- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04868578
PPI Supplementation to Fight ECtopIc Calcification in PXE (PROPHECI-PPI)
Efficacy of PyROphosPHate Supplementation to Fight ECtopIc Calcification in PseudoXanthoma Elasticum - PROPHECI-PPI Study
Study Overview
Status
Conditions
Intervention / Treatment
Study Type
Enrollment (Anticipated)
Phase
- Not Applicable
Contacts and Locations
Study Contact
- Name: Georges LEFTHERIOTIS, PUPH
- Phone Number: 33 4 92 03 29 41
- Email: leftheriotis.g@chu-nice.fr
Study Contact Backup
- Name: Sophie Bonnet
- Email: bonnet.s@chu-nice.fr
Study Locations
-
-
-
Angers, France, 49000
- Recruiting
- Angers Hospital
-
Contact:
- ludovic Martin, PHD
- Phone Number: 33 2 41 35 34 19
- Email: LuMartin@chu-angers.fr
-
Principal Investigator:
- ludovic Martin
-
Sub-Investigator:
- Emmanuel Letavernier
-
Sub-Investigator:
- Samir Henni
-
Nice, France, 06000
- Recruiting
- Nice hospital
-
Contact:
- Georges leftheriotis
- Phone Number: 04 92 03 29 41
- Email: leftheriotis.g@chu-nice.Fr
-
Principal Investigator:
- georges Leftheriotis
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
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.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Double
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: PPI
the patient take PPI caps
|
patient take PPI every day during 12 months
|
Placebo Comparator: PPI placebo
The patient take PPI placebo
|
patient take PPI placebo every day during 12 months
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Arterial calcification score
Time Frame: 12 months
|
Using manufacturer dedicated software, the investigator, will determine the calcification score from CT images according to the validated Agatston score method.
A threshold of 130 Hounsfield Units for calcium detection will be used for the study.
Voxels above this threshold representing arterial wall calcifications will be manually identified and selected by the observer on the CT image.
Calcifications mass in the studied arterial segments will be analyzed and quantified.
The length and antero-posterior mean diameter of each arterial segment will be determined for further normalization of the calcification score to the arterial wall surface (data expressed as HU/mm2 of arterial surface).
The intraclass correlation and Bland-Altman coefficient for inter-observer reliability of the total peripheral artery calcium mass measurement will be determined by scoring 10 random scans by two independent investigators.
|
12 months
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
clinical observation of dermatological changes
Time Frame: 12 months
|
number of patient with dematological changes. Dermatological changes will be monitored by a dermatoscopic study of the skin according to the following protocol: Images will be acquired from commonly affected areas (neck, axilla, antecubital fossae, armpits and periumbilical area) by a contact dermatoscope (SD) with non-polarized light and recorded, as previously described . The investigators will ensure that the very same areas are imaged at baseline and at the end of the study. The surface of SD elementary changes featuring yellow papulae (presenting as "dots" or "reticular networks") on digitized images will be compared using an open image processing software (ImageJ, https://imagej.nih.gov/ij/index.html NIH). |
12 months
|
clinical observation of ophtalmologic changes
Time Frame: 12 Montths
|
Number of patietns with ophtalmologic changes.
These changes will be monitored (blinded to treatment) by the occurrence of subretinal neovascularization events, the frequency of anti-VEGF administration and the best-corrected visual acuity (BCVA).
A sub-retinal neovascularization event will be defined as any of the following that provide an indication to start or intensify anti-VEGF injections to prevent (further) visual impairment: 1) retinal bleeding suspected to be caused by sub-retinal neovascularization (if needed confirmed by fluorescein angiography); 2) a significant increase in sub retinal or intraretinal fluid; and/or 3) growth of a sub retinal neovascular complex.
These events will be scored by the trained ophthalmologist of the PXE reference center (Dr Th Bresson) who will be blinded for the treatment.
|
12 Montths
|
vascular changes
Time Frame: 12 months
|
Ankle Brachial Index (ABI) at rest: Systolic arterial blood pressure will be recorded using a pneumatic cuff wrapped around the ankle. An ultrasound probe allows recording the blood flow in the left and right ankle arteries (namely posterior and anterior tibial arteries) and brachial arteries supine at rest. The ABI is determined by the ratio between the lowest ankle artery pressure and the highest brachial artery blood pressure. A peripheral arterial occlusive disease (PAOD) is defined according to the actual recommendations: an ABI <0.90) and an incompressible arterial wall reflecting mediacalcosis defined by ABI>1.40. |
12 months
|
Collaborators and Investigators
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Anticipated)
Study Completion (Anticipated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- 19-APN-01
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Pseudoxanthoma Elasticum
-
Fundación Pública Andaluza para la Investigación...UnknownGrönblad-Stranberg Disease (Pseudoxanthoma Elasticum)Spain
-
Tianjin Eye HospitalCompletedElasticum, Incomplete PseudoxanthomaChina
-
University Hospital, AngersCompletedPseudoxanthoma ElasticumFrance
-
Mark LebwohlCompletedPseudoxanthoma ElasticumUnited States
-
University Hospital, AngersUnknown
-
Daiichi Sankyo, Inc.PXE InternationalActive, not recruitingPseudoxanthoma ElasticumUnited States, Netherlands
-
Daiichi Sankyo, Inc.Thomas Jefferson University; PXE InternationalCompletedPseudoxanthoma ElasticumUnited States
-
Tampere University HospitalUMC Utrecht; Hungarian Academy of SciencesWithdrawnPseudoxanthoma Elasticum
-
University Hospital, BonnUnknown
-
University Hospital, AngersCompletedPseudoxanthoma Elasticum | EmploymentFrance
Clinical Trials on study treatment PPI
-
University of Colorado, DenverCystic Fibrosis FoundationTerminatedCystic FibrosisUnited States
-
Dartmouth-Hitchcock Medical CenterWithdrawnGastro Esophageal Reflux Disease PPI Non-Responders
-
Presidio Pharmaceuticals, Inc.CompletedPharmacokinetic Assessments in Healthy VolunteersUnited States
-
Presidio Pharmaceuticals, Inc.CompletedPharmacokinetic Assessments in Healthy VolunteersUnited States
-
ShireCompletedGastroesophageal Reflux DiseaseUnited States, Czechia, Germany, Latvia, Poland, Romania
-
Kartal Kosuyolu Yuksek Ihtisas Education and Research...Unknown
-
University of Southern CaliforniaWithdrawn
-
Al-Azhar UniversityNot yet recruitingPenile Prosthesis Infection | Penile Prosthesis; Complications, Infection or Inflammation
-
PRAECIS Pharmaceuticals Inc.TerminatedSolid Tumors | Non-Hodgkin's Lymphoma,United States
-
Yonsei UniversityCompletedChronic Cough Caused by Gastroesophageal RefluxKorea, Republic of