Response to Oral Lansoprazole of Inorganic Pyrophosphate Levels in Patients With Grönblad-Stranberg Disease (Pseudoxanthoma Elasticum)
试验速览
- 阶段
- 4 期
- 发起方
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Changes in PPi,NPP1-3 and TNAP
研究概览
简要总结
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)
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
盲法说明
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
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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.
研究组 & 干预措施
1. Randomized placebo-controlled, parallel-group study, crossover-design
干预措施: Lansoprazole 30mg (Drug)
1. Randomized placebo-controlled, parallel-group study, crossover-design
干预措施: Placebo (Drug)
2. Randomized placebo-controlled, parallel-group study, crossover-design
干预措施: Lansoprazole 30mg (Drug)
2. Randomized placebo-controlled, parallel-group study, crossover-design
干预措施: Placebo (Drug)
结局指标
主要结局
Changes in PPi,NPP1-3 and TNAP
时间窗: 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
次要结局
未报告次要终点