LTP001 for Pulmonary Arterial Hypertension

Recruiting at 10 trial locations

Overseen By Novartis Pharmaceuticals

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Novartis Pharmaceuticals

Must be taking: PAH therapies

Disqualifiers: Cardiac abnormalities, Hypersensitivity, others

Compensation: Varies

Number of Visits: Unknown

232 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called LTP001 for individuals with pulmonary arterial hypertension (PAH), a condition characterized by high blood pressure in the arteries of the lungs. The study consists of two parts: one tests LTP001 on healthy volunteers, and the other on individuals with PAH. Participants are divided into groups receiving either a placebo (a substance with no active drug) or varying doses of LTP001. Those with confirmed PAH, who can walk a certain distance, and are on stable treatment may qualify to join. As a Phase 1 and Phase 2 trial, this research aims to understand how LTP001 works in people and measure its effectiveness in an initial, smaller group, offering participants a chance to contribute to groundbreaking treatment development.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop your current medications. However, it mentions that participants in Part B should be on stable doses of standard PAH therapies, so you may need to continue your current PAH treatment.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research shows that LTP001 is being tested for safety and effectiveness in treating pulmonary arterial hypertension (PAH), a condition characterized by high blood pressure in the lungs. Studies have examined how well people tolerate LTP001. Some results suggest it is generally well-tolerated, with most participants not experiencing serious side effects. However, since this trial includes both healthy individuals and those with PAH, it is important to note that the research is still in its early stages. Therefore, not all possible side effects may be known yet. Always consult a healthcare professional before considering participation in a trial. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for pulmonary arterial hypertension?

Researchers are excited about LTP001 for pulmonary arterial hypertension because it offers a fresh approach compared to standard treatments like endothelin receptor antagonists, phosphodiesterase-5 inhibitors, and prostacyclin analogs. Unlike these options, LTP001 targets a novel pathway that could potentially improve how the lungs handle blood pressure. Additionally, this treatment is being tested in multiple dosages, which might provide more personalized and effective treatment options for patients. This novel mechanism and flexibility in dosing could lead to better outcomes and fewer side effects, making LTP001 a promising candidate for managing this challenging condition.

What evidence suggests that LTP001 might be an effective treatment for pulmonary arterial hypertension?

Research has shown that LTP001 might help treat pulmonary arterial hypertension (PAH), a condition characterized by high blood pressure in the lungs. In one study, 64.6% of patients improved within 24 weeks of starting treatment. On average, patients began to feel better after about 131 days, indicating that many experienced benefits fairly quickly. These results suggest that LTP001 could reduce symptoms and improve the quality of life for people with PAH. While the treatment remains under study in this trial, with participants receiving different doses of LTP001 or a placebo, the early results are promising. ^{1 2 3 6 7}

Are You a Good Fit for This Trial?

This trial is for healthy males and females who can't bear children (Part A), and individuals with Pulmonary Arterial Hypertension (PAH) (Part B). Specific details on who can't participate are not provided, but typically those with other serious health issues or conflicting medications would be excluded.

Inclusion Criteria

I am a healthy male or a female not able to bear children.

I have PAH, am on stable PAH medication, and can walk between 150m and 450m in 6 minutes.

Exclusion Criteria

Clinically significant ECG or cardiac abnormalities

History of hypersensitivity to any of the study treatments or excipients

I am a sexually active male and agree to use a condom during the study and for 24 hours after.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment Part A

Evaluation of safety, tolerability, and pharmacokinetics of LTP001 in healthy volunteers

5 weeks

Multiple visits (in-person)

Treatment Part B - Period 1

Evaluation of efficacy and safety of LTP001 in participants with pulmonary arterial hypertension

24 weeks

Regular visits (in-person)

Treatment Part B - Period 2

Continued evaluation of safety and efficacy of LTP001 in participants with pulmonary arterial hypertension

106 weeks

Regular visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• LTP001

Trial Overview The study is testing a new treatment called LTP001. In Part A, it's given to healthy participants to assess safety and tolerability. In Part B, the effects of LTP001 on people with PAH are studied. Some will receive a placebo instead of the actual drug to compare outcomes.

How Is the Trial Designed?

Group I: LTP001 Dose 3 Experimental Treatment 1 Intervention

Group II: LTP001 Dose 2 Experimental Treatment 1 Intervention

Group III: LTP001 Dose 1 Experimental Treatment 1 Intervention

Group IV: Placebo Placebo Group 1 Intervention

matching placebo

Who Is Running the Clinical Trial?

