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Oral Pyrophosphate Absorption in PXE Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04441671
Recruitment Status : Withdrawn (Withdrawn)
First Posted : June 22, 2020
Last Update Posted : April 22, 2021
Sponsor:
Collaborators:
UMC Utrecht
Hungarian Academy of Sciences
Information provided by (Responsible Party):
Tampere University Hospital

Study Description
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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.

Condition or disease Intervention/treatment Phase
Pseudoxanthoma Elasticum Drug: Disodium Pyrophosphate Phase 2

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.

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
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
Absorption trial
Other Name: Na2H2PPi


Outcome Measures
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Primary Outcome Measures :
  1. Concentration area under the curve 0-t of pyrophosphate [ Time Frame: two days ]
  2. Maximal concentration of pyrophosphate [ Time Frame: two days ]
    Cmax

  3. Timepoint of maximal pyrophosphate concentration [ Time Frame: two days ]
    Tmax


Secondary Outcome Measures :
  1. Concentration area under the curve 0-t of phosphate [ Time Frame: two days ]
    AUC0-t

  2. Maximal concentration of phosphate [ Time Frame: two days ]
    Cmax

  3. Timepoint of maximal phosphate concentration [ Time Frame: two days ]
    Tmax


Eligibility Criteria
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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
Contacts and Locations
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Information from the National Library of Medicine

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


Sponsors and Collaborators
Tampere University Hospital
UMC Utrecht
Hungarian Academy of Sciences
Investigators
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Principal Investigator: Pasi I Nevalainen, MD, PhD Tampere University Hospital
More Information
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Responsible Party: Tampere University Hospital
ClinicalTrials.gov Identifier: NCT04441671    
Other Study ID Numbers: R19017M
2019-002109-24 ( EudraCT Number )
KLnro 38/2019 ( Other Identifier: Fimea )
First Posted: June 22, 2020    Key Record Dates
Last Update Posted: April 22, 2021
Last Verified: April 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Plan Description: Only anonymized data can be shared

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Pseudoxanthoma Elasticum
Hemostatic Disorders
Vascular Diseases
Cardiovascular Diseases
Hemorrhagic Disorders
Hematologic Diseases
 Skin Abnormalities
Congenital Abnormalities
Skin Diseases, Genetic
Genetic Diseases, Inborn
Connective Tissue Diseases
Skin Diseases