Dichloroacetate (DCA) for the Treatment of Pulmonary Arterial Hypertension
Status:
Completed
Trial end date:
2013-09-01
Target enrollment:
Participant gender:
Summary
Hypothesis: The small molecule and metabolic modulator Dichloroacetate (DCA) is safe,
tolerated as a potential therapy in patients with moderate or severe Pulmonary Arterial
Hypertension (PAH).
This is a Phase I, two centre study in subjects with PAH WHO functional class III-IV whose
symptoms have been clinically stable on their prescribed medical treatment (which includes
endothelin and/or phosphodiesterase type 5 inhibitors) for 8 weeks prior to enrollment. Such
patients will be given either DCA 3.0 mg/kg BID (group I), 6.25 mg/kg BID (group II) or 12.5
mg/kg BID (group III) as an additional treatment for 16 weeks. The design is open-label with
the subjects acting as their own controls.
Primary endpoint is the safety and tolerability of DCA. Secondary end points include: a)
functional capacity including a change in the 6 minute walk form baseline, b) change in
pulmonary vascular resistance (measured by right heart catheterization), c) right ventricular
volumes and mass (measured by MRI), d) NT-proBNP levels changed from baseline, e) change in
FDG-glucose uptake in the lung and right ventricle (measured by PET) and f) change in quality
of life indices.
15 evaluable patients in each site are expected to be included.