NCT05308472

Study of Bitopertin to Evaluate the Safety, Tolerability, Efficacy, and PPIX Concentrations in Participants With EPP

(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)

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 75 (actual)
• Sponsor: Disc Medicine, Inc · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

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.

Conditions

• Erythropoietic Protoporphyria

Interventions

Type	Name	Description
DRUG	DISC-1459	Oral dose level 1, once a day for 120 days
DRUG	DISC-1459	Oral dose level 2, once a day for 120 days
DRUG	Placebo	Oral dose, once a day for 120 days

Timeline

• Start date: 2022-10-31
• Primary completion: 2024-02-20
• Completion: 2024-08-23
• First posted: 2022-04-04
• Last updated: 2026-01-08
• Results posted: 2026-01-08

Locations

9 sites across 1 country: United States

Regulatory

• FDA-regulated drug study