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

NCT04660461 · Status: UNKNOWN · Phase: PHASE4 · Type: INTERVENTIONAL · Enrollment: 20

Last updated 2020-12-09

No results posted yet for this study

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:

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)

Conditions

  • Grönblad-Stranberg Disease (Pseudoxanthoma Elasticum)

Interventions

DRUG

Lansoprazole 30mg

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

DRUG

Placebo

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

Sponsors & Collaborators

  • Fundación Pública Andaluza para la Investigación de Málaga en Biomedicina y Salud

    lead OTHER

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Model
CROSSOVER

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-02-04
Primary Completion
2021-06-24
Completion
2021-06-24

Countries

  • Spain

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04660461 on ClinicalTrials.gov