Repositioning imatinib for pulmonary arterial hypertension (REPIPAH)
Research type
Research Study
Full title
Repositioning oral imatinib for pulmonary arterial hypertension (REPIPAH).
IRAS ID
1010855
Contact name
Martin Wilkins
Contact email
Sponsor organisation
Imperial College London
ISRCTN Number
ISRCTN94603622
Research summary
Imatinib is a potential treatment for pulmonary arterial hypertension (PAH), a rare condition in which a narrowing of the vessels carrying blood through the lungs increases resistance to blood flow, putting an increased work load on the heart.
A Phase 3 clinical trial in PAH suggested that 400mg daily reduces the resistance and the pressure but is poorly tolerated. We have recently completed a dose-finding study in PAH and found that imatinib 200mg daily is better tolerated by patients and reduces pulmonary artery pressure and improves exercise capacity. Furthermore, the effects appear to persist for days after the drug has been cleared from the body, suggesting that imatinib may have disease-modifying properties and be suitable for intermittent weekly dosing.
In this new study, we will investigate in more detail the time course of the response to imatinib and, in an effort to improve tolerability, examine the effect of dosing less frequently than once daily. We will invite into the study suitable patients with PAH attending NHS national pulmonary hypertension referral centres . We will invite patients who will have been living with implanted devices that provide daily reports of pulmonary artery pressure and physical activity. The devices are as part of their clinical management. In this study, patients will continue with their prescribed licensed therapies and imatinib will be added to their background treatment for up to 24 weeks, starting with 200mg daily. In patients who respond, the dose of imatinib will be reduced to 200mg once weekly and they will be closely observed using remote monitoring. Thereafter, the frequency of dosing may be increased, as necessary, to a maximum of 200mg once daily, to maintain a beneficial response. The expectation is that we will identify the optimal dosing regimen for patients with PAH.
REC name
South Central - Berkshire B Research Ethics Committee
REC reference
24/SC/0345
Date of REC Opinion
20 Dec 2024
REC opinion
Further Information Favourable Opinion