Not recruitingRare disease

Study of Afamelanotide for Patients with Erythropoietic Protoporphyria (EPP)

Verified siteRegistered drugNo placebo

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial focuses on a condition called Erythropoietic Protoporphyria (EPP), a rare genetic disorder that causes sensitivity to sunlight, leading to painful skin reactions. The study will use a treatment called afamelanotide, which is administered as an implant under the skin. Afamelanotide is a synthetic hormone designed to help reduce the skin's sensitivity to light in people with EPP.

The purpose of the study is to understand how afamelanotide behaves in the body, specifically looking at how it is absorbed, distributed, and eliminated. This is known as pharmacokinetics. The study will involve both adolescents and adults with EPP to compare how the treatment works in these different age groups.

Participants in the study will receive the afamelanotide implant and will be monitored over a period of time to gather information on how the treatment affects their condition. The study aims to provide valuable insights into the effectiveness and safety of afamelanotide for managing symptoms of EPP.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initial assessment
Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes verifying age requirements, which are between 12 and 70 years for patients with erythropoietic protoporphyria (EPP).
Patients must agree not to use any medications during the trial and must be able to understand and sign the informed consent form. For adolescents, a parent or legal representative must also sign.
Step 2
Administration of medication
The medication used in this study is afamelanotide, administered as a SCENESSE 16 mg implant. The implant is placed subcutaneously, which means it is inserted under the skin.
The purpose of this step is to evaluate how the body absorbs and processes the medication, known as pharmacokinetics.
Step 3
Monitoring and evaluation
After the administration of the implant, patients are monitored to assess the pharmacokinetic profile of afamelanotide. This involves measuring the maximum concentration of the drug in the blood (Cmax) and the area under the concentration-time curve (AUC(0-t)).
These measurements help determine how the medication is processed in both adolescent and adult patients.
Step 4
Completion of study
The study is estimated to conclude by July 26, 2024. At the end of the study, data collected will be analyzed to understand the pharmacokinetics of afamelanotide in patients with EPP.

Who can join the trial?

5 criteria

Must be diagnosed with Erythropoietic Protoporphyria (EPP), a condition that affects the skin and blood.
Adults aged between 18 and 70 years or adolescents aged between 12 and 17 years can participate.
Must agree not to use any medications during the study.
Must be able to understand and sign a written document called the Informed Consent Form. For adolescents, a parent or legal representative must also sign.
Must be able and willing to follow the study's rules and requirements.

Who cannot join the trial?

8 criteria

Patients who do not have a confirmed diagnosis of Erythropoietic Protoporphyria (EPP). EPP is a condition that affects the skin and blood.
Patients who are not within the specified age range for the study. The study includes adolescents and adults.
Patients who are not able to follow the study procedures or attend the required visits.
Patients who are pregnant or breastfeeding.
Patients who have other medical conditions that might interfere with the study or make participation unsafe.
Patients who are taking medications that could affect the study results.
Patients who have participated in another clinical trial recently.
Patients who have a history of allergic reactions to the study medication or similar drugs.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
Responsive
Rotterdam, The Netherlands
Katholieke Universiteit te Leuven
Responsive
Leuven, Belgium

Trial locations

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Investigated drugs

Afamelanotide is a medication being studied for its effects in patients with Erythropoietic Protoporphyria (EPP). This study aims to understand how the body absorbs, distributes, and processes afamelanotide in both adolescent and adult patients with EPP. The goal is to compare how the medication behaves in these two different age groups to ensure its safety and effectiveness.

Afamelanotide

What is already known about the treatment

Afamelanotide – Afamelanotide is administered as a subcutaneous implant, which is placed under the skin. It is currently being studied in clinical trials for its effectiveness in treating Erythropoietic Protoporphyria (EPP), a rare genetic condition. The medication works by stimulating the production of melanin, a natural pigment in the skin, which helps protect against damage from sunlight. Afamelanotide is classified as a melanocortin receptor agonist, and it is being evaluated for its pharmacokinetics, or how the drug is absorbed, distributed, metabolized, and excreted in the body.

Investigated diseases

Erythropoietic Protoporphyria (EPP) – Erythropoietic Protoporphyria is a rare genetic disorder that affects the production of heme, a component of hemoglobin. This condition leads to an accumulation of protoporphyrin in the blood, which can cause sensitivity to sunlight. Individuals with EPP often experience painful skin reactions when exposed to sunlight, including burning, itching, and swelling. These symptoms can occur within minutes of sun exposure and may last for several hours. Over time, repeated sun exposure can lead to chronic skin changes and scarring. EPP is typically diagnosed in childhood, as symptoms often appear early in life.