SODIUM THIOSULFATE PENTAHYDRATE for Ectopic ossification (iPPSD2) | Calcinosis / ectopic calcification secondary to dermatomyositis | Calcinosis / ectopic calcification secondary to systemic sclerosis

Inclusion criteria

• {"criterion_text":"- 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 ≤ 30 years old\n- Indication of STS infusion validated by a multidisciplinary committee, based on the significant morbidity and/or functional impact of the targeted calcification/ossification\n- Patient with no planned surgery of the calcifications/ossifications for the twelve coming months\n- 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. Men with women of childbearing potential partners should use condoms during the whole treatment period and until 91 days after the last injection\n- Informed consent signed by the patient / parents\n- Patient affiliated to (or beneficiary of) the social security system"}

Exclusion criteria

• {"criterion_text":"- Allergy to STS, sulfites or one of the excipients used\n- Pregnant, parturient or breastfeeding woman\n- Contraindication to local injection of STS\n- Anticoagulant therapy\n- Patient deprived of freedom by a court judgment or an administrative decision\n- Patient undergoing psychiatric care under coercion\n- Legally protected adult patients (guardianship / curatorship)\n- Adult patient unable to give consent\n- Patient placed under judicial protection\n- Ongoing treatment by STS, including topical forms"}

Primary endpoints

• {"endpoint_text":"- 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","definition_or_measurement_approach":"Volume change measured by CT-scan measurements comparing treated calcifications/ossifications at start of STS treatment (M6) and end of treatment (M12), reported as percentage of volume evolution for each disease group."}

Secondary endpoints

• {"endpoint_text":"- 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","definition_or_measurement_approach":"Absolute volumes measured on CT-scan at M0, M6, and M12."}
• {"endpoint_text":"- (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","definition_or_measurement_approach":"(i) AE collection with causality, severity and seriousness adjudication, including discontinuation or dose reduction; (ii) trabecular bone density measured by Hounsfield units on CT at M0, M6, M12; (iii) pain related to administration recorded (scales not specified here)."}
• {"endpoint_text":"- 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","definition_or_measurement_approach":"Hounsfield unit measurements of the treated calcifications/ossifications on CT at M0, M6 and M12."}
• {"endpoint_text":"- Percentage of patients with a 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.","definition_or_measurement_approach":"Pain measured by HEDEN for ages 2-7 (clinically relevant change ≥2) and VAS for >7 years (change ≥2); percentage of patients meeting these thresholds between M0-M6 and M6-M12."}
• {"endpoint_text":"- Percentage of patients with a 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","definition_or_measurement_approach":"Quality of life assessed by PedsQL for ages 2-18 (clinically relevant change ≥5) and SF-36 for >18 years (change ≥20); percentage of patients meeting these thresholds between M0-M6 and M6-M12."}

France

Earliest CTIS Part Ii Submission Date

04-03-2024

Latest Decision Or Authorization Date

13-03-2026

Processing Time Days

739

Number Of Sites

12

Number Of Participants

40

Primary sponsor

Full Name

Centre Hospitalier Et Universitaire De Limoges

Organisation Type

Hospital/Clinic/Other health care facility

Country Of Registered Address

France