Study of Bitopertin in Participants With EPP or XLP (APOLLO)
The goal of this clinical trial is to learn if bitopertin works and is safe to treat EPP or XLP in participants 12 years or older. The main questions it aims to answer are: - Whether bitopertin increases pain-free sunlight exposure after 6 months of treatment in partici...
Age: 12 years - 66+
Gender: All
HELIOS: Open-Label, Long-Term Extension Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With EPP or XLP
This is an open-label, long-term extension study to investigate the safety, tolerability
and efficacy of DISC-1459 in participants with EPP.
Age: 12 years - 66+
Gender: All
Study of Bitopertin to Evaluate the Safety, Tolerability, Efficacy, and PPIX Concentrations in Participants With 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 portio...
Age: 18 years - 66+
Gender: All
Longitudinal Study of the Porphyrias
The objective of this protocol is to conduct a longitudinal multidisciplinary investigation of the human porphyrias including the natural history, morbidity, pregnancy outcomes, and mortality in people with these disorders.
Age: Birth - 66+
Gender: All