Erythropoietic Protoporphyria — Clinical Trials

MONDO:00016765 active·2 recruitingLast updated: Mar 19, 2026, 12:05 AM

Trial Landscape

Erythropoietic Protoporphyria has an emerging clinical trial program, driven by drug treatment.

Recruiting

Active Trials

By Approach

Drug Treatment5

By Phase

Phase 2

Phase 3

Sponsor Types

Industry5

Top Sponsors

Disc Medicine, Inc

Portal Therapeutics, Inc.

Tanabe Pharma America, Inc.

Trial Locations

United States5 trials

Australia4 trials

France3 trials

Netherlands3 trials

Spain3 trials

United Kingdom3 trials

Canada2 trials

Czechia2 trials

Germany2 trials

Italy2 trials

Trials often recruit across multiple countries, so totals exceed the trial count.

5 Trials

Phase 3Recruiting

NCT06910358

Study of Bitopertin in Participants With EPP or XLP (APOLLO)

Sponsor:Disc Medicine, IncOrphan Designation

United StatesStarted: Apr 2025Est. completion: Nov 2026Updated: Feb 2026

Phase 3Recruiting

NCT05005975

Extension Study to Evaluate Safety and Tolerability of Oral Dersimelagon (MT-7117) in Subjects With Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP)

Sponsor:Tanabe Pharma America, Inc.

United StatesStarted: Aug 2021Est. completion: Dec 2027Updated: Dec 2025

Phase 2Enrolling by Invitation

NCT05883748

HELIOS: Open-Label, Long-Term Extension Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With EPP or XLP

Sponsor:Disc Medicine, IncOrphan Designation

United StatesStarted: Aug 2023Est. completion: Jun 2028Updated: Dec 2025

Phase 2Enrolling by Invitation

NCT06971900

GATEWAY: A Phase 2a Study of PORT-77 in Adults With Erythropoietic Protoporphyria

Sponsor:Portal Therapeutics, Inc.Orphan Designation

United StatesStarted: Apr 2025Est. completion: Jan 2026Updated: May 2025

Phase 3Active, Not Recruiting

NCT06144840

INcreased Sun Exposure Without Pain In Research Participants With EPP or XLP

Sponsor:Tanabe Pharma America, Inc.

United StatesStarted: Dec 2023Est. completion: Jun 2026Updated: Dec 2025

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Data sourced from ClinicalTrials.gov via Kisho's automated pipeline. Trials refreshed every 4 hours for monitored diseases.

Last sync: March 19, 2026 at 12:05 AM