LTP001 for Pulmonary Arterial Hypertension

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

Trial Summary

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.

What data supports the effectiveness of the drug LTP001 for treating pulmonary arterial hypertension?

The research does not provide direct evidence about LTP001, but it mentions that similar drugs like riociguat and ambrisentan have shown effectiveness in improving outcomes for pulmonary arterial hypertension, suggesting that LTP001 might also be effective. ^{1 2 3 4 5}

What is the purpose of this trial?

A study to learn about the treatment LTP001 in healthy participants (Part A) and in participants with PAH (Part B)

Eligibility Criteria

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

I do not have any health conditions that could affect how my body handles medication.

I have a health condition that could increase my risk in a study.

Timeline

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

Treatment Details

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.

Participant Groups

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

Findings from Research

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]

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]

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]

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]

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]

References

1. England 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. [2022]

2. Switzerland pubmed.ncbi.nlm.nih.gov

Time to clinical improvement: an appropriate surrogate endpoint for pulmonary arterial hypertension medication trials. [2023]

3. China pubmed.ncbi.nlm.nih.gov

[Efficacy of oral bosentan for treatment of congenital heart disease-associated pulmonary arterial hypertension]. [2018]

4. United States pubmed.ncbi.nlm.nih.gov

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

5. United States pubmed.ncbi.nlm.nih.gov

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