Oral Pyrophosphate Absorption in PXE Disease
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|ClinicalTrials.gov Identifier: NCT04441671|
Recruitment Status : Withdrawn (Withdrawn)
First Posted : June 22, 2020
Last Update Posted : April 22, 2021
|Condition or disease||Intervention/treatment||Phase|
|Pseudoxanthoma Elasticum||Drug: Disodium Pyrophosphate||Phase 2|
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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||0 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Oral Disodiumpyrophosphate (Na2H2PPi) Absorption in Pseudoxanthoma Elasticum (PXE)|
|Actual Study Start Date :||December 8, 2020|
|Actual Primary Completion Date :||April 1, 2021|
|Actual Study Completion Date :||April 1, 2021|
Experimental: 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
Drug: Disodium Pyrophosphate
Other Name: Na2H2PPi
- Concentration area under the curve 0-t of pyrophosphate [ Time Frame: two days ]
- Maximal concentration of pyrophosphate [ Time Frame: two days ]Cmax
- Timepoint of maximal pyrophosphate concentration [ Time Frame: two days ]Tmax
- Concentration area under the curve 0-t of phosphate [ Time Frame: two days ]AUC0-t
- Maximal concentration of phosphate [ Time Frame: two days ]Cmax
- Timepoint of maximal phosphate concentration [ Time Frame: two days ]Tmax
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04441671
|Principal Investigator:||Pasi I Nevalainen, MD, PhD||Tampere University Hospital|