Oral Pyrophosphate Absorption in PXE Disease

NCT04441671 | Withdrawn Phase 2

• 3 other identifiers: R19017M2019-002109-24KLnro 38/2019
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Pseudoxanthoma Elasticum

Outcome Measures

Primary Outcomes (3)

• Concentration area under the curve 0-t of pyrophosphate

  two days

• Maximal concentration of pyrophosphate

  Cmax

  two days

• Timepoint of maximal pyrophosphate concentration

  Tmax

  two days

Secondary Outcomes (3)

• Concentration area under the curve 0-t of phosphate

  two days

• Maximal concentration of phosphate

  two days

• Timepoint of maximal phosphate concentration

  two days

Study Arms (1)

Open label

EXPERIMENTAL

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

Interventions

Disodium Pyrophosphate DRUG

Absorption trial

Also known as: Na2H2PPi

Open label

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Adults (\>18 yrs)
• clinically and genetically proven PXE
• Body mass index (BMI) 18,5-34,9 kg/m2

You may not qualify if:

• Special groups according to researchers' decision.
• Pregnancy
• No effective contraception in females in child-bearing age.

Sponsors & Collaborators

• Tampere University Hospital: lead
• UMC Utrecht: collaborator
• Hungarian Academy of Sciences: collaborator

MeSH Terms

Conditions

Pseudoxanthoma Elasticum

Interventions

sodium pyrophosphate

Condition Hierarchy (Ancestors)

Hemostatic Disorders; Vascular Diseases; Cardiovascular Diseases; Hemorrhagic Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Skin Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases

Study Officials

• Pasi I Nevalainen, MD, PhD

  Tampere University Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 20, 2020
• First Posted: June 22, 2020
• Study Start: December 8, 2020
• Primary Completion: April 1, 2021
• Study Completion: April 1, 2021
• Last Updated: April 22, 2021

Record last verified: 2021-04

Data Sharing

• IPD Sharing: Will not share