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Study on the Safety and Tolerability of Oral Dersimelagon for Patients 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 (EPP) and X-Linked Protoporphyria (XLP). These are genetic disorders that cause sensitivity to sunlight, leading to painful skin reactions. The study is testing a medication called Dersimelagon, also known by its code name MT-7117. This medication is taken orally in the form of a tablet.

The purpose of the study is to evaluate the long-term safety and tolerability of Dersimelagon in individuals with EPP or XLP. Participants in the study will take the medication over an extended period, and their health will be monitored to ensure the treatment is safe and well-tolerated. The study will involve regular check-ups, including physical exams and laboratory tests, to track any changes in health and to identify any potential side effects.

Throughout the study, participants will be closely observed for any treatment-emergent adverse events, which are any new or worsening health issues that occur during the study. This includes serious adverse events and any other health concerns that may arise. The study aims to provide valuable information on the long-term use of Dersimelagon for managing EPP and XLP, contributing to better understanding and treatment of these conditions.

1 enrollment

Upon joining the study, eligibility is confirmed based on specific criteria. This includes providing written consent and, for adolescents, both assent and parental consent.

Eligibility also requires completion of a previous study phase, a minimum body weight, and the ability to travel to study sites for scheduled visits.

Female participants must have a negative pregnancy test and agree to use effective contraception methods.

2 medication administration

The study involves taking an oral medication called dersimelagon, also known as MT-7117 Formulation Code C, in the form of a tablet.

The medication is administered orally, but specific dosage and frequency details are not provided in the available information.

3 monitoring and assessments

Throughout the study, various health assessments are conducted to monitor safety and tolerability.

These assessments include checking for any adverse events, conducting physical examinations, and measuring vital signs such as blood pressure and body temperature.

Laboratory tests are performed to evaluate blood and urine samples, including liver function tests.

An electrocardiogram (ECG) is used to monitor heart activity, and any changes in skin moles are assessed by a dermatologist.

4 study duration

The study is designed as a long-term extension, with an estimated end date in June 2025.

Participants are expected to remain in the study for its entire duration, attending all scheduled visits and assessments.

Who Can Join the Study?

  • Subjects must provide written informed consent to participate. For teenagers, both their agreement and parental consent are required.
  • Subjects must have completed a previous study called MT-7117-G01 through Week 58.
  • Subjects must have a body weight of at least a certain amount (please refer to the study protocol for full details).
  • Subjects must be willing and able to travel to the study sites for all scheduled visits.
  • In the investigator’s opinion, subjects must be able to understand the nature of the study and any risks involved, and be willing to cooperate and follow the study’s rules and requirements, including travel.
  • Female subjects must not be breastfeeding and must have a negative urine pregnancy test at the start of the study before receiving the first dose of the study drug.
  • Female subjects who can have children and male subjects with partners who can have children must agree to use two effective methods of birth control, including a barrier method. For female subjects, one method must be highly effective.

Who Cannot Join the Study?

  • Patients who do not have Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP) cannot participate. These are specific conditions related to how the body processes certain substances, leading to skin sensitivity to sunlight.
  • Patients who are not within the specified age ranges cannot participate. The study includes certain age groups, so if you are outside these, you may not be eligible.
  • Patients who are not part of the specified clinical trial groups cannot participate. This means the study is looking for people with specific characteristics or conditions.
  • Both male and female patients are eligible, so gender is not a reason for exclusion.
  • Patients who are considered part of a vulnerable population may not be eligible. This term refers to groups of people who might need special protection or consideration in research studies.

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 Clinic De Barcelona Barcelona Spain
Charite Universitaetsmedizin Berlin KöR Berlin Germany
Karolinska University Hospital Solna Sweden
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
Iveatp Iapqwlvv Fxbdgcxpnomrk Ottqgzmvzdo Rome Italy
Inabryqkw Fga Cvomptby Ait Ereiricypptk Mzbokjav Prague Czechia
Htkea Btshnb Hd Bergen Norway
Evejppv Uzbqddarwxte Maempha Chzuifs Ryxovrter (vvuwiez Myk 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
13.04.2022
Czechia Czechia
Not recruiting
13.04.2022
France France
Not recruiting
13.04.2022
Germany Germany
Not recruiting
13.04.2022
Italy Italy
Not recruiting
13.04.2022
Norway Norway
Not recruiting
13.04.2022
Poland Poland
Not recruiting
13.04.2022
Spain Spain
Not recruiting
13.04.2022
Sweden Sweden
Not recruiting
13.04.2022
The Netherlands The Netherlands
Not recruiting
13.04.2022

Trial locations

Investigated drugs:

Dersimelagon (MT-7117) is an oral medication being studied for its safety and tolerability in people with Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP). These are conditions that cause sensitivity to sunlight, leading to pain and skin damage. The trial aims to understand how well patients can tolerate this medication over a long period and to ensure it is safe for use.

Erythropoietic Protoporphyria – This is a rare genetic disorder that affects the production of heme, a component of hemoglobin. It leads to a buildup of protoporphyrin in the blood, which can cause sensitivity to sunlight. Individuals with this condition often experience painful skin reactions after exposure to sunlight, including burning, itching, and swelling. Over time, repeated sun exposure can lead to changes in skin texture and appearance. The condition is usually diagnosed in childhood and can significantly impact daily activities due to the need to avoid sunlight.

X-Linked Protoporphyria – This is a genetic disorder similar to erythropoietic protoporphyria, but it is linked to the X chromosome. It also results in the accumulation of protoporphyrin in the blood, causing sensitivity to sunlight. Affected individuals may experience severe pain, redness, and swelling of the skin after sun exposure. The condition can lead to chronic skin changes and scarring over time. It is typically identified in early childhood and requires careful management to avoid sun exposure. The disorder is more common in males due to its X-linked inheritance pattern.

Trial ID:
2024-514466-38-00
Protocol code:
MT-7117-A-301
NCT ID:
NCT05005975
Trial Phase:
Therapeutic confirmatory (Phase III)

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