavi 10 mg tablets

PRD2387738 · Product

Active substance: Ruxolitinib
Substance synonyms: INCB018424, INC-424, INCB-018424
Pharmaceutical form: TABLET
Route of administration: ORAL
Max daily dose: 25 mg milligram(s)
Max total dose: 25 mg milligram(s)
Max treatment duration: 54 Month(s)
Authorisation status: Authorised
ATC code: L01EJ01 — -
Marketing authorisation: EU/1/12/773/016
MA holder: NOVARTIS EUROPHARM LIMITED
MA country: EU
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: Yes
Modification description: The drug product is packaged in a different packaging presentation relative to the commercially available product. The clinical supplies are packaged in HDPE bottles with child resistant closures.

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Novartis Pharma AG

Sponsor organisation: Novartis Pharma AG
Address: Lichtstrasse 35
City: Basel
Postcode: 4056
Country: Switzerland

Scientific contact point

Organisation: Novartis Pharma AG
Contact name: Novartis Pharma Arzneimittel GmbH

Public contact point

Organisation: Novartis Pharma AG
Contact name: Novartis Pharma Arzneimittel GmbH

Third parties 5

Organisation	City, country	Duties
IQVIA Limited ORG-100008655	Reading, United Kingdom	On site monitoring
Syneos Health Inc. ORG-100008382	Morrisville, United States	On site monitoring
Parexel International (IRL) Limited ORG-100022780	Dublin 2, Ireland	Code 12
Mag. Andreas Raffeiner GmbH ORG-100043223	Walding, Austria	Code 8
Icon Clinical Research Limited ORG-100008322	Dublin 18, Ireland	On site monitoring

Locations

9 EU/EEA countries · 79 investigational sites

By country

Country	MS status	Planned subjects	Sites
Austria	Authorised, recruitment pending	7	4
Belgium	Authorised, recruitment pending	5	3
Czechia	Authorised, recruitment pending	12	5
France	Authorised, recruitment pending	19	17
Germany	Authorised, recruitment pending	35	10
Italy	Authorised, recruitment pending	43	16
Netherlands	Authorised, recruitment pending	5	2
Poland	Authorised, recruitment pending	13	7
Spain	Authorised, recruitment pending	34	15
Rest of world United States, Turkey, Brazil, Argentina, Canada, Japan, Malaysia, India, United Kingdom, Taiwan, Australia, China, Korea, Republic of, Switzerland	—	288	—

Investigational sites

Austria

4 sites · Authorised, recruitment pending

Noe LGA Gesundheit Region Mitte GmbH

2102: Klinische Abteilung für Innere Medizin 1, Dunant-Platz 1, 3100, St. Poelten

Gemeinnuetzige Salzburger Landeskliniken Betriebsgesellschaft mbH

2100: Univ. Klinik für Innere Medizin III, Muellner Hauptstrasse 48, 5020, Salzburg

Hanusch Krankenhaus Der Wiener Gebietskrankenkasse

2103: Medizinische Abteilung für Hämatologie und Onkologie, Heinrich-Collin-Strasse 30, Penzing, Vienna

Medical University Of Graz

2101: Abteilung für Hämatologie, Neue Stiftingtalstrasse 6, 8010, Graz

Belgium

3 sites · Authorised, recruitment pending

Algemeen Ziekenhuis Delta

2201: Hematology, Deltalaan 1, 8800, Roeselare

Ziekenhuis Aan De Stroom

2200: Hematology, Kempenstraat 100, 2030, Antwerp

Cliniques Universitaires Saint-Luc

2202: Hematology, Hippokrateslaan 10, Batiment 54, Sint-Lambrechts-Woluwe

Czechia

5 sites · Authorised, recruitment pending

Institute Of Hematology And Blood Transfusion

2300, NA, U Nemocnice 2094/1, Nove Mesto, Prague

Fakultni Nemocnice Hradec Kralove

2301, IV. interni hematologicka klinika, Sokolska 581, Novy Hradec Kralove, Hradec Kralove

University Hospital Olomouc

2303, Hemato-onkologicka klinika, Zdravotniku 248/7, 779 00, Olomouc

Fakultni Nemocnice Brno

2302, Interni hematologicka a onkologicka klinika, Jihlavska 340/20, Bohunice, Brno

