PPI Supplementation to Fight ECtopIc Calcification in PXE

Launched by CENTRE HOSPITALIER UNIVERSITAIRE DE NICE · Apr 27, 2021

Keywords

ClinConnect Summary

This clinical trial is studying a new potential treatment for a rare condition called Pseudoxanthoma Elasticum (PXE), which causes abnormal calcification in the body’s connective tissues. People with PXE may experience skin changes, vision problems, and issues with blood circulation and kidney health. Researchers want to see if taking a specific supplement called inorganic pyrophosphate (PPi) can help increase its levels in the blood and reduce the effects of PXE. The trial will compare the effects of PPi with a placebo (a non-active treatment) to determine its safety and effectiveness.

To participate in this study, individuals need to be between 18 and 64 years old and have a confirmed diagnosis of PXE. Women of childbearing age must be using effective birth control during the study. Participants will be monitored closely, and they will take the PPi supplement or placebo daily for a set period. It’s important to note that certain health conditions, like kidney problems or vitamin deficiencies, may prevent someone from joining the trial. Overall, this study aims to explore new treatment options for those living with PXE, offering hope for improved management of their condition.

Gender

ALL

Eligibility 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.