Bitopertin for Erythropoietic Protoporphyria
(APOLLO Trial)
Trial Summary
Will I have to stop taking my current medications?
The trial requires a washout period (time without taking certain medications) of at least 2 months for afamelanotide and dersimelagon before screening. You also cannot use opioids for more than 7 days in the 2 months before screening or during the study, and you must avoid certain drugs or herbal remedies that affect liver enzymes for 28 days before starting the trial and throughout the study.
How does the drug Bitopertin differ from other treatments for erythropoietic protoporphyria?
What is the purpose of this trial?
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.
Research Team
Will Savage, MD, PhD
Principal Investigator
Disc Medicine
Eligibility Criteria
This trial is for individuals aged 12 or older with a diagnosis of EPP or XLP, confirmed by genetic testing or porphyrin analysis. They must have liver function within certain limits and agree to use effective birth control. Participants need to weigh at least 32 kg if under 18, or have a BMI over 18.5 if adult.Inclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive either bitopertin or a placebo for 6 months to evaluate efficacy, safety, and tolerability
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- Bitopertin
Find a Clinic Near You
Who Is Running the Clinical Trial?
Disc Medicine, Inc
Lead Sponsor