Fakultni Nemocnice Ostrava

2304, Klinika hematoonkologie, 17. Listopadu 1790/5, Poruba, Ostrava

France

17 sites · Authorised, recruitment pending

Centre Hospitalier Universitaire De Bordeaux

2406: Hématologie, Avenue De Magellan, 33600, Pessac

Chorale Du Centre Hospitalier De Lens

2416: Hématologie, 99 Route De La Bassee, 62300, Lens

Centre Hospitalier Universitaire De Saint Etienne

2408: Hématologie et thérapie cellulaire, Avenue Albert Raimond, 42270, Saint Priest En Jarez

Centre Hospitalier Regional De Marseille

2417: Hématologie, 147 Boulevard Baille, 13005, Marseille

Hospices Civils De Lyon

2415: Hématologie, 165 Chemin Du Grand Revoyet, 69310, Pierre Benite

Centre Hospitalier Universitaire De Poitiers

2410: Hématologie oncologie, 2 Rue De La Miletrie, 86000, Poitiers

Hopital Saint Louis

2403: Hématologie, 1 Avenue Claude Vellefaux, 75010, Paris

Centre Hospitalier Regional Et Universitaire De Brest

2409: Hématologie, Boulevard Tanguy Prigent, 29200, Brest

Centre Hospitalier Universitaire De Nimes

2414: Hématologie, Place Du Professeur Robert Debre, 30029, Nimes Cedex 9

Centre Hospitalier Universitaire D'Angers

2412: Hématologie, 4 Rue Larrey, 49100, Angers

Centre Hospitalier Universitaire Amiens Picardie

2402: Hématologie, 1 Rond Point Du Pr Christian Cabrol, 80054, Amiens Cedex 1

Centre Hospitalier Universitaire De Toulouse

2407: Hématologie, 1 Avenue Irene Joliot Curie, 31059, Toulouse Cedex 9

Centre Hospitalier Universitaire De Rennes

2411: Hématologie, 2 Rue Henri Le Guilloux, 35000, Rennes

Centre Hospitalier Universitaire De Nice

2413: Hématologie, 151 Route De Saint Antoine, 06200, Nice

CHRU De Nancy

2400: Hématologie, 29 Avenue Du Mal De Lattre De Tassigny, 54000, Nancy

Assistance Publique Hopitaux De Paris

2405: Hématologie, 78 Rue Du General Leclerc, 94270, Le Kremlin-Bicetre

Les Hopitaux Universitaires De Strasbourg

2401: Hématologie, 1 Avenue Moliere, Bp 49, Strasbourg Cedex 2

Germany

10 sites · Authorised, recruitment pending

Universitaetsklinikum Essen AöR

#2506: Klinik für Haematologie und Stammzelltransplantation, Hufelandstrasse 55, Holsterhausen, Essen

Gemeinschaftspraxis Haematologie Onkologie

#2508: Gemeinschaftspraxis Haematologie und Onkologie, Arnoldstrasse 18, Johannstadt-Nord, Dresden

Universitaetsklinikum Erlangen AöR

#2503: Haematologie u. internistische Onkologie, Ulmenweg 18, Innenstadt, Erlangen

Universitaetsklinikum Jena KöR

#2505:Klinik fuer Innere Medizin II Abteilung für Haematologie und Onkologie, Am Klinikum 1, Lobeda, Jena

Muehlenkreiskliniken AöR

#2507: Universitätsklinik für Hämatologie, Onkolologie, Gerinnungsstörungen und Palliativmedizin, Hans-Nolte-Strasse 1, Haeverstaedt, Minden

University Medical Center Hamburg-Eppendorf

#2510:Zentrum für Onkologie Studienzentrale der II. Medizinischen Klinik, Martinistrasse 52, Eppendorf, Hamburg

Medizinische Hochschule Hannover

#2523:Klinik für Hämatologie, Hämostaseologie, Onkologie und Stammzelltransplantation, Carl-Neuberg-Strasse 1, Gross Buchholz, Hanover

