Clinical trial • Phase II • Cardiology

HUMAN IGG4 KAPPA MONOCLONAL ANTIBODY AGAINST PLATELET-DERIVED GROWTH FACTOR SUBUNIT B for Pulmonary arterial hypertension

Phase II trial of HUMAN IGG4 KAPPA MONOCLONAL ANTIBODY AGAINST PLATELET-DERIVED GROWTH FACTOR SUBUNIT B for Pulmonary arterial hypertension.

Overview

Trial Therapeutic Area
Cardiology
Trial Disease
Pulmonary arterial hypertension
Trial Stage
Phase II
Drug Modality
Monoclonal antibody

Key dates

Initial CTIS Submission Date
21-11-2025
First CTIS Authorization Date
18-03-2026

Trial design

Randomised, placebo to regn13335 (placebo comparator). background standard of care (soc) therapy on a stable dose and regimen is permitted as described in the protocol. (no dose or schedule for placebo specified in provided data.)-controlled Phase II trial across 30 sites in Belgium, France, Germany and others.

Randomised
Yes
Comparator
Placebo to REGN13335 (placebo comparator). Background Standard Of Care (SOC) therapy on a stable dose and regimen is permitted as described in the protocol. (No dose or schedule for placebo specified in provided data.)
Target Sample Size
52

Eligibility

Recruits 52 adults.

Inclusion criteria

  • {"criterion_text":"- Documented clinical diagnosis of PAH (Group 1 PH according to the 7th World Symposium on Pulmonary Hypertension (WSPH))"}
  • {"criterion_text":"- WHO functional class II or III (slight to marked limitation of functional status due to PAH)"}
  • {"criterion_text":"- Receiving background Standard Of Care (SOC) therapy for PAH on a stable dose and regimen, as determined by the investigator, as described in the protocol"}
  • {"criterion_text":"- PVR ≥400 dynes∙sec/cm^5 (5 Wood units) based on Right Heart Catheterization (RHC) during the screening period"}
  • {"criterion_text":"- Has 6MWD ≥150 and ≤550 meters repeated twice during screening as described in the protocol"}
  • {"criterion_text":"- Note: Other protocol-defined inclusion criteria apply"}

Exclusion criteria

  • {"criterion_text":"- Has Group 2 (PH associated with left heart disease), Group 3 (PH associated with lung diseases and/or hypoxia), Group 4 (PH associated with pulmonary artery obstructions), or Group 5 (PH with unclear and/or multifactorial mechanisms) PH according to the 7th WSPH"}
  • {"criterion_text":"- Note: Other protocol-defined exclusion criteria apply"}
  • {"criterion_text":"- Pulmonary Arterial Wedge Pressure (PAWP) >15 mm Hg by RHC during the screening period"}
  • {"criterion_text":"- History of left-sided heart disease and/or clinically significant cardiac disease, as described in the protocol"}
  • {"criterion_text":"- Obstructive lung disease defined as Forced Expiratory Volume in 1 second (FEV1)/Forced Vital Capacity <0.7 and FEV1 <70% of the predicted value as described in the protocol"}
  • {"criterion_text":"- Evidence of interstitial lung disease as defined in the protocol"}
  • {"criterion_text":"- Evidence of chronic thromboembolic pulmonary disease or acute pulmonary embolism as described in the protocol"}
  • {"criterion_text":"- Participants requiring anticoagulation and/or antiplatelet therapy for an underlying medical condition as described in the protocol"}
  • {"criterion_text":"- Has any history of intracranial bleeding or any history of elevated intracranial pressure"}
  • {"criterion_text":"- Has any history of bleeding meeting criteria as described in the protocol"}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Change from baseline in Pulmonary Vascular Resistance (PVR)","definition_or_measurement_approach":"Change from baseline in PVR; PVR is assessed based on Right Heart Catheterization (RHC) measurements during screening and follow-up as described in the protocol"}

