NCT05764265

Extension Study of Efficacy and Safety of LTP001 in Pulmonary Arterial Hypertension Participants

Terminated Phase 2 Results posted Last updated 27 January 2026

What this trial tests

Phase 2 trial testing LTP001 in Pulmonary Arterial Hypertension in 31 participants. Terminated before completion.

Timeline

27 March 2023

Primary endpoint
26 April 2024

14 May 2024

Quick facts

Lead sponsor	Novartis Pharmaceuticals
Phase	Phase 2
Status	Terminated
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	31
Start date	27 March 2023
Primary completion	26 April 2024
Estimated completion	14 May 2024
Sites	12 locations across Netherlands, United Kingdom, Germany, Poland, Argentina, United States, Spain

Drugs / interventions tested

• LTP001 — full drug profile →

Conditions studied

• Pulmonary Arterial Hypertension — all drugs for Pulmonary Arterial Hypertension →

Sponsor

Novartis Pharmaceuticals — full company profile →

Who can join

Adults 18 to 75, any sex, with Pulmonary Arterial Hypertension. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) Primary · Up to approximately 45 weeks

Incidence and severity of adverse events (AEs) by treatment group, including changes in the vital signs, electrocardiogram and laboratory results qualifying and reported as AEs. Due to the study termination, no patient reached Week 52. At the end of treatment visit, final safety assessments were performed.

AEs

Group	Value	95% CI
LTP001 6 mg (Actual Treatment in CLTP001A12201)	52.2
LTP001 6 mg (Placebo in CLTP001A12201)	62.5

Serious AEs

Group	Value	95% CI
LTP001 6 mg (Actual Treatment in CLTP001A12201)	17.4
LTP001 6 mg (Placebo in CLTP001A12201)	0

Change From Baseline in Average Cardiac Output (CO) at Week 26 Secondary · Baseline, Week 26

Right heart catheterization (RHC) assessment was performed to assess several hemodynamic variables in pulmonary hypertension, including CO.

Group	Value	95% CI
LTP001 6 mg (Actual Treatment in CLTP001A12201)	-0.111	± 0.2153
LTP001 6 mg (Placebo in CLTP001A12201)	-0.065	± 0.5916

Change From Baseline in Mean Pulmonary Artery (PA) Pressure at Week 26 Secondary · Baseline, Week 26

Right heart catheterization (RHC) assessment was performed to assess several hemodynamic variables in pulmonary hypertension, including PA pressure.

Group	Value	95% CI
LTP001 6 mg (Actual Treatment in CLTP001A12201)	3.8	± 8.18
LTP001 6 mg (Placebo in CLTP001A12201)	-1.5	± 7.78

Change From Baseline in Pulmonary Capillary Wedge Pressure (PCWP) at Week 26 Secondary · Baseline, Week 26

Right heart catheterization (RHC) assessment was performed to assess several hemodynamic variables in pulmonary hypertension, including pulmonary capillary wedge pressure (PCWP).

Group	Value	95% CI
LTP001 6 mg (Actual Treatment in CLTP001A12201)	-1.0	± 1.79
LTP001 6 mg (Placebo in CLTP001A12201)	-0.5	± 0.71

Change From Baseline in Right Heart Catheterization Pulmonary Vascular Resistance (PVR) at Week 26 Secondary · Baseline, Week 26

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

Group	Value	95% CI
LTP001 6 mg (Actual Treatment in CLTP001A12201)	100.058	± 95.5879
LTP001 6 mg (Placebo in CLTP001A12201)	-7.445	± 34.8250

Change From Baseline in Right Atrium (RA) Pressures at Week 26 Secondary · Baseline, Week 26

The Right Heart Catheterization (RHC) assessment was performed to assess several hemodynamic variables in pulmonary hypertension, including RA pressures.

Group	Value	95% CI
LTP001 6 mg (Actual Treatment in CLTP001A12201)	-1.5	± 6.22
LTP001 6 mg (Placebo in CLTP001A12201)	0.0	± 2.83

Change From Baseline in Systemic Vascular Resistance (SVR) at Week 26 Secondary · Baseline, Week 26

The Right Heart Catheterization (RHC) assessment was performed to assess several hemodynamic variables in pulmonary hypertension, including SVR.