Ambulantes Zentrum für Hematology und Onkologie

#2522: Haematology and Onccology, Karl-Liebknecht-Strasse 9, 03046, Cottbus

Universitaetsklinikum Aachen AöR

#2524:CIO – A Klinik für Hämatologie, Onkologie, Hämostaseologie und Stammzelltransplantation, Pauwelsstrasse 30, 52074, Aachen

Heidelberg University

#2511:III. Medizinische Klinik Haematologie und Onkologie, Theodor-Kutzer-Ufer 1-3, Wohlgelegen, Mannheim

Italy

16 sites · Authorised, recruitment pending

Istituto Oncologico Veneto

2608: UOC OncoematologiaIOV IRCCS Castelfranco Veneto c/o Osp San Giacomo, Via Dei Carpani 16/z, 31033, Castelfranco Veneto

Azienda Ospedaliero-Universitaria Policlinico Umberto I

2612: U.O.C. Ematologia, Viale Del Policlinico 155, 00161, Rome

Azienda Ospedaliera Universitaria Integrata Verona

2611: Centro Ricerche Cliniche di Verona S.r.l.8° piano Lotto B, Piazzale Ludovico Antonio Scuro 10, 37134, Verona

Azienda Ospedaliero-Universitaria Maggiore Della Carita

2602: S.C.D.U. Ematologia, Corso Giuseppe Mazzini 18, 28100, Novara

Azienda Ospedaliera Ordine Mauriziano Di Torino

2603: S.C.D.U. Ematologia e Terapie Cellulari, Via Ferdinando Magellano 1, 10128, Turin

Fondazione IRCCS San Gerardo Dei Tintori

2604: Divisione di Ematologia Adulti e Unità Trapiantodi Midollo, Via Giovanbattista Pergolesi 33, 20900, Monza

Azienda Ospedaliero-Universitaria Di Bologna IRCCS Istituto Di Ricerca E Di Cura A Carattere Scientifico

2606: U.O.C. di EmatologiaPad. 8 Dip. di Oncologia e Ematologia, Via Pietro Albertoni 15, 40138, Bologna

Azienda Sanitaria Universitaria Friuli Centrale

2610: Clinica Ematologica, Piazzale Santa Maria Della Misericordia 15, 33100, Udine

Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico

2607: U.O.C. Ematologia, Via Francesco Sforza 35, 20122, Milan

Istituto Romagnolo Per Lo Studio Dei Tumori Dino Amadori IRST S.r.l.

2600: SSDB Ematologia e Trapianti CSE, Via Piero Maroncelli 40, 47014, Meldola

Azienda Ospedaliero-Universitaria San Luigi Gonzaga

2609: S.C.D.U. Medicina Internaad indirizzo Ematologico, Regione Gonzole 10, 10043, Orbassano

Azienda Ospedaliero Universitaria Careggi

2601: Centro di Ricerca e Innovazione per le MalattieMieloproliferative (CRIMM), Largo Giovanni Alessandro Brambilla 3, 50134, Florence

Azienda Ospedaliera di Padova

2614: U.O.C. Ematologia e Immunologia Clinica, Via Nicolo' Giustiniani 2, 35128, Padova

Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone

2615: U.O. di Ematologia Divisione di Oncologia, Via Del Vespro 129, 90127, Palermo

Azienda Sanitaria Locale Roma 2

2605: U.O.C. Ematologia, Piazzale Dell' Umanesimo 10, 00144, Rome

Azienda Ospedaliera Universitaria Federico II Di Napoli

2613: U.O.C. Ematologia e Trapianti di Midollo Osseo, Via Sergio Pansini 5, 80131, Naples

Netherlands

2 sites · Authorised, recruitment pending

Universitair Medisch Centrum Utrecht

2701 : Hematology, Heidelberglaan 100, 3584 CX, Utrecht

Amsterdam UMC Stichting

2700 : Hematology, De Boelelaan 1117, 1081 HV, Amsterdam

Poland

7 sites · Authorised, recruitment pending

Medicover Integrated Clinical Services Sp. z o.o.

