E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Grönblad-Stranberg disease (Pseudoxanthoma Elasticum) |
Enfermedad de Grönblad-Stranberg (Pseudoxantoma Elasticum) |
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E.1.1.1 | Medical condition in easily understood language |
Grönblad-Stranberg disease (Pseudoxanthoma Elasticum ) |
Enfermedad de Grönblad-Stranberg (Pseudoxantoma Elasticum) |
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E.1.1.2 | Therapeutic area | Body processes [G] - Metabolic Phenomena [G03] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 19.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10037150 |
E.1.2 | Term | Pseudoxanthoma elasticum |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic disorders |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
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). |
Verificar los cambios del PPi plasmático, y las principales moléculas que los regulan (ENPP1-3, TNAP) tras la administración oral de lansoprazol en pacientes con diagnóstico de Pseudoxantoma Elasticum (PXE). |
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E.2.2 | Secondary objectives of the trial |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal 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. |
1.- Pacientes ≥18 años diagnosticados de PXE según los criterios de 2010 de PLOMP et al (10) en seguimiento en la Consulta de Medicina Interna del Hospital Virgen de la Victoria de Málaga y en la Consulta de Medicina Interna del Hospital Virgen de la Macarena de Sevilla y que cumplan, al menos, dos de los siguientes criterios: A. Lesiones retinianas del tipo de piel de naranja y/o estrías angioides. B. Lesiones cutáneas consistentes en pápulas o placas amarillentas en la cara lateral del cuello y/o flexuras del cuerpo (axilas, codos, rodillas) o alteraciones en la biopsia de piel con fragmentación y/o conglomerados de y/o calcificación de las fibras elásticas. C. Una mutación patogénica de los dos alelos del gen ABCC6. |
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E.4 | Principal 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 |
1.- Negativa al consentimiento informado. 2.- Dieta vegetariana o dietas extremas. 3.- Embarazo o intención del mismo en los meses de duración del estudio. 4.- Edad < 18 años. 5.- Hipersensibilidad conocida a los medicamentos tipos “prazoles” o inhibidores de la bomba de protones. 6.- Ingesta de medicamentos que puedan interferir con los IBP (antifúngicos y atazanavir). 7.- Toma previa de inhibidores de la bomba de protones, salvo periodo de lavado de 15 días si la situación clínica del paciente lo permite |
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E.5 End points |
E.5.1 | 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). |
Verificar los cambios del PPi plasmático, y las principales moléculas que los regulan (ENPP1-3, TNAP) tras la administración oral de lansoprazol en pacientes con diagnóstico de Pseudoxantoma Elasticum (PXE). |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
8th and 16th week |
Semanas 8 y 16 |
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E.5.2 | Secondary end point(s) |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | No |
E.6.5 | Efficacy | No |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | Yes |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 2 |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 2 |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 0 |
E.8.9.1 | In the Member State concerned months | 24 |
E.8.9.1 | In the Member State concerned days | 0 |