Response to Oral Lansoprazole of Inorganic Pyrophosphate Levels in Patients With Grönblad-Stranberg Disease (Pseudoxanthoma Elasticum) (FIMPXE2016-01)
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| ClinicalTrials.gov Identifier: NCT04660461 |
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Recruitment Status :
Recruiting
First Posted : December 9, 2020
Last Update Posted : December 9, 2020
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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:
- Date of Birth
- Sex.
- Physical examination (anthropometry and vital signs)
- Date of first symptom.
- Date of final diagnosis
- 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).
- Skin affectation (yellowish papules or plaques on the side of the neck or other areas of flexure and lax skin).
- 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.)
- History of renal lithiasis, arterial hypertension, diabetes mellitus, treatments, smoking and dyslipidemia.
- 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)
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Grönblad-Stranberg Disease (Pseudoxanthoma Elasticum) | Drug: Lansoprazole 30mg Drug: Placebo | Phase 4 |
| Study Type : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 20 participants |
| Allocation: | Randomized |
| Intervention Model: | Crossover Assignment |
| Intervention Model Description: | 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 |
| Masking: | Double (Participant, Investigator) |
| Masking Description: | 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 |
| Primary Purpose: | Treatment |
| Official Title: | Response to Oral Lansoprazole of Inorganic Pyrophosphate Levels in Patients With Grönblad-Stranberg Disease (Pseudoxanthoma Elasticum) |
| Actual Study Start Date : | February 4, 2020 |
| Estimated Primary Completion Date : | June 24, 2021 |
| Estimated Study Completion Date : | June 24, 2021 |
| Arm | Intervention/treatment |
|---|---|
| Experimental: 1. Randomized placebo-controlled, parallel-group study, crossover-design |
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 |
| Experimental: 2. Randomized placebo-controlled, parallel-group study, crossover-design |
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 |
- 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
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| Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Patients ≥18 years old
- Patients diagnosed with PXE according to 2010 criteria by PLOMP et al.
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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.
Exclusion Criteria:
- 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04660461
| Contact: Gloria Luque | 951291977 | gloria.luque@gmail.com |
| Spain | |
| Hospital Universitario Virgen de la Victoria | Recruiting |
| Málaga, Spain | |
| Contact: Pedro Valdivieso, MD | |
| Principal Investigator: Pedro Valdivieso | |
| Sub-Investigator: Juan Carlos Carrillo | |
| Responsible Party: | Fundación Pública Andaluza para la Investigación de Málaga en Biomedicina y Salud |
| ClinicalTrials.gov Identifier: | NCT04660461 |
| Other Study ID Numbers: |
EudraCT 2016-004021-16 FIM-PXE-2016-01 ( Other Identifier: FIMABIS ) |
| First Posted: | December 9, 2020 Key Record Dates |
| Last Update Posted: | December 9, 2020 |
| Last Verified: | November 2020 |
| Studies a U.S. FDA-regulated Drug Product: | No |
| Studies a U.S. FDA-regulated Device Product: | No |
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Grönblad-Stranberg Disease Pseudoxathoma Elasticum |
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Pseudoxanthoma Elasticum Hemostatic Disorders Vascular Diseases Cardiovascular Diseases Hemorrhagic Disorders Hematologic Diseases Skin Abnormalities Congenital Abnormalities Skin Diseases, Genetic Genetic Diseases, Inborn |
Connective Tissue Diseases Skin Diseases Lansoprazole Dexlansoprazole Anti-Ulcer Agents Gastrointestinal Agents Proton Pump Inhibitors Enzyme Inhibitors Molecular Mechanisms of Pharmacological Action |