SIMD 2019 Poster: Phase I clinical evaluation of CNSA-001 (sepiapterin), a novel pharmacological treatment for phenylketonuria and tetrahydrobiopterin deficiencies, in healthy volunteers

The results of a randomized, double-blind, placebo- and active treatment-controlled study are presented in this poster at the 2019 Society for Inherited Metabolic Disorders (SIMD) Annual Meeting in Seattle

  • The study design, pharmacokinetic data and safety and tolerability of CNSA-001, a preparation of sepiapterin which is being investigated for treatment of BH4-responsive phenylketonuria (PKU) and primary BH4 deficiencies, are reported in this poster
  • 83 healthy volunteers were randomized into cohorts to receive different doses of CNSA-001, active control (sapropterin dihydrochloride) or placebo: a single administration in Part A, with multiple doses given in Part B. Safety and tolerability results are also presented
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