2801 : NA, Ul. Stefana Batorego 18-22, 87-100, Torun

Uniwersytecki Szpital Kliniczny Nr 4 W Lublinie

2805 : Oddział Wieloprofilowy Zachowawczy, Ul. Dra Kazimierza Jaczewskiego 8, 20-090, Lublin

Instytut Hematologii I Transfuzjologii

2802 : Klinika Hematologii, Onkologiczne Centrum Wsparcia Badań Klinicznych, Ul. Indiry Gandhi 14, 02-776, Warsaw

Aidport Sp. z o.o.

2803 : NA, Ul Ksiedza Stanisława Kozierowskiego 24, 60-185, Skorzewo

Specjalistyczny Szpital Im. Dra Alfreda Sokolowskiego

2800 : Oddział Hematologiczny, Ul. Alfreda Sokolowskiego 4, 58-309, Walbrzych

Uniwersyteckie Centrum Kliniczne

2804 : Klinika Hematologii i Transplantologii, Ul. Mariana Smoluchowskiego 17, 80-214, Gdansk

Pratia Hematologia Sp. z o.o.

2806 : Pratia Onkologia Katowice, Ul. Tadeusza Kosciuszki 92, 40-519, Katowice

Spain

15 sites · Authorised, recruitment pending

Hospital Universitario Donostia

2914: Hematology, Pasealeku Doct. Begiristain 109, 20014, Donostia

Institut Catala D'oncologia

2909: Hematology, Avinguda De La Gran Via De L'hospitalet 199-203, 08908, L'hospitalet De Llobregat

