Trial intelligence / Profile preview

Etidronate for Prevention of Ectopic Mineralization in PXE

NCT05832580Phase 3

Key facts

Status: Recruiting
Start: Apr 2023
Primary completion: Mar 2027
Countries: 1
Modality: Small Molecules

Drugs: etidronate
Conditions: pseudoxanthoma elasticum (PXE)

Overview

Phase 3, randomised, double-blind trial in pseudoxanthoma elasticum (PXE) comparing Etidronate with Placebo (2 arms). Primary endpoint: Arterial calcification scores in the legs and the carotid syphon at 24 months. Started Apr 2023; status: recruiting.

Design

2 arms

Study design: RandomizedDouble-blind

Etidronate
Etidronate 20mg/kg daily for 2 weeks every 12 weeks
Placebo
Matching placebo capsules

Who it enrols

Patients aged 18-50 with a definitive diagnosis of Pseudoxanthoma Elasticum (PXE) based on Plomp criteria (skin, eye, genetic).

Endpoints

Primary

Arterial calcification scores in the legs and the carotid syphon24 months

Secondary

Visual acuity24 months
Contrast sensitivity24 months
Reflectivity of Bruch's membrane24 months
Length of angioid streaks24 months
Area of angioid streaks24 months
Area of atrophy24 months
Intra cranial velocity pulsatility index24 months
Elastin degradation of the skin24 months

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