150 Participants Needed

Bitopertin for Erythropoietic Protoporphyria

(APOLLO Trial)

Recruiting at 6 trial locations
DM
Overseen ByDisc Medicine Clinical Trials
Pivotal Trial (Near Approval)This treatment is in the last trial phase before FDA approval
Prior Safety DataThis treatment has passed at least one previous human 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?

Bitopertin is unique because it targets the heme biosynthesis pathway, potentially addressing the underlying cause of erythropoietic protoporphyria by influencing iron metabolism and protoporphyrin IX levels, unlike other treatments that mainly focus on managing symptoms like photosensitivity.12345

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

WS

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.
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Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive either bitopertin or a placebo for 6 months to evaluate efficacy, safety, and tolerability

6 months
Regular study visits for assessments

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

  • Bitopertin
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
2Treatment groups
Experimental Treatment
Placebo Group
Group I: DISC-1459 oral doseExperimental Treatment1 Intervention
Group II: PlaceboPlacebo Group1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

Disc Medicine, Inc

Lead Sponsor

Trials
6
Recruited
580+

References

Loss-of-function ferrochelatase and gain-of-function erythroid-specific 5-aminolevulinate synthase mutations causing erythropoietic protoporphyria and x-linked protoporphyria in North American patients reveal novel mutations and a high prevalence of X-linked protoporphyria. [2021]
Excessive erythrocyte PPIX influences the hematologic status and iron metabolism in patients with dominant erythropoietic protoporphyria. [2016]
New mutation identified in two sisters with adult-onset erythropoietic protoporphyria. [2013]
beta-Carotene in the treatment of erythropoietic protoporphyria. A short review. [2013]
Phototesting in erythropoietic protoporphyria trials: A systematic review. [2023]
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