Hospital Universitario Quironsalud Madrid

2912: Hematology, Calle De Diego De Velazquez 1, 28223, Pozuelo De Alarcon

El Hospital Universitario De Gran Canaria Dr. Negrin

2902: Hematology, Barranco De La Ballena S N, 35010, Las Palmas De Gran Canaria

University Clinical Hospital Virgen De La Arrixaca

2908: Hematology, Carretera Madrid-Cartagena S/N, El Palmar, Murcia

Hospital General Universitario Gregorio Maranon

2907: Hematology, Calle Del Doctor Esquerdo 46, 28009, Madrid

Hospital Universitari Vall D Hebron

2900: Hematology, Passeig De La Vall D'Hebron 119-129, 08035, Barcelona

Hospital Clinic De Barcelona

2901: Hematology, Calle Villarroel 170, 08036, Barcelona

Hospital General Universitario Dr. Balmis

2906: Hematology, Avinguda Del Pintor Baeza 12, 03010, Alicante

Hospital Universitario Regional De Malaga

2904: Hematology, Avenida De Carlos De Haya S/N, 29010, Malaga

Area De Salud De Leon Y El Bierzo

2913: Hematology, Calle Altos De Nava S/N, 24008, Leon

Hospital Universitario De Navarra

2915: Hematology, Irunlarrea Kalea 3, 31008, Pamplona

Hospital Universitario De Salamanca

2905: Hematology, Paseo De San Vicente 58-182, 37007, Salamanca

Hospital Universitario Ramon Y Cajal

2903: Hematology, Carretera Del Colmenar Viejo Km 9 100, Por El Pardo, Madrid

Institut Catala D'oncologia

2911: Hematology, Avinguda De Franca S/n, 17007, Girona

Results and documents

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

Documents 99 files

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

Type	Title	Version
Protocol (for publication)	D1_Protocol - Signature Page_2025-523555-66_1_English_Red	08Oct2025
Protocol (for publication)	D1_Protocol_2025-523555-66_1_English_Red	00
Protocol (for publication)	D4_Patient-facing document - PRO_1_Note to Assessor_NonRed	03Feb2026
Recruitment arrangements (for publication)	K1_Recruitment Arrangements - Country_1_AT_English_NonRed	v1.0
Recruitment arrangements (for publication)	K1_Recruitment Arrangements - Country_1_AT_German_NonRed	v1.0
Recruitment arrangements (for publication)	K1_Recruitment Arrangements - Country_1_BE_English_NonRed	6Jan2026
Recruitment arrangements (for publication)	K1_Recruitment Arrangements - Country_1_CZ_NonRed	V01
Recruitment arrangements (for publication)	K1_Recruitment Arrangements - Country_1_DE_English_NonRed	v01
Recruitment arrangements (for publication)	K1_Recruitment Arrangements - Country_1_ES_Spanish_NonRed	v2.0
Recruitment arrangements (for publication)	K1_Recruitment Arrangements - Country_1_FR_NonRed	V00
Recruitment arrangements (for publication)	K1_Recruitment Arrangements - Country_1_IT_English_NonRed	1.0
Recruitment arrangements (for publication)	K1_Recruitment Arrangements - Country_1_NL_English_NonRed	V01
Recruitment arrangements (for publication)	K1_Recruitment Arrangements - Country_2_AT_German_NonRed	v1.0
Recruitment arrangements (for publication)	K1_Recruitments Arrangements_1_PL_Polish_NonRed	2.0
Recruitment arrangements (for publication)	K2_Advertisements - Country_1_BE_Dutch_NonRed	V1.0
Recruitment arrangements (for publication)	K2_Advertisements - Country_1_BE_French_NonRed	V1.0
Recruitment arrangements (for publication)	K2_Advertisements - Country_1_CZ_Czech_NonRed	V1.0
Recruitment arrangements (for publication)	K2_Advertisements - Country_1_DE_German_NonRed	v0
Recruitment arrangements (for publication)	K2_Advertisements - Country_1_ES_Spanish_NonRed	v1.0
Recruitment arrangements (for publication)	K2_Advertisements - Country_1_FR_French_NonRed	01
Recruitment arrangements (for publication)	K2_Advertisements - Country_1_IT_Italian_NonRed	1.0
Recruitment arrangements (for publication)	K2_Advertisements - Country_1_NL_Dutch_NonRed	V00
Recruitment arrangements (for publication)	K2_Advertisements - Country_1_PL_Polish_NonRed	1.0
Recruitment arrangements (for publication)	K2_Advertisements - Country_2_BE_Dutch_NonRed	V1.0
Recruitment arrangements (for publication)	K2_Advertisements - Country_2_BE_French_NonRed	V1.0
Recruitment arrangements (for publication)	K2_Advertisements - Country_2_CZ_Czech_NonRed	V1.0
Recruitment arrangements (for publication)	K2_Advertisements - Country_2_DE_German_NonRed	v00
Recruitment arrangements (for publication)	K2_Advertisements - Country_2_IT_Italian_NonRed	1.0
Recruitment arrangements (for publication)	K2_Advertisements - Country_2_NL_Dutch_NonRed	V00
Recruitment arrangements (for publication)	K2_Advertisements - Country_2_PL_Polish_NonRed	1.0
Recruitment arrangements (for publication)	K2_Advertisements - Country_3_CZ_Czech_NonRed	V1.0
Recruitment arrangements (for publication)	K2_Advertisements - Country_3_DE_German_NonRed	v1.0
