RecruitingPHASE3INTERVENTIONAL

Extension Study to Evaluate Safety and Tolerability of Oral Dersimelagon (MT-7117) in Subjects With Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP)

This research is a continuation study checking the long-term safety and how well people tolerate a medicine called dersimelagon (also known as MT-7117). It's for individuals living with Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP), which are rare conditions that make your skin very sensitive to light. This study is specifically for people who have already taken part in previous studies involving this medicine. The main goal is to keep an eye on any side effects and understand how the body handles the medicine over a longer period. It's a 'Phase 3' study, meaning it's one of the final steps before a medicine might become widely available.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

Tanabe Pharma America, Inc.

Enrolment target

301

Start

10 Aug 2021

Estimated completion

01 Dec 2027

EPP XLP

What is this study about?

This study is focused on two rare inherited conditions called Erythropoietic Protoporphyria (EPP) and X-Linked Protoporphyria (XLP). People with these conditions experience severe pain, burning, and itching when their skin is exposed to sunlight or bright artificial light. This can significantly impact their daily lives, as they often have to avoid light to prevent these painful reactions.

The medicine being studied is called dersimelagon (or MT-7117). It's hoped that this medicine could help people with EPP and XLP manage their symptoms and live more comfortably. Since this medicine has already been studied in earlier trials, this particular study is an 'extension' or follow-up. Its main purpose is to gather more information about the medicine's long-term safety and to see how well people tolerate it over extended periods.

By continuing to monitor participants who have already received dersimelagon, researchers can better understand any potential long-term side effects and how consistently the medicine is tolerated. This information is crucial for making sure the medicine is safe and effective if it becomes available to everyone in the future. Knowing more about long-term use helps doctors and patients make informed decisions.

Key takeaways

This study is for people with EPP or XLP who have already tried the medicine dersimelagon.
It aims to understand the long-term safety and side effects of dersimelagon.
Participants must be aged 12-75 and able to attend study site visits.
Careful monitoring will be done for any changes in health.
Your contribution helps advance understanding of EPP/XLP treatment.
You can stop participating at any time.

Who may be eligible?

This study is for people aged 12 to 75 who have either Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP). A key requirement is that you must have already taken part in one of the previous studies for dersimelagon (MT-7117) and completed it. This ensures that everyone joining this long-term safety study has already been introduced to the medicine.

You also need to be able to travel to the study sites for appointments and be willing to follow all the study rules. For female participants, if you could become pregnant, you'll need to have a negative pregnancy test and agree to use two effective methods of contraception during the study. The same applies to male participants whose partners could become pregnant.

There are also some reasons why you might not be able to join. For example, if you have other skin conditions caused by light exposure that aren't EPP or XLP. Also, if you have serious liver problems or certain abnormal blood test results related to your liver, you might not be eligible. The study team will check all these details carefully to make sure it's safe for you to take part.

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.

I am between 12 and 75 years old.
I have been diagnosed with EPP or XLP.
I have already completed a previous study in which I received dersimelagon (MT-7117).
I am able and willing to travel to the study clinic for appointments.
If I identify as female and could become pregnant, I am not pregnant and agree to use two effective birth control methods.
If I identify as male and my partner could become pregnant, we agree to use two effective birth control methods.

Answer every question to see your result.

What does participation involve?

If you decide to join this study, you will continue to take the study medicine, dersimelagon, by mouth. You will need to visit the study site regularly for check-ups and assessments. These visits will help the medical team monitor your health, how the medicine is affecting you, and if you're experiencing any side effects. They may involve blood tests, physical examinations, and questions about how you are feeling and how your condition is affecting you.

The specific number of visits and the total length of your participation will depend on the study's design, but it is an extension study, so it will involve long-term follow-up. The study team will explain the full schedule to you before you agree to take part. You'll need to be able to travel to the study site for all your appointments.

Potential risks and benefits

Participating in this study might offer the benefit of continuing to receive a medicine that could help manage your EPP or XLP, and it contributes valuable information to the medical community about its long-term safety. However, like all medicines, dersimelagon may have side effects, and some are not yet fully known, especially with long-term use. The study team will closely monitor you for any potential issues. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (51)

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· Recruiting
University of California at San Francisco - CSF Porphyria Center
Verified postcode
San Francisco, United States· Not yet recruiting
University Of Miami School Of Medicine, Center For Liver Diseases
Verified postcode
Miami, United States· Not yet recruiting
Massachusetts General Hospital
Verified postcode
Boston, United States· Not yet recruiting
MetroBoston Clinical Partners, LLC
Verified postcode
Brighton, United States· Recruiting
Henry Ford Health System
Verified postcode
Detroit, United States· Not yet recruiting
Kansas City Research Institute
Verified postcode
Kansas City, United States· Recruiting
Icahn School of Medicine at Mount Sinai (ISSMS)-The Mount Sinai Hospital (MSH)
Verified postcode
New York, United States· Recruiting
Wake Forest University Baptist Health
Verified postcode
Winston-Salem, United States· Recruiting
Cleveland Clinic - Taussig Cancer Institute
Verified postcode
Cleveland, United States· Not yet recruiting
Remington-Davis Clinical Research
Verified postcode
Columbus, United States· Recruiting
Thomas Jefferson University
Verified postcode
Philadelphia, United States· Recruiting

Common questions

What is EPP or XLP?

They are rare conditions that make your skin very sensitive to light, causing pain and burning when exposed to sun or bright light.

Who is this study for?

It's for people aged 12 to 75 with EPP or XLP who have already taken part in a previous study for dersimelagon (MT-7117).

What is dersimelagon (MT-7117)?

It's the experimental medicine being studied to see its long-term safety and how well people tolerate it for EPP/XLP.

Do I have to travel for the study?

Yes, you need to be able to travel to the study sites for all your scheduled appointments.

What if I decide I don't want to continue?

You can leave the study at any time, for any reason, and it won't affect your regular medical care.

How to find out more

Clinical Trials Information Desk, To prevent mis-communication,

information.US@mb.tanabe-pharma.com please e-mail Official trial record

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