Pulmonary Arterial HypertensionPhase 2NCT05135000
Study of Efficacy and Safety of LTP001 in Pulmonary Arterial Hypertension
A Phase 2 study testing LTP001 in people with Pulmonary Arterial Hypertension, run by Novartis Pharmaceuticals at 15 sites in 7 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Pulmonary Arterial Hypertension
- What is being tested?
- LTP001
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 47 participants
- Where
- 15 sitesin 7 countries
- Study dates
- 30 Jun 2022to 25 Apr 2024
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Show all 14Show fewer
- 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
- 6MWD 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
Who cannot join
- Participants with pulmonary hypertension (PH) in the Clinical Classification Groups 2-5 (WHO), and any PAH Group 1 subgroups not covered by Inclusion Criterion #4.
- 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).
Trial locations and contacts
15 sites in 7 countries.
15 sites in 7 countries
Germany3 sites
Novartis Investigative Site
Berlin, 13353
Novartis Investigative Site
Dresden, 01307
Novartis Investigative Site
Heidelberg, 69120
Poland3 sites
Novartis Investigative Site
Krakow, 31 202
Novartis Investigative Site
Lodz, 91-347
Novartis Investigative Site
Wroclaw, 50-556
Spain3 sites
Novartis Investigative Site
Barcelona, Catalonia, 08036
Novartis Investigative Site
Madrid, 28041
Novartis Investigative Site
Málaga, Andalusia, 29010
United States2 sites
Medical Univ of South Carolina
Charleston, South Carolina, 29425
Pulmonary Associates PA
Mesa, Arizona, 85206
United Kingdom2 sites
Novartis Investigative Site
London, SW3 6PH
Novartis Investigative Site
Sheffield, South Yorkshire, S10 2JF
About this study
Official title: A Randomized, Participant- and Investigator-blinded, Placebo-controlled Study to Investigate Efficacy, Safety, and Tolerability of LTP001 in Participants With Pulmonary Arterial Hypertension
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.
Read the detailed description
This study was a non-confirmatory, randomized, participant- and investigator-blinded, placebo controlled trial evaluating the efficacy and safety of LTP001 on top of standard of care in participants with PAH.
The study included a screening period of up to 8 weeks, followed by a 24-week treatment phase with daily dosing and visits scheduled approximately every 4 weeks. One follow-up visit, which also served as the end-of-study visit, was conducted approximately 30 days after the conclusion of the treatment phase. The total duration of the study, from the beginning of the screening period to the end-of-study visit, was approximately 37 weeks.
A total of 44 participants were planned to be randomized in a 3:1 ratio to receive either LTP001 6 mg or placebo.
- Conditions
- Pulmonary Arterial Hypertension
- Being tested
- LTP001, Placebo
- Keywords
- Pulmonary Hypertension
- Medical subject headings
- Pulmonary Arterial Hypertension, Hypertension, Pulmonary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LTP001Drug
LTP001, 6 mg, was administered orally once daily in the morning
PlaceboDrug
Placebo to LTP001 was administered once daily in the morning
| Study group | Role | What they receive |
|---|---|---|
| LTP001 | Experimental | Participants received LTP001, 6 mg, oral capsules, once daily in the morning for approximately 24 weeks Receives: Drug: LTP001 |
| Placebo | Placebo comparator | Participants received LTP001 placebo capsules matching LTP001 orally once daily in the morning for approximately 24 weeks Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Baseline, Week 25
Secondary outcomes (15)
Change From Baseline in Six Minute Walk Distance (6MWD)
6MWD test measures the distance that a patient can walk on a flat, hard surface in a period of 6 minutes
Measured: Baseline, Weeks 13 and 25
Change From Baseline in Right Atrium (RA) Pressures at Week 25
The Right Heart Catheterization (RHC) assessment was performed to assess several hemodynamic variables in pulmonary hypertension, including RA pressures.
Measured: Baseline, Week 25
Change From Baseline in Pulmonary Capillary Wedge Pressure at Week 25
Right heart catheterization (RHC) assessment was performed to assess several hemodynamic variables in pulmonary hypertension, including pulmonary capillary wedge pressure (PCWP).
Measured: Baseline, Week 25
Change From Baseline in Mean Pulmonary Artery Pressure at Week 25
Right heart catheterization (RHC) assessment was performed to assess several hemodynamic variables in pulmonary hypertension, including pulmonary artery pressure.
