Using PPI to Treat Pseudoxanthoma Elasticum
Efficacy of PyROphosPHate Supplementation to Fight ECtopIc Calcification in PseudoXanthoma Elasticum - PROPHECI-PPI Study
This study is testing if taking a supplement called pyrophosphate can help adults with Pseudoxanthoma Elasticum reduce harmful calcification in their bodies.
Quick facts
| Phase | Not applicable |
|---|---|
| Study type | Interventional |
| Enrollment | 99 (estimated) |
| Ages | 18 Years to 65 Years |
| Sex | All |
| Sponsor | Centre Hospitalier Universitaire de Nice Academic / other |
| Locations | 2 sites (Angers and 1 other locations) |
| Trial ID | NCT04868578 on ClinicalTrials.gov |
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.
Who should consider this trial
Good fit: Ideal candidates for this study are adults aged 18 to 65 with a confirmed diagnosis of Pseudoxanthoma Elasticum.
Not a fit: Patients with renal insufficiency, osteomalacia, or those who are pregnant or lactating may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly improve the quality of life for patients suffering from PXE by reducing ectopic calcifications.
How similar studies have performed: While the approach of using pyrophosphate supplementation is novel for PXE, similar strategies targeting ectopic calcification have shown promise in other conditions.
Eligibility criteria
Show full inclusion / exclusion criteria
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 this trial is running
Angers and 1 other locations
- 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
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.