HELIOS: Open-Label, Long-Term Extension Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With EPP or XLP
NCT ID: NCT05883748
Last Updated: 2025-12-18
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ENROLLING_BY_INVITATION
PHASE2/PHASE3
230 participants
INTERVENTIONAL
2023-08-31
2028-06-30
Brief Summary
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Detailed Description
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Conditions
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Keywords
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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DISC-1459 Oral Dose Level 1
Oral dose, once a day
DISC-1459
DISC-1459 dose level 1
DISC-1459 Oral Dose Level 2
Oral dose, once a day
DISC-1459
DISC-1459 dose level 2
Interventions
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DISC-1459
DISC-1459 dose level 1
DISC-1459
DISC-1459 dose level 2
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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 Criteria
* 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
12 Years
ALL
No
Sponsors
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Disc Medicine, Inc
INDUSTRY
Responsible Party
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Principal Investigators
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Will Savage, MD PhD
Role: STUDY_DIRECTOR
Disc Medicine
Locations
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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
Countries
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Other Identifiers
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DISC-1459-501
Identifier Type: -
Identifier Source: org_study_id