Clinical trial • Phase I/II • Respiratory

LTP001 for Pulmonary arterial hypertension

Phase I/II trial of LTP001 for Pulmonary arterial hypertension.

Overview

Trial Therapeutic Area
Respiratory
Trial Disease
Pulmonary arterial hypertension
Trial Stage
Phase I/II
Drug Modality
Small molecule

Key dates

Initial CTIS Submission Date
30-04-2025
First CTIS Authorization Date
22-08-2025

Trial design

Randomised, placebo (placebo to ltp001 00mg hard gelatin capsule) plus standard-of-care (soc); active comparator arm: ltp001 plus soc (dose and schedule not specified in provided documents).-controlled, adaptive Phase I/II trial in Belgium, Czechia, Germany and others.

Randomised
Yes
Comparator
Placebo (Placebo to LTP001 00mg hard gelatin capsule) plus standard-of-care (SoC); active comparator arm: LTP001 plus SoC (dose and schedule not specified in provided documents).
Adaptive
True (Part A uses single and multiple ascending dose escalation; Part B Period 1 is designed to characterize dose-response. Specific interim analysis or stopping rules are not specified in the provided text.)
Single Multiple Or Escalation Dose Combined
Yes
Target Sample Size
53
Trial Duration For Participant
540

Eligibility

Recruits 53 Vulnerable population selected. Signed informed consent must be obtained prior to participation. Study includes specific informed consent documents for pregnant participants and pregnant partners and pre-ICF telephone data consent; assent procedures are not described..

Pregnancy Exclusion
Part B: Women of child-bearing potential unless they are using highly effective methods of contraception
Vulnerable Population
Vulnerable population selected. Signed informed consent must be obtained prior to participation. Study includes specific informed consent documents for pregnant participants and pregnant partners and pre-ICF telephone data consent; assent procedures are not described.

Inclusion criteria

  • {"criterion_text":"- Part A: Signed informed consent must be obtained prior to participation in the study. Part B: Confirmed diagnosis of pulmonary arterial hypertension\n- Part A: Healthy male and non-childbearing potential female participants Part B: Pre-randomization PVR ≥400 dyn.sec.cm\n- Part A: In good health in the opinion of the investigator and as determined by medical history, physical examination, vital signs, ECG, and laboratory tests. Part B: Treatment with stable doses of standard-of-care PAH therapies\n- Part A: Able to read, speak, and understand the local language, to understand and comply with the requirements of the study. Part B: 6MWD ≥ 150 m and ≤450 m"}

Exclusion criteria

  • {"criterion_text":"- Part A: History or current diagnosis of clinically significant ECG or cardiac abnormalities indicating significant risk of safety for participants. Part B: Any surgical or medical condition which may place the participant at higher risk from his/her participation in the study\n- Part A: Use of any investigational drugs within 5 half-lives or within 30 days or until the expected pharmacodynamic effect has returned to baseline prior to initial dosing, whichever is longer. Part B: Women of child-bearing potential unless they are using highly effective methods of contraception\n- Part A: History of hypersensitivity to the investigational compound/compound class or excipients being used in this study. Part B: Sexually active males unwilling to use a condom during intercourse while taking study treatment and for 24 hours after stopping study treatment.\n- Part B: History of hypersensitivity to any of the study treatments or excipients"}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Part A: All safety endpoints, including vital signs, ECG, central lab evaluations and AEs up to and including the EoS visit. Part B: (Period 1) Change in PVR from baseline to 24 weeks (Period 2): All safety endpoints, including vital signs, ECG, central lab evaluations, treatment-emergent AEs and discontinuations due to AEs.","definition_or_measurement_approach":"Part A safety endpoints assessed by vital signs, ECG, central laboratory evaluations and adverse event reporting up to the end-of-study visit. Part B Period 1 primary efficacy endpoint is change in pulmonary vascular resistance (PVR) from baseline to 24 weeks; Period 2 primary endpoints are safety endpoints measured by vital signs, ECG, central labs, treatment-emergent AEs and discontinuations due to AEs."}

