HELIOS: An Open-Label, Long-Term Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants with Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP).

Condition studied

Erythropoietic Protoporphyria (EPP), X-Linked Protoporphyria (XLP)

Investigational medicinal product(s)

Eligibility

Primary endpoint

Safety and tolerability of bitopertin, as assessed by the incidence of treatmentemergent adverse events (TEAEs), vital signs, physical examinations, and clinical laboratory parameters.

Endpoint detail

1. Change from baseline in daylight tolerance, as assessed by total hours spent in sunlight without pain and average time to first prodromal syndrome in sunlight., 2. Percent change from baseline in whole blood metal-free PPIX concentrations., 3. Plasma bitopertin concentrations.