Response to oral lansoprazole in patients with Pseudoxanthoma Elasticum

• Register: EUCTR
• Last refreshed on: 7 January 2019
• Main ID: EUCTR2016-004021-16-ES
• Date of registration: 10/03/2017
• Prospective Registration: Yes
• Primary sponsor: Fundación Pública Andaluza para la Investigación de Málaga en Biomedicina y Salud (FIMABIS)
• Public title: Response to oral lansoprazole in patients with Pseudoxanthoma Elasticum
• Scientific title: Response to oral lansoprazole in inorganic pyrophosphate levels in patients with Grönblad-Stranberg disease (Pseudoxanthoma Elasticum) - FIM-PXE-2016-01
• Date of first enrolment: 31/10/2018
• Target sample size: 20
• Recruitment status: Authorised-recruitment may be ongoing or finished
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2016-004021-16
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes; Randomised: yes; Open: no; Single blind: no; Double blind: yes; Parallel group: no; Cross over: yes; Other: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: yes; Other: no; Number of treatment arms in the trial: 2
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

Spain

Contacts

• Name: Gloria Luque
• Affiliation: FIMABIS

• Name: Gloria Luque
• Affiliation: FIMABIS

Key inclusion & exclusion criteria

Inclusion criteria:
1.- Patients =18 years diagnosed of PXE according to the 2010 criteria of PLOMP et al (10) in follow-up at the Internal Medicine Consultation of the Virgen de la Victoria Hospital in Málaga and at the Internal Medicine Consultation of the Hospital Virgen de la Macarena de Sevilla and that meet at least two of the following criteria:
A. Retinal lesions peau d'orange type and / or angioid streaks.
B. Skin lesions consisting of papules or yellowish plaques on the lateral side of the neck and / or body flexures (axilary folds, elbows, knees) or alterations in the skin biopsy with fragmentation and / or conglomerates of and / or elastic fiber calcification.
C. A pathogenic mutation of the two alleles of the ABCC6 gene.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 20
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 20

Exclusion criteria:
1.- Refuse to give informed consent.
2.- Vegetarian diet or extreme diets.
3.- Pregnancy or intention during study period.
4.- Age <18 years.
5.- Known hypersensitivity to "prazoles" or proton pump inhibitors.
6.- Ingestion of medications that may interfere with PPIs (antifungal and atazanavir).
7.- Previous treatment with proton pump inhibitors, except for a 15 day wash period if the patient's clinical situation allows it


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]

Grönblad-Stranberg disease (Pseudoxanthoma Elasticum)
MedDRA version: 19.1 Level: PT Classification code 10037150 Term: Pseudoxanthoma elasticum System Organ Class: 10010331 - Congenital, familial and genetic disorders

Intervention(s)

Trade Name: Lansoprazole
Pharmaceutical Form: Capsule
INN or Proposed INN: Lansolprazole
CAS Number: 103577-45-3
Other descriptive name: LANSOPRAZOLE
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 30-
Pharmaceutical form of the placebo: Capsule
Route of administration of the placebo: Oral use

Primary Outcome(s)

Secondary Objective: Not applicable

Timepoint(s) of evaluation of this end point: 8th and 16th week

Main Objective: Verify the changes in plasmatic PPi and the main molecules that regulate them (ENPP1-3, TNAP) after oral administration of lansoprazole in patients with Pseudoxanthoma Elasticum (PXE).

Primary end point(s): Verify the changes in plasmatic PPi and the main molecules that regulate them (ENPP1-3, TNAP) after oral administration of lansoprazole in patients with Pseudoxanthoma Elasticum (PXE).

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: Not applicable

Secondary end point(s): Not applicable

Secondary ID(s)

FIM-PXE-2016-01

Source(s) of Monetary Support

FIMABIS