Potential pulmonary arterial hypertension (PAH) therapies currently in development

TherapyClinical trial identifierClinical trial designPrimary end-pointsTreatment durationStatus (October 2016)
Therapies targeting inflammation and immunity
 UbenimexNCT02664558Phase II, multicentre, randomised, double-blind, placebo-controlled trial in PAH patientsChange in PVR24 weeksRecruiting [17]
NCT02736149Phase II, open-label, multicentre, extension study in PAH patientsFrequency of adverse events∼1 yearNot yet recruiting [18]
 RituximabNCT01086540Phase II, randomised, double-blind, placebo-controlled trial in PAH-SSc patientsChange in PVR24 weeksRecruiting [19]
 TocilizumabNCT02676947Phase II, open-label trial in PAH patientsIncidence and severity of adverse events; change in PVR6 monthsRecruiting [20]
Therapies targeting mitochondrial dysfunction
 Bardoxolone methylNCT02036970Phase II, double-blind, randomised, interventional trial in pulmonary hypertension Group I, II or V patientsChange in 6MWD16 weeksPreliminary results published [21]
NCT02657356Phase III, double-blind, early interventional trial in PAH-CTD patientsChange in 6MWD24 weeksRecruiting [22]
 GS-4997NCT02234141Phase II, dose-ranging, randomised, double-blind, placebo-controlled trial in PAH patientsChange in PVR24 weeksOngoing, not recruiting [23]
Therapies targeting BMPR2 signalling
 TacrolimusNCT01647945Phase II, double-blind, randomised trial in PAH patientsFrequency of adverse events16 weeksTerminated due to limited funding/slow patient recruitment; follow-up multicentre phase IIb efficacy trial planned [24]
Therapies targeting iron deficiency
 Ferinject (ferric carboxymaltose)NCT01447628Phase II, double-blind, randomised, interventional trial in IPAH, HPAH and anorexigen-associated PAH patientsChange in PVR and exercise capacity24 weeksRecruiting [25]
NCT01847352Single-blind, nonrandomised, interventional, trial in healthy volunteers who met iron-deficient or iron-replete criteriaChange in PASP following i.v. iron infusion1 weekCompleted: April 2014 [26, 27]
 Ferrous sulfate (oral dietary  iron supplement)NCT01446848Interventional, open-label study in IPAH patients with iron deficiencyChange in zinc protoporphyrin level; change in serum ferritin level12 weeksCompleted: August 2014 [28]
Pulmonary artery denervation
 Pulmonary arterial denervation procedurechiCTR-ONC-12002085Phase II, observational, unblinded, nonrandomised study in PAH and PAH-CTD patientsChange in PASP and 6MWD24 weeksCompleted: April 2014 [29]
NCT02525926Single-blind, randomised, interventional efficacy study in PAH patientsMean pulmonary artery pressure26 weeksRecruiting [30]
  • PVR: pulmonary vascular resistance (as measured by right heart catheterisation); PAH-SSc: PAH associated with systemic sclerosis; 6MWD: 6-min walk distance; PAH-CTD: PAH associated with connective tissue disease; BMPR2: bone morphogenetic protein receptor type 2; IPAH: idiopathic PAH; HPAH: heritable PAH; PASP: pulmonary artery systolic pressure.