Active not recruitingPHASE3INTERVENTIONAL

INcreased Sun Exposure Without Pain In Research Participants With EPP or XLP

This research study is investigating a new treatment called Dersimelagon for individuals aged 12 to 75 who have either Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP). These conditions make skin very sensitive to sunlight, often causing pain, burning, tingling, or itching. The main goal is to find out if Dersimelagon can extend the time people with EPP or XLP can spend in the sun before these uncomfortable symptoms begin, and to see if it makes these symptoms less severe. Participants will either receive the new medication or a placebo (a dummy pill) to compare the effects. The study hopes to improve the quality of life for those affected by these challenging sun-sensitive conditions.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Tanabe Pharma America, Inc.

Enrolment target

165

Start

11 Dec 2023

Estimated completion

01 Jun 2026

Erythropoietic Protoporphyria (EPP)X-Linked Protoporphyria (XLP)

What is this study about?

This study is about understanding how a new medicine, called Dersimelagon (also known as MT-7117), can help people with two rare conditions: Erythropoietic Protoporphyria (EPP) and X-Linked Protoporphyria (XLP). If you have one of these conditions, you know how difficult it can be. Your skin is extra sensitive to sunlight, and even a short time outside can cause painful symptoms like burning, tingling, itching, or stinging.

The main idea of this research is to see if Dersimelagon can make a real difference. Specifically, the researchers want to find out if taking this medicine helps people with EPP or XLP spend more time in the sun before they start feeling these early, uncomfortable symptoms. They also want to check if the medicine can make those symptoms less severe when they do appear. Improving your ability to be outdoors without discomfort could significantly improve your daily life and activities.

To do this, some people will receive Dersimelagon, and others will receive a placebo, which looks just like the real medicine but has no active ingredients. This helps researchers fairly compare how effective the new medicine is. The study is currently in Phase 3, which means it's one of the final steps before a medicine might be considered for wider use.

Key takeaways

The study is for people with EPP or XLP aged 12-75.
It tests a new medication, Dersimelagon, against a placebo.
The main goal is to see if it helps reduce sun sensitivity and pain.
Participants need to be willing to visit clinics and get sun exposure.
You can stop participating at any time.
This is a 'Phase 3' study, meaning it's a key stage for new medicines.

Who may be eligible?

To join this study, you need to have a confirmed diagnosis of Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP). You must be between 12 and 75 years old. It's important that you're able to come to the study locations for all appointments and are willing to spend time outdoors in the sun as much as you can during the study.

There are also some conditions that would prevent you from taking part. For example, if you have other skin conditions caused by light sensitivity (apart from EPP or XLP), or if you have certain liver problems. You wouldn't be able to join if you have a history of certain cancers like melanoma, squamous cell, or basal cell skin cancer.

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.

Do I have a confirmed diagnosis of EPP or XLP?
Am I between 12 and 75 years old?
Am I able to travel to study appointments?
Am I willing to spend time outdoors in the sun during the study?
Do I have any other light-sensitive skin conditions or certain liver problems?
Have I had melanoma or other significant skin cancers?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you'll first be asked to sign a consent form, which means you understand the study and agree to participate. If you're under 18, both you and your parents will need to agree. You'll have regular visits to the study clinics for checks and to receive the study medication. This medication will be a pill you take, either Dersimelagon or a placebo (a dummy pill). You'll be asked to try and get outside in the sun as much as possible during daylight hours throughout the study. We won't know exactly how long the study will last for each person based on this summary, but it will involve several visits, medication, and follow-up checks.

Potential risks and benefits

Taking part in a study like this could potentially offer benefits, such as finding out if Dersimelagon can help you spend more time in the sun without painful symptoms. However, there are also potential risks, including side effects from the medication (though these are not detailed here), and the time commitment involved. You might also receive the placebo and not the active drug. It's very important to remember that you are free to leave the study at any time, for any reason, without it affecting your usual medical care.

Locations (35)

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

Marvel Clinical Research, LLC
Verified postcode
Huntington Beach, United States
University of California at San Francisco
Verified postcode
San Francisco, United States
University Of Miami School Of Medicine, Center For Liver Diseases
Verified postcode
Miami, United States
MGH
Verified postcode
Boston, United States
MetroBoston Clinical Partners, LLC
Verified postcode
Brighton, United States
Henry Ford Health System
Verified postcode
Detroit, United States
Kansas City Research Institute
Verified postcode
Kansas City, United States
Icahn School of Medicine at Mount Sinai (ISMMS) - The Mount Sinai Hospital (MSH)
Verified postcode
New York, United States
Wake Forest University Baptist Health
Verified postcode
Winston-Salem, United States
Remington-Davis Clinical Research
Verified postcode
Columbus, United States
Einstein Medical Center (EMC)
Verified postcode
Philadelphia, United States
The University of Texas Medical Branch (UTMB)
Verified postcode
Galveston, United States

Common questions

What are EPP and XLP?

They are rare conditions where your skin becomes very sensitive to sunlight, often causing pain, burning, or itching when exposed to the sun.

What is Dersimelagon?

Dersimelagon is a new experimental medication being tested to see if it can help people with EPP or XLP spend more time in the sun without uncomfortable symptoms.

What is a placebo?

A placebo is a 'dummy' pill that looks just like the study medication but doesn't contain any active ingredients. This helps researchers compare the real medicine's effects.

Will I definitely get the new medicine?

No, you might receive the new medicine or the placebo. The study is set up this way to fairly test the medicine's effectiveness.

How old do I need to be to join?

You need to be between 12 and 75 years old to be considered for this study.

How to find out more

Official trial record

Always speak to your GP or specialist before deciding to take part in a study.