Secondary endpoints

  • {"endpoint_text":"- Part A: Cmax, Tmax, AUClast, AUCinf and T1/2 of LTP001 Part B: (Period 1) Change in 6MWD from baseline to 24 weeks. (Period 2) Change in 6MWD from baseline to 18 months","definition_or_measurement_approach":"Part A: plasma PK parameters (Cmax, Tmax, AUClast, AUCinf, T1/2) for LTP001. Part B: change in six-minute walk distance (6MWD) from baseline to 24 weeks (Period 1) and from baseline to 18 months (Period 2)."}
  • {"endpoint_text":"- Part B Period 2: Number of participants experiencing an improvement in WHO FC from baseline to 18 months","definition_or_measurement_approach":"Proportion/count of participants with improvement in WHO functional class from baseline to 18 months."}
  • {"endpoint_text":"- Part B (Period 1): Change in WHO FC from baseline to 24 weeks","definition_or_measurement_approach":"Change in WHO functional class from baseline to 24 weeks."}
  • {"endpoint_text":"- Part B (Period 1): All safety endpoints, including vital signs, ECG, clinical laboratory evaluations, treatment-emergent adverse events and discontinuations due to adverse events","definition_or_measurement_approach":"Safety assessments including vital signs, ECG, clinical laboratory evaluations, treatment-emergent AEs and discontinuations due to AEs."}

Recruitment

Digital Remote Recruitment
True (telephone pre-ICF consent, eCOA use and reminders, email communication for patient services and electronic consent-related materials are included in country documents).
Planned Sample Size
53
Recruitment Window Months
39
Consent Approach
Signed informed consent must be obtained from each participant prior to participation. Pre-ICF telephone data consent is used for initial contact. Dedicated main informed consent forms and supplementary/optional ICFs (e.g., pregnancy follow-up, partner ICF, optional additional research, home health services) are provided. Country-specific ICFs are available in local languages as evidenced by published ICF documents (examples include English, French, Dutch, German, Greek, Italian, Czech, Polish, Portuguese, Romanian, Spanish, Latvian, Russian).

Methods

  • Dr-to-Dr letters: direct letters to physicians to inform and recruit patients (documents present for multiple countries including BE, CZ, DE, GR, IT, LV, PT, RO, ES, PL, FR).
  • Dr-to-Patient / Referring-Dr-to-Patient letters: letters sent via clinicians to patients (country-specific versions present: BE, CZ, DE, GR, IT, RO, ES, PT, PL, LV, etc.).
  • Patient recruitment brochures / fact sheets / posters: printed patient-facing materials and posters for public/clinic display (country-specific versions present for BE, CZ, DE, FR, GR, IT, LV, PT, RO, ES, PL, etc.).
  • GP letters / Referrals: communications targeted at general practitioners to refer eligible patients (documents present, e.g., IT).
  • Pre-ICF Telephone Data Consent: telephone outreach and pre-screening consent via telephone (documents titled 'Pre-ICF Telephone Data Consent' present for BE, CZ, DE, GR, IT, PT, RO, ES, PL, FR, LV).
  • Patient Services / Concierge services / Home Trial Services: patient support and home trial service arrangements to facilitate recruitment and participation (documents and third-party responsibilities indicate Patient Services, Home Trial Services and Concierge Services).

Geography

Total Number Of Sites
51
Total Number Of Participants
68

Belgium

Earliest CTIS Part Ii Submission Date
29-07-2025
Latest Decision Or Authorization Date
27-08-2025
Processing Time Days
29
Number Of Sites
2
Number Of Participants
1

Sites

Site Name
UZ Leuven
Department Name
Department of Pneumology
Contact Person Name
Laurent Godinas
Contact Person Email
Laurent.godinas@uzleuven.be
Site Name
Hopital Erasme
Department Name
Department of Cardiology
Contact Person Name
Jean-Luc Vachiéry

Czechia

Earliest CTIS Part Ii Submission Date
11-07-2025
Latest Decision Or Authorization Date
26-08-2025
Processing Time Days
46
Number Of Sites
2
Number Of Participants
5

