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

NCT04660461 | Unknown Phase 4

• 2 other identifiers: EudraCT 2016-004021-16FIM-PXE-2016-01
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

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:

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

Conditions

Grönblad-Stranberg Disease (Pseudoxanthoma Elasticum)

Keywords

Grönblad-Stranberg Disease; Pseudoxathoma Elasticum

Outcome Measures

Primary Outcomes (1)

• 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

  8 weeks, 18 weeks

Study Arms (2)

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

EXPERIMENTAL

Drug: Lansoprazole 30mg; Drug: Placebo

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

EXPERIMENTAL

Drug: Lansoprazole 30mg; Drug: Placebo

Interventions

Lansoprazole 30mg DRUG

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

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

Placebo DRUG

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

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

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

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

Study Sites (1)

Hospital Universitario Virgen de la Victoria

Málaga, Spain

RECRUITING

Related Publications (35)

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MeSH Terms

Conditions

Pseudoxanthoma Elasticum

Interventions

Lansoprazole

Condition Hierarchy (Ancestors)

Hemostatic Disorders; Vascular Diseases; Cardiovascular Diseases; Hemorrhagic Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Skin Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases

Intervention Hierarchy (Ancestors)

2-Pyridinylmethylsulfinylbenzimidazoles; Sulfoxides; Sulfur Compounds; Organic Chemicals; Pyridines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Benzimidazoles; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Central Study Contacts

Gloria Luque

CONTACT

951291977; gloria.luque@gmail.com

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Masking Details: 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
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: 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

Study Record Dates

• First Submitted: December 3, 2020
• First Posted: December 9, 2020
• Study Start: February 4, 2020
• Primary Completion: June 24, 2021
• Study Completion: June 24, 2021
• Last Updated: December 9, 2020

Record last verified: 2020-11

Locations

ES (1)