Study of Efficacy and Safety of LTP001 in Pulmonary Arterial Hypertension

NCT05135000 | Terminated Phase 2 Results DMC FDA Drug

• 1 other identifier: CLTP001A12201
• Study Type: interventional
• Target: 47
• Locations: 7 countries
• Sites: 15

Brief Summary

The purpose of this study was to explore the efficacy and safety of LTP001 in participants with pulmonary arterial hypertension (PAH) to determine if LTP001 had an adequate clinical profile to warrant further clinical development in this indication.

Conditions

Pulmonary Arterial Hypertension

Keywords

Pulmonary Hypertension

Outcome Measures

Primary Outcomes (1)

• Change From Baseline in Right Heard Catheterization Pulmonary Vascular Resistance (PVR) at Week 25

  PVR was defined as the resistance against blood flow from the pulmonary artery to the left atrium measured in dyn.s.cm-5

  Baseline, Week 25

Secondary Outcomes (15)

• Change From Baseline in Six Minute Walk Distance (6MWD)

  Baseline, Weeks 13 and 25

• Change From Baseline in Right Atrium (RA) Pressures at Week 25

  Baseline, Week 25

• Change From Baseline in Pulmonary Capillary Wedge Pressure at Week 25

  Baseline, Week 25

• Change From Baseline in Mean Pulmonary Artery Pressure at Week 25

  Baseline, Week 25

• Change From Baseline in Average Cardiac Output (CO) at Week 25

  Baseline, Week 25

Study Arms (2)

LTP001

EXPERIMENTAL

Participants received LTP001, 6 mg, oral capsules, once daily in the morning for approximately 24 weeks

Drug: LTP001

Placebo

PLACEBO COMPARATOR

Participants received LTP001 placebo capsules matching LTP001 orally once daily in the morning for approximately 24 weeks

Drug: Placebo

Interventions

LTP001 DRUG

LTP001, 6 mg, was administered orally once daily in the morning

LTP001

Placebo DRUG

Placebo to LTP001 was administered once daily in the morning

Placebo

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• History of PAH belonging to one of the following subgroups of the Clinical Classification Group 1 (WHO):
• participants with idiopathic pulmonary arterial hypertension (IPAH)
• Hereditary pulmonary arterial hypertension
• Congenital heart disease (surgically repaired at least 12 months prior to screening)
• drug or toxin induced (for example, anorexigen, or methamphetamine use).
• Resting mean pulmonary arterial pressure (mPAP) \> 25 mmHg; pulmonary capillary wedge pressure (PCWP) or left ventricular end diastolic pressure \< 15 mmHg, as determined by right heart catheterization within 20 days of randomization.
• Pulmonary Vascular Resistance \> 6 Wood units (480 dynes s/cm-5), as determined by right heart catheterization within 20 days of randomization.
• WHO Functional Class II-III
• MWD must be between 150 and 550 m (inclusive). The qualifying test needs to be within 20 days of randomization. To meet the above criterion additional six minute walk test (6MWT) may be performed up to a maximum of 3 tests in total prior to dosing; the minimal time difference between two tests should be at least 4 h.
• Standard of care therapy which is stable at least 6 weeks prior to RHC and qualifying 6MWT assessment within 20 days of randomization. Standard of care includes one or more of the following treatments:
• prostacyclin analogues and receptor agonists (if I.V., dose adjustments must be within 20% of initial stable dose)
• endothelin receptor antagonists (ERAs)
• phosphodiesterase type 5 inhibitors (PDE5i)
• soluble guanylate cyclase (sGC) stimulators

You may not qualify if:

• Participants with a history of left sided heart disease, chronic left sided heart failure, congenital or acquired valvular disease compromising left ventricular function and/or pulmonary venous hypertension or symptomatic coronary disease (non-symptomatic, revascularized coronary artery disease would be acceptable).
• Participants with obstructive lung disease defined as: FEV1/FVC \< 60% and FEV1 \< 60% of predicted value after bronchodilator administration as well as participants with moderate or severe restrictive lung disease: Total Lung Capacity \< 70% of predicted value. Testing must have occurred within 24months of screening. If historical testing is not available, then lung function testing must be conducted during the screening period.
• Acute or chronic impairment (other than dyspnea), which would limit the ability to comply with study requirements, including interference with physical activity and execution of study procedures such as 6MWT (e.g., angina pectoris, claudication, musculoskeletal disorder, multiple sclerosis, need for walking aids).

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (15)

Pulmonary Associates PA

Mesa, Arizona, 85206, United States

Location

Medical Univ of South Carolina

Charleston, South Carolina, 29425, United States

Location

Novartis Investigative Site

Caba, Buenos Aires, C1025ABI, Argentina

Location

Novartis Investigative Site

Berlin, 13353, Germany

Location

Novartis Investigative Site

Dresden, 01307, Germany

Location

Novartis Investigative Site

Heidelberg, 69120, Germany

Location

Novartis Investigative Site

Amsterdam, 1081 HV, Netherlands

Location

Novartis Investigative Site

Krakow, 31 202, Poland

Location

Novartis Investigative Site

Lodz, 91-347, Poland

Location

Novartis Investigative Site

Wroclaw, 50-556, Poland

Location

Novartis Investigative Site

Málaga, Andalusia, 29010, Spain

Location

Novartis Investigative Site

Barcelona, Catalonia, 08036, Spain

Location

Novartis Investigative Site

Madrid, 28041, Spain

Location

Novartis Investigative Site

Sheffield, South Yorkshire, S10 2JF, United Kingdom

Location

Novartis Investigative Site

London, SW3 6PH, United Kingdom

Location

Related Links

• A Plain Language Trial Summary is available on www.novctrd.com

MeSH Terms

Conditions

Pulmonary Arterial Hypertension; Hypertension, Pulmonary

Condition Hierarchy (Ancestors)

Lung Diseases; Respiratory Tract Diseases; Hypertension; Vascular Diseases; Cardiovascular Diseases

Results Point of Contact

• Title: Study Director
• Organization: Novartis Pharmaceuticals

Novartis.email@Novartis.com

+ 1 862 778 8300

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 16, 2021
• First Posted: November 26, 2021
• Study Start: June 30, 2022
• Primary Completion: March 20, 2024
• Study Completion: April 25, 2024
• Last Updated: January 13, 2026
• Results First Posted: May 15, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will share

Locations

PL (3); DE (3); AR (1); ES (3); GB (2); NL (1); US (2)