Sites

Site Name
Institute For Clinical And Experimental Medicine
Department Name
Klinika kardiologie Ambulance plicní hypertenze
Contact Person Name
Hikmet Al-Hiti
Contact Person Email
hial@medicon.cz
Site Name
Vseobecna Fakultni Nemocnice V Praze
Department Name
II. interní klinika kardiologie a angiologie VFN a 1. LF UK Centrum pro plicní hypertenzi
Contact Person Name
Pavel Jansa
Contact Person Email
pavel.jansa@vfn.cz

Germany

Earliest CTIS Part Ii Submission Date
14-08-2025
Latest Decision Or Authorization Date
26-08-2025
Processing Time Days
12
Number Of Sites
8
Number Of Participants
7

Sites

Site Name
DRK Kliniken Berlin
Department Name
Medizinische Klinik II
Contact Person Name
Christian Opitz
Contact Person Email
c.opitz@drk-kliniken-berlin.de
Site Name
Universitaet Des Saarlandes
Department Name
Innere Medizin V – Pneumologie, Allergologie, Beatmungs- und Umweltmedizin
Contact Person Name
Albert Omlor
Contact Person Email
Albert.Omlor@uks.eu
Site Name
Justus-Liebig-Universitaet Giessen
Department Name
Medizinische Klinik II, Ambulanz für pulmonale Hypertonie
Contact Person Name
Ardeschir Ghofrani
Site Name
Evangelische Lungenklinik Berlin Krankenhausbetriebs gGmbH
Contact Person Name
Christian Grohé
Contact Person Email
Christian.Grohe@jsd.de
Site Name
Universitaetsmedizin Greifswald KöR
Department Name
Klinik und Poliklinik für Innere Medizin B Innere Medizin, Pneumologie
Contact Person Name
Ralf Ewert
Contact Person Email
ewert@uni-greifswald.de
Site Name
Technische Universitaet Dresden
Department Name
Medizinische Klinik I / Pneumologisches
Contact Person Name
Michael Halank
Site Name
Universitaetsklinikum Regensburg AöR
Department Name
Klinik und Poliklinik für Innere Medizin II/Kardiologie
Contact Person Name
Stefan Stadler
Contact Person Email
stefan.stadler@ukr.de
Site Name
Thoraxklinik Heidelberg gGmbH
Department Name
Universitätsklinikum Heidelberg Zentrum für pulmonale Hypertonie
Contact Person Name
Ekkehard Grünig

Greece

Earliest CTIS Part Ii Submission Date
19-05-2025
Latest Decision Or Authorization Date
26-08-2025
Processing Time Days
99
Number Of Sites
3
Number Of Participants
6

Sites

Site Name
University General Hospital Attikon
Department Name
2nd Critical Care Department
Contact Person Name
Iraklis Tsagkaris
Contact Person Email
itsagkaris@med.uoa.gr
Site Name
University General Hospital Of Thessaloniki Ahepa
Department Name
1st Cardiology Clinic
Contact Person Name
Georgios Giannakoulas
Contact Person Email
g.giannakoulas@gmail.com
Site Name
Evangelismos S.A.
Department Name
1st Department of Critical Care and Pulmonary Hypertension Clinic
Contact Person Name
Anastasia Anthi
Contact Person Email
anastasia.anthi1@gmail.com

Italy

Earliest CTIS Part Ii Submission Date
26-06-2025
Latest Decision Or Authorization Date
25-08-2025
Processing Time Days
60
Number Of Sites
8
Number Of Participants
16

