(AURORA) a Randomized, Double-blind, Placebo-Controlled Study of Bitopertin to Evaluate the Safety, Tolerability, Efficacy, and Protoporphyrin IX (PPIX) Concentrations in Participants with Erythropoietic Protoporphyria (EPP)
This is a Phase 2, multi-center, double-blind, placebo-controlled, parallel group study of bitopertin to evaluate the safety, tolerability, efficacy, and PPIX concentration change in participants with EPP. Participants may roll over to an open label extension portion after completing the double-blind treatment period.
• Aged 18 years or older at the time of signing the informed consent form (ICF).
• Diagnosis of EPP, based on medical history by ferrochelatase ( FECH) genotyping or by biochemical porphyrin analysis.
• Body weight ≥50 kg.
• Washout of at least 2 months prior to Screening of afamelanotide and dersimelagon, if applicable.
• Aspartate aminotransferase (AST) and alanine transaminase (ALT) \<2× upper limit of normal (ULN) and total bilirubin \<ULN (unless documented Gilbert syndrome) at Screening. Albumin \>lower limit of normal (LLN).