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
Enrolling by Invitation
PHASE2Ages 12+InterventionalTreatmentDisc Medicine, Inc
~230 participants
Updated 2026-07-23 on ClinicalTrials.gov
What's tested:DISC-1459
At a glance
Recruiting sites
0 of 21 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Incidence of treatment-emergent adverse events
Measured over up to 5 Years
+5 more outcomes measured
Conditions
Where it's being run
21 sites across 19 statesMassachusetts2
France2
Alabama1
California1
Florida1
New York1
North Carolina1
Ohio1
Study leadership
- Will Savage, MD PhD · STUDY_DIRECTOR · Disc Medicine
Who to contact
This trial hasn't published a contact. View it on ClinicalTrials.gov
Do you actually qualify for this trial?
Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.
Eligibility criteria
Inclusion
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.
Exclusion
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
What this trial measures
- Incidence of treatment-emergent adverse eventsup to 5 Years
- Incidence of clinically abnormal vital signsup to 5 Years
- Incidence of clinically abnormal physical examup to 5 Years
- Incidence of abnormal laboratory test resultsup to 5 Years
- Assessment of Patient Health Questionnaire (PHQ-8)up to 5 Years
The Patient Health Questionnaire (PHQ-8), an 8-item participant-report measure for screening for depression and for establishing depression severity. The total score ranges from 0-24, with a higher score indicating greater depression symptom severity.
- Assessment of C-SSRSup to 5 Years
The C-SSRS is a clinician-rated scale that assesses suicidality from ideation to behaviors and monitors the potential emergence of suicidality in clinical studies.