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出境医 / 临床实验 / Calcinosis Reduction by Pyrophosphate in SSC

Calcinosis Reduction by Pyrophosphate in SSC

Study Description
Brief Summary:
Calcinosis, i.e. crystal-like nodules are troublesome complication of systemic sclerosis, an autoimmune disease. Pyrophosphate inhibits its formation is laborytory. We would like to test if orally administered pyrophosphate prevents calcinosis formation.

Condition or disease Intervention/treatment Phase
Systemic Sclerosis Scleroderma Dietary Supplement: Pyrophosphate Dietary Supplement: Placebo Not Applicable

Detailed Description:
Calcinosis, the formation of hydroxyapatite subcutaneous nodules, often complicates systemic sclerosis. There is no standard treatment for it. Based upon our preclinical experienc, we would like to test it orally administered pyrophosphate inhibits calciosis.
Study Design
Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 60 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single (Investigator)
Primary Purpose: Treatment
Official Title: The Effect of Orally Administered Pyrophosphate on Calcinosis Formation in Systematic Sclerosis
Estimated Study Start Date : August 1, 2021
Estimated Primary Completion Date : June 30, 2024
Estimated Study Completion Date : June 30, 2024
Arms and Interventions
Arm Intervention/treatment
Experimental: Pyrophosphate Dietary Supplement: Pyrophosphate
50 mg/kg bwt once daily in gelatine capsules.
Placebo Comparator: Placebo Dietary Supplement: Placebo
Glucose in gelatine capsules.
Outcome Measures
Primary Outcome Measures :
  1. Change in the size of calcinosis nodues. [ Time Frame: 52 weeks+ 16 weeks ]
    Volumetric assessment with low energy CT


Secondary Outcome Measures :
  1. Change of the severity of sympthoms caused by calcinosis [ Time Frame: 52 weeks+ 16 weeks ]
    Visual analogue scale (0-100)

  2. Change of the activity of calcinosis [ Time Frame: 52 weeks+ 16 weeks ]
    Visual analogue scale (0-100)

  3. Change of the size of calcinosis by ultrasound [ Time Frame: 52 weeks+ 16 weeks ]
    Volumetric assessment by ultrasound


Eligibility Criteria
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Ages Eligible for Study:   18 Years to 70 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • to meet ACR/ EULAR classification criteria

Exclusion Criteria:

  • severe upper minfestation of SSC
  • hypo- or hyperthyreoidism
  • QT prolongation on ECG
Contacts and Locations

Contacts
Layout table for location contacts
Contact: László Kovács, MD, PhD +36309081611 kovacs.laszlo@med.u-szeged.hu
Contact: Márta Bocskai, MD +3662341520 bocskai.marta@med.u-szeged.hu

Sponsors and Collaborators
Szeged University
Research Centre for Natural Sciences
University of Debrecen Dept. of Rheumatology
Investigators
Layout table for investigator information
Principal Investigator: László Kovács, MD, Ph.D. Szeged University
Study Director: András Váradi, PhD, DSc Department of Enzymology, Research Centre for Natural Sciences
Tracking Information
First Submitted Date  ICMJE July 14, 2021
First Posted Date  ICMJE July 19, 2021
Last Update Posted Date July 19, 2021
Estimated Study Start Date  ICMJE August 1, 2021
Estimated Primary Completion Date June 30, 2024   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: July 16, 2021) 		Change in the size of calcinosis nodues. [ Time Frame: 52 weeks+ 16 weeks ]
Volumetric assessment with low energy CT
Original Primary Outcome Measures  ICMJE Same as current
Change History No Changes Posted
Current Secondary Outcome Measures  ICMJE
 (submitted: July 16, 2021)
  • Change of the severity of sympthoms caused by calcinosis [ Time Frame: 52 weeks+ 16 weeks ]
    Visual analogue scale (0-100)
  • Change of the activity of calcinosis [ Time Frame: 52 weeks+ 16 weeks ]
    Visual analogue scale (0-100)
  • Change of the size of calcinosis by ultrasound [ Time Frame: 52 weeks+ 16 weeks ]
    Volumetric assessment by ultrasound
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Calcinosis Reduction by Pyrophosphate in SSC
Official Title  ICMJE The Effect of Orally Administered Pyrophosphate on Calcinosis Formation in Systematic Sclerosis
Brief Summary Calcinosis, i.e. crystal-like nodules are troublesome complication of systemic sclerosis, an autoimmune disease. Pyrophosphate inhibits its formation is laborytory. We would like to test if orally administered pyrophosphate prevents calcinosis formation.
Detailed Description Calcinosis, the formation of hydroxyapatite subcutaneous nodules, often complicates systemic sclerosis. There is no standard treatment for it. Based upon our preclinical experienc, we would like to test it orally administered pyrophosphate inhibits calciosis.
Study Type  ICMJE Interventional
Study Phase  ICMJE Not Applicable
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single (Investigator)
Primary Purpose: Treatment
Condition  ICMJE
  • Systemic Sclerosis
  • Scleroderma
Intervention  ICMJE
  • Dietary Supplement: Pyrophosphate
    50 mg/kg bwt once daily in gelatine capsules.
  • Dietary Supplement: Placebo
    Glucose in gelatine capsules.
Study Arms  ICMJE
  • Experimental: Pyrophosphate
    Intervention: Dietary Supplement: Pyrophosphate
  • Placebo Comparator: Placebo
    Intervention: Dietary Supplement: Placebo
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 Not yet recruiting
Estimated Enrollment  ICMJE
 (submitted: July 16, 2021) 		60
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE June 30, 2024
Estimated Primary Completion Date June 30, 2024   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • to meet ACR/ EULAR classification criteria

Exclusion Criteria:

  • severe upper minfestation of SSC
  • hypo- or hyperthyreoidism
  • QT prolongation on ECG
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 70 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: László Kovács, MD, PhD +36309081611 kovacs.laszlo@med.u-szeged.hu
Contact: Márta Bocskai, MD +3662341520 bocskai.marta@med.u-szeged.hu
Listed Location Countries  ICMJE Not Provided
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04966416
Other Study ID Numbers  ICMJE CALC-001
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
Product Manufactured in and Exported from the U.S.: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party László Kovács MD, PhD, Szeged University
Study Sponsor  ICMJE Szeged University
Collaborators  ICMJE
  • Research Centre for Natural Sciences
  • University of Debrecen Dept. of Rheumatology
Investigators  ICMJE
Principal Investigator: László Kovács, MD, Ph.D. Szeged University
Study Director: András Váradi, PhD, DSc Department of Enzymology, Research Centre for Natural Sciences
PRS Account Szeged University
Verification Date July 2021

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