Oral Pyrophosphate Absorption in PXE Disease

NCT ID: NCT04441671

Last Updated: 2021-04-22

Basic Information

• Recruitment Status: WITHDRAWN
• Clinical Phase: PHASE2
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-12-08
• Study Completion Date: 2021-04-01

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.

Conditions

Pseudoxanthoma Elasticum

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Open label

Disodiumpyrophosphate, capsuled powder, First day: 30 mg/kg fasting at 08.00 and with standard mixed meal at 12.00 Second day: 50 mg/kg fasting at 08.00 and with standard mixed meal at 12.00

Group Type: EXPERIMENTAL

Disodium Pyrophosphate

Intervention Type: DRUG

Absorption trial

Interventions

Disodium Pyrophosphate

Absorption trial

Intervention Type: DRUG

Other Intervention Names

Na2H2PPi

Eligibility Criteria

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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 80 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

UMC Utrecht

OTHER

Sponsor Role: collaborator

Hungarian Academy of Sciences

OTHER

Sponsor Role: collaborator

Tampere University Hospital

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Pasi I Nevalainen, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Tampere University Hospital

Other Identifiers

2019-002109-24

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

KLnro 38/2019

Identifier Type: OTHER

Identifier Source: secondary_id

R19017M

Identifier Type: -

Identifier Source: org_study_id