CLTP001A12201E1
Research type
Research Study
Full title
An open-label extension study to investigate efficacy, safety and tolerability of LTP001 in participants with pulmonary arterial hypertension
IRAS ID
1006633
Contact name
Angela Muehlenbroich
Contact email
Sponsor organisation
Novartis Pharma AG
Eudract number
2022-002007-38
Research summary
The purpose of this study is to find out the long-term safety and efficacy of LTP001 in patients with Pulmonary Arterial Hypertension (PAH) who took part in the CLTP001A12201 study. It offers them the opportunity to receive LTP001 (whether they received LTP001 or placebo) for up to a year.
Pulmonary arterial hypertension (PAH) affects the heart and lungs. It is characterised by abnormally high blood pressure (hypertension) in the pulmonary artery, the blood vessel that carries blood from the heart to the lungs. Symptoms include shortness of breath (dyspnoea) during exercise, and fainting spells. The symptoms tend to get worse over time and may also include dizziness, swelling (oedema) of the ankles or legs, chest pain, and a racing pulse.
Some cases of PAH are inherited, but most cases of PAH occur in individuals with no family history of the disorder. PAH can also occur secondary to underlying conditions such as connective tissue diseases, HIV infection, chronic haemolytic anaemia, and congenital heart disease, or be induced by certain drugs and toxins.
PAH affects 15-50 per million people worldwide and there is a need for disease-modifying therapy. Current treatment is focused on managing the symptoms with supportive care, and may include both surgery and medications. Lung transplantation is an option in the most severe cases.
The study will last approx. 57 weeks from baseline to final visit, and will run in 3 NHS sites in the UK; the global target is 40 patients with PAH, aged between 18 and 75 years. It will involve a 52 week extension treatment period with LTP001, and study assessments include blood samples, ECGs, echocardiograms, right heart catheterisation, 6-minute Walk Test, an optional wearable activity monitoring device, and symptom diary and questionnaires.REC name
South West - Central Bristol Research Ethics Committee
REC reference
23/SW/0036
Date of REC Opinion
22 Aug 2023
REC opinion
Further Information Favourable Opinion