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COMPLETEDPHASE2

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)

NCT05308472•Disc Medicine, Inc

View on ClinicalTrials.gov

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.

Eligibility

Age

18 - No limit years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•1. Aged 18 years or older at the time of signing the informed consent form (ICF).
•2. Diagnosis of EPP, based on medical history by ferrochelatase ( FECH) genotyping or by biochemical porphyrin analysis.
•3. Body weight ≥50 kg.
•4. Washout of at least 2 months prior to Screening of afamelanotide and dersimelagon, if applicable.
•5. Aspartate aminotransferase (AST) and alanine transaminase (ALT) \<2× upper limit of normal (ULN) and total bilirubin \<ULN (unless documented Gilbert syndrome) at Screening. Albumin \>lower limit of normal (LLN).

Exclusion Criteria

•Medical History:
•1. Major surgery within 8 weeks before Screening or incomplete recovery from any previous surgery.
•2. Other than EPP, an inherited or acquired red cell disease associated with anemia.
•3. A history or known allergic reaction to any investigational product excipients or history of anaphylaxis to any food or drug.
•4. History of liver transplantation.
•5. History of alcohol dependence or excessive alcohol consumption, as assessed by the Investigator.
•6. Human immunodeficiency virus (HIV), active Hepatitis B, or C.
•7. Other medical or psychiatric condition or laboratory finding not specifically noted above that, in the judgment of the Investigator or Sponsor, would put the participant at unacceptable risk or otherwise preclude the participant from participating in the study
•8. Condition or concomitant medication that would confound the ability to interpret clinical, clinical laboratory, or participant diary data, including a major psychiatric condition that has had an exacerbation or required hospitalization in the last 6 months.
•Treatment History:
+6 more criteria

Locations (9)

University of Alabama Hospital

Birmingham, Alabama, United States

University of California San Francisco

San Francisco, California, United States

University of Miami Miller School of Medicine

Miami, Florida, United States

Massachusetts General Hospital

Boston, Massachusetts, United States

Mount Sinai Hospital

New York, New York, United States

Atrium Health Wake Forest Baptist

Winston-Salem, North Carolina, United States

Einstein Medical Center

Philadelphia, Pennsylvania, United States

University of Texas

Galveston, Texas, United States

Fred Hutchinson Cancer Center

Seattle, Washington, United States

Key Dates

Start Date

October 31, 2022

Primary Completion Date

February 20, 2024

Completion Date

August 23, 2024

Last Updated

January 8, 2026

Enrollment

ACTUAL participants

Conditions

Erythropoietic Protoporphyria

Interventions

DISC-1459

DRUG

DISC-1459

DRUG

Placebo

DRUG

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

NCT06910358

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

View study

ENROLLING_BY_INVITATIONPHASE2

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

NCT06971900

Erythropoietic Protoporphyria (EPP)

View study

COMPLETED

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: January 8, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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

Inclusion Criteria

Exclusion Criteria

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

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

Phase II Confirmatory Study in Erythropoietic Protoporphyria (EPP)

Multicentre Phase III Erythropoietic Protoporphyria Study

Sorbent Therapy of the Cutaneous Porphyrias

Effect of Isoniazid on Protoporphyrin Levels in Erythropoietic Protoporphyria