Pyrophosphate therapy in PXE disease

Pseudoxanthoma elasticum – supplementation therapy of pyrophosphate deficiency

Status

Not yet recruiting

Phases

Phase 2Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2018-001492-20-FI

Enrollment

100

Registered

2018-06-11

Start date

2018-06-20

Completion date

Unknown

Last updated

2018-08-20

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Pseudoxanthoma elasticum

Interventions

Trade Name: SODIUM ACID PYROPHOSPHATE SAPP ROR 28 FOOD GRADE Product Name: SODIUM ACID PYROPHOSPHATE SAPP ROR 28 FOOD GRADE Product Code: CAS No.: 7758-16-9 Pharmaceutical Form:

Eligibility

Sex/Gender

All

Inclusion criteria

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

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.

Design outcomes

Primary

Measure	Time frame
Main Objective: To stop vascular calcification and its complications To study pyrophosphate absorption kinetics ;Secondary Objective: ;Primary end point(s): Area of retinal changes Change in symptoms of claudication Absorption study: time of normalization of plasma pyrophosphate concentration after oral pyrophosphate;Timepoint(s) of evaluation of this end point: 12, 24 and 36 months Absorption study: first visit	—

Measure

Time frame

Main Objective: To stop vascular calcification and its complications To study pyrophosphate absorption kinetics ;Secondary Objective: ;Primary end point(s): Area of retinal changes Change in symptoms of claudication Absorption study: time of normalization of plasma pyrophosphate concentration after oral pyrophosphate;Timepoint(s) of evaluation of this end point: 12, 24 and 36 months Absorption study: first visit

—

Secondary

Measure	Time frame
Secondary end point(s): Ankle to brachial index change Major amputations Visual acuity radiologic changes in lower extremity arteries ;Timepoint(s) of evaluation of this end point: 12, 24 and 36 months	—

Countries

Finland

Contacts

Public ContactPasi Nevalainen

Tampere university hospital

pasi.nevalainen@pshp.fi358331166006

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026