Subject information and informed consent form (for publication)	L1_ICF - Follow up for pregnant participant_1_CZ_Czech_NonRed	v00.00.01
Subject information and informed consent form (for publication)	L1_ICF - Follow up for pregnant participant_1_DE_German_Red	v00.00.00
Subject information and informed consent form (for publication)	L1_ICF - Follow up for pregnant participant_1_ES_Spanish_NonRed	v00.00.01
Subject information and informed consent form (for publication)	L1_ICF - Follow up for pregnant participant_1_FR_French_Red	V00.00.00
Subject information and informed consent form (for publication)	L1_ICF - Follow up for pregnant participant_1_IT_Italian_NonRed	00.00.00
Subject information and informed consent form (for publication)	L1_ICF - Follow up for pregnant participant_1_NL_Dutch_NonRed	V00.00.00
Subject information and informed consent form (for publication)	L1_ICF - Follow up for pregnant participant_1_PL_Polish_NonRed	00.00.00
Subject information and informed consent form (for publication)	L1_ICF - Follow up for pregnant partner of participant_1_BE_Dutch_NonRed	v00.00.00
Subject information and informed consent form (for publication)	L1_ICF - Follow up for pregnant partner of participant_1_BE_English_NonRed	v00.00.00
Subject information and informed consent form (for publication)	L1_ICF - Follow up for pregnant partner of participant_1_BE_French_NonRed	v00.00.00
Subject information and informed consent form (for publication)	L1_ICF - Follow up for pregnant partner of participant_1_CZ_Czech_NonRed	v00.00.01
Subject information and informed consent form (for publication)	L1_ICF - Follow up for pregnant partner of participant_1_DE_German_Red	v00.00.00
Subject information and informed consent form (for publication)	L1_ICF - Follow up for pregnant partner of participant_1_ES_Spanish_NonRed	v00.00.01
Subject information and informed consent form (for publication)	L1_ICF - Follow up for pregnant partner of participant_1_IT_Italian_NonRed	00.00.00
Subject information and informed consent form (for publication)	L1_ICF - Follow up for pregnant partner of participant_1_NL_Dutch_NonRed	V00.00.00
Subject information and informed consent form (for publication)	L1_ICF - Follow up for pregnant partner of participant_1_PL_Polish_NonRed	00.00.00
Subject information and informed consent form (for publication)	L1_ICF - Info Sheet Female Partner_1_BE_Dutch_NonRed	v00.00.00
Subject information and informed consent form (for publication)	L1_ICF - Info Sheet Female Partner_1_BE_English_NonRed	v00.00.00
Subject information and informed consent form (for publication)	L1_ICF - Info Sheet Female Partner_1_BE_French_NonRed	v00.00.00
Subject information and informed consent form (for publication)	L1_ICF - Info Sheet Female Partner_1_CZ_Czech_NonRed	v00.00.01
Subject information and informed consent form (for publication)	L1_ICF - Info Sheet Female Partner_1_DE_German_NonRed	v00.00.00
Subject information and informed consent form (for publication)	L1_ICF - Info Sheet Female Partner_1_ES_Spanish_NonRed	v00.00.00
Subject information and informed consent form (for publication)	L1_ICF - Info Sheet Female Partner_1_FR_French_Red	V00.00.00
Subject information and informed consent form (for publication)	L1_ICF - Info Sheet Female Partner_1_IT_Italian_NonRed	00.00.00
Subject information and informed consent form (for publication)	L1_ICF - Info Sheet Female Partner_1_PL_Polish_NonRed	00.00.00
Subject information and informed consent form (for publication)	L1_ICF - Main ICF - Adult_1_AT_German_Red	v00.00.03
Subject information and informed consent form (for publication)	L1_ICF - Main ICF - Adult_1_BE_Dutch_Red	v00.00.01
Subject information and informed consent form (for publication)	L1_ICF - Main ICF - Adult_1_BE_English_Red	v00.00.01
Subject information and informed consent form (for publication)	L1_ICF - Main ICF - Adult_1_BE_French_Red	v00.00.01
Subject information and informed consent form (for publication)	L1_ICF - Main ICF - Adult_1_CZ_Czech_Red	v00.00.01
Subject information and informed consent form (for publication)	L1_ICF - Main ICF - Adult_1_DE_German_Red	v00.00.02
Subject information and informed consent form (for publication)	L1_ICF - Main ICF - Adult_1_ES_Spanish_Red	v00.00.01
Subject information and informed consent form (for publication)	L1_ICF - Main ICF - Adult_1_FR_French_Red	V00.00.00
Subject information and informed consent form (for publication)	L1_ICF - Main ICF - Adult_1_IT_Italian_Red	00.00.01
Subject information and informed consent form (for publication)	L1_ICF - Main ICF - Adult_1_NL_Dutch_Red	V00.00.00
Subject information and informed consent form (for publication)	L1_ICF - Main ICF - Adult_1_PL_Polish_NonRed	00.00.00