Secondary endpoints

  • {"endpoint_text":"- Occurrence of Treatment-Emergent Adverse Events (TEAEs)","definition_or_measurement_approach":"Standard reporting of treatment-emergent adverse events collected throughout the study period as per protocol safety assessments"}
  • {"endpoint_text":"- Severity of TEAEs","definition_or_measurement_approach":"Severity graded according to protocol-specified criteria (e.g., CTCAE or study definitions) as collected in safety assessments"}
  • {"endpoint_text":"- Change from baseline in circulating N-Terminal pro-B-type Natriuretic Peptide (NT-proBNP) concentrations","definition_or_measurement_approach":"Change from baseline in circulating NT-proBNP measured in plasma samples using validated laboratory assays"}
  • {"endpoint_text":"- Change from baseline in mean pulmonary artery pressure","definition_or_measurement_approach":"Change from baseline in mean pulmonary artery pressure assessed by hemodynamic measurements (e.g., RHC) per protocol"}
  • {"endpoint_text":"- Change from baseline in cardiac output","definition_or_measurement_approach":"Change from baseline in cardiac output measured by hemodynamic assessments as specified in the protocol"}
  • {"endpoint_text":"- Change from baseline in cardiac index","definition_or_measurement_approach":"Change from baseline in cardiac index measured via hemodynamic methods per protocol"}
  • {"endpoint_text":"- Change from baseline in right atrial pressure","definition_or_measurement_approach":"Change from baseline in right atrial pressure measured by RHC or protocol-specified hemodynamic assessment"}
  • {"endpoint_text":"- Change from baseline in 6-Minute Walk Distance (6MWD)","definition_or_measurement_approach":"Change from baseline in distance walked during standardized 6-minute walk test (6MWD) performed per protocol"}
  • {"endpoint_text":"- Concentrations of functional REGN13335 in plasma","definition_or_measurement_approach":"Plasma concentrations of functional REGN13335 measured using validated bioanalytical assays"}
  • {"endpoint_text":"- Concentrations of total soluble Platelet-Derived Growth Factor-B (PDGF-B) ligands in plasma","definition_or_measurement_approach":"Plasma concentrations of total soluble PDGF-B ligands measured using validated biomarker assays"}

Recruitment

Digital Remote Recruitment
True, recruitment uses digital/remote methods including banner ads, basic website materials, telemedicine support, and eCOA/electronic materials (vendors listed for telemedicine and eCOA)
Planned Sample Size
52
Recruitment Window Months
28
Consent Approach
Informed consent is obtained using subject information and informed consent forms (Main ICF and related ICFs). Multiple language versions of ICFs and supporting consent materials are provided (document titles include EN, FR, NL, LV, RU, CZ, DE, PL, ES). A Pregnant Partner ICF and other subject-specific ICFs are included. Consent is obtained from each adult participant; materials and procedures are country-specific as per the 'Recruitment and Informed Consent Procedure' documents.

Methods

  • Online banner ads and basic website materials (document titles include 'Banner Ads' and 'Basic Website 5 pg')
  • Study brochures and printed recruitment materials ('Study Brochure' documents)
  • Site-based recruitment supported by local recruitment and informed consent procedure documents (country-specific 'Recruitment and Informed Consent Procedure' files)
  • Telemedicine support for recruitment and participant interactions (Datacubed Health listed with role 'Telemedicine')
  • Recruitment and retention materials provided by a vendor (Clariness GmbH responsible for 'Recruitment and Retention Materials')

Geography

Total Number Of Sites
30
Total Number Of Participants
47

Belgium

Earliest CTIS Part Ii Submission Date
25-02-2026
Latest Decision Or Authorization Date
18-03-2026
Processing Time Days
21
Number Of Sites
2
Number Of Participants
2

Sites

Site Name
Hopital Erasme
Department Name
056001:Cardiologie
Principal Investigator Name
Jean-Luc Vachiery
Principal Investigator Email
jean.luc.vachiery@hubruxelles.be
Contact Person Name
Jean-Luc Vachiery
Site Name
UZ Leuven
Department Name
056002:Pneumologie
Principal Investigator Name
Marion Delcroix
Principal Investigator Email
marion.delcroix@uzleuven.be
Contact Person Name
Marion Delcroix
Contact Person Email
marion.delcroix@uzleuven.be

France

Earliest CTIS Part Ii Submission Date
09-03-2026
Latest Decision Or Authorization Date
20-03-2026
Processing Time Days
11
Number Of Sites
6
Number Of Participants
9

