Treatment of Ectopic Mineralization in Pseudoxanthoma Elasticum.

Phase 2

Completed

• Conditions: Pseudoxanthoma Elasticum; 10007510; 10047060; 10047066

• Registration Number: NL-OMON41676
• Lead Sponsor: niversitair Medisch Centrum Utrecht

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 11 June 2024

Recruitment & Eligibility

• Status: Completed
• Sex: Not specified
• Target Recruitment: 74

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

1. Subjects who are unable or unwilling to sign an informed consent.
2. Severe renal impairment (estimated creatinine clearance/eGFR of <30 ml/min/1.73m2 calculated using CKD-EPI equation).
3. Known abnormality of the esophagus that would interfere with the passage of the drug, such as a oesophagus stenosis.
4. Patients with osteomalacy
5. Patients with chronic diarrhea (>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 <2,20 mmol/L and ionised calcium < 1.15)*.
11. Vitamin D deficiency <35 nmol/L*.
*After correcting the hypocalcemia or vitamin D deficiency a participant is again suitable for participation in the TEMP trial, as long as the participant meets the inclusion criteria.

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>The primary endpoint is the percentage change in 18F-NaF-uptake after 12 months<br /><br>treatment with etidronate 20 mg/kg compared with placebo. </p><br>

Secondary Outcome Measures

Name	Time	Method
<p>Secondary endpoints are the percentage change in 18F-NaF-uptake in other<br /><br>arteries than the femoral artery, ophthalmological changes , changes in<br /><br>vascular stiffness, changes in bone mineral density, dermatological changes,<br /><br>changes in quality of life and changes in serum calcium and phosphate, changes<br /><br>in MRI brain lesions, vascular brain flow, pulsatility and brain tissue<br /><br>perfusion, changes in cognitive function.</p><br>