Measured: Baseline, Week 25
Change From Baseline in Average Cardiac Output (CO) at Week 25
Right heart catheterization (RHC) assessment was performed to assess several hemodynamic variables in pulmonary hypertension, including cardiac output (CO).
Measured: Baseline, Week 25
Change From Baseline in Fractional Area Change (FAC)
Key right ventricular (RV) function endpoints such as RV fractional area change (RV FAC) were assessed with echocardiography.
Measured: Baseline, Weeks 5, 13, and 25
Change From Baseline in Peak Velocity of Excursion (RV S')
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.
Measured: Baseline, Weeks 5, 13, and 25
Change From Baseline in Tricuspid Annular Plane Systolic Excursion (TAPSE)
Key right ventricular (RV) function endpoints such as tricuspid annular plane systolic excursion (TAPSE) were assessed with echocardiography.
Measured: Baseline, Weeks 5, 13, and 25
Change From Baseline in Tricuspid Annular Systolic Velocity (TASV)
Key right ventricular (RV) function endpoints such as tricuspid annular systolic velocity (TASV) were assessed with echocardiography.
Measured: Baseline, Weeks 5, 13 and 25
Change From Baseline in EmPHasis-10
emPHasis-10 is a questionnaire with 10 questions designed to determine how pulmonary hypertension affects a participant's life. Each item is scored on a scale of 0 to 5, with a total score ranging from 0 to 50. A higher score indicates worse quality of life.
Measured: Baseline, Weeks 13 and 25
Change From Baseline in Pulmonary Arterial Hypertension-Symptoms and Impact (PAH-SYMPACT)
PAH-SYMPACT is a questionnaire used to assess pulmonary arterial hypertension symptoms and their impact. Individual item scores range from 0 to 4. Total score is calculated as the sum of the scores for the individual items divided by the number of items. A higher score indicates more severe symptoms/impacts.
Measured: Baseline, Weeks 13 and 25
Maximum Observed Blood Concentrations (Cmax) for LTP001
The maximum (peak) observed blood drug concentration after single dose administration.
Measured: Day 1 and Week 25 at 15, 45, and 120 minutes post-dose
Time to Reach Maximum Blood Concentrations (Tmax) of LTP001
The time to reach maximum (peak) blood drug concentration after single dose administration.
Measured: Day 1 and Week 25 at 15, 45, and 120 minutes post-dose
Time to Clinical Worsening
Time to any of the following:
- Death
- Hospital stay greater than 24 hours due to worsening of pulmonary arterial hypertension
- Worsening of PAH resulting in need for lung transplantation or balloon atrial septostomy
- Initiation of parenteral prostanoid therapy, initiation of oxygen therapy, initiation of any other pulmonary arterial hypertension-specific therapies or need for increase of diuretics for more than 4 weeks due to worsening of pulmonary arterial hypertension
- Significant drop in six minute walk distance
Measured: Baseline up to approximately 30 weeks
Change From Baseline in N-terminal Fragment of the Prohormone B-type Natriuretic Peptide (NT-ProBNP)
NT-proBNP is a blood biomarker to assess right ventricular distress.
Measured: Baseline to Week 29
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Nov 2021
- Study start30 Jun 2022
- Primary completion20 Mar 2024
- Study completion25 Apr 2024
Why it stopped: Sponsor Decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Pulmonary Arterial HypertensionStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 47 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05135000
- Sponsor's protocol ID
- CLTP001A12201
- Status last verified
- Dec 2025
- First submitted
- 16 Nov 2021
- Last updated
- 13 Jan 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
Study at a glance
From the registry record, last updated by the sponsor on 13 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Participant- and Investigator-blinded, Placebo-controlled Study to Investigate Efficacy, Safety, and Tolerability of LTP001 in Participants With Pulmonary Arterial Hypertension
- ClinicalTrials.gov ID
- NCT05135000
- Acronym / sponsor ID
- CLTP001A12201
- Conditions
- Pulmonary Arterial Hypertension
- Keywords
- Pulmonary Hypertension
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Sponsor Decision
- Enrolment
- 47 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 30 Jun 2022 (actual)
- Primary completion
- 20 Mar 2024 (actual)
- Study completion
- 25 Apr 2024 (actual)
- First posted
- 26 Nov 2021 (actual)
- Results
- Posted 15 May 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
This study was a non-confirmatory, randomized, participant- and investigator-blinded, placebo controlled trial evaluating the efficacy and safety of LTP001 on top of standard of care in participants with PAH.