Sites

Site Name
Centre Hospitalier Universitaire Grenoble Alpes
Department Name
250004:Cardiologie
Principal Investigator Name
Helene Bouvaist
Principal Investigator Email
hbouvaist@chu-grenoble.fr
Contact Person Name
Helene Bouvaist
Contact Person Email
hbouvaist@chu-grenoble.fr
Site Name
Les Hopitaux Universitaires De Strasbourg
Department Name
250002:Pneumologie
Principal Investigator Name
Marianne Riou
Principal Investigator Email
marianne.riou@chru-strasbourg.fr
Contact Person Name
Marianne Riou
Site Name
Assistance Publique Hopitaux De Paris
Department Name
250001: Pneumologie
Principal Investigator Name
Olivier Sitbon
Principal Investigator Email
olivier.sitbon@aphp.fr
Contact Person Name
Olivier Sitbon
Contact Person Email
olivier.sitbon@aphp.fr
Site Name
Centre Hospitalier Universitaire De Toulouse
Department Name
250006: Pneumologie
Principal Investigator Name
Grégoire Prevot
Principal Investigator Email
prevot.g@chu-toulouse.fr
Contact Person Name
Grégoire Prevot
Contact Person Email
prevot.g@chu-toulouse.fr
Site Name
Centre Hospitalier Universitaire De Bordeaux
Department Name
250003:Pneumologie
Principal Investigator Name
Arnaud Maurac
Principal Investigator Email
arnaud.maurac@chu-bordeaux.fr
Contact Person Name
Arnaud Maurac
Contact Person Email
arnaud.maurac@chu-bordeaux.fr
Site Name
Centre Hospitalier Universitaire De Saint Etienne
Department Name
250005:Médecine Vasculaire et Thérapeutiques
Principal Investigator Name
Laurent Bertoletti
Principal Investigator Email
laurent.bertoletti@chu-st-etienne.fr
Contact Person Name
Laurent Bertoletti

Germany

Earliest CTIS Part Ii Submission Date
10-03-2026
Latest Decision Or Authorization Date
26-03-2026
Processing Time Days
16
Number Of Sites
5
Number Of Participants
7

Sites

Site Name
Universitaetsmedizin Greifswald KöR
Department Name
276001; Zentrum für Innere Medizin, Lungenheilkunde und Infektionskrankheiten
Principal Investigator Name
Ralf Ewert
Principal Investigator Email
ewert@uni-greifswald.de
Contact Person Name
Ralf Ewert
Contact Person Email
ewert@uni-greifswald.de
Site Name
Universitaetsmedizin der Johannes Gutenberg-Universitaet Mainz KöR
Department Name
276006; Hautklinik und Poliklinik
Principal Investigator Name
Kai-Helge Schmidt
Principal Investigator Email
kai_helge.schmidt@unimedizin-mainz.de
Contact Person Name
Kai-Helge Schmidt
Site Name
Martin-Luther-Universitaet Halle-Wittenberg
Department Name
276005; Innere Medizin
Principal Investigator Name
Stephan Eisenmann
Principal Investigator Email
stephan.eisenmann@uk-halle.de
Contact Person Name
Stephan Eisenmann
Contact Person Email
stephan.eisenmann@uk-halle.de
Site Name
Krankenhaus Neuwittelsbach
Department Name
276004; Pulmonology
Principal Investigator Name
Hanno Leuchte
Principal Investigator Email
prof.leuchte@krankenhaus-neuwittelsbach.de
Contact Person Name
Hanno Leuchte
Site Name
Justus-Liebig-Universitaet Giessen
Department Name
276003; Medizinische Klinik und Poliklinik II
Principal Investigator Name
Ardeschir Hossein Ghofrani
Contact Person Name
Ardeschir Hossein Ghofrani

Latvia

Earliest CTIS Part Ii Submission Date
02-03-2026
Latest Decision Or Authorization Date
18-03-2026
Processing Time Days
16
Number Of Sites
1
Number Of Participants
3

Sites

Site Name
Pauls Stradins Clinical University Hospital
Department Name
428001: Department No.24
Principal Investigator Name
Andris Skride
Principal Investigator Email
andris.skride@gmail.com
Contact Person Name
Andris Skride
Contact Person Email
andris.skride@gmail.com

