NCT05764265

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

An Open-label Extension Study to Investigate Efficacy, Safety and Tolerability of LTP001 in Participants With Pulmonary Arterial Hypertension

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 31 (actual)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

This was a non-randomized, open-label extension study of LTP001 for participants with PAH who completed the parent Study CLTP001A12201. Eligible participants were presented with the opportunity to enroll in the extension study at the end of treatment visit of the parent study. Participants in the extension study were planned to receive a once-daily dose of LTP001 for 52 weeks regardless of their parent study treatment (i.e. LTP001 or placebo). The study duration was planned up to 54 weeks with a treatment duration up to 52 weeks and maximum 2-week transition period from the CLTP001A12201 study. The visit frequency was planned to include visits at Weeks 1, 5, 13, 26, 39, 52, and 54 along with optional visits at the discretion of the Investigators at Weeks 9 and 17. Due to the study termination, no patient reached Week 52. After the termination announcement, following the instruction to immediately stop treatment for all participants, an end-of-treatment (EOT) visit was conducted. Sites were advised to complete protocol-required assessments based on investigator judgement and patient willingness to undergo procedures, with a primary focus on ensuring a safe exit from the study. Most sites performed only a few safety assessments, and only a minimal number of patients completed an echocardiogram.

Conditions

• Pulmonary Arterial Hypertension

Interventions

Type	Name	Description
DRUG	LTP001	LTP001, 6 mg, was administered orally once daily in the morning

Timeline

• Start date: 2023-03-27
• Primary completion: 2024-04-26
• Completion: 2024-05-14
• First posted: 2023-03-10
• Last updated: 2026-01-27
• Results posted: 2025-12-26

Locations

12 sites across 7 countries: United States, Argentina, Germany, Netherlands, Poland, Spain, United Kingdom

Regulatory

• FDA-regulated drug study