Group	Value	95% CI
LTP001 6 mg (Actual Treatment in CLTP001A12201)	166.748	± 128.6202
LTP001 6 mg (Placebo in CLTP001A12201)	-49.710	± 28.5388

Change From Baseline in Six Minute Walk Distance (6MWD) Secondary · Baseline, Week 26, up to 39 weeks (EOT)

6MWD test measures the distance that a participant can walk on a flat, hard surface in a period of 6 minutes. Due to the study termination, no patient reached Week 52. At the end of treatment (EOT) visit, final safety assessments were performed based on investigator judgement and patient willingness to undergo procedures.

Week 26 n=6,2

Group	Value	95% CI
LTP001 6 mg (Actual Treatment in CLTP001A12201)	-33.7	± 55.60
LTP001 6 mg (Placebo in CLTP001A12201)	-9.0	± 25.46

EOT n=16,2

Group	Value	95% CI
LTP001 6 mg (Actual Treatment in CLTP001A12201)	-6.3	± 50.21
LTP001 6 mg (Placebo in CLTP001A12201)	-1.0	± 1.41

Change From Baseline in Tricuspid Annular Plane Systolic Excursion (TAPSE) Secondary · Baseline, Week 26, up to 39 weeks (EOT)

Key right ventricular (RV) function endpoints such as tricuspid annular plane systolic excursion (TAPSE) were assessed with echocardiography. Due to the study termination, no patient reached Week 52. At the end of treatment (EOT) visit, final safety assessments were performed based on investigator judgement and patient willingness to undergo procedures. Only a minimal number of patients completed an echocardiogram (Echo).

Week 26 n=6,2

Group	Value	95% CI
LTP001 6 mg (Actual Treatment in CLTP001A12201)	0.03	± 0.175
LTP001 6 mg (Placebo in CLTP001A12201)	0.00	± 0.283

EOT n=7,0

Group	Value	95% CI
LTP001 6 mg (Actual Treatment in CLTP001A12201)	0.01	± 0.682

Change From Baseline in Tricuspid Annular Plane Systolic Velocity (TASV) Secondary · Baseline, Week 26, up to 39 weeks (EOT)

Key right ventricular (RV) function endpoints such as tricuspid annular systolic velocity (TASV) were assessed with echocardiography. Due to the study termination, no patient reached Week 52. At the end of treatment (EOT) visit, final safety assessments were performed based on investigator judgement and patient willingness to undergo procedures. Only a minimal number of patients completed an echocardiogram (Echo).

Week 26 n=6,2

Group	Value	95% CI
LTP001 6 mg (Actual Treatment in CLTP001A12201)	-2.2	± 3.43
LTP001 6 mg (Placebo in CLTP001A12201)	-0.5	± 0.71

EOT n=7,0

Group	Value	95% CI
LTP001 6 mg (Actual Treatment in CLTP001A12201)	-2.6	± 1.90

Change From Baseline in Peak Velocity of Excursion (RV S') Secondary · Baseline, Week 26, up to 39 weeks (EOT)

Key right ventricular (RV) function per echocardiography. The terms Tricuspid Annular Systolic Velocity (TASV) and Peak Velocity of Excursion (RV S') are synonymous in echocardiography to describe the peak systolic velocity of the lateral tricuspid annulus. Including both TASV and RV S' as separate secondary endpoints was an oversight in the protocol as the data, calculation, and analyses for both (TASV and RV S') are identical. Therefore, the TASV and RV S' data in this results disclosure are the same. Due to the study termination, no patient reached Week 52. At the end of treatment (EOT) vis

Week 26 n=6,2

Group	Value	95% CI
LTP001 6 mg (Actual Treatment in CLTP001A12201)	-2.2	± 3.43
LTP001 6 mg (Placebo in CLTP001A12201)	-0.5	± 0.71

EOT n=7,0

Group	Value	95% CI
LTP001 6 mg (Actual Treatment in CLTP001A12201)	-2.6	± 1.90

Change From Baseline in Fractional Area Change (FAC) Secondary · Baseline, Week 26, up to 39 weeks (EOT)

Key right ventricular (RV) function endpoints such as RV fractional area change (RV FAC) were assessed with echocardiography. Due to the study termination, no patient reached Week 52. At the end of treatment (EOT) visit, final safety assessments were performed based on investigator judgement and patient willingness to undergo procedures. Only a minimal number of patients completed an echocardiogram (Echo).