Subject information and informed consent form (for publication)	L1_ICF - Optional Assessment_1_CZ_Czech_Red	v00.00.01
Subject information and informed consent form (for publication)	L1_ICF - Optional Assessment_1_ES_Spanish_Red	v00.00.00
Subject information and informed consent form (for publication)	L1_ICF - Optional Assessment_1_IT_Italian_Red	00.00.00
Subject information and informed consent form (for publication)	L1_ICF - Optional Assessment_1_PL_Polish_NonRed	v00.00.01
Subject information and informed consent form (for publication)	L1_ICF - Optional_1_AT_German_NonRed	v00.00.01
Subject information and informed consent form (for publication)	L1_ICF - Optional_2_AT_German_NonRed	v00.00.01
Subject information and informed consent form (for publication)	L1_ICF - Optional_3_AT_German_NonRed	v00.00.01
Subject information and informed consent form (for publication)	L1_ICF - Pregnancy Follow up Parent Legal Guardian_1_FR_French_Red	V00.00.00
Subject information and informed consent form (for publication)	L1_ICF - Research_1_DE_German_Red	00.00.01
Subject information and informed consent form (for publication)	L1_ICF - Separate Data Protection Consent_1_CZ_Czech_NonRed	v00.00.01
Subject information and informed consent form (for publication)	L1_List of submitted documents Part II_1_CZ_NonRed	v1.0
Subject information and informed consent form (for publication)	L1_Patient Card_1_CZ_Czech_NonRed	24Jan2024
Subject information and informed consent form (for publication)	L1_Patient Card_1_DE_German_NonRed	v01
Subject information and informed consent form (for publication)	L1_Subject Info Sheet or Other Info_1_AT_German_Red	v1.1
Subject information and informed consent form (for publication)	L1_Subject Info Sheet or Other Info_1_ES_Spanish_NonRed	v1.0
Subject information and informed consent form (for publication)	L1_Subject Info Sheet or Other Info_2_AT_German_NonRed	v4.0
Subject information and informed consent form (for publication)	L1_Subject Info Sheet or Other Info_3_AT_German_NonRed	v4.0
Subject information and informed consent form (for publication)	L1_Subject Info Sheet or Other Info_4_AT_German_NonRed	v5Dec2012
Subject information and informed consent form (for publication)	L2_ICF Procedure_1_AT_English_NonRed	v1.0
Subject information and informed consent form (for publication)	L2_ICF Procedure_1_BE_English_Red	6Jan2026
Subject information and informed consent form (for publication)	L2_ICF Procedure_1_DE_English_NonRed	v01
Subject information and informed consent form (for publication)	L2_ICF Procedure_1_ES_Spanish_NonRed	v1.0
Synopsis of the protocol (for publication)	D1_Protocol Summary in Lay Language_2025-523555-66-00_1_CZ_Czech_NonRed	1
Synopsis of the protocol (for publication)	D1_Protocol Summary in Lay Language_2025-523555-66-00_1_English_NonRed	v01
Synopsis of the protocol (for publication)	D1_Protocol Summary in Lay Language_2025-523555-66-00_1_ES_Spanish_NonRed	00
Synopsis of the protocol (for publication)	D1_Protocol Summary in Lay Language_2025-523555-66-00_1_FR_French_NonRed	V01
Synopsis of the protocol (for publication)	D1_Protocol Summary in Lay Language_2025-523555-66-00_1_IT_Italian_NonRed	00
Synopsis of the protocol (for publication)	D1_Protocol Summary in Lay Language_2025-523555-66-00_1_NL_Dutch_NonRed	V00
Synopsis of the protocol (for publication)	D1_Protocol Summary in Lay Language_2025-523555-66-00_1_PL_Polish_NonRed	0.1
Synopsis of the protocol (for publication)	D1_Protocol Summary in Technical Language_2025-523555-66-00_1_AT_German_Red	V00
Synopsis of the protocol (for publication)	D1_Protocol Summary in Technical Language_2025-523555-66-00_1_CZ_Czech_Red	V1.0

Application history

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

#	Type	Code	Submitted	Reference MS	Conclusion	Decision date
1	INITIAL	IN	2026-02-06	Germany	Acceptable with conditions 2026-05-29	2026-05-29

A study of pelabresib and ruxolitinib compared to placebo and ruxolitinib in adult patients with myelofibrosis who have not received prior treatment with a Janus kinase inhibitor.

Overview

Key facts

External identifiers

Trial design

Main objective

Secondary objectives 12

Conditions and MedDRA coding

Eligibility criteria

Inclusion criteria 7

Exclusion criteria 6

Endpoints

Primary endpoints 1

Secondary endpoints 12

Investigational products

Test 2

Placebo 1

Auxiliary 9

Sponsors and contacts

Novartis Pharma AG

Scientific contact point

Public contact point

Third parties 5

Locations

By country

Investigational sites

Results and documents

Documents 99 files

Application history

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