Oral Pyrophosphate Absorption in PXE Disease

Pseudoxanthoma Elasticum Clinical Trial

Official title:

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

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT04441671
• Other study ID #: R19017M
• Secondary ID: 2019-002109-24KL
• Status: Withdrawn
• Phase: Phase 2
• Start date: December 8, 2020
• Est. completion date: April 1, 2021

Study information

• Verified date: April 2021
• Source: Tampere University Hospital
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Est. completion date: April 1, 2021
• Est. primary completion date: April 1, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 80 Years

Eligibility

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.

Related Conditions & MeSH terms

• Pseudoxanthoma Elasticum

Intervention

Drug:
• Disodium Pyrophosphate
Absorption trial

Locations

Country	Name	City	State
n/a

Sponsors (3)

Lead Sponsor	Collaborator
Tampere University Hospital	Hungarian Academy of Sciences, UMC Utrecht

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Concentration area under the curve 0-t of pyrophosphate		two days
Primary	Maximal concentration of pyrophosphate	Cmax	two days
Primary	Timepoint of maximal pyrophosphate concentration	Tmax	two days
Secondary	Concentration area under the curve 0-t of phosphate	AUC0-t	two days
Secondary	Maximal concentration of phosphate	Cmax	two days
Secondary	Timepoint of maximal phosphate concentration	Tmax	two days