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Study on the Effects of Dersimelagon Phosphate for Adults and Adolescents with Erythropoietic Protoporphyria or X-Linked Protoporphyria

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What is this study about?

This clinical trial is focused on studying two rare conditions: Erythropoietic Protoporphyria and X-Linked Protoporphyria. These are conditions that cause sensitivity to sunlight, leading to symptoms like burning, tingling, itching, or stinging when exposed to sunlight. The study is testing a new treatment called MT-7117, which is taken as a tablet. The purpose of the study is to see how well MT-7117 works in delaying the start and reducing the severity of these symptoms when patients are exposed to sunlight.

Participants in the study will be randomly assigned to receive either the MT-7117 tablet or a placebo, which looks like the MT-7117 tablet but does not contain the active medication. The study will last for a period of 16 weeks, during which participants will take the tablets and report their experiences with sunlight exposure and any symptoms they feel. The study aims to understand if MT-7117 can help people with these conditions spend more time in the sun without experiencing discomfort.

Throughout the study, participants will be asked to keep track of their sunlight exposure and any symptoms they experience. They will also provide feedback on their overall impression of any changes they notice. The study will help determine if MT-7117 is a safe and effective treatment option for people with Erythropoietic Protoporphyria or X-Linked Protoporphyria, potentially improving their quality of life by allowing them to enjoy more time outdoors.

1 joining the study

Upon joining the study, participants are required to provide written informed consent. For minors, both the minor’s assent and legal representative’s consent are necessary.

Participants must have a confirmed diagnosis of erythropoietic protoporphyria (EPP) or X-linked protoporphyria (XLP) and be between 12 and 75 years old.

2 baseline visit

During the baseline visit, female participants must undergo a urine pregnancy test to ensure a negative result before receiving the first dose of the study drug.

Participants are expected to demonstrate an understanding of the study’s nature and any associated risks, and agree to comply with protocol requirements, including travel and sunlight exposure.

3 treatment phase

Participants will be randomly assigned to receive either the study drug, MT-7117 (Formulation Code C), or a placebo. The medication is administered orally in tablet form.

The treatment period lasts for 16 weeks, during which participants will take the medication as directed by the study protocol.

4 monitoring and assessments

Throughout the 16-week treatment period, participants will be monitored for changes in sunlight exposure time to the first prodromal symptom, such as burning, tingling, itching, or stinging.

Participants will also report any sunlight-induced pain events and non-prodrome phototoxic reactions.

5 end of treatment evaluation

At the end of the 16-week period, participants will undergo an evaluation to assess the primary endpoint, which is the change from baseline in average daily sunlight exposure time to the first prodromal symptom.

Secondary endpoints include the Patient Global Impression of Change (PGIC) and the total number of sunlight-induced pain events and phototoxic reactions.

Who Can Join the Study?

  • Participants must provide written consent to join the study. If the participant is a minor, both the minor’s agreement and a legal representative’s consent are needed.
  • Both males and females with a confirmed diagnosis of Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP) can participate. These are specific medical conditions related to sensitivity to sunlight.
  • Participants should be between 12 and 75 years old at the time of screening.
  • Participants must be willing and able to travel to the study locations for all scheduled visits.
  • The study investigator must believe that the participant can understand the study’s nature and any risks involved, and is willing to follow the study’s rules and requirements, including traveling and being exposed to direct sunlight as much as possible.
  • Female participants must not be breastfeeding and must have a negative urine pregnancy test before receiving the first dose of the study medication.
  • Female participants who can have children and male participants with partners who can have children must currently use or be willing to use two effective methods of birth control.

Who Cannot Join the Study?

  • Individuals who do not have a diagnosis of Erythropoietic Protoporphyria or X-Linked Protoporphyria. These are specific conditions related to sensitivity to sunlight.
  • Individuals who are not within the specified age ranges for the study. The study is open to certain age groups, so being outside these ranges would exclude participation.
  • Individuals who are not able to follow the study procedures or comply with the study requirements.
  • Individuals who have other medical conditions that might interfere with the study or pose a risk to their health if they participate.
  • Individuals who are currently participating in another clinical trial or have recently participated in one, as this could affect the study results.
  • Individuals who are pregnant or breastfeeding, as the study may not be safe for them or their baby.
  • Individuals who have a history of allergic reactions to the study medication or similar medications.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Centre Hospitalier Universitaire De Bordeaux Bordeaux France
Azienda Ospedaliero Universitaria Di Sassari Sassari Italy

Other Sites

Site Name City Country Status
Azienda Ospedaliero Universitaria Di Modena Modena Italy
Hospital Universitario 12 De Octubre Madrid Spain
Instytut Hematologii I Transfuzjologii Warsaw Poland
Istituto Di Ricovero E Cura A Carattere Scientifico Materno Infantile Burlo Garofolo Trieste Italy
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico Milan Italy
Ente Ospedaliero Ospedali Galliera Di Genova Genoa Italy
Hospital General Universitario De Valencia Valencia Spain
Centre Hospitalier Universitaire De Nantes Nantes France
University Multiprofile Hospital For Active Treatment St. Ivan Rilski EAD Sofia Bulgaria
Hopital Beaujon Clichy France
Universita Degli Studi Di Brescia Brescia Italy
Iahabp Ixdoqkzj Fcwscgehsldib Ooheuvcyahm Rome Italy
Iuzwpiukq Fgf Cifyybzi Asg Ehvbwdcfktkw Mozlswye Prague Czechia
Ebdwhho Uehgztmzdyuy Mipquhr Ciphgbq Rfvzkpmvn (zcrosfc Mco Rotterdam The Netherlands

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Bulgaria Bulgaria
Not recruiting
29.03.2024
Czechia Czechia
Not recruiting
29.03.2024
France France
Not recruiting
29.03.2024
Italy Italy
Not recruiting
29.03.2024
Poland Poland
Not recruiting
29.03.2024
Spain Spain
Not recruiting
29.03.2024
The Netherlands The Netherlands
Not recruiting
29.03.2024

Trial locations

Investigated drugs:

MT-7117 is a medication being studied for its ability to help people with Erythropoietic Protoporphyria (EPP) or X-linked Protoporphyria (XLP). These are conditions that cause painful skin reactions when exposed to sunlight. The study is looking at how well MT-7117 can delay the start and reduce the severity of symptoms like burning, tingling, itching, or stinging that occur when patients are exposed to sunlight.

Erythropoietic Protoporphyria – This is a rare genetic disorder that affects the body’s ability to produce heme, a component of hemoglobin. It is characterized by a deficiency in the enzyme ferrochelatase, leading to the accumulation of protoporphyrin in the blood. Individuals with this condition experience severe pain and skin reactions when exposed to sunlight, often described as burning, tingling, itching, or stinging. These symptoms can occur within minutes of sun exposure and may persist for hours. Over time, repeated sun exposure can lead to chronic skin changes and scarring. The condition is typically diagnosed in childhood or early adulthood.

X-Linked Protoporphyria – This is a genetic disorder similar to erythropoietic protoporphyria, but it is linked to mutations on the X chromosome. It also results in the accumulation of protoporphyrin in the blood, causing sensitivity to sunlight. Affected individuals experience painful skin reactions, such as burning, tingling, itching, or stinging, shortly after sun exposure. These symptoms can be severe and may lead to avoidance of sunlight. The disorder is more common in males due to its X-linked inheritance pattern. Over time, chronic exposure can lead to skin damage and scarring.

Trial ID:
2023-506735-15-00
Protocol code:
MT-7117-A-302
Trial Phase:
Therapeutic confirmatory (Phase III)

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