Patient presenting with: - ectopic ossification secondary to iPPSD2 or - ectopic calcification secondary to dermatomyositis or - ectopic calcification secondary to systemic sclerosis MedDRA version: 20.0 Level: LLT Classification code 10006935 Term: Calcification and ossification, unspecified System Organ Class: 100000004867
Conditions
Interventions
Trade Name: Sodium Thiosulfate 10%
Product Name: Sodium thiosulfate
Pharmaceutical Form: Solution for injection
INN or Proposed INN: Sodium thiosulfate
Sponsors
CHU de LIMOGES
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Patient presenting with: ? ectopic calcification secondary to dermatomyositis or ? ectopic calcification secondary to systemic sclerosis And aged 18 years old or higher OR ? ectopic ossification secondary to iPPSD2 And aged = 2 years old and = 17 years old And after the specific authorization of ANSM and CPP - Indication of STS injection validated by a multidisciplinary committee, based on the significant morbidity and/or functional impact of the targeted calcification/ossification - Patient with no planned surgery of the calcifications/ossifications for the twelve coming months -Women of childbearing potential on highly effective contraception (such as hormonal contraception, intrauterine device, intrauterine hormone-releasing system, bilateral tubal occlusion, vasectomised partner or sexual abstinence). Contraception will be extended up to one month after the last injection. - Informed consent signed by the patient / parents - Patient affiliated to (or beneficiary of) the social security system Are the trial subjects under 18? yes Number of subjects for this age range: 10 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 30 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: - Allergy to STS, sulfites or one of the excipients used - Contraindication to local injection of STS - Anticoagulant therapy - Pregnant, parturient or breastfeeding woman - Patient deprived of freedom by a court judgment or an administrative decision - Patient undergoing psychiatric care under coercion - Legally protected adult patients (guardianship / curatorship) - Patient unable to give consent - Patient placed under judicial protection
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate, after a run-in period of 6 months, the efficacy of a 6- month treatment of 10% STS solution when locally injected for the treatment of calcifications (secondary to dermatomyositis or systemic sclerosis) and ectopic ossifications (secondary to iPPSD2);Primary end point(s): Percentage of volume evolution of the treated calcifications / ossifications between the beginning (M6) and the end (M12) of STS treatment, in each of the three diseases (dermatomyositis, systemic sclerosis and iPPSD2), evaluated on CT-scan measurements.;Timepoint(s) of evaluation of this end point: month 12; Secondary Objective: To evaluate in each disease (dermatomyositis, systemic sclerosis and iPPSD2): 1) the evolution of ectopic calcification/ossification volume between (i) time of inclusion, (ii) the end of the run-in period (i.e. 6 months after inclusion) and (iii) after 6 months of local injections of STS) 2) the safety of local injections of STS during 6 months of treatment 3) the evolution of the ectopic calcification/ossification density in between (i) time of inclusion, (ii) the end of the run-in period (6 months after inclusion) and (iii) after 6 months of local injections of STS 4) the percentage of clinically pertinent variation of pain after the 6 month run-in period and after the 6 months of local injections of STS 5) the percentage of clinically pertinent variation of quality of life after the 6-month run-in period and after the 6 months of local injections of STS | — |
Secondary
| Measure | Time frame |
|---|---|
| Timepoint(s) of evaluation of this end point: Month 6 Month 12 ; Secondary end point(s): 1) Volume of the treated calcifications / ossifications at (i) inclusion (M0), the end of the run-in period (M6) and after 6 months of local injections of STS (M12) in each disease, evaluated on CT-scan measurements. 2) (i) Adverse events (clinical and biological): causality, severity, and seriousness (outcome, drug discontinuation or drug reduction due to AE) during the 6 months of STS treatment.(ii) Trabecular bone density calculated through measurements of Hounsfield units level within the bone facing the treated area on M0, M6 and M12 CT evaluations. (iii) pain associated with STS infusion or injections. 3) Hounsfield density of the treated ectopic calcifications/ossifications at (i) inclusion (M0), the end of the run-in period (M6) and after 6 months of local injections of STS (M12), evaluated on CT-scan measurements. 4) Percentage of clinically pertinent variation in pain evaluated with pain scales: difference in HEDEN = 2 (2-7 years old), difference in VAS score = 2 (> 7 years old) between M0-M6 and M6-M12. 5) Percentage of clinically pertinent variation in quality of life evaluated with quality of life scales: difference in PedsQL Scale = 5 (2 years old -18 years old, using appropriates reports), difference in SF36 score = 20 (> 18 years old) between M0-M6 and M6-M12. | — |
Countries
France
Contacts
Public ContactDirection Research and Innovation
CHU de LIMOGES
Outcome results
None listed