Study 301 is a multinational, randomized, placebo-controlled, induction-design Phase III trial evaluating up to 118 patients. The trial is approximately four weeks in duration and includes an initial open-label dose titration period of up to two weeks, after which patients undergo a seven-day washout period prior to randomization into a seven-day double-blind treatment period.
During the open label dose titration period, all patients will be titrated to maximum therapeutic benefit with up to 600mg tid of Droxidopa and must demonstrate both a blood pressure and symptomatic improvement. Patients not responding to treatment during the titration period will be excluded from the trial.
All patients demonstrating improvement will then undergo a seven day washout period prior to being equally randomized (N=59) to receive either their respective effective doses of active drug or placebo for one week. At the end of the one week blinded, treatment period, patients are evaluated for changes in symptomatic benefit relative to that recorded at randomization.
The primary endpoint will be the relative symptomatic change, as measured by the mean score of Item 1 (dizziness or light-headedness) of the orthostatic hypotension symptom assessment (OHSA), seven days following randomization either to continued therapy with Droxidopa or to placebo.
The OHSA scale is a validated scale designed to rate symptoms occurring specifically as a result of low blood pressure and uses an 11-point scale (zero to 10), with more severe symptoms scoring higher.
Simon Pedder, president and CEO of Chelsea, said: “We are confident in the design and strength of both of our Phase III trials and believe that together, the results of Studies 301 and 302 should provide a solid basis to support approval of Droxidopa in this indication.”