Spain

Earliest CTIS Part Ii Submission Date
23-03-2026
Latest Decision Or Authorization Date
26-03-2026
Processing Time Days
3
Number Of Sites
7
Number Of Participants
9

Sites

Site Name
Hospital Costa Del Sol
Department Name
724005; Cardiology
Principal Investigator Name
Rafael Bravo Marques
Principal Investigator Email
rafabravomarques@gmail.com
Contact Person Name
Rafael Bravo Marques
Contact Person Email
rafabravomarques@gmail.com
Site Name
Hospital Universitario Marques De Valdecilla
Department Name
724004; Pulmonology
Principal Investigator Name
Jose Cifrian Martinez
Principal Investigator Email
josemanuel.cifrian@scsalud.es
Contact Person Name
Jose Cifrian Martinez
Contact Person Email
josemanuel.cifrian@scsalud.es
Site Name
Hospital Clinic De Barcelona
Department Name
724007; Neumología
Principal Investigator Name
Isabel Blanco
Principal Investigator Email
iblanco2@clinic.cat
Contact Person Name
Isabel Blanco
Contact Person Email
iblanco2@clinic.cat
Site Name
Hospital Universitario 12 De Octubre
Department Name
724002; Cardiology
Principal Investigator Name
Pilar Escribano Subías
Principal Investigator Email
pilar.escribano.subias@gmail.com
Contact Person Name
Pilar Escribano Subías
Site Name
Hospital Universitario De Salamanca
Department Name
724006; Medicina Interna
Principal Investigator Name
Sergio Cadenas Menendez
Principal Investigator Email
sercam.2007@gmail.com
Contact Person Name
Sergio Cadenas Menendez
Contact Person Email
sercam.2007@gmail.com
Site Name
Hospital Universitari Vall D Hebron
Department Name
724001; Pneumology
Principal Investigator Name
Manuel López Meseguer
Principal Investigator Email
manuel.lopez@vallhebron.cat
Contact Person Name
Manuel López Meseguer
Contact Person Email
manuel.lopez@vallhebron.cat
Site Name
Hospital Universitario De Toledo
Department Name
724003; Cardiology
Principal Investigator Name
Maria Lazaro Salvador
Principal Investigator Email
maria.lasal@gmail.com
Contact Person Name
Maria Lazaro Salvador
Contact Person Email
maria.lasal@gmail.com

Poland

Earliest CTIS Part Ii Submission Date
20-02-2026
Latest Decision Or Authorization Date
27-03-2026
Processing Time Days
35
Number Of Sites
6
Number Of Participants
12

Sites

Site Name
Centrum Medyczne "Hipokrates" S.C. Elżbieta i Grzegorz Grześk
Department Name
616006: Cardiology
Principal Investigator Name
Grzegorz Grzesk
Principal Investigator Email
grzegorz.grzesk@interia.pl
Contact Person Name
Grzegorz Grzesk
Contact Person Email
ggrzesk@cm.umk.pl
Site Name
Wojewodzki Specjalistyczny Szpital Im Dr Wl Bieganskiego
Department Name
616003: Oddzial Kardiologiczny Klinika Kardiologii Katedry Kardiologii UM w Lodzi
Principal Investigator Name
Radoslaw Krecki
Principal Investigator Email
rkrecki@gmail.com
Contact Person Name
Radoslaw Krecki
Contact Person Email
rkrecki@gmail.com
Site Name
Krakowski Szpital Specjalistyczny Im. Sw. Jana Pawla II
Department Name
616004: Oddzial Kliniczny Chorob Serca i Naczyn z Pododdzialem Intensywnego Nadzoru Kardiologicznego
Principal Investigator Name
Grzegorz Kopec
Principal Investigator Email
grzegorzkrakow1@gmail.com
Contact Person Name
Grzegorz Kopec
Contact Person Email
grzegorzkrakow1@gmail.com
Site Name
Uniwersytecki Szpital Kliniczny W Bialymstoku
Department Name
616001: Klinika Kardiologii i Chorob Wewnetrznych z OINK
Principal Investigator Name
Karol Kaminski
Principal Investigator Email
fizklin@wp.pl
Contact Person Name
Karol Kaminski
Contact Person Email
fizklin@wp.pl
Site Name
Gornoslaskie Centrum Medyczne Im Prof. Leszka Gieca Sląskiego Uniwersytetu Medycznego W Katowicach
Department Name
616005: II Oddzial Kardiologii
Principal Investigator Name
Grzegorz Smolka
Principal Investigator Email
gsmolka@me.com
Contact Person Name
Grzegorz Smolka
Contact Person Email
gsmolka@me.com
Site Name
Uniwersytecki Szpital Kliniczny W Poznaniu
Department Name
616002: Ośrodek Wczesnych Faz
Principal Investigator Name
Tatiana Mularek-Kubzdela
Principal Investigator Email
tatianamularek@wp.pl
Contact Person Name
Tatiana Mularek-Kubzdela
Contact Person Email
tatianamularek@wp.pl

