Porphyria non-acute – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Fri, 12 Jun 2026 11:22:34 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Porphyria non-acute – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 Long-term study of bitopertin safety and effectiveness in patients with erythropoietic protoporphyria or X-linked protoporphyria https://clinicaltrials.eu/trial/long-term-study-of-bitopertin-safety-and-effectiveness-in-patients-with-erythropoietic-protoporphyria-or-x-linked-protoporphyria/ Thu, 30 Apr 2026 13:08:39 +0000 https://clinicaltrials.eu/trial/long-term-study-of-bitopertin-safety-and-effectiveness-in-patients-with-erythropoietic-protoporphyria-or-x-linked-protoporphyria/ This study involves two rare blood conditions called Erythropoietic Protoporphyria and X-Linked Protoporphyria. These conditions cause a buildup of a substance in the blood that makes the skin extremely sensitive to sunlight, leading to pain and discomfort when exposed to light. People with these conditions often experience burning pain within minutes of sun exposure, which can severely limit their daily activities and quality of life. The treatment being tested in this study is called Bitopertin, which is also known by its code name DISC-1459. This medication comes as a film-coated tablet that is taken by mouth.

The purpose of this study is to examine the long-term safety and tolerability of bitopertin in people with these light-sensitive conditions. The study will look at how well the medication is tolerated over an extended period and whether it causes any unwanted effects. Additionally, the study will measure how the treatment affects the ability to spend time in daylight without experiencing pain and will track changes in the levels of the substance in the blood that causes the light sensitivity.

This is a long-term study that will follow participants who have already taken part in a previous study of bitopertin. Participants will continue taking the medication for up to 52 weeks, with the maximum daily dose being 60 milligrams. During the study, regular check-ups will be conducted to monitor overall health, including physical examinations and blood tests. The study will also track how much time participants can spend in sunlight without pain and how quickly symptoms appear after sun exposure, as well as measure the concentration of the medication in the blood to understand how the body processes it.

]]> Study of bitopertin to improve sunlight tolerance in adults with erythropoietic protoporphyria or X-linked protoporphyria https://clinicaltrials.eu/trial/study-of-bitopertin-to-improve-sunlight-tolerance-in-adults-with-erythropoietic-protoporphyria-or-x-linked-protoporphyria/ Wed, 29 Apr 2026 15:07:12 +0000 https://clinicaltrials.eu/trial/study-of-bitopertin-to-improve-sunlight-tolerance-in-adults-with-erythropoietic-protoporphyria-or-x-linked-protoporphyria/ This study focuses on two rare genetic conditions: Erythropoietic Protoporphyria (EPP) and X-Linked Protoporphyria (XLP). These disorders cause extreme sensitivity to sunlight, where exposure can lead to painful skin reactions. The study will test a medication called Bitopertin, given as a film-coated tablet that patients take by mouth.

The purpose of this research is to determine if Bitopertin can help patients spend more time in sunlight without experiencing pain and reduce certain harmful substances in their blood. The study will compare Bitopertin against a placebo to evaluate how well it works and how safe it is for patients with these conditions.

During the six-month study, participants will take either Bitopertin or placebo tablets daily. They will keep track of their time spent in sunlight and any skin reactions they experience. Regular blood tests will be performed to measure changes in blood components related to their condition. The study will monitor how well patients tolerate the medication and any side effects that may occur.

]]> Study on the Safety and Tolerability of Oral Dersimelagon for Patients with Erythropoietic Protoporphyria or X-Linked Protoporphyria https://clinicaltrials.eu/trial/study-on-the-safety-and-tolerability-of-oral-dersimelagon-for-patients-with-erythropoietic-protoporphyria-or-x-linked-protoporphyria/ Wed, 29 Apr 2026 15:01:42 +0000 https://clinicaltrials.eu/trial/study-on-the-safety-and-tolerability-of-oral-dersimelagon-for-patients-with-erythropoietic-protoporphyria-or-x-linked-protoporphyria/ 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.

]]> Study of Afamelanotide for Patients with Erythropoietic Protoporphyria (EPP) https://clinicaltrials.eu/trial/study-of-afamelanotide-for-patients-with-erythropoietic-protoporphyria-epp/ Wed, 29 Apr 2026 14:56:52 +0000 https://clinicaltrials.eu/trial/study-of-afamelanotide-for-patients-with-erythropoietic-protoporphyria-epp/ This clinical trial focuses on a condition called Erythropoietic Protoporphyria (EPP), a rare genetic disorder that causes sensitivity to sunlight, leading to painful skin reactions. The study will use a treatment called afamelanotide, which is administered as an implant under the skin. Afamelanotide is a synthetic hormone designed to help reduce the skin’s sensitivity to light in people with EPP.

The purpose of the study is to understand how afamelanotide behaves in the body, specifically looking at how it is absorbed, distributed, and eliminated. This is known as pharmacokinetics. The study will involve both adolescents and adults with EPP to compare how the treatment works in these different age groups.

Participants in the study will receive the afamelanotide implant and will be monitored over a period of time to gather information on how the treatment affects their condition. The study aims to provide valuable insights into the effectiveness and safety of afamelanotide for managing symptoms of EPP.

]]> Study on the Effects of Dersimelagon Phosphate for Adults and Adolescents with Erythropoietic Protoporphyria or X-Linked Protoporphyria https://clinicaltrials.eu/trial/study-on-the-effects-of-dersimelagon-phosphate-for-adults-and-adolescents-with-erythropoietic-protoporphyria-or-x-linked-protoporphyria/ Wed, 29 Apr 2026 14:47:54 +0000 https://clinicaltrials.eu/trial/study-on-the-effects-of-dersimelagon-phosphate-for-adults-and-adolescents-with-erythropoietic-protoporphyria-or-x-linked-protoporphyria/ 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.

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