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

Launched by DISC MEDICINE, INC · Mar 27, 2025

Keywords

ClinConnect Summary

This clinical trial, called APOLLO, is studying a medication called bitopertin to see if it helps people aged 12 and older with Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP). The main goals are to find out if bitopertin can allow participants to spend more time in the sun without pain after six months and to measure changes in a specific substance in the blood related to these conditions. Participants will receive either bitopertin or a placebo (a look-alike pill with no active drug) and will need to fill out daily questionnaires and attend regular study visits.

To be eligible for this trial, participants must be at least 12 years old and have a confirmed diagnosis of EPP or XLP. They need to be able to show that they can keep a daily diary of sun exposure and meet certain health criteria, including maintaining a healthy weight. The study is currently recruiting, so if you or someone you know meets these criteria and is interested, it could be a chance to contribute to important research while potentially benefiting from the treatment.

Gender

ALL

Eligibility criteria

• 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) 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:
• • 1. Major surgery within 8 weeks before screening or incomplete recovery from any previous surgery.
• • 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.