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APOLLO: A Randomized, Double-Blind, Placebo-Controlled Study of Bitopertin to Evaluate the Efficacy, Safety, and Tolerability in Participants with Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP)
EU CTIS ID: 2024-520407-27-00
What this study is testing
To assess average pain-free sunlight exposure tolerance after 6 months of treatment To assess changes in whole-blood metal-free protoporphyrin IX (PPIX) levels after 6 months of treatment To assess safety and tolerability
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 1. Aged 12 years or older at the time of study consent.
- 10. Able to comply with all study procedures.
- 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 [CCI] through Day [CCI], 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.
You likely can't join if
- 1. Major surgery within 8 weeks before screening or incomplete recovery from any previous surgery.
- 10. Prior exposure to bitopertin.
- 11. Concurrent or planned treatment with afamelanotide or dersimelagon during the study period.
- 12. 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.
- 13. New treatment for anemia, including initiation of iron supplementation, within 1 month of screening.
- 14. 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 [CCI} days prior to the first dose and throughout the study.
See the full eligibility criteria
Who can join
- 1. Aged 12 years or older at the time of study consent.
- 10. Able to comply with all study procedures.
- 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 [CCI] through Day [CCI], 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.
- 8. Negative pregnancy test (females of childbearing potential) at screening (Days [CCI] to [CCI]) AND baseline (Day 1), prior to dosing.
- 9. Able to understand the study aims, procedures, and requirements, and provide written informed consent (and assent if necessary).
What rules you out
- 1. Major surgery within 8 weeks before screening or incomplete recovery from any previous surgery.
- 10. Prior exposure to bitopertin.
- 11. Concurrent or planned treatment with afamelanotide or dersimelagon during the study period.
- 12. 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.
- 13. New treatment for anemia, including initiation of iron supplementation, within 1 month of screening.
- 14. 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 [CCI} days prior to the first dose and throughout the study.
- 15. Current or planned treatment with antipsychotic medication.
- 16. Hemoglobin <10 g/dL at screening.
- 17. Participation in other interventional clinical studies within 30 days prior to screening.
- 18. If female, pregnant, planning to become pregnant, or breastfeeding.
- 2. Other than EPP or XLP, an inherited intrinsic or extrinsic red cell disease associated with anemia, eg, G6PD, hemoglobinopathy, membranopathy, or immune or nonimmune hemolytic anemia. Any comorbid hematologic disease must be deemed acceptable by the Sponsor.
- 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. A positive HIV or viral hepatitis test result should be discussed between the Investigator and Sponsor prior to enrollment.
- 7. [CCI]
- 8. 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.
- 9. 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.
The study team makes the final eligibility decision.
Where it's taking place
- Canada
- United Kingdom
- United States
- Australia
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years, 0-17 years, 65+ years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Canada; United Kingdom; United States; Australia. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.