In short
Clinical trials are studying Ltp001 in people with idiopathic pulmonary fibrosis and pulmonary arterial hypertension, as well as in healthy volunteers in one part of a study. These trials are looking at safety, tolerability, and possible benefit, with outcomes such as lung function and pulmonary vascular resistance.
Key points
- Ltp001 is being studied in two clinical trials, one in idiopathic pulmonary fibrosis and one in pulmonary arterial hypertension. The IPF study is Phase 2 and looked at whether treatment could improve forced vital capacity, a measure of lung function, over 26 weeks. The PAH study is Phase 1 and includes both healthy participants in Part A and people with WHO functional class II-III PAH in Part B. That study focuses on safety, tolerability, and change in pulmonary vascular resistance. Together, these trials are testing both possible benefit and safety in different patient groups.
Trial overview
Two interventional studies are investigating Ltp001 in different groups of participants. One study is in people with idiopathic pulmonary fibrosis, or IPF, and the other study includes healthy volunteers in Part A and people with pulmonary arterial hypertension, or PAH, in Part B. The IPF study is Phase 2 and was completed, while the other study is Phase 1 and was authorised.
Idiopathic pulmonary fibrosis study
The IPF trial studied whether investigational products, including Ltp001 and placebo, could improve lung function in participants with idiopathic pulmonary fibrosis. The study was designed to compare treatment with placebo over 26 weeks of treatment. Its main goal was to assess efficacy, which means whether the treatment may work for the disease.
This study enrolled 109 participants and was completed. The primary endpoint was the change from baseline to the end of treatment in Forced Vital Capacity (FVC), expressed as percent predicted. Percent predicted means the result is compared with what would be expected for a healthy person of the same age, sex, height, and background.
Healthy volunteers and PAH study
The second study is a Phase 1 trial that looks at safety and tolerability of Ltp001. Part A includes healthy participants and is noted as not applicable to the EU, for information only. Part B includes participants with pulmonary arterial hypertension and is focused on both treatment effects and safety.
In Part A, the study assesses single and multiple doses of Ltp001 in healthy participants and measures safety endpoints such as vital signs, ECG, central lab evaluations, and adverse events up to the end-of-study visit. In Part B, Period 1 evaluates the dose-response relationship of Ltp001 plus standard-of-care compared with placebo plus standard-of-care in participants with WHO functional class II-III PAH. Period 2 then continues to assess safety and tolerability, including treatment-emergent adverse events and discontinuations due to adverse events.
This study has an enrollment of 121 participants and is authorised.
Outcomes and endpoints
The main outcome in the IPF study is the change in FVC from baseline after 26 weeks of treatment. This tells researchers whether lung function changed during the study.
In the PAH study, the main outcome in Period 1 is the change in PVR from baseline to 24 weeks. PVR is a measure of how hard it is for blood to flow through the blood vessels in the lungs. In Period 2, the study tracks safety endpoints such as vital signs, ECG, central lab tests, treatment-emergent adverse events, and stopping treatment because of adverse events.
Who could participate
The IPF study was for participants with idiopathic pulmonary fibrosis. The PAH study had two different groups: healthy volunteers in Part A and participants with WHO functional class II-III PAH in Part B. WHO functional class II-III means symptoms affect daily activity to a moderate degree.
- IPF participants: People with idiopathic pulmonary fibrosis who joined a Phase 2 treatment study.
- Healthy volunteers: People without the target disease who helped researchers study early safety in Part A.
- PAH participants: People with pulmonary arterial hypertension in WHO functional class II-III who took part in Part B.
What the trials measured
These trials focused on whether Ltp001 could help in the studied diseases and whether it could be given safely. In IPF, the key measure was lung function using FVC. In PAH, the key measure was PVR, along with safety checks such as vital signs, ECG, lab tests, and adverse events.
The studies used placebo comparison in both settings, which helps show whether changes are likely due to Ltp001 rather than chance or other care. The PAH study also used standard-of-care treatment, meaning the usual treatment given for the condition.