Study of Bitopertin in Participants With EPP or XLP (APOLLO)

Last updated: May 8, 2025
Sponsor: Disc Medicine, Inc
Overall Status: Active - Recruiting

Phase

3

Condition

N/A

Treatment

DISC-1459

Placebo

Clinical Study ID

NCT06910358
DISC-1459-301
2024-520407-27-00
  • Ages > 12
  • All Genders

Study Summary

The goal of this clinical trial is to learn if bitopertin works and is safe to treat EPP or XLP in participants 12 years or older. The main questions it aims to answer are:

  • 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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. Aged 12 years or older at the time of study consent.

  2. Diagnosis of EPP or XLP, based on medical history by ferrochelatase (FECH) oraminolevulinic acid synthase 2 (ALAS2) genotyping or by biochemical porphyrinanalysis.

  3. Minimum daily Sun Exposure Diary compliance ≥85% on Days -14 through Day -1,inclusive, during screening, and at least 1 successfully completed Sun ExposureChallenge (adults only, as this assessment is optional for adolescents) orhistorical recall of time to prodrome

  4. Body weight ≥32 kg (ages 12 to <18 years), body mass index ≥18.5 kg/m2 (ages ≥18years) 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 havepartners of childbearing potential and females of childbearing potential duringscreening, while taking study drug, and for at least 30 days after the last dose ofstudy drug).

Exclusion

Exclusion Criteria:

  1. Major surgery within 8 weeks before screening or incomplete recovery from anyprevious surgery.

  2. Other than EPP or XLP, an inherited intrinsic or extrinsic red cell diseaseassociated 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 theInvestigator.

  6. Active human immunodeficiency virus (HIV), active hepatitis B or C.

  7. Other medical or psychiatric condition or laboratory finding not specifically notedabove that, in the judgment of the Investigator or Sponsor, would put theparticipant at unacceptable risk or otherwise preclude the participant fromparticipating in the study.

  8. Condition or concomitant medication that would confound the ability to interpretclinical, clinical laboratory, or participant diary data, including a majorpsychiatric condition that has had an exacerbation or required hospitalization inthe last 6 months. Treatment History:

  9. Prior exposure to bitopertin.

  10. Concurrent or planned treatment with afamelanotide or dersimelagon during the studyperiod.

  11. Treatment with opioids for any period >7 days in the 2 months prior to screening oranticipated to require opioid use for >7 days at any point during the study.

  12. New treatment for anemia, including initiation of iron supplementation, within 1month of screening.

  13. Current or planned use of any drugs or herbal remedies known to be strong ormoderate inhibitors or inducers of cytochrome P450 (CYP)3A4 enzymes for 28 daysprior 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 toscreening.

  17. If female, pregnant or breastfeeding.

Study Design

Total Participants: 150
Treatment Group(s): 2
Primary Treatment: DISC-1459
Phase: 3
Study Start date:
April 04, 2025
Estimated Completion Date:
November 30, 2026

Connect with a study center

  • Marvel Clinical Research

    Huntington Beach, California 92647
    United States

    Active - Recruiting

  • Massachusetts General Hospital

    Boston, Massachusetts 02115
    United States

    Active - Recruiting

  • MetroBoston Clinical Partners

    Boston, Massachusetts 02135
    United States

    Active - Recruiting

  • Remington-Davis Clinical Research

    Columbus, Ohio 43215
    United States

    Active - Recruiting

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