The study included a screening period of up to 8 weeks, followed by a 24-week treatment phase with daily dosing and visits scheduled approximately every 4 weeks. One follow-up visit, which also served as the end-of-study visit, was conducted approximately 30 days after the conclusion of the treatment phase. The total duration of the study, from the beginning of the screening period to the end-of-study visit, was approximately 37 weeks.
A total of 44 participants were planned to be randomized in a 3:1 ratio to receive either LTP001 6 mg or placebo.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| LTP001 | Experimental | Drug: LTP001Participants received LTP001, 6 mg, oral capsules, once daily in the morning for approximately 24 weeks |
| Placebo | Placebo comparator | Drug: PlaceboParticipants received LTP001 placebo capsules matching LTP001 orally once daily in the morning for approximately 24 weeks |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (14 listed)
- History of PAH belonging to one of the following subgroups of the Clinical Classification Group 1 (WHO):
- 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
- 6MWD 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.
Key exclusion criteria (4 listed)
- Participants with pulmonary hypertension (PH) in the Clinical Classification Groups 2-5 (WHO), and any PAH Group 1 subgroups not covered by Inclusion Criterion #4.
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| LTP001LTP001, 6 mg, was administered orally once daily in the morning | Drug | LTP001 |
| PlaceboPlacebo to LTP001 was administered once daily in the morning | Drug | Placebo |
Primary outcome
Change From Baseline in Right Heard Catheterization Pulmonary Vascular Resistance (PVR) at Week 25
Time frame: Baseline, 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
15 secondary outcomes
Change From Baseline in Six Minute Walk Distance (6MWD)
Time frame: Baseline, Weeks 13 and 25
Change From Baseline in Right Atrium (RA) Pressures at Week 25
Time frame: Baseline, Week 25
Change From Baseline in Pulmonary Capillary Wedge Pressure at Week 25
Time frame: Baseline, Week 25
Change From Baseline in Mean Pulmonary Artery Pressure at Week 25
Time frame: Baseline, Week 25
Change From Baseline in Average Cardiac Output (CO) at Week 25
Time frame: Baseline, Week 25
Change From Baseline in Fractional Area Change (FAC)
Time frame: Baseline, Weeks 5, 13, and 25
Change From Baseline in Peak Velocity of Excursion (RV S')
Time frame: Baseline, Weeks 5, 13, and 25
Change From Baseline in Tricuspid Annular Plane Systolic Excursion (TAPSE)
Time frame: Baseline, Weeks 5, 13, and 25
Change From Baseline in Tricuspid Annular Systolic Velocity (TASV)
Time frame: Baseline, Weeks 5, 13 and 25
Change From Baseline in EmPHasis-10
Time frame: Baseline, Weeks 13 and 25
Change From Baseline in Pulmonary Arterial Hypertension-Symptoms and Impact (PAH-SYMPACT)
Time frame: Baseline, Weeks 13 and 25
Maximum Observed Blood Concentrations (Cmax) for LTP001
Time frame: Day 1 and Week 25 at 15, 45, and 120 minutes post-dose
Time to Reach Maximum Blood Concentrations (Tmax) of LTP001
Time frame: Day 1 and Week 25 at 15, 45, and 120 minutes post-dose
Time to Clinical Worsening
Time frame: Baseline up to approximately 30 weeks
Change From Baseline in N-terminal Fragment of the Prohormone B-type Natriuretic Peptide (NT-ProBNP)
Time frame: Baseline to Week 29
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Jan 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- A Plain Language Trial Summary is available on www.novctrd.comLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. 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. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
15 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing LTP001 in people with Pulmonary Arterial Hypertension, run by Novartis Pharmaceuticals at 15 sites in 7 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 14 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 13 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in Germany, Poland, Spain, United States, United Kingdom and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 15 May 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change From Baseline in Right Heard Catheterization Pulmonary Vascular Resistance (PVR) at Week 25 (time frame: Baseline, Week 25). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Jan 2026.