Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

MT-7117-A-302 Study: A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Adults and Adolescents with Erythropoietic Protoporphyria or X-Linked Protoporphyria

This important study is investigating a new medication called MT-7117 for grown-ups and young people living with Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP). These are rare conditions that make skin very sensitive and painful in sunlight. The main goal is to see if MT-7117 can increase the amount of time people can spend in the sun before they feel burning, tingling, or itching sensations. Researchers will also check how patients feel overall about their condition improving and count how many painful or uncomfortable reactions to sunlight occur during the study. This is a "Phase 3" trial, meaning it's a key step to see if the medicine really works and is safe before it can be widely used.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Tanabe Pharma America Inc.

Enrolment target

Start

11 Mar 2024

Erythropoietic Protoporphyria or X-Linked Protoporphyria

What is this study about?

This study is about finding a better way to help people who have rare conditions called Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP). If you have one of these, your skin can become very painful, tingly, or itchy when exposed to sunlight. This can make everyday activities, like going outside, very difficult.

The researchers are testing a new medicine called MT-7117. They want to see if this medicine can help people with EPP or XLP spend more time in the sun without getting those uncomfortable skin feelings. They will be measuring how much longer a person can stay in the sun before they start to feel early warning signs like burning, tingling, or itching, especially during the middle of the day. They'll compare this new medicine to a dummy pill (placebo) to truly understand its effects.

Beyond simply spending more time in the sun, the study will also look at how patients feel their condition is changing and improving. They'll also keep track of how many painful skin reactions or other kinds of sun-related problems happen during the study. The aim is to find a treatment that can significantly improve the daily lives of people living with these challenging conditions.

Key takeaways

This study is testing a new medicine (MT-7117) for rare sun sensitivity conditions.
It aims to help people with EPP or XLP spend more time in the sun without discomfort.
Participants will receive either the new medicine or a dummy pill for 16 weeks.
The study assesses how daily sun exposure time and pain events change.
It's a Phase 3 study, a crucial step before potential widespread use.

Who may be eligible?

To join this study, participants need to be adults or teenagers, aged 18 years or older. There is no upper age limit, meaning older adults can also take part. Both men and women are welcome to participate.

To be eligible, you must have been diagnosed with either Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP). These are the specific conditions the new medicine is designed to help.

There might be other specific health requirements or conditions that would prevent someone from joining, but these would be discussed in detail by the study doctor. It's always best to talk to the study team directly to confirm if you might be a good fit.

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)?
Or have you been diagnosed with X-Linked Protoporphyria (XLP)?
Are you able to attend regular clinic visits?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would be given either the new medicine, MT-7117, or a dummy pill (placebo). You wouldn't know which one you are taking. You would take the medication regularly for 16 weeks.

Throughout these 16 weeks, you would attend several appointments at the study clinic. During these visits, the study team would monitor your health, ask about your symptoms, and perform some tests. You would be asked to record information about your daily sunlight exposure and any skin symptoms you experience.

After the initial 16-week treatment period, there would likely be follow-up appointments to continue checking on your health and how the treatment has affected you in the longer term. The total duration of your participation, including follow-up, would be fully explained by the study team.

Potential risks and benefits

Taking part in a clinical trial offers the potential benefit of accessing a new treatment before it's widely available, which might improve your symptoms or quality of life if the medicine works. However, there are also potential risks; the new medicine might cause side effects, which could be mild or more serious, or it might not work for you. Since you could receive a dummy pill, there's a chance you might not get the active treatment. You have the right to leave the study at any time, for any reason, without it affecting your usual medical care.

Locations (7)

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

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Unverified
Spain
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Unverified
Bulgaria
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Unverified
Netherlands
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Unverified
France
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Unverified
Czechia
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Unverified
Poland
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Unverified
Italy

Common questions

What is MT-7117?

MT-7117 is a new medicine being tested to help people with Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP) manage their sunlight sensitivity.

What does 'prodromal symptom' mean?

These are early warning signs you feel just before a full reaction. In this study, it refers to burning, tingling, itching, or stinging sensations on your skin from sun exposure.

Will I know if I'm getting the real medicine or a dummy pill?

No, this is a 'double-blind' study, meaning neither you nor your study doctor will know whether you are receiving MT-7117 or the placebo (dummy pill).

How long will the treatment part of the study last?

The main treatment period where you take the study medication will last for 16 weeks (about 4 months).

What is 'Phase 3' of a clinical trial?

Phase 3 is an important stage where a medicine is tested on a larger group of people to confirm how well it works, its safety, and compare it to existing treatments or a placebo before it can be approved for wider use.