Study of Bitopertin in Participants With EPP or XLP (APOLLO)
NCT ID: NCT06910358
Last Updated: 2026-02-12
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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RECRUITING
PHASE3
150 participants
INTERVENTIONAL
2025-04-04
2026-11-30
Brief Summary
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* Whether bitopertin increases pain-free sunlight exposure after 6 months of treatment in participants with EPP or XLP.
* How PPIX concentration levels change from before bitopertin treatment to after 6 months of treatment.
Researchers will compare bitopertin to a placebo look-alike substance that contains no drug.
Participants will complete daily questionnaires and attend study visits for assessments.
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Placebo
Placebo
Oral dose, once a day for 24 weeks
DISC-1459 oral dose
DISC-1459
Oral dose, once a day for 24 weeks
Interventions
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Placebo
Oral dose, once a day for 24 weeks
DISC-1459
Oral dose, once a day for 24 weeks
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
2. Diagnosis of EPP or XLP, based on medical history by ferrochelatase (FECH) or aminolevulinic acid synthase 2 (ALAS2) genotyping or by biochemical porphyrin analysis.
3. Minimum daily Sun Exposure Diary compliance ≥85% on Days -14 through Day -1, inclusive, during screening, and at least 1 successfully completed Sun Exposure Challenge (adults only, as this assessment is optional for adolescents) or historical recall of time to prodrome
4. Body weight ≥32 kg (ages 12 to \<18 years), body mass index ≥18.5 kg/m2 (ages ≥18 years) at screening.
5. Washout of at least 2 months prior to screening of afamelanotide and dersimelagon, if applicable.
6. Aspartate aminotransferase and alanine transaminase \<3× upper limit of normal (ULN)and total bilirubin \<2× ULN (unless documented Gilbert syndrome) at screening. Albumin \>lower limit of normal (LLN).
7. Willing to practice highly effective methods of birth control (both males who have partners of childbearing potential and females of childbearing potential during screening, while taking study drug, and for at least 30 days after the last dose of study drug).
Exclusion Criteria
2. Other than EPP or XLP, an inherited intrinsic or extrinsic red cell disease associated with anemia.
3. Known hypersensitivity to any component of the study drug.
4. History of liver transplantation or anticipated need for liver transplantation.
5. History of alcohol dependence or excessive alcohol consumption, as assessed by the Investigator.
6. Active 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:
9. Prior exposure to bitopertin.
10. Concurrent or planned treatment with afamelanotide or dersimelagon during the study period.
11. 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.
12. New treatment for anemia, including initiation of iron supplementation, within 1 month of screening.
13. Current or planned use of any drugs or herbal remedies known to be strong or moderate inhibitors or inducers of cytochrome P450 (CYP)3A4 enzymes for 28 days prior to the first dose and throughout the study.
14. Current or planned treatment with antipsychotic medication.
Laboratory Exclusions:
15. Hemoglobin \<10 g/dL at screening.
Miscellaneous:
16. Participation in other interventional clinical studies within 30 days prior to screening.
17. If female, pregnant or breastfeeding.
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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Marvel Clinical Research
Huntington Beach, California, 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
Henry Ford Health System
Detroit, Michigan, United States
Mount Sinai Hospital
New York, New York, United States
Wake Forest University
Winston-Salem, North Carolina, United States
Remington-Davis Clinical Research
Columbus, Ohio, United States
University of Texas Medical Branch
Galveston, Texas, United States
University of Washington
Seattle, Washington, United States
Royal Prince Alfred Hospital
Camperdown, New South Wales, Australia
The Royal Melbourne Hospital
Parkville, Victoria, Australia
University of Alberta
Edmonton, Alberta, Canada
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, The Netherlands, Netherlands
Helse Bergen
Bergen, Norway, Norway
Hospital Clinic de Barcelona
Barcelona, Spain, Spain
Karolinska University Hospital
Stockholm, Sweden, Sweden
Guy's and St Thomas' NHS Foundation Trust
London, England, United Kingdom
Countries
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Central Contacts
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Facility Contacts
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Role: backup
Role: backup
Other Identifiers
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DISC-1459-301
Identifier Type: -
Identifier Source: org_study_id
2024-520407-27-00
Identifier Type: CTIS
Identifier Source: secondary_id
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