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

Enrolling by invitation · Phase 2/Phase 3

Conditions studied: Erythropoietic Protoporphyria

In brief

This is an open-label, long-term extension study to investigate the safety, tolerability and efficacy of DISC-1459 in participants with EPP.

Key facts

Study ID

NCT05883748

Run by

Disc Medicine, Inc

People needed

230

Starts

2023-08-31

Expected to finish

2028-06-01

Last updated by the study team

2026-07-23

Who can join

Age: 12 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Participants with diagnosis of EPP who are participating (or who have participated) in a prior Disc Medicine bitopertin study and who have completed the randomized treatment phase and End-of-Study visit
• Aged ≥12 years upon study consent
• Body weight ≥32 kg for participants <18 years of age and BMI ≥18.5 kg/m2 for adult participants
• Willing to practice highly effective methods of birth control (both males who have partners of childbearing potential and females of childbearing potential during the study, while taking study drug, and for at least 30 days after the last dose of study drug.
• Negative urine or serum pregnancy test (females of childbearing potential).
• Able to understand the study aims, procedures, and requirements, and provide written informed consent (and assent if necessary).
• Able to comply with all study procedures.

You may not qualify if…

• Participants who have an ongoing SAE from a clinical study that is assessed by the investigator as related to bitopertin
• Other medical or psychiatric condition or laboratory finding not specifically noted above that, in the judgement of the Investigator or Sponsor, would put the participant at unacceptable risk or otherwise preclude participation 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
• Planned treatment with afamelanotide or dersimelagon during the study
• Planned use of any drugs or herbal remedies known to be strong inhibitors or inducers of cytochrome p450 (CYP)3A4 enzymes throughout the study
• If female, pregnant, or breastfeeding
• Participation in any other clinical protocol or investigational trial, other than Disc Medicine bitopertin trials, that involves administration of experimental therapy and/or therapeutic devices within 30 days of Day 1
• Score of PHQ-8 ≥10 at screening or imminent suicidal risk identified by the C-SSRS as defined as suicidal ideation with intent (Grade 4 or 5) within the last year or any suicidal behavior within the last 5 years.
• Consumption of grapefruit/Seville orange and products containing these for 14 days prior to first dose of study drug and throughout the study

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
• MetroBoston Clinical Partners — Boston, Massachusetts, United States
• Mount Sinai Hospital — New York, New York, United States
• Atrium Health Wake Forest Baptist — Winston-Salem, North Carolina, United States
• Remington-Davis Clinical Research — Columbus, Ohio, United States
• Einstein Medical Center — Philadelphia, Pennsylvania, United States
• University of Texas — Galveston, Texas, United States
• Fred Hutchinson Cancer Center — Seattle, Washington, United States
• Royal Prince Alfred Hospital — Camperdown, New South Wales, Australia
• The Royal Melbourne Hospital — Parkville, Victoria, Australia
• UZ Leuven — Leuven, Belgium
• CHU de Nantes - Hôtel Dieu, Service de dermatologie — Nantes, France, France
• Centre d'Investigation Clinique (CIC) Hôpital Bichat - Claude-Bernard — Paris, France, France
• Charité - Universitätsmedizin Berlin, Institute of Allergology — Berlin, Germany, Germany
• Klinikum Chemnitz gGmbH — Chemnitz, Saxony, Germany
• Erasmus MC — Rotterdam, Netherlands
• Hospital Clinic de Barcelona — Barcelona, Spain, Spain
• Guy's and St Thomas' NHS Foundation Trust — London, England, United Kingdom

Full record on ClinicalTrials.gov

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