Actively Recruiting

Phase Not Applicable
Age: 18Years - 65Years
All Genders
ID04868578

Efficacy of PyROphosPHate Supplementation to Fight ECtopIc Calcification in PseudoXanthoma Elasticum - PROPHECI-PPI Study

Led by Centre Hospitalier Universitaire de Nice · Updated on 2024-05-17

99

Participants Needed

2

Research Sites

37 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

This research investigates Pseudoxanthoma Elasticum (PXE), a rare inherited metabolic disorder causing progressive calcification in connective tissues that affects the skin, eyes, blood vessels, and kidneys. PXE results from a gene loss-of-function that lowers plasma levels of inorganic pyrophosphate (PPi), an important anti-calcifying molecule. The trial aims to evaluate the safety and effectiveness of daily oral PPi salt supplements compared to placebo in patients with PXE. Participants in this study will be randomly assigned to receive either PPi supplement capsules or placebo capsules once daily for 12 months. This double-blind trial compares these two groups to assess whether PPi supplementation can impact disease progression. The study is led by the Centre Hospitalier Universitaire de Nice and includes careful monitoring and follow-up during the treatment period. Throughout the trial, participants will undergo clinical evaluations focusing on arterial calcification scores after 12 months, along with assessments of skin and eye changes and vascular health. The research team will monitor safety, symptoms, and treatment adherence during regular visits. The total participation lasts for the 12-month treatment period, and progress will be measured using detailed clinical observations and imaging.

CONDITIONS

Brief Title

PPI Supplementation to Fight ECtopIc Calcification in PXE

Who Can Participate

Age: 18Years - 65Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Patients aged over 18 years and under 65 years, both men and women, with clinically and biologically confirmed PXE (genotype and phenotype) by international criteria
  • Women of childbearing age must use effective contraception during the study
  • Patient must be affiliated with social security insurance
  • Signed informed consent is required
  • Patient must have no acute complications related or unrelated to PXE at the time of study
Not Eligible

You will not qualify if you...

  • Renal insufficiency defined by renal clearance less than 30 ml/min/1.73 m²
  • Patients with osteomalacia
  • Patients with chronic diarrhea lasting more than 1 month
  • Pregnancy, lactating, or women planning pregnancy within three years
  • Any other medical condition deemed exclusionary by the Principal Investigator
  • Use of bisphosphonate within the last 5 years
  • Hypocalcemia (calcium <2.20 mmol/L and ionized calcium <1.15 mmol/L)
  • Vitamin D deficiency less than 35 nmol/L
  • Enrollment in another interventional clinical trial that could interfere
  • Patients over 18 years protected by law
  • After correcting hypocalcemia and/or vitamin D deficiency, patients may be re-evaluated for eligibility

AI-Screening

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

1 visit (in-person)

Treatment

Duration - 12 months

Participants take either PPI or a placebo daily for 12 months as part of the study treatment.

Regular visits for assessments during the 12 months

Trial Site Locations

Total: 2 locations

1

Angers Hospital

Angers, France, 49000

Actively Recruiting

2

Nice Hospital

Nice, France, 06000

Actively Recruiting

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Research Team

G

Georges LEFTHERIOTIS, PUPH

S

Sophie Bonnet

How is the study designed?

Study Type

INTERVENTIONAL

Masking

DOUBLE

Allocation

RANDOMIZED

Model

PARALLEL

Primary Purpose

TREATMENT

Number of Arms

2

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Published Research Related To This Trial

The PROPHECI trial: a phase II, double-blind, placebo-controlled, randomized clinical trial for the treatment of pseudoxanthoma elasticum with oral pyrophosphate.

Laetitia Clotaire, Isabelle Rubera, Christophe Duranton...

https://pubmed.ncbi.nlm.nih.gov/39881395