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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Recruitment Status : Withdrawn (Withdrawn)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

Tracking Information
First Submitted Date  ICMJE January 20, 2020
First Posted Date  ICMJE June 22, 2020
Last Update Posted Date April 22, 2021
Actual Study Start Date  ICMJE December 8, 2020
Actual Primary Completion Date April 1, 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 21, 2021)
  • 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
Original Primary Outcome Measures  ICMJE
 (submitted: June 18, 2020)
Absorption pharmacokinetics of pyrophosphate (PPi) [ Time Frame: two days ]
AUC0-t
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: April 21, 2021)
  • 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
Original Secondary Outcome Measures  ICMJE
 (submitted: June 18, 2020)
  • Absorption pharmacokinetics of pyrophosphate (PPi) [ Time Frame: two days ]
    Cmax
  • Absorption pharmacokinetics of pyrophosphate (PPi) [ Time Frame: two days ]
    Tmax
  • Absorption pharmacokinetics of phosphate (Pi) [ Time Frame: two days ]
    AUC0-t
  • Absorption pharmacokinetics of phosphate (Pi) [ Time Frame: two days ]
    Cmax
  • Absorption pharmacokinetics of phosphate (Pi) [ Time Frame: two days ]
    Tmax
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Oral Pyrophosphate Absorption in PXE Disease
Official Title  ICMJE Oral Disodiumpyrophosphate (Na2H2PPi) Absorption in Pseudoxanthoma Elasticum (PXE)
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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Pseudoxanthoma Elasticum
Intervention  ICMJE Drug: Disodium Pyrophosphate
Absorption trial
Other Name: Na2H2PPi
Study Arms  ICMJE 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
Intervention: Drug: Disodium Pyrophosphate
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Withdrawn
Actual Enrollment  ICMJE
 (submitted: April 21, 2021)
0
Original Estimated Enrollment  ICMJE
 (submitted: June 18, 2020)
12
Actual Study Completion Date  ICMJE April 1, 2021
Actual Primary Completion Date April 1, 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 80 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Not Provided
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04441671
Other Study ID Numbers  ICMJE R19017M
2019-002109-24 ( EudraCT Number )
KLnro 38/2019 ( Other Identifier: Fimea )
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Plan Description: Only anonymized data can be shared
Current Responsible Party Tampere University Hospital
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Tampere University Hospital
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE
  • UMC Utrecht
  • Hungarian Academy of Sciences
Investigators  ICMJE
Principal Investigator: Pasi I Nevalainen, MD, PhD Tampere University Hospital
PRS Account Tampere University Hospital
Verification Date April 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP