Ongoing, recruitingPhase I and Phase II (Integrated)- OtherInterventional

A Study to Evaluate the Pharmacokinetics of Afamelanotide in Patients with Erythropoietic Protoporphyria (EPP).

This research study is about a medicine called SCENESSE, used for adults who have a rare genetic skin condition known as Erythropoietic Protoporphyria, or EPP. People with EPP are very sensitive to sunlight, which can cause painful skin reactions. The study wants to understand how the body handles SCENESSE – how quickly it gets into the system, how long it stays, and how it eventually leaves. This is important to help doctors understand the best way to use this medicine. SCENESSE is given as a small implant under the skin. All adults are welcome to participate.

At a glance

Status

Ongoing, recruiting

Phase

Phase I and Phase II (Integrated)- Other

Sponsor

Clinuvel Europe Limited

Enrolment target

Start

20 Feb 2024

Erythropoietic Protoporphyria (EPP)

What is this study about?

This study is designed for adults living with a condition called Erythropoietic Protoporphyria, often shortened to EPP. EPP is a rare, inherited condition that makes a person's skin extremely sensitive to sunlight and strong artificial light. Even short exposure can cause painful burning, itching, swelling, and redness. It can significantly affect daily life, as people often have to avoid light exposure.

The main goal of this particular study is to learn more about a medicine called SCENESSE (the active part of which is afamelanotide). We want to understand how the body processes this medication. This involves looking at how much of the medicine gets into the bloodstream, how long it stays there, and how it eventually leaves the body. This information, often called 'pharmacokinetics', helps us understand how the medicine works within the body.

SCENESSE is given as a small implant, which is placed just under the skin. By understanding how the body handles the medicine, we can make sure it's used as effectively and safely as possible for people with EPP. This study is an early stage of research, combining what we call Phase I and Phase II, which means we are still gathering important information about the medicine's behaviour.

Key takeaways

This study is for adults with Erythropoietic Protoporphyria (EPP).
It investigates how the medicine SCENESSE moves through the body.
SCENESSE is given as a small implant under the skin.
Participation involves clinic visits and blood sample collections.
The study helps understand how to use SCENESSE most effectively.
Your decision to join or leave is voluntary and respected.

Who may be eligible?

This study is looking for volunteers who are adults, meaning you must be 18 years old or older. There is no upper age limit, so people of all adult ages can take part.

We are accepting both men and women into this study. The most important requirement is that you have been diagnosed with Erythropoietic Protoporphyria (EPP), which is the specific condition this medicine is designed to help.

Before you can join, the study team will ask you some questions and do a few tests to make sure the study is a safe and good fit for you. They will explain everything clearly, so you understand if you meet all the necessary requirements.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you 18 years old or older?
Have you been diagnosed with Erythropoietic Protoporphyria (EPP)?
Are you able to attend clinic visits as required?
Are you willing to have blood samples taken?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will receive the SCENESSE medicine as a small implant placed under your skin. This is a common way to give this type of medicine.

Throughout the study, you will have several scheduled visits to the clinic or hospital. During these visits, the study team will take blood samples at specific times. These blood samples are very important because they help us measure how much of the SCENESSE medicine is in your body and how it's being processed over time. You might also have other health checks to make sure you are doing well.

The total length of your involvement in the study will be explained in detail by the research team. After receiving the implant, there will be a period of follow-up visits to monitor your progress and continue to collect the necessary information. All procedures will be clearly explained before you agree to participate.

Potential risks and benefits

Taking part in any medical study has potential benefits and risks. A potential benefit could be contributing to a better understanding of how SCENESSE works in the body for people with EPP, which could lead to improved care in the future. However, it's important to remember that this study focuses on how the medicine moves through the body, not necessarily on treating your EPP directly. There might be side effects from the medicine or discomfort from procedures like blood tests or the implant. The study team will explain all known risks to you. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (2)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Belgium
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Unverified
Netherlands

Common questions

What is EPP?

EPP (Erythropoietic Protoporphyria) is a rare inherited condition that makes your skin very sensitive to light, causing painful reactions.

What is SCENESSE?

SCENESSE is a medicine being studied for EPP. It's given as a small implant under the skin.

Why are they doing this study?

This study aims to understand how your body handles SCENESSE – how much gets in, how long it stays, and how it leaves, to use it best for EPP.

Will this study cure my EPP?

No, this study is about understanding how the medicine works in the body, not about curing EPP, though it may help improve future treatments.

Do I have to take part?

No, taking part is completely voluntary. You can decide not to join or leave at any time without it affecting your medical care.