Sites

Site Name
Azienda Ospedaliero Universitaria Di Sassari
Department Name
Cardiology
Contact Person Name
Gavino Casu
Contact Person Email
gavino.casu@aouss.it
Site Name
Azienda Sanitaria Locale TO4
Department Name
Cardiology
Contact Person Name
Walter Grosso Marra
Contact Person Email
wrossomarra@aslto.iemonte.it
Site Name
Istituto Mediterraneo Per I Trapianti E Terapie Ad Alta Specializzazione S.r.l. I.S.M.E.T.T. S.r.l.
Department Name
Cardiology
Contact Person Name
Patrizio Vitulo
Contact Person Email
pvitulo@ismett.edu
Site Name
Azienda Ospedaliero-Universitaria Policlinico Umberto I
Department Name
Cardiology
Contact Person Name
Roberto Badagliacca
Site Name
Azienda Sanitaria Universitaria Giuliano Isontina
Department Name
Cardiology
Contact Person Name
Marco Confalonieri
Site Name
Fondazione IRCCS Policlinico San Matteo
Department Name
Cardiology
Contact Person Name
Stefano Ghio
Contact Person Email
s.ghio@smatteo.pv.it
Site Name
Azienda Ospedaliero Universitaria Ospedali Riuniti
Department Name
Cardiology
Contact Person Name
Michele Correale
Contact Person Email
Michele.correale@libero.it
Site Name
Fondazione IRCCS San Gerardo Dei Tintori
Department Name
Cardiology
Contact Person Name
Giuseppe Paciocco
Contact Person Email
giuseppe.paciocco@unimib.it

Latvia

Earliest CTIS Part Ii Submission Date
28-07-2025
Latest Decision Or Authorization Date
26-08-2025
Processing Time Days
29
Number Of Sites
1
Number Of Participants
7

Sites

Site Name
Pauls Stradins Clinical University Hospital
Department Name
Rare Disease Unit; Latvian Cardiology Center
Contact Person Name
Andris Skride
Contact Person Email
andris.skride@gmail.com

Portugal

Earliest CTIS Part Ii Submission Date
17-07-2025
Latest Decision Or Authorization Date
22-08-2025
Processing Time Days
36
Number Of Sites
2
Number Of Participants
2

Sites

Site Name
Unidade Local De Saude De Almada-Seixal E.P.E.
Department Name
Cardiology department
Contact Person Name
Sofia Alegria
Site Name
Unidade Local De Saude De Santa Maria E.P.E.
Department Name
Cardiology department
Contact Person Name
Rui Plácido
Contact Person Email
placidorui@gmail.com

Romania

Earliest CTIS Part Ii Submission Date
19-05-2025
Latest Decision Or Authorization Date
27-08-2025
Processing Time Days
100
Number Of Sites
2
Number Of Participants
2

Sites

Site Name
Clinical Hospital Of Infectious Diseases And Pneumophysiology Dr.Victor Babes Timisoara
Department Name
Sectia clinica Pneumologie II
Contact Person Name
Ovidiu Fira-Mladinescu
Contact Person Email
mladinescu@umft.ro
Site Name
Institutul De Pneumoftiziologie Marius Nasta
Department Name
Sectia Clinica Pneumologie Vl
Contact Person Name
Tudor Constantinescu
Contact Person Email
Tudor_constantinescu@yahoo.com

Spain

Earliest CTIS Part Ii Submission Date
28-07-2025
Latest Decision Or Authorization Date
26-08-2025
Processing Time Days
29
Number Of Sites
8
Number Of Participants
8

Sites

Site Name
Complexo Hospitalario Universitario De Santiago
Contact Person Name
Maria Alvarez Barredo
Site Name
Complejo Hospitalario Universitario Insular Materno Infantil
Contact Person Name
Francisco Javier Guerra Ramons
Site Name
University Hospital Virgen Del Rocio S.L.
Contact Person Name
Remedios Otero Candelera
Site Name
Hospital Costa Del Sol
Contact Person Name
Rafael Bravo Marques
Contact Person Email
rafabravomarques@gmail.com
Site Name
Hospital Del Mar
Contact Person Name
Diego Rodriguez Chiaradia
Contact Person Email
darodriguez@hmar.cat
Site Name
Hospital Universitari Vall D Hebron
Contact Person Name
Cristina Berastegui Garcia
Site Name
Hospital Universitario De Toledo
Contact Person Name
Maria Lazaro Salvador
Contact Person Email
maria.lasal@gmail.com
Site Name
University Hospital Virgen Del Rocio (duplicate entry previously listed?)
Contact Person Name
Remedios Otero Candelera