Czechia

Earliest CTIS Part Ii Submission Date
24-02-2026
Latest Decision Or Authorization Date
20-04-2026
Processing Time Days
55
Number Of Sites
3
Number Of Participants
5

Sites

Site Name
University Hospital Olomouc
Department Name
203003:III. Interni klinika
Principal Investigator Name
Jan Precek
Principal Investigator Email
jan.precek@seznam.cz
Contact Person Name
Jan Precek
Contact Person Email
jan.precek@seznam.cz
Site Name
Vseobecna Fakultni Nemocnice V Praze
Department Name
203002:II. Interni klinika
Principal Investigator Name
Pavel Jansa
Principal Investigator Email
pavel.jansa@vfn.cz
Contact Person Name
Pavel Jansa
Contact Person Email
pavel.jansa@vfn.cz
Site Name
Institute For Clinical And Experimental Medicine
Department Name
203001:Klinika kardiologie
Principal Investigator Name
Hikmet Al-Hiti
Principal Investigator Email
hial@medicon.cz
Contact Person Name
Hikmet Al-Hiti
Contact Person Email
hial@medicon.cz

Sponsor

Primary sponsor

Full Name
Regeneron Pharmaceuticals Inc.
Organisation Type
Pharmaceutical company
Country Of Registered Address
United States

Contract research organisations

Name
Parexel International (IRL) Limited
Responsibilities
CRO

Third parties

  • {"country":"Slovakia","full_name":"SanaClis s.r.o.","duties_or_roles":"Drug Distribution","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Cardiovascular Clinical Sciences Inc.","duties_or_roles":"Central Reading (RHC) & Clinical Event Adjudication","organisation_type":"Pharmaceutical company"}
  • {"country":"Ireland","full_name":"Parexel International (IRL) Limited","duties_or_roles":"CRO","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Datacubed Health Inc.","duties_or_roles":"Telemedicine","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Yprime LLC","duties_or_roles":"","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"Poland","full_name":"Medicover Integrated Clinical Services Sp. z o.o.","duties_or_roles":"Specialty Lab","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"United States","full_name":"Transperfect Translations International Inc.","duties_or_roles":"Translations","organisation_type":"Pharmaceutical company"}
  • {"country":"France","full_name":"Mapi Research Trust","duties_or_roles":"Translations - PAH SYMPACT","organisation_type":"Pharmaceutical company"}
  • {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"Central Lab","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"WCG Clinical Inc.","duties_or_roles":"IDMC Vendor","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Simpleshow USA Corp.","duties_or_roles":"OLE IP preparation and administration training video","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"Germany","full_name":"Clariness GmbH","duties_or_roles":"Recruitment and Retention Materials","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"United States","full_name":"Bioclinica Inc.","duties_or_roles":"Imaging - ECHO","organisation_type":"Laboratory/Research/Testing facility"}

Investigational products

Investigational Product Name
REGN13335
Active Substance
HUMAN IGG4 KAPPA MONOCLONAL ANTIBODY AGAINST PLATELET-DERIVED GROWTH FACTOR SUBUNIT B
Modality
Monoclonal antibody
Routes Of Administration
INTRAVENOUS/SUBCUTANEOUS/INTRAMUSCULAR
Route
INTRAVENOUS/SUBCUTANEOUS/INTRAMUSCULAR
Investigational Product Name
Placebo to REGN13335
Modality
Other

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