Pseudoxanthoma Elasticum
Conditions
Interventions
Subjects will be randomized to either treatment with etidronate during one year
(cyclical 20 mg/kg for 2 weeks on and 10 weeks of) or placebo.
Sponsors
Universitair Medisch Centrum Utrecht
Eligibility
Age
18 Years to 99 Years
Inclusion criteria
Inclusion criteria: 1. All patients should be at least 18 years old. 2. A participant must meet the revised diagnostic criteria of Plomp et al. for the diagnosis of PXE 3. In all patients evidence of arterial calcification should be available.
Exclusion criteria
Exclusion criteria: 1. Subjects who are unable or unwilling to sign an informed consent. 2. Severe renal impairment (estimated creatinine clearance/eGFR of 1 month) 6. Known sensitivity to etidronate.;7. Pregnant, lactating or fertile women who might wish to become pregnant within three years. 8. Any other medical or social condition that, in the opinion of the Principal Investigator, might put the subject at risk of harm during the study or might adversely affect the interpretation of the study data. 9. Use of bisphosphonate during last 5 years. 10. Hypocalcemia (calcium
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary endpoint is the percentage change in 18F-NaF-uptake after 12 months treatment with etidronate 20 mg/kg compared with placebo. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary endpoints are the percentage change in 18F-NaF-uptake in other arteries than the femoral artery, ophthalmological changes , changes in vascular stiffness, changes in bone mineral density, dermatological changes, changes in quality of life and changes in serum calcium and phosphate, changes in MRI brain lesions, vascular brain flow, pulsatility and brain tissue perfusion, changes in cognitive function. | — |
Countries
Netherlands
Outcome results
None listed