Poland

Earliest CTIS Part Ii Submission Date
29-07-2025
Latest Decision Or Authorization Date
01-09-2025
Processing Time Days
34
Number Of Sites
3
Number Of Participants
6

Sites

Site Name
Krakowski Szpital Specjalistyczny Im. Sw. Jana Pawla II
Department Name
Oddział Kliniczny Chorób Serca i Naczyń z Pododdziałem Intensywnego Nadzoru Kardiologicznego
Contact Person Name
Grzegorz Kopeć
Contact Person Email
grzegorzkrakow1@gmail.com
Site Name
Wojewodzki Szpital Specjalistyczny Im. Stefana Kardynala Wyszynskiego Samodzielny Publiczny Zaklad Opieki Zdrowotnej W Lublinie
Department Name
Oddział Kardiologii – Pododdział Intensywnego Nadzoru Kardiologicznego – Pododdział Reh. Kardiol.
Contact Person Name
Piotr Błaszczak
Contact Person Email
lubkard@szpital.lublin.pl
Site Name
Uniwersytecki Szpital Kliniczny W Poznaniu
Department Name
Uniwersytecki Ośrodek Wczesnych Faz
Contact Person Name
Tatiana Mularek-Kubzdela
Contact Person Email
tatianamularek@wp.pl

France

Earliest CTIS Part Ii Submission Date
19-05-2025
Latest Decision Or Authorization Date
26-11-2025
Processing Time Days
191
Number Of Sites
12
Number Of Participants
8

Sites

Site Name
Centre Hospitalier Regional Et Universitaire De Brest
Department Name
Hôpital de la Cavale Blanche Service de Pneumologie
Contact Person Name
Cécile TROMEUR
Contact Person Email
cecile.tromeur@chu-brest.fr
Site Name
Centre Hospitalier Universitaire Grenoble Alpes
Department Name
Service dePneumologie Physiologie - CHUGA
Contact Person Name
Bruno DEGANO
Contact Person Email
bdegano@chu-grenoble.fr
Site Name
Centre Hospitalier Universitaire De Saint Etienne
Department Name
Hôpital Nord Service de Médecine Vasculaire et Thérapeutique
Contact Person Name
Laurent BERTOLETTI
Site Name
CHRU De Nancy
Department Name
Département de Pneumologie
Contact Person Name
Ari CHAOUAT
Contact Person Email
ari.chaouat@chru-nancy.fr
Site Name
Centre Hospitalier Universitaire De Montpellier
Department Name
Hôpital Arnaud de Villene Service des Maladies Respiratoires
Contact Person Name
Arnaud BOURDIN
Contact Person Email
a-bourdin@chu-montpellier.fr
Site Name
Centre Hospitalier Universitaire De Toulouse
Department Name
Hôpital Larrey Service de Pneumologie
Contact Person Name
Grégoire PREVOT
Contact Person Email
prevot.g@chu-toulouse.fr
Site Name
Les Hopitaux Universitaires De Strasbourg
Department Name
Service de Pneumolologie
Contact Person Name
Marianne RIOU
Site Name
Assistance Publique Hopitaux De Paris
Department Name
Service de Pneumologie et Réanimation Respiratoire
Contact Person Name
Olivier SITBON
Contact Person Email
olivier.sitbon@aphp.fr
Site Name
Centre Hospitalier Universitaire De Poitiers
Department Name
Service de Pneumologie
Contact Person Name
Etienne-Marie JUTANT
Site Name
Centre Hospitalier Universitaire D'Angers
Department Name
Département de Pneumologie
Contact Person Name
Frédéric GAGNADOUX
Contact Person Email
rgagnadoux@chu-angers.fr
Site Name
Centre Hospitalier Universitaire De Bordeaux
Department Name
Hôpital Haut Leveque Service de Pneumologie
Contact Person Name
Arnaud MAURAC
Contact Person Email
arnaud.maurac@chu-bordeaux.fr
Site Name
Centre Hospitalier Universitaire De Nice
Department Name
Service de Cardiologie
Contact Person Name
Emile FERRARI
Contact Person Email
ferrari.e@chu-nice.fr

