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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

About this trial

This is an interventional treatment trial for Systemic Sclerosis focused on measuring calcinosis, pyrophosphate

Eligibility Criteria

18 Years - 70 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Studies a U.S. FDA-regulated Device Product

Product Manufactured in and Exported from the U.S.

Data Monitoring Committee

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

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

kovacs.laszlo@med.u-szeged.hu

First Name & Middle Initial & Last Name or Official Title & Degree

Márta Bocskai, MD

Phone

+3662341520

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

background

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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