Systemic SclerosisNCT04966416
Calcinosis Reduction by Pyrophosphate in SSC
A study testing Pyrophosphate in people with Systemic Sclerosis and Scleroderma, run by Szeged University.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Systemic Sclerosis, Scleroderma
- What is being tested?
- Pyrophosphate
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 60 participants
- Where
- No sites listed yet
- Study dates
- 1 Aug 2023to 30 Jun 2026
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- to meet ACR/ EULAR classification criteria
Who cannot join
- severe upper minfestation of SSC
- hypo- or hyperthyreoidism
- QT prolongation on ECG
Trial locations and contacts
No sites are listed yet.
About this study
Official title: The Effect of Orally Administered Pyrophosphate on Calcinosis Formation in Systematic Sclerosis
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.
Read the 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.
- Conditions
- Systemic Sclerosis, Scleroderma
- Being tested
- Pyrophosphate, Placebo
- Keywords
- calcinosis, pyrophosphate
- Medical subject headings
- Scleroderma, Systemic, Scleroderma, Diffuse, Calcinosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PyrophosphateDietary supplement
50 mg/kg bwt once daily in gelatine capsules.
PlaceboDietary supplement
Glucose in gelatine capsules.
| Study group | Role | What they receive |
|---|---|---|
| Pyrophosphate | Experimental | Disodium pyrophosphate Receives: Dietary Supplement: Pyrophosphate |
| Placebo | Placebo comparator | Glucose Receives: Dietary Supplement: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in the size of calcinosis nodues.
Volumetric assessment with low energy CT
Measured: 52 weeks+ 16 weeks
Secondary outcomes (3)
Change of the severity of sympthoms caused by calcinosis
Visual analogue scale (0-100)
Measured: 52 weeks+ 16 weeks
Change of the activity of calcinosis
Visual analogue scale (0-100)
Measured: 52 weeks+ 16 weeks
Change of the size of calcinosis by ultrasound
Volumetric assessment by ultrasound
Measured: 52 weeks+ 16 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Jul 2021
- Study start1 Aug 2023 (estimated)
- Primary completion30 Jun 2025 (estimated)
- Study completion30 Jun 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Systemic SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Investigator
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Szeged University
- Sponsor type
- University, hospital or other
- Collaborators
- Research Centre for Natural Sciences, University of Debrecen Dept. of Rheumatology
- Responsible party
- Principal investigator, László Kovács MD, PhD, Szeged University
- Investigators
- László Kovács, MD, Ph.D., Principal investigator, Szeged UniversityAndrás Váradi, PhD, DSc, Study director, Department of Enzymology, Research Centre for Natural Sciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04966416
- Sponsor's protocol ID
- CALC-001
- Status last verified
- May 2023
- First submitted
- 14 Jul 2021
- Last updated
- 25 May 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
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.
PubMed 34152415Dedinszki 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.
PubMed 28701330
Study at a glance
From the registry record, last updated by the sponsor on 25 May 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Effect of Orally Administered Pyrophosphate on Calcinosis Formation in Systematic Sclerosis
- ClinicalTrials.gov ID
- NCT04966416
- Acronym / sponsor ID
- CALC-001
- Conditions
- Systemic Sclerosis, Scleroderma
- Keywords
- calcinosis, pyrophosphate
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- Szeged UniversityUniversity, hospital or other
- Collaborators
- Research Centre for Natural Sciences, University of Debrecen Dept. of Rheumatology
- Study start
- 1 Aug 2023 (estimated)
- Primary completion
- 30 Jun 2025 (estimated)
- Study completion
- 30 Jun 2026 (estimated)
- First posted
- 19 Jul 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Pyrophosphate | Experimental | Dietary Supplement: PyrophosphateDisodium pyrophosphate |
| Placebo | Placebo comparator | Dietary Supplement: PlaceboGlucose |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- to meet ACR/ EULAR classification criteria
Key exclusion criteria (3 listed)
- severe upper minfestation of SSC
- hypo- or hyperthyreoidism
- QT prolongation on ECG
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Pyrophosphate50 mg/kg bwt once daily in gelatine capsules. | Dietary supplement | Pyrophosphate |
| PlaceboGlucose in gelatine capsules. | Dietary supplement | Placebo |
Primary outcome
Change in the size of calcinosis nodues.
Time frame: 52 weeks+ 16 weeks
Volumetric assessment with low energy CT
3 secondary outcomes
Change of the severity of sympthoms caused by calcinosis
Time frame: 52 weeks+ 16 weeks
Change of the activity of calcinosis
Time frame: 52 weeks+ 16 weeks
Change of the size of calcinosis by ultrasound
Time frame: 52 weeks+ 16 weeks
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 May 2023.
- Background: PubMed 34152415Hsu 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.
- Background: PubMed 28701330Dedinszki 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
László Kovács, MD, Ph.D. principal investigator
Szeged University
András Váradi, PhD, DSc study director
Department of Enzymology, Research Centre for Natural Sciences
Questions about this trial
What is this clinical trial studying?
A study testing Pyrophosphate in people with Systemic Sclerosis and Scleroderma, run by Szeged University.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 25 May 2023. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact László Kovács, MD, PhD (+36309081611, kovacs.laszlo@med.u-szeged.hu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in the size of calcinosis nodues. (time frame: 52 weeks+ 16 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04966416, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 May 2023.