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Clinical Trials

Date: 2017-12-21

Type of information: Results

phase: 1

Announcement: results

Company: Ablynx (Belgium)

Product: caplacizumab

Action mechanism:

  • nanobody. Caplacizumab is a bivalent anti-von Willebrand Factor (vWF) Nanobody. vWF is implicated in thrombotic thrombocytopenic purpura (TTP), a rare disease where pre-cursors of vWF (ultra-large vWF multimers; UL-vWF) are present in the blood of patients and lead to blood clot formation and potentially life-threatening pathology. Caplacizumab inhibits platelet binding to UL-vWF and thus has the potential to prevent the formation of these string-like clots in the blood of patients with acquired TTP.
  • Caplacizumab received orphan drug designation in the US and EU in 2009 and could be the first drug specifically approved for the treatment of acquired TTP as an adjunct to plasma exchange.

Disease: acquired thrombotic thrombocytopenic purpura (TTP)

Therapeutic area: Autoimmune diseases - Rare diseases

Country: USA

Trial details:

  • The goal of this Phase I study is to assess the safety, tolerability, PK and PD profiles and immunogenicity of caplacizumab in healthy Japanese subjects, before initiating studies in Japanese patients with aTTP. The study consists of single ascending dose and multiple dose parts with blinded safety reviews prior to proceeding to a higher dose or multiple dosing. In total, 60 healthy Japanese and Caucasian subjects will be enrolled into the trial. (NCT03172208)
 

Latest news:

  • • On December 21, 2017, Ablynx announced that the single and multiple dose Phase I study demonstrated comparable PK of caplacizumab in Japanese and Caucasian subjects. The Phase I single centre study enrolled 60 healthy Japanese and Caucasian subjects and consisted of single ascending dose and multiple dose parts. At all doses studied, the PK of caplacizumab in the Japanese population were similar to those observed in Caucasians. Caplacizumab was well-tolerated in all groups and its safety profile was consistent with its mechanism of action. Dr Robert K. Zeldin, Chief Medical Officer at Ablynx, commented: “We are very pleased with these results as they enable us to bridge available clinical data for caplacizumab between Japanese and Caucasian populations. This is a very important step in making caplacizumab available to Japanese patients suffering from aTTP. We look forward to discussing these results, together with the recent Phase III HERCULES study results of caplacizumab, with the Japanese Regulatory Agency and aligning on a path forward for regulatory submission in Japan.”
  • • On June 26, 2017, Ablynx announced that the first Japanese healthy volunteers have been dosed in the Phase I single centre, randomised, double-blind, placebo-controlled study of caplacizumab, its first-in-class anti-von Willebrand factor (vWF) Nanobody® being developed for the treatment of acquired thrombotic thrombocytopenic purpura (aTTP). The goal of this Phase I study is to assess the safety, tolerability, PK and PD profiles and immunogenicity of caplacizumab in healthy Japanese subjects, before initiating studies in Japanese patients with aTTP. The study consists of single ascending dose and multiple dose parts with blinded safety reviews prior to proceeding to a higher dose or multiple dosing . In total, 60 healthy Japanese and Caucasian subjects will be enrolled into the trial. The company is looking forward to reporting the results of this study before the end of 2017. Abalynx will also report the top line results from its Phase III HERCULES study in 145 patients with acquired TTP. This study is evaluating the efficacy and safety of caplacizumab in patients with aTTP when administered in addition to the standard-of-care. These results are expected to support a BLA filing in the United States in 2018.

Is general: Yes