Calcinosis Reduction by Pyrophosphate in SSC
Primary Purpose
Systemic Sclerosis, Scleroderma
Status
Not yet recruiting
Phase
Not Applicable
Locations
Study Type
Interventional
Intervention
Pyrophosphate
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Systemic Sclerosis focused on measuring calcinosis, pyrophosphate
Eligibility Criteria
Inclusion Criteria:
- to meet ACR/ EULAR classification criteria
Exclusion Criteria:
- severe upper minfestation of SSC
- hypo- or hyperthyreoidism
- QT prolongation on ECG
Sites / Locations
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
Pyrophosphate
Placebo
Arm Description
Disodium pyrophosphate
Glucose
Outcomes
Primary Outcome Measures
Change in the size of calcinosis nodues.
Volumetric assessment with low energy CT
Secondary Outcome Measures
Change of the severity of sympthoms caused by calcinosis
Visual analogue scale (0-100)
Change of the activity of calcinosis
Visual analogue scale (0-100)
Change of the size of calcinosis by ultrasound
Volumetric assessment by ultrasound
Full Information
NCT ID
NCT04966416
First Posted
July 14, 2021
Last Updated
May 23, 2023
Sponsor
Szeged University
Collaborators
Research Centre for Natural Sciences, University of Debrecen Dept. of Rheumatology
1. Study Identification
Unique Protocol Identification Number
NCT04966416
Brief Title
Calcinosis Reduction by Pyrophosphate in SSC
Official Title
The Effect of Orally Administered Pyrophosphate on Calcinosis Formation in Systematic Sclerosis
Study Type
Interventional
2. Study Status
Record Verification Date
May 2023
Overall Recruitment Status
Not yet recruiting
Study Start Date
August 1, 2023 (Anticipated)
Primary Completion Date
June 30, 2025 (Anticipated)
Study Completion Date
June 30, 2026 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Szeged University
Collaborators
Research Centre for Natural Sciences, University of Debrecen Dept. of Rheumatology
4. Oversight
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
Data Monitoring Committee
No
5. 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.
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Systemic Sclerosis, Scleroderma
Keywords
calcinosis, pyrophosphate
7. Study Design
Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Parallel Assignment
Masking
Investigator
Allocation
Randomized
Enrollment
60 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Pyrophosphate
Arm Type
Experimental
Arm Description
Disodium pyrophosphate
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Glucose
Intervention Type
Dietary Supplement
Intervention Name(s)
Pyrophosphate
Intervention Description
50 mg/kg bwt once daily in gelatine capsules.
Intervention Type
Dietary Supplement
Intervention Name(s)
Placebo
Intervention Description
Glucose in gelatine capsules.
Primary Outcome Measure Information:
Title
Change in the size of calcinosis nodues.
Description
Volumetric assessment with low energy CT
Time Frame
52 weeks+ 16 weeks
Secondary Outcome Measure Information:
Title
Change of the severity of sympthoms caused by calcinosis
Description
Visual analogue scale (0-100)
Time Frame
52 weeks+ 16 weeks
Title
Change of the activity of calcinosis
Description
Visual analogue scale (0-100)
Time Frame
52 weeks+ 16 weeks
Title
Change of the size of calcinosis by ultrasound
Description
Volumetric assessment by ultrasound
Time Frame
52 weeks+ 16 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
70 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
to meet ACR/ EULAR classification criteria
Exclusion Criteria:
severe upper minfestation of SSC
hypo- or hyperthyreoidism
QT prolongation on ECG
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
László Kovács, MD, PhD
Phone
+36309081611
Email
kovacs.laszlo@med.u-szeged.hu
First Name & Middle Initial & Last Name or Official Title & Degree
Márta Bocskai, MD
Phone
+3662341520
Email
bocskai.marta@med.u-szeged.hu
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
László Kovács, MD, Ph.D.
Organizational Affiliation
Szeged University
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
András Váradi, PhD, DSc
Organizational Affiliation
Department of Enzymology, Research Centre for Natural Sciences
Official's Role
Study Director
12. IPD Sharing Statement
Citations:
PubMed Identifier
34152415
Citation
Hsu VM, Kozak E, Li Q, Bocskai M, Schlesinger N, Rosenthal A, McClure ST, Kovacs L, Balint L, Szamosi S, Szucs G, Carns M, Aren K, Goldberg I, Varadi A, Varga J. Inorganic pyrophosphate is reduced in patients with systemic sclerosis. Rheumatology (Oxford). 2022 Mar 2;61(3):1158-1165. doi: 10.1093/rheumatology/keab508.
Results Reference
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PubMed Identifier
28701330
Citation
Dedinszki D, Szeri F, Kozak E, Pomozi V, Tokesi N, Mezei TR, Merczel K, Letavernier E, Tang E, Le Saux O, Aranyi T, van de Wetering K, Varadi A. Oral administration of pyrophosphate inhibits connective tissue calcification. EMBO Mol Med. 2017 Nov;9(11):1463-1470. doi: 10.15252/emmm.201707532.
Results Reference
background
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Calcinosis Reduction by Pyrophosphate in SSC
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