Using PPI to Treat Pseudoxanthoma Elasticum

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This clinical trial investigates the use of pyrophosphate supplementation to address ectopic calcification in patients with Pseudoxanthoma Elasticum (PXE), a rare genetic disorder. The study aims to evaluate the efficacy of this treatment compared to a placebo in individuals diagnosed with PXE. Participants will be monitored for changes in calcification and overall health outcomes. The trial includes adults aged 18 to 65 who meet specific diagnostic criteria for PXE.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Patients (\>18 years and \<65 years), men and women, presenting a clinically and biologically authenticated PXE (genotype + phenotype) according to the international diagnostic criteria (26).
* Efficient contraceptive method in woman of childbearing age at inclusion and during the overall study
* Patient affiliated to a social security insurance
* Signed informed consent
* Patient without acute complication linked or not to the pathology at the time of the study

NON-INCLUSION CRITERIA

* Renal insufficiency (i.e. defined by a renal clearance \<30ml / min / 1.73 m²)
* Patients with osteomalacia
* Patients with chronic diarrhea (\> 1 month)
* Pregnancy, lactating or fertile women who may wish to become pregnant within three years.
* Any other medical condition that may be considered in the opinion of the Principal Investigator.
* Use of bisphosphonate during last 5 years.
* Hypocalcemia (calcium \<2.20 mmol/L and ionized calcium \<1.15 mmol/L) \*.
* Vitamin D deficiency \<35 nmol/L \*
* Enrollment in another inteventional clinical trial which could interfere with the present study
* patients (\>18 years) protected by law

  * After correcting the hypocalcemia and/or vitamin D deficiency, a participant is again suitable for participation in the trial, as long as the participant meets the inclusion criteria.

Where it's running

• Angers Hospital
  Angers, France Recruiting
• Nice Hospital
  Nice, France Recruiting

Study contacts

• Study coordinator: Georges LEFTHERIOTIS, PUPH
• Email: leftheriotis.g@chu-nice.fr
• Phone: 33 4 92 03 29 41

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Purinergic compounds for people with pseudoxanthoma elasticum

  This trial will try purinergic compounds to see if restoring pyrophosphate-related signals can reduce tissue calcification in adults with genetically confirmed pseudoxanthoma elasticum.

  Recruiting Centre Hospitalier Universitaire de Nice · Angers and 1 other locations · NCT07323082

• Tracking PXE-related changes at the back of the eye over time

  This study will see if repeated high-resolution eye imaging can track changes in Bruch's membrane, choriocapillaris blood flow, and retinal atrophy in people with PXE.

  Recruiting University Hospital, Bonn · Bonn, North Rhine-Westphalia · NCT07048106

• Choriocapillaris blood flow differences in people with pseudoxanthoma elasticum versus healthy adults

  We will test whether a noninvasive eye scan (OCT angiography) shows reduced choriocapillaris blood flow in adults with pseudoxanthoma elasticum compared with matched healthy adults.

  Recruiting University Hospital, Angers · Angers · NCT07006649

• Optimizing recruitment and follow-up for people with pseudoxanthoma elasticum

  This project will try new ways of recruiting and following people with PXE treated at the national reference center to see if care and monitoring improve.

  Recruiting University Hospital, Angers · Angers · NCT06636344

• Preventing calcification in patients with Pseudoxanthoma Elasticum

  This study is testing if a medication called etidronate can help young adults with Pseudoxanthoma Elasticum reduce harmful calcification in their arteries over two years.

  Recruiting Phase 3 UMC Utrecht · Utrecht · NCT05832580

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.