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Study on Preventing Arterial Calcification in Young Patients with Pseudoxanthoma Elasticum Using Etidronate

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a rare genetic condition called Pseudoxanthoma Elasticum (PXE). PXE affects the skin, eyes, and blood vessels, leading to changes in the body's connective tissues. The trial will investigate the effects of a treatment using Etidronate Disodium, which is a type of medication known as a bisphosphonate. This medication is being tested to see if it can prevent or slow down the buildup of calcium in the arteries, which is a common problem in people with PXE.

The purpose of the study is to determine if taking Etidronate Disodium for 24 months can stop the progression of calcium deposits in the arteries of the legs and neck. Participants in the study will be randomly assigned to receive either the Etidronate Disodium capsules or a placebo, which looks like the medication but does not contain the active ingredient. The study is designed to be double-blind, meaning neither the participants nor the researchers will know who is receiving the actual medication or the placebo during the trial.

Throughout the study, various tests will be conducted to monitor changes in the participants' arteries and overall health. These tests include imaging techniques like computed tomography (CT) scans and ultrasound to measure calcium buildup and artery thickness. The study will also assess the impact of the treatment on eye health, skin condition, and overall quality of life. The trial aims to provide valuable insights into whether Etidronate Disodium can be an effective treatment for managing PXE and its associated complications.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial visit

    Upon joining the study, an initial visit is scheduled. During this visit, eligibility is confirmed based on specific criteria, such as age and a definitive diagnosis of pseudoxanthoma elasticum.

    A comprehensive health assessment is conducted, including a review of medical history and a physical examination. This ensures that participation in the trial is safe and appropriate.

  2. Step 2

    Medication administration

    Participants are randomly assigned to receive either the study medication, etidronate disodium, or a placebo. The medication is provided in the form of oral capsules, with a dosage of 400 mg.

    The capsules are taken orally as directed, with the frequency and duration specified by the study protocol. The treatment period lasts for 24 months.

  3. Step 3

    Regular follow-up visits

    Regular follow-up visits are scheduled throughout the 24-month treatment period. These visits are essential for monitoring health and assessing the effects of the medication.

    During these visits, various tests and assessments are conducted, including imaging studies and blood tests, to evaluate changes in arterial calcification and other health parameters.

  4. Step 4

    Final assessment

    At the end of the 24-month treatment period, a final assessment is conducted. This includes a comprehensive evaluation of health and any changes in the condition being studied.

    The final assessment helps determine the effectiveness of the treatment in halting the progression of arterial calcification and other related health outcomes.

Who can join the trial?

9 criteria

  • Be between 18 years and 55 years old.
  • Have a definitive diagnosis of Pseudoxanthoma Elasticum (PXE). This means meeting at least two criteria from different categories: skin, eye, or genetics.
  • For the skin category:
    • Have yellowish bumps or plaques on the side of the neck or areas where the skin folds.
    • Have a skin biopsy showing changes in elastic fibers, such as fragmentation, clumping, and calcification.
    • For the eye category:
      • Have a "peau d'orange" appearance of the retina, which looks like the surface of an orange.
      • Have one or more angioid streaks, which are lines in the retina, each at least as long as the diameter of the optic disk.
      • For the genetics category:
        • Have a harmful mutation in both copies of the ABCC6 gene.
        • Have a first-degree relative (parent, sibling, or child) who independently meets the diagnostic criteria for PXE.
        • Women who can become pregnant must use effective birth control.

Who cannot join the trial?

3 criteria

  • Patients who do not have the condition called Pseudoxanthoma Elasticum cannot participate.
  • Patients who are not within the specified age range cannot participate. The age range is not specified here, but it is important to check if you qualify.
  • Patients who are part of a vulnerable population cannot participate. This usually means groups that need special protection, like children or people with certain disabilities.
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Investigated drugs

Etidronate is a medication used in this clinical trial to see if it can stop or slow down the buildup of calcium in the blood vessels of young patients with a condition called pseudoxanthoma elasticum. This condition can cause calcium to deposit in places it shouldn't, like in the arteries, which can lead to health problems. The trial aims to find out if taking etidronate for 24 months can prevent this unwanted calcium buildup, particularly in the legs and certain parts of the neck. By doing so, it hopes to improve the health and quality of life for people with this condition.

What is already known about the treatment

Etidronate – Etidronate is administered orally in the form of tablets and is currently being studied for its potential to prevent abnormal mineral deposits in the body, specifically in a condition called pseudoxanthoma elasticum. This medication is being evaluated in clinical trials to see if it can stop the progression of calcium buildup in certain arteries over a 24-month period. Etidronate works by interfering with the process that leads to the hardening of tissues due to calcium deposits, which is its main therapeutic use. It belongs to a class of drugs known as bisphosphonates, which are typically used to manage bone disorders.

Investigated diseases

Pseudoxanthoma Elasticum – Pseudoxanthoma Elasticum is a genetic disorder that affects connective tissue, primarily impacting the skin, eyes, and cardiovascular system. It is characterized by the progressive calcification and fragmentation of elastic fibers in the body. In the skin, this leads to the development of yellowish papules and plaques, often in areas of flexure such as the neck and armpits. In the eyes, it can cause changes in the retina, leading to vision problems. The cardiovascular system may experience arterial calcification, which can affect blood flow and lead to complications. The progression of the disease varies among individuals, with symptoms typically worsening over time.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-512133-33-00Protocol code24U-1056Estimated enrolment76 patientsSponsorUniversitair Medisch Centrum Utrecht

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On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

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