Bitopertin for Erythropoietic Protoporphyria

(APOLLO Trial)

Recruiting at 1 trial location

Overseen By Disc Medicine Clinical Trials

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Recruiting

Sponsor: Disc Medicine, Inc

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

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

I have been diagnosed with EPP or XLP through genetic testing or porphyrin analysis.

My liver tests are within the required range.

I have kept a sun exposure diary with at least 85% accuracy for the last 14 days.

Treatment Details

Interventions

• Bitopertin (Other)

Trial Overview The study tests bitopertin against a placebo in people with EPP/XLP to see if it allows them more pain-free time in sunlight after six months and how it affects PPIX levels. It involves daily questionnaires and regular visits for assessments.

Participant Groups

Group I: DISC-1459 oral dose Experimental Treatment 1 Intervention

Group II: Placebo Placebo Group 1 Intervention

Who Is Running the Clinical Trial?

Disc Medicine, Inc

Lead Sponsor

Trials

6

Recruited

580+