Oral Pyrophosphate Absorption in PXE Disease

Oral Disodiumpyrophosphate (Na2H2PPi) Absorption in Pseudoxanthoma Elasticum (PXE)

Status

Withdrawn

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT04441671

Enrollment

Registered

2020-06-22

Start date

2020-12-08

Completion date

2021-04-01

Last updated

2021-04-22

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

Conditions

Pseudoxanthoma Elasticum

Brief summary

Pyrophosphate is an endogenous, non-toxic metabolite inhibiting soft tissue calcification. The aim of our study is to find optimal dosing and safety of oral disodium-PPi (Na2H2PPi). Absorption curves (pharmacokinetics), AUC0-t, Cmax and Tmax for PPi and phosphate will be provided for healthy controls and PXE-patients both fasting and with standard meal intake.

Detailed description

Phase II oral capsulized disodium-PPi (Na2H2PPi) powder absorption study in subjects with PXE (n=8-12) will be done in the ward of Internal Medicine of Tampere University Hospital and University Medical Center Utrecht. After a 10-hour fast at 8.00 a.m. 30 mg/kg (first day) and 50 mg/kg (second day) capsulized dose of PPi will be given with 2 dl water. At 12.00 a.m. another capsulized 30 mg/kg (first day) or 50 mg/kg (second day) single dose of PPi with a standard mixed meal (lunch) will be given with 2 dl water at the time when a subject starts eating. Plasma and urine sampling include plasma and spot urine electrolytes, creatinine, and pyrophosphate. Plasma sampling will be done at 0, 15, 30, 60, 120 and 240 min after ingestion of PPi. Urine spot sample will be taken at 0 and 240 min after ingestion of PPi. Physical activity is restricted. Side-effects will be recorded.

Interventions

DRUGDisodium Pyrophosphate

Absorption trial

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to 80 Years

Healthy volunteers

Inclusion criteria

* Adults (\>18 yrs) * clinically and genetically proven PXE * Body mass index (BMI) 18,5-34,9 kg/m2

Exclusion criteria

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

Design outcomes

Primary

Measure	Time frame	Description
Concentration area under the curve 0-t of pyrophosphate	two days	—
Maximal concentration of pyrophosphate	two days	Cmax
Timepoint of maximal pyrophosphate concentration	two days	Tmax

Secondary

Measure	Time frame	Description
Concentration area under the curve 0-t of phosphate	two days	AUC0-t
Maximal concentration of phosphate	two days	Cmax
Timepoint of maximal phosphate concentration	two days	Tmax

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026