What side effects are associated with this type of intervention?

Common treatments for Erythropoietic Protoporphyria (EPP) include beta-carotene, afamelanotide, and other photoprotective agents. Known side effects of these treatments can include skin discoloration, nausea, headache, and fatigue. For Glycine Transporter 1 Inhibitors like Bitopertin, potential side effects observed in other contexts may include gastrointestinal disturbances, dizziness, and changes in mood or behavior. Patients should discuss these potential side effects with their healthcare provider to manage and mitigate risks effectively.

What prior FDA approvals exist?

As of now, the FDA has approved afamelanotide (Scenesse) for the treatment of Erythropoietic Protoporphyria (EPP). Afamelanotide is a melanocortin receptor agonist that helps increase melanin production, providing photoprotection for patients with EPP. There are no FDA-approved treatments specifically similar to Bitopertin, a Glycine Transporter 1 Inhibitor, for EPP. However, ongoing research and clinical trials, such as the study of Bitopertin, aim to explore new therapeutic options for managing this condition.

What other types of research exist?

Currently, there are no specific novel alternatives to Bitopertin for Erythropoietic Protoporphyria (EPP) mentioned in the provided context. Patients should consult with their healthcare provider to explore ongoing clinical trials or emerging therapies that may be suitable for their condition.

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Other

DISC-1459 for Erythropoietic Protoporphyria

Phase 2

Waitlist Available

Research Sponsored by Disc Medicine, Inc

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Summary

This trial is testing bitopertin, a drug that might help people with EPP by changing the levels of a substance in their blood. The study aims to see if it is safe and effective for these patients.

Who is the study for?

Adults over 18 with Erythropoietic Protoporphyria (EPP), weighing at least 50 kg, and normal albumin levels can join this trial. They must not have used certain medications recently and should have stable liver function tests. People with severe allergies, recent major surgery, psychiatric conditions requiring hospitalization within six months, or those using strong CYP3A4 enzyme affecting drugs cannot participate.

What is being tested?

The study is testing Bitopertin's safety and effectiveness in treating EPP compared to a placebo. Participants will be randomly assigned to receive either the drug or placebo in a double-blind setup where neither they nor the researchers know who gets what until after the results are collected.

What are the potential side effects?

While specific side effects for Bitopertin are not listed here, common ones may include reactions at the injection site, gastrointestinal issues like nausea or diarrhea, potential liver enzyme changes, fatigue, headache or allergic reactions.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Trial Design

4Treatment groups

Experimental Treatment

Placebo Group

Group I: Open-Label Extension (optional)Experimental Treatment1 Intervention

Group II: DISC-1459 oral dose level 2Experimental Treatment1 Intervention

Group III: DISC-1459 oral dose level 1Experimental Treatment1 Intervention

Group IV: PlaceboPlacebo Group1 Intervention

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Bitopertin

Not yet FDA approved

Do I qualify?Apply below to find out

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Erythropoietic Protoporphyria (EPP) is commonly treated with agents that reduce the accumulation of protoporphyrin IX (PPIX) in the body, which is responsible for the photosensitivity and liver complications seen in EPP patients. Treatments like afamelanotide, a melanocortin receptor agonist, work by increasing melanin production, providing a protective barrier against UV radiation. Another approach involves the use of beta-carotene, which acts as an antioxidant to reduce photosensitivity. Bitopertin, a Glycine Transporter 1 Inhibitor, is being studied for its potential to modulate heme synthesis by reducing the availability of glycine, a substrate in the heme biosynthesis pathway. This could lower PPIX levels, thereby alleviating symptoms. Understanding these mechanisms is crucial for EPP patients as it helps in selecting appropriate therapies that target the underlying biochemical abnormalities, improving their quality of life.

A Phase 3 Study of Enarodustat in Anemic Patients with CKD not Requiring Dialysis: The SYMPHONY ND Study.[Infantile pyknocytosis: A cause of noenatal hemolytic anemia. Is recombinant erythropoietin an alternative to transfusion?].