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

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:

  1. Date of Birth
  2. Sex.
  3. Physical examination (anthropometry and vital signs)
  4. Date of first symptom.
  5. Date of final diagnosis
  6. 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).
  7. Skin affectation (yellowish papules or plaques on the side of the neck or other areas of flexure and lax skin).
  8. 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.)
  9. History of renal lithiasis, arterial hypertension, diabetes mellitus, treatments, smoking and dyslipidemia.
  10. 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)

Study Overview

Study Type

Interventional

Enrollment (Anticipated)

20

Phase

  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

      • Málaga, Spain
        • Recruiting
        • Hospital Universitario Virgen de la Victoria
        • Contact:
          • Pedro Valdivieso, MD
        • Principal Investigator:
          • Pedro Valdivieso
        • Sub-Investigator:
          • Juan Carlos Carrillo

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

16 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Crossover Assignment
  • Masking: Double

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: 1. Randomized placebo-controlled, parallel-group study, crossover-design
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
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
Experimental: 2. Randomized placebo-controlled, parallel-group study, crossover-design
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
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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Changes in PPi,NPP1-3 and TNAP
Time Frame: 8 weeks, 18 weeks
changes in plasma PPi, and the main molecules that regulate it (NPP1-3, TNAP) after oral administration of lansoprazole in patients diagnosed with PXE
8 weeks, 18 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

February 4, 2020

Primary Completion (Anticipated)

June 24, 2021

Study Completion (Anticipated)

June 24, 2021

Study Registration Dates

First Submitted

December 3, 2020

First Submitted That Met QC Criteria

December 3, 2020

First Posted (Actual)

December 9, 2020

Study Record Updates

Last Update Posted (Actual)

December 9, 2020

Last Update Submitted That Met QC Criteria

December 3, 2020

Last Verified

November 1, 2020

More Information

Terms related to this study

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

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.

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