NCT04660461: FIMPXE2016-01

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

Status unknown Phase 4 Last updated 9 December 2020

What this trial tests

Phase 4 trial testing Lansoprazole 30mg in Grönblad-Stranberg Disease (Pseudoxanthoma Elasticum) in 20 participants. Status unknown.

Timeline

4 February 2020

Primary endpoint
24 June 2021

24 June 2021

Quick facts

Lead sponsor	Fundación Pública Andaluza para la Investigación de Málaga en Biomedicina y Salud
Phase	Phase 4
Status	Status unknown
Study type	INTERVENTIONAL
Allocation	randomized
Design	crossover
Masking	double
Primary purpose	treatment
Enrollment	20
Start date	4 February 2020
Primary completion	24 June 2021
Estimated completion	24 June 2021
Sites	1 location across Spain

Drugs / interventions tested

• Lansoprazole 30mg — full drug profile →
• Placebo

Conditions studied

• Grönblad-Stranberg Disease (Pseudoxanthoma Elasticum) — all drugs for Grönblad-Stranberg Disease (Pseudoxanthoma Elasticum) →

Sponsor

Fundación Pública Andaluza para la Investigación de Málaga en Biomedicina y Salud — full company profile →

Who can join

18 and older, any sex, with Grönblad-Stranberg Disease (Pseudoxanthoma Elasticum). Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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

Publications & conference data

5 peer-reviewed publications reference this trial (live from Europe PMC):

1. Inorganic Pyrophosphate Deficiency Syndromes and Potential Treatments for Pathologic Tissue Calcification.
   Ralph D, van de Wetering K, Uitto J, Li Q. · · 2022 · cited 37× · PMID 35182493 · DOI 10.1016/j.ajpath.2022.01.012
2. The PROPHECI trial: a phase II, double-blind, placebo-controlled, randomized clinical trial for the treatment of pseudoxanthoma elasticum with oral pyrophosphate.
   Clotaire L, Rubera I, Duranton C, Gal J, et al · · 2025 · cited 11× · PMID 39881395 · DOI 10.1186/s13063-024-08666-w
3. Lansoprazole Increases Inorganic Pyrophosphate in Patients with Pseudoxanthoma Elasticum: A Double-Blind, Randomized, Placebo-Controlled Crossover Trial.
   Murcia Casas B, Carrillo Linares JL, Baquero Aranda I, Rioja Villodres J, et al · · 2023 · cited 6× · PMID 36902331 · DOI 10.3390/ijms24054899
4. The Purinergic Nature of Pseudoxanthoma Elasticum.
   Kauffenstein G, Martin L, Le Saux O. · · 2024 · cited 5× · PMID 38392293 · DOI 10.3390/biology13020074
5. Diabetes-induced vascular calcification is associated with low pyrophosphate and its oral supplementation prevents calcification in diabetic mice.
   Fülöp K, Kozák E, Tőkési N, Kocsis L, et al · · 2026 · PMID 41081331 · DOI 10.1002/2211-5463.70141