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

Completed · Phase 2 · Has a placebo group

Conditions studied: Erythropoietic Protoporphyria

In brief

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.

Key facts

Study ID: NCT05308472
Run by: Disc Medicine, Inc
People needed: 75
Starts: 2022-10-31
Expected to finish: 2024-08-23
Last updated by the study team: 2026-01-08

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

Aged 18 years or older at the time of signing the informed consent form (ICF).
Diagnosis of EPP, based on medical history by ferrochelatase ( FECH) genotyping or by biochemical porphyrin analysis.
Body weight ≥50 kg.
Washout of at least 2 months prior to Screening of afamelanotide and dersimelagon, if applicable.
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).

You may not qualify if…

Medical History:
Major surgery within 8 weeks before Screening or incomplete recovery from any previous surgery.
Other than EPP, an inherited or acquired red cell disease associated with anemia.
A history or known allergic reaction to any investigational product excipients or history of anaphylaxis to any food or drug.
History of liver transplantation.
History of alcohol dependence or excessive alcohol consumption, as assessed by the Investigator.
Human immunodeficiency virus (HIV), active Hepatitis B, or C.
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
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:
Concurrent or planned treatment with afamelanotide or dersimelagon during the study period.
Treatment with opioids for any period >7 days in the 2 months prior to screening or anticipated to require opioid use for >7 days at any point during the study.
New treatment for anemia, including initiation of iron supplementation, in the 2 months prior to Screening.
Current or planned use of any drugs or herbal remedies known to be strong inhibitors or inducers of CYP3A4 enzymes for 28 days prior to the first dose and throughout the study.
Laboratory Exclusions:
Hemoglobin <10 g/dL at Screening.

Where it is running

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

Full record on ClinicalTrials.gov

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