CLTP001A12201 – PAH PoC Study
Research type
Research Study
Full title
A randomized, participant- and investigator-blinded, placebo-controlled study to investigate efficacy, safety and tolerability of LTP001 in participants with pulmonary arterial hypertension
IRAS ID
1004298
Contact name
Angela Muehlenbroich
Contact email
Sponsor organisation
Novartis Pharma AG
Eudract number
2021-000670-28
Clinicaltrials.gov Identifier
Research summary
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.
This study will show whether LTP001 reduces pulmonary artery pressure and thus may control or reverse disease progression, and also improve patient symptoms. This information will help decide if LTP001 could be developed further as a treatment for this condition. No other drug with a similar action has been tested in clinical studies so far.
The study will last approx. 37 weeks from screening 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 24 week treatment period with LTP001 or placebo, and study assessments include blood and sputum samples, respiratory tests, ECG, echocardiogram, right heart catheterisation, 6-minute Walk Test, an optional wearable activity monitoring device, and symptom diary and questionnaires.
REC name
South Central - Berkshire Research Ethics Committee
REC reference
21/SC/0372
Date of REC Opinion
29 Dec 2021
REC opinion
Further Information Favourable Opinion