A Study to Learn About the Treatment LTP001 in Healthy Participants (Part A) and in Participants With PAH (Part B)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Novartis Pharma AG

Participant type

Healthy volunteers, Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Respiratory Tract Diseases [C08]

Trial duration

18 Sep 2025 → ongoing

Decision date (initial)

2025-08-27

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Novartis Pharma AG

External identifiers

EU CT number

2024-513613-12-00

WHO UTN

U1111-1320-5027

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Others, Safety, Pharmacokinetic, Efficacy

Part A: To assess the safety and tolerability of single and multiple doses of LTP001 in healthy participants
Part B:
Period 1: To characterize the dose-response relationship of LTP001 plus standard-of-care (SoC) compared to placebo plus SoC in participants with WHO functional class (FC) II-III PAH
Period 2: To assess the safety and tolerability of LTP001

Secondary objectives 4

1. Part A: To assess the plasma pharmacokinetics (PK) of single and multiple doses of LTP001 in healthy participants Part B: Period 1: To evaluate the effect of LTP001 plus SoC on exercise capacity as measured by the 6-minute walk test Period 2: Exercise capacity as measured by the 6-minute walk test
2. Part B Period 2: Disease progression as measured by change in WHO FC
3. Part B Period 1: Disease progression as measured by change in WHO FC
4. Part B Period 1: Safety and tolerability

Conditions and MedDRA coding

Part A: Healthy volunteers (Part A is not applicable to the EU; for information only) Part B: pulmonary arterial hypertension

Regulatory references

Scientific advice from competent authorities

Food And Drug Administration

Plan to share IPD

Yes

IPD plan description

Novartis is committed to sharing with qualified external researchers, access to patient level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

1. Part A: Signed informed consent must be obtained prior to participation in the study. Part B: Confirmed diagnosis of pulmonary arterial hypertension
2. Part A: Healthy male and non-childbearing potential female participants Part B: Pre-randomization PVR ≥400 dyn.sec.cm
3. 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
4. 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 4

1. 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
2. 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
3. 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.
4. Part B: History of hypersensitivity to any of the study treatments or excipients

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. 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.

Secondary endpoints 4

1. 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
2. Part B Period 2: Number of participants experiencing an improvement in WHO FC from baseline to 18 months
3. Part B (Period 1): Change in WHO FC from baseline to 24 weeks
4. Part B (Period 1): All safety endpoints, including vital signs, ECG, clinical laboratory evaluations, treatment-emergent adverse events and discontinuations due to adverse events

Investigational products

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

Test 2

Placebo 2

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 15

Locations

11 EU/EEA countries · 50 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Oversight and notifications

Regulatory notifications under CTR Articles 38, 52, 53, 54 and 77

Corrective measures 1 · Art. 77 CTR

Results and documents

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

Documents 181 files

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

Application history

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