Sponsor

Primary sponsor

Full Name
Novartis Pharma AG
Organisation Type
Pharmaceutical company
Country Of Registered Address
Switzerland

Contract research organisations

Name
PPD Development LP / PPD International Holdings LLC / PPD Global Ltd. / PPD Global Limited
Responsibilities
Multiple operational roles including patient recruitment services, global clinical supplies and regulatory support (sponsorDuties codes present; contact: EUCTRInquiry.sm@ppd.com)
Name
Icon Clinical Research Limited
Responsibilities
Imaging (RHC) Central-reader and other clinical operations (sponsorDuties codes: 13, 15)
Name
Parexel International (IRL) Limited
Responsibilities
Clinical services (sponsorDuties code: 12)
Name
Medidata Solutions Inc.
Responsibilities
Data management/eCOA related services (sponsorDuties code: 7)
Name
4g Clinical LLC
Responsibilities
Operational support (sponsorDuties code: 3)

Third parties

  • {"country":"United Kingdom","full_name":"The Doctors Laboratory Limited","duties_or_roles":"sponsorDuties codes: [4]; contact email: pm@tdlpathology.com","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"United States","full_name":"PPD Development LP","duties_or_roles":"sponsorDuties codes: [4]; contact email: EUCTRInquiry.sm@ppd.com","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"sponsorDuties codes: [7]; contact email: info@medidata.com","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"sponsorDuties codes: [13, 15]; responsibilities include Imaging (RHC) Central-reader; contact email: colby.hawthorne@iconplc.com","organisation_type":"Pharmaceutical company"}
  • {"country":"India","full_name":"Veeda Clinical Research Limited","duties_or_roles":"sponsorDuties codes: [4]; contact email: info@veedacr.com","organisation_type":"Pharmaceutical company"}
  • {"country":"Belgium","full_name":"PPD International Holdings LLC","duties_or_roles":"sponsorDuties codes: [4]; contact email: EUCTRInquiry.sm@ppd.com","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Bioclinica Inc.","duties_or_roles":"sponsorDuties codes: [13, 15]; responsibilities include Imaging (Echo) Central Reader; contact email: customercare@clario.com","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"United States","full_name":"RWS Life Sciences Inc.","duties_or_roles":"sponsorDuties codes: [15]; responsibilities include PRO formatting and translations; contact email: LV.Sales@rws.com","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"sponsorDuties codes: [11, 15]; responsibilities include eCOA and Cardiac Safety (ECG); contact email: amy.weisguth@clario.com","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"4g Clinical LLC","duties_or_roles":"sponsorDuties codes: [3]; contact email: info@4gclinical.com","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"Ireland","full_name":"Parexel International (IRL) Limited","duties_or_roles":"sponsorDuties codes: [12]; contact email: Clinicaltrial.Enquiries@parexel.com","organisation_type":"Pharmaceutical company"}
  • {"country":"Greece","full_name":"PPD Global Ltd.","duties_or_roles":"sponsorDuties codes: [12,5]; contact email: approvalsgr.sm@thermofisher.com","organisation_type":"Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"PPD Global Limited","duties_or_roles":"sponsorDuties codes: [1,10,11,12,13,14,15,2,5,6,8,9]; includes Patient Recruitment Services, Global Clinical Supplies (ancillary items); contact email: EUCTRInquiry.sm@ppd.com","organisation_type":"Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"Synexus Clinical Research Acquisitions Limited","duties_or_roles":"sponsorDuties codes: [15]; responsibilities include Home Trial Services; contact email: hometrialservices.sm@ppd.com","organisation_type":"Pharmaceutical company"}

Investigational products

Investigational Product Name
LTP001
Active Substance
LTP001
Modality
Small molecule
Routes Of Administration
ORAL
Route
ORAL
Authorisation Status
Authorised (prodAuthStatus 1)
Investigational Product Name
Placebo to LTP001 00mg hard gelatin capsule
Modality
Other
Combination Treatment
Yes

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