Week 26 n=6,1

Group	Value	95% CI
LTP001 6 mg (Actual Treatment in CLTP001A12201)	-0.87	± 6.162
LTP001 6 mg (Placebo in CLTP001A12201)	7.30	± NA

EOT n=6,0

Group	Value	95% CI
LTP001 6 mg (Actual Treatment in CLTP001A12201)	0.97	± 5.290

Adverse events — posted to ClinicalTrials.gov

Time frame: Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

LTP001 6 mg (Actual Treatment in CLTP001A12201)

Serious: 4/23 (17%)

Deaths: 0/23

LTP001 6 mg (Placebo in CLTP001A12201)

Serious: 0/8 (0%)

Deaths: 0/8

Total

Serious: 4/31 (13%)

Deaths: 0/31

Serious adverse events (4 terms)

Reaction	System	LTP001 6 mg (Actual Treatm…	LTP001 6 mg (Placebo in CL…	Total
Pulmonary arterial hypertension	Respiratory, thoracic and mediastinal disorders	—	—	—
Medical device site haemorrhage	General disorders and administration site conditions	—	—	—
Device related infection	Infections and infestations	—	—	—
Disseminated gonococcal infection	Infections and infestations	—	—	—

Other adverse events (15 terms — click to expand)

Reaction	System	LTP001 6 mg (Actual Treatm…	LTP001 6 mg (Placebo in CL…	Total
Nasopharyngitis	Infections and infestations	—	—	—
Arthralgia	Musculoskeletal and connective tissue disorders	—	—	—
Headache	Nervous system disorders	—	—	—
Nasal congestion	Respiratory, thoracic and mediastinal disorders	—	—	—
Lymphadenopathy	Blood and lymphatic system disorders	—	—	—
Eczema eyelids	Eye disorders	—	—	—
Fatigue	General disorders and administration site conditions	—	—	—
Bartholinitis	Infections and infestations	—	—	—
Bronchitis	Infections and infestations	—	—	—
Tooth abscess	Infections and infestations	—	—	—
Musculoskeletal pain	Musculoskeletal and connective tissue disorders	—	—	—
Musculoskeletal stiffness	Musculoskeletal and connective tissue disorders	—	—	—
Dizziness	Nervous system disorders	—	—	—
Insomnia	Psychiatric disorders	—	—	—
Epistaxis	Respiratory, thoracic and mediastinal disorders	—	—	—

Most-reported serious reactions: Pulmonary arterial hypertension, Medical device site haemorrhage, Device related infection, Disseminated gonococcal infection.

Data from ClinicalTrials.gov NCT05764265 adverse events section.

Sponsor's own description

The purpose of this study was to measure the long-term safety and efficacy profile of LTP001 in participants with pulmonary arterial hypertension (PAH). The study offered participants who had completed the CLTP001A12201 double-blind parent study in PAH an opportunity to receive LTP001 (whether they were on LTP001 or not). Unblinding of the treatment received in CLTP001A12201 was generally not needed but could occur on request by the investigator.

Publications & conference data

4 peer-reviewed publications reference this trial (live from Europe PMC):

1. Mitochondrial network dynamics in pulmonary disease: Bridging the gap between inflammation, oxidative stress, and bioenergetics.
   Pokharel MD, Garcia-Flores A, Marciano D, Franco MC, et al · · 2024 · cited 73× · PMID 38295575 · DOI 10.1016/j.redox.2024.103049
2. Medical Management of Pulmonary Arterial Hypertension: Current Approaches and Investigational Drugs.
   Jin Q, Chen D, Zhang X, Zhang F, et al · · 2023 · cited 8× · PMID 37376028 · DOI 10.3390/pharmaceutics15061579
3. Emerging significance of E3 ubiquitin ligases and Deubiquitinases in pulmonary hypertension.
   Ding CW, Qiu JY, Shen H, Yan Y, et al · · 2026 · PMID 41957583 · DOI 10.1186/s10020-026-01478-5
4. [Heritable pulmonary arterial hypertension].
   Eichstaedt CA, Shaukat M, Grünig E. · · 2024 · PMID 38771375 · DOI 10.1007/s00108-024-01718-y