Oral Pyrophosphate Absorption in PXE Disease

Phase 1

• Conditions: Pseudoxanthoma elasticum; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2019-002109-24-FI
• Lead Sponsor: Tampere University Hospital

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-07-01
• Last Update Posted: 5 April 2021

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 10

Inclusion Criteria

Adults (>18 yrs) when they have a clinically and genetically proven PXE.
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 12
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Special groups according to researchers' decision. Pregnancy. No effective contraception in females in child-bearing age.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: Phase II trial: to find optimal dosing and safety of oral disodium-<br>PPi. Absorption curves (pharmacokinetics) will be provided for PXE-patients.;Secondary Objective: Not applicaple;Primary end point(s): absorption curves and adverse events;Timepoint(s) of evaluation of this end point: Two days of absoprtion kinetics

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): None