Pyrophosphate therapy in PXE disease

Phase 1

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

• Registration Number: EUCTR2018-001492-20-FI
• Lead Sponsor: Tampere university hospital

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2018-06-11
• Last Update Posted: 20 August 2018

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 100

Inclusion Criteria

Adults (>18 yrs) are included when they have a clinically proven PXE. We will use the data from our earlier registry study to select patients with either mild to severe retinal changes or signs of an occlusive vascular disease.
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 80
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 20

Exclusion Criteria

Pregnancy, non-compliance or special groups according to researchers decision. Those without any signs of retinal or vascular disease are excluded. A fertile female without proper contraception.

Study & Design

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