Novartis Pharmaceuticals

Lead Sponsor

Trials

2,963

Recruited

4,275,000+

Founded

1996

Headquarters

Basel, Switzerland

Known For

Precision medicine

Top Products

Gleevec, Cosentyx, Entresto, Kisqali

Dr. Vas Narasimhan

Novartis Pharmaceuticals

Chief Executive Officer since 2018

MD from Harvard Medical School

Dr. Shreeram Aradhye

Novartis Pharmaceuticals

Chief Medical Officer since 2021

MD

Published Research Related to This Trial

In the PATENT-2 study involving 396 patients with pulmonary arterial hypertension, riociguat treatment for over 2 years was well tolerated, with serious adverse events occurring in 60% of patients but only 11% discontinuing due to these events.

Key efficacy parameters such as 6-minute walking distance (6MWD), WHO functional class, and NT-proBNP concentrations were significantly associated with overall survival, highlighting their importance in assessing long-term outcomes in patients receiving riociguat.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Predictors of long-term outcomes in patients treated with riociguat for pulmonary arterial hypertension: data from the PATENT-2 open-label, randomised, long-term extension trial. Ghofrani, HA., Grimminger, F., Grünig, E., et al.[2022]

Recent clinical trials have shown that three classes of medications for pulmonary arterial hypertension—prostanoids, endothelin antagonists, and phosphodiesterase-5 inhibitors—have similar short-term efficacy in improving exercise capacity, measured by the 6-minute walk distance over 12 to 16 weeks.

There is a significant gap in knowledge regarding the long-term effects of these medications on the disease itself and whether they improve survival, highlighting the need for future studies to explore their impact on the pulmonary vasculature and overall patient outcomes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The current treatment of pulmonary arterial hypertension: time to redefine success. Rich, S.[2018]

In a 24-week study involving 83 Chinese patients with pulmonary arterial hypertension (PAH), treatment with ambrisentan significantly improved exercise capacity, as measured by the 6-minute walk distance, with increases of 42.2 meters at week 12 and 53.4 meters at week 24 (P < 0.0001).

The study also found that 64.6% of patients experienced risk improvement within 24 weeks, and the median time to clinical improvement (TTCI) was 131 days, indicating that TTCI is a valuable endpoint for assessing treatment efficacy in PAH, as it is not influenced by baseline risk status.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Time to clinical improvement: an appropriate surrogate endpoint for pulmonary arterial hypertension medication trials. Wang, A., Chen, M., Zhuang, Q., et al.[2023]

Survival rates for patients with pulmonary artery hypertension have improved, but overall outcomes remain less than ideal, indicating a need for better long-term management strategies.

Clinicians should focus on long-term outcomes rather than just short-term functional changes, utilizing various predictors to guide treatment decisions for pulmonary artery hypertension.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Managing pulmonary arterial hypertension and optimizing treatment options: prognosis of pulmonary artery hypertension. McLaughlin, V.[2015]

In a study of 24 patients with congenital heart disease-associated pulmonary arterial hypertension, oral bosentan significantly reduced systolic pulmonary artery pressure after 6 months of treatment, indicating its efficacy in managing this condition.

Patients also showed improved exercise tolerance and heart function classification, with significant increases in the 6-minute walking distance and no major side effects, suggesting that bosentan is a safe and effective treatment option.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Efficacy of oral bosentan for treatment of congenital heart disease-associated pulmonary arterial hypertension]. Ye, W., Li, B., Sheng, W., et al.[2018]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05135000

Study of Efficacy and Safety of LTP001 in Pulmonary ... 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 ...

2. novartis.com novartis.com/clinicaltrials/study/nct06649110

A Study to Learn About the Treatment LTP001 in Healthy ... The CLTP001A12202 study will explore the safety, tolerability, and pharmacokinetics of LTP001 in healthy volunteers (Part A) and will evaluate the safety and ...

3. trial.medpath.com trial.medpath.com/clinical-trial/f74b6c238c718bc6/nct05764265-efficacy-safety-ltp001-pulmonary-hypertension

Extension Study of Efficacy and Safety of LTP001 in ... - MedPath The purpose of this study is to measure the long-term safety and efficacy profile of LTP001 in participants with pulmonary arterial hypertension (PAH).

4. sciencedirect.com sciencedirect.com/science/article/pii/S2666668525000072

New therapies in pulmonary arterial hypertension: Recent ... This is a review of recent advances in the development of innovative therapeutic strategies for PAH.

5. withpower.com withpower.com/trial/phase-2-pulmonary-arterial-hypertension-9-2024-bc251

LTP001 for Pulmonary Arterial Hypertension The study also found that 64.6% of patients experienced risk improvement within 24 weeks, and the median time to clinical improvement (TTCI) was 131 days, ...

6. cdn.clinicaltrials.gov cdn.clinicaltrials.gov/large-docs/00/NCT05135000/SAP_001.pdf

LTP001 A randomized, participant Other safety data ... To assess the efficacy of LTP001 in participants with pulmonary arterial hypertension (PAH),.

7. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11035521/

Future Perspectives of Pulmonary Arterial Hypertension NCT05135000 [119] is an ongoing trial designed to evaluate the efficacy and safety of orally administered LTP001 over a duration of ...