Badanie wpływu etidronianu disodowego na postęp kalcyfikacji tętnic u młodych pacjentów z pseudoxanthoma elasticum

Pseudoxanthoma Elasticum (PXE) is a rare condition that affects the skin, eyes, and blood vessels. It can lead to changes in the skin, vision problems, and hardening of the arteries. This study is looking at whether a medication called etidronate disodium, taken as an oral capsule, can help prevent the buildup of minerals in the arteries of people with PXE. The study will compare the effects of etidronate disodium to a placebo, which is a substance with no active medication.

The main goal of the study is to see if taking etidronate disodium for 24 months can stop the progression of mineral buildup in the arteries of the legs and neck. Participants will be randomly assigned to receive either the medication or a placebo, and neither the participants nor the researchers will know who is receiving which treatment. This is known as a double-blind study. The study will use various tests, such as low-dose CT scans and other imaging techniques, to measure changes in the arteries and other areas affected by PXE.

Throughout the study, researchers will also look at other effects of the treatment, such as changes in vision, skin condition, and overall quality of life. The study aims to provide more information on whether etidronate disodium can be an effective treatment for people with PXE, potentially improving their health and well-being. The study is expected to continue until the end of 2026.