Ectopic soft tissue calcifications or ossifications can complicate the course of numerous diseases; most of them are rare or very rare. Even if the clinical, radiological and pathological presentation of ectopic calcifications and ossifications are different, the same hypotheses are discussed considering their hypothetical pathophysiology. Indeed, high calcium phosphate product, local cellular lesions and abnormal transdifferentiation of mesenchymal cells are regularly evoked when pathophysiology of such calcifications or ossifications are discussed. Apart from several case reports that have not been confirmed so far, no medical treatments are available, leading to significant pain and impairment of quality of life for patients. Therefore, only surgical treatment can be proposed when the volume or the consequences of these calcifications/ossifications become too important. Sodium thiosulfate (STS) is currently used as a cyanide poisoning antagonist and a chemoprotectant against adverse effects of several chemotherapies such as Cisplatin. Numerous case reports and several studies have revealed the potential interest of STS in the treatment of uremic induced vascular or soft tissues calcifications. Recently, our group has developed an expertise in the use of STS for the treatment of ectopic soft tissue calcifications or ossifications. Considering these promising preliminary data, and their limits, we developed a strategy to treat soft tissue calcifications or ossifications based on a local administration of STS. The first results of this therapeutic strategy are highly promising and the local or systemic safety is satisfactory so far. These preliminary data also reported by others deserve to be confirmed in a prospective study. We propose in this project to conduct a prospective open controlled phase II trial in order to assess the efficacy