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Response to Oral Lansoprazole of Inorganic Pyrophosphate Levels in Patients With Grönblad-Stranberg Disease (Pseudoxanthoma Elasticum) (FIMPXE2016-01)

Primary Purpose

Grönblad-Stranberg Disease (Pseudoxanthoma Elasticum)

Status
Unknown status
Phase
Phase 4
Locations
Spain
Study Type
Interventional
Intervention
Lansoprazole 30mg
Placebo
Sponsored by
Fundación Pública Andaluza para la Investigación de Málaga en Biomedicina y Salud
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Grönblad-Stranberg Disease (Pseudoxanthoma Elasticum) focused on measuring Grönblad-Stranberg Disease, Pseudoxathoma Elasticum

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. Patients ≥18 years old
  2. Patients diagnosed with PXE according to 2010 criteria by PLOMP et al.
  3. At least, patients meet two of the following criteria:

    1. Retinal lesions such as Orange peel and/or Angioid streaks
    2. Skin lesions such as papules or yellowish plaques on the lateral face of the neck and / or flexures of the body (armpits, elbows, knees) or alterations in the skin biopsy with fragmentation and / or conglomerates of and / or calcification of the elastic fibers.
    3. A pathogenic mutation of the two alleles of the ABCC6 gene.

Exclusion Criteria:

  1. Refusal of informed consent.
  2. Vegetarian diet or extreme diets.
  3. Pregnancy or its intention during the months of the study.
  4. Age <18 years old
  5. Known hypersensitivity to "prazoles" or proton pump inhibitors.
  6. Intake of medications that may interfere with Lansoprazole and that cannot be withdrawn or modified in its form of administration to avoid such interference.
  7. Prior taking of proton pump inhibitors, except for a wash out period of 15 days if the clinical situation of the patient allows it.

Sites / Locations

  • Hospital Universitario Virgen de la VictoriaRecruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Experimental

Arm Label

1. Randomized placebo-controlled, parallel-group study, crossover-design

2. Randomized placebo-controlled, parallel-group study, crossover-design

Arm Description

Outcomes

Primary Outcome Measures

Changes in PPi,NPP1-3 and TNAP
changes in plasma PPi, and the main molecules that regulate it (NPP1-3, TNAP) after oral administration of lansoprazole in patients diagnosed with PXE

Secondary Outcome Measures

Full Information

First Posted
December 3, 2020
Last Updated
December 3, 2020
Sponsor
Fundación Pública Andaluza para la Investigación de Málaga en Biomedicina y Salud
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1. Study Identification

Unique Protocol Identification Number
NCT04660461
Brief Title
Response to Oral Lansoprazole of Inorganic Pyrophosphate Levels in Patients With Grönblad-Stranberg Disease (Pseudoxanthoma Elasticum)
Acronym
FIMPXE2016-01
Official Title
Response to Oral Lansoprazole of Inorganic Pyrophosphate Levels in Patients With Grönblad-Stranberg Disease (Pseudoxanthoma Elasticum)
Study Type
Interventional

2. Study Status

Record Verification Date
November 2020
Overall Recruitment Status
Unknown status
Study Start Date
February 4, 2020 (Actual)
Primary Completion Date
June 24, 2021 (Anticipated)
Study Completion Date
June 24, 2021 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Fundación Pública Andaluza para la Investigación de Málaga en Biomedicina y Salud

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

Brief Summary
Protocol code and version: FIM-PXE-2016-01 Version 1.4 Trial title: "Response to oral lansoprazole of inorganic pyrophosphate levels in patients with Grönblad- Stranberg disease (Pseudoxanthoma Elasticum)" Trial design: Double-blind, placebo-controlled, randomised, two-stage crossover clinical trial, with each patient serving as their own control and reducing the number of patients to confirm our hypothesis. Principal Investigator: Dr. Pedro Valdivielso Felices Participating centres Virgen de la Victoria's Universitary Hospital in Malaga and Virgen de la Macarena's Universitary Hospital in Seville. Duration of the trial: 12 months Expected start date: December 2019 Objectives: Principal:To verify the changes in plasma PPi, and the main molecules that regulate it (NPP1-3, TNAP) after oral administration of lansoprazole in patients diagnosed with PXE. Description of treatment: Selection:20 patients who meet all the criteria for inclusion and none for exclusion. Randomisation and 1st stage: Patients will receive lansoprazole 30mg/day or their placebo for 8 weeks. Wash-out: After 8 weeks, all treatment will be suspended for 15 days. 2nd stage (crossed): Treatment is crossed, each patient serves as his or her own control. Evaluation variables: Date of Birth Sex. Physical examination (anthropometry and vital signs) Date of first symptom. Date of final diagnosis Ocular affectation (orange peel skin, complete striae angioides, lucentis, corrected visual acuity, cataracts, intraocular pressure, fundus (vascular flow, optic nerve drusen, retinal atrophy, neovascular membranes, macular thickness, colloid thickness). Skin affectation (yellowish papules or plaques on the side of the neck or other areas of flexure and lax skin). Vascular affectation (intermittent claudication clinic, angina and/or episode of acute myocardial infarction and/or non-embolic ischemic stroke, surgical or percutaneous revascularisation, cardiac murmur,10.) History of renal lithiasis, arterial hypertension, diabetes mellitus, treatments, smoking and dyslipidemia. Specific biochemical variables: Inorganic pyrophosphate (IPP) NPP1 and NPP2.3: activity and mass concentration of the enzyme Non-specific tissue alkaline phosphatase (NTAP) and PHA. Osteocalcin: To check possible side effects on bone metabolism. 5'-Nucleotidas General analytical parameters (haemoglobin, haematocrit, MVC, MHC, platelets, neutrophils, prothrombin activity, TPTA, thrombin time, ferritin, PCR, glycaemia, urea, creatinine, cholesterol, triglycerides, HDL cholesterol, LDL cholesterol, uric acid, calcium, phosphorus, alkaline phosphatase, PTH). By means of routine clinical laboratory techniques. Number of patients: TOTAL : 20 patients(Competitive recruitment)

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Grönblad-Stranberg Disease (Pseudoxanthoma Elasticum)
Keywords
Grönblad-Stranberg Disease, Pseudoxathoma Elasticum

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Crossover Assignment
Model Description
Clinical trial, double blind, placebo controlled, randomized and crossed groups of patients in two stages, that receive a treatment with lansoprazole and placebo during 8 weeks with wash-ot period of 15 days
Masking
ParticipantInvestigator
Masking Description
The masking of the drug to produce the cecum will be carried out by the Pharmacy on University Hospital Virgen de la Macarena accredited by the AEMPS. The medication for this clinical trial will be prepared by the Pharmacy on University Hospital Virgen de la Macarena in Seville ; the lansoprazole capsules and their placebo will be re-encapsulated in larger ones to maintain the cecum. The proposed doses used are those used in mild/moderate gastro-oesophageal diseases
Allocation
Randomized
Enrollment
20 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
1. Randomized placebo-controlled, parallel-group study, crossover-design
Arm Type
Experimental
Arm Title
2. Randomized placebo-controlled, parallel-group study, crossover-design
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Lansoprazole 30mg
Intervention Description
Subjects are randomized to recieve Lansoprazole 30mg/day or placebo during 8 weeks. After a washing-out period of 15 days, subjects will receive the other treatment (cross-over design) during 8 weeks
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Subjects are randomized to recieve Lansoprazole 30mg/day or placebo during 8 weeks. After a washing-out period of 15 days, subjects will receive the other treatment (cross-over design) during 8 weeks
Primary Outcome Measure Information:
Title
Changes in PPi,NPP1-3 and TNAP
Description
changes in plasma PPi, and the main molecules that regulate it (NPP1-3, TNAP) after oral administration of lansoprazole in patients diagnosed with PXE
Time Frame
8 weeks, 18 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Patients ≥18 years old Patients diagnosed with PXE according to 2010 criteria by PLOMP et al. At least, patients meet two of the following criteria: Retinal lesions such as Orange peel and/or Angioid streaks Skin lesions such as papules or yellowish plaques on the lateral face of the neck and / or flexures of the body (armpits, elbows, knees) or alterations in the skin biopsy with fragmentation and / or conglomerates of and / or calcification of the elastic fibers. A pathogenic mutation of the two alleles of the ABCC6 gene. Exclusion Criteria: Refusal of informed consent. Vegetarian diet or extreme diets. Pregnancy or its intention during the months of the study. Age <18 years old Known hypersensitivity to "prazoles" or proton pump inhibitors. Intake of medications that may interfere with Lansoprazole and that cannot be withdrawn or modified in its form of administration to avoid such interference. Prior taking of proton pump inhibitors, except for a wash out period of 15 days if the clinical situation of the patient allows it.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Gloria Luque
Phone
951291977
Email
gloria.luque@gmail.com
Facility Information:
Facility Name
Hospital Universitario Virgen de la Victoria
City
Málaga
Country
Spain
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Pedro Valdivieso, MD
First Name & Middle Initial & Last Name & Degree
Pedro Valdivieso
First Name & Middle Initial & Last Name & Degree
Juan Carlos Carrillo

12. IPD Sharing Statement

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Response to Oral Lansoprazole of Inorganic Pyrophosphate Levels in Patients With Grönblad-Stranberg Disease (Pseudoxanthoma Elasticum)

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