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).

Status

Not yet recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2025-523275-27-00

Enrollment

Registered

2026-02-12

Start date

Unknown

Completion date

Unknown

Last updated

2026-02-13

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

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

Brief summary

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

Detailed description

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.

Interventions

DRUGBitopertin

Eligibility

Sex/Gender

All

Age

0 Years to No maximum

Design outcomes

Primary

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

Secondary

Measure	Time frame
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.	—

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 14, 2026