The Prevention of Systemic Ectopic Mineralization in Pseudoxanthoma Elasticum
ConditionPseudoxanthoma Elasticum
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age18-50
SponsorUMC Utrecht
About this trial
The goal of this randomized clinical trial is to assess the effect of etidronate on ectopic calcification in relatively young patients with Pseudoxanthoma elasticum. The main question it aims to answer are:
What is the difference in the arterial calcification scores in the legs and the carotid syphon measured on low-dose CT scan after 24 months of treatment compared to baseline between etidronate and placebo.
Participants will be asked to do take etidronate or placebo for 24 months.
Eligibility criteria
Qualifiers
1. Be between 18 years and 50 years.
Skin
Yellowish papules and/or plaques on the lateral side of the neck and/or flexural areas of the body or
Increase of morphologically altered elastin with fragmentation, clumping and calcification of elastic fibers in a skin biopsy taken.
Disqualifiers
Patients that are unable or unwilling to sign for informed consent.
Pregnant, lactating, or fertile women who might wish to become pregnant within three years.**
Patients with an estimated glomerular filtration rate below 30 ml/min/1.73m2 according to the CKD-EPI equation.31
Patients with a known abnormality of the oesophagus that would interfere with passage of the drug (e.g. oesophagus stenosis).
Trial design
Treatments tested in this trial
- Etidronate
- Placebo
Treatment groups
76 Participants
are divided into 2 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov