Health TrialsbyHealthPanel

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

This trial does not accept healthy volunteers

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

Design model

Parallel

Treatments tested in this trial

  • Etidronate

    Drug

    Etidronate in capsules of 200 mg or 400 mg.

  • Placebo

    Drug

    Placebo in similar capsules

Treatment groups

76 Participants
are divided into 2 treatment groups
Group A: EtidronateExperimental treatment 1 intervention
Group B: PlaceboPlacebo comparator 1 intervention

Trial outcomes

Primary outcomes

1

Arterial calcification

The difference in arterial calcification volume measured (uL) on CT-scans between the etidronate and placebo group 24 months.

Time frame
24 months.

Secondary outcomes

1

Ophtalmology: Visual acuity

Difference in visual acuity at phtalmologic exam.

Time frame
24 months.
2

Ophtalmology: Contrast sensitivity.

Difference in contrast sensitivity at ophtalmologic exam.

Time frame
24 months.
3

Ophtalmology: Reflectivity of Bruch's membrane

Difference in normalized reflectivity in Bruch's membrane as measured with SD-optical coherence tomography, between the etidronate and placebo group after 24 months.

Time frame
24 months.
4

Ophtalmology: Length of angioid streaks.

Length of angioid streaks in the fundus, between the etidronate and placebogroup after 24 months.

Time frame
24 months.

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

UMC Utrecht

Lead sponsor

Source ClinicalTrials.gov
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