Ablynx initiates Phase II clinical trial for thrombotic thrombocytopenic purpura

01-Oct-2010 - Belgium

Ablynx announced that it has opened recruitment in its first clinical trial centre for an open-label, randomised Phase II study for patients with acquired thrombotic thrombocytopenic purpura (TTP). This Phase II study will be conducted in Europe and North America and it is planned to enroll over 100 patients. Ablynx's anti-von Willebrand factor (vWF) Nanobody(®) was granted Orphan Drug Designation for the treatment of TTP in May 2009, by both the U.S. food and Drug Administration and the European Commission.

During the Phase II study, the Nanobody will be administered to TTP patients as an adjunct to standard plasma exchange therapy. The primary endpoint of the study will be the time to response, defined by the normalisation of platelet count and the blood marker lactate dehydrogenase (LDH). The study goal is to see a significant reduction in the time to response for the anti-vWF treated population compared with placebo treatment. It is anticipated that this could translate into a shorter duration of life-threatening episode, a reduction of the number of plasma exchanges required and the amount of plasma product administered.

Dr Edwin Moses, CEO and Chairman of Ablynx commented: "There is currently no drug treatment approved specifically for TTP. Patients are often hospitalised for up to two weeks and have to receive frequent plasma exchanges which are costly and can lead to serious side effects such as allergic reactions or infections. Despite the plasma exchange treatment, the condition is very serious with up to 20% mortality. We believe that our anti-vWF Nanobody may reduce the number of days a TTP patient is hospitalised for plasma exchange thereby reducing the overall number of plasma transfusions and increasing the quality of life for these patients." He added: "This is the third Nanobody programme entering Phase II clinical trials."

https://www.bionity.com/en/news/123698/ablynx-initiates-phase-ii-clinical-trial-for-thrombotic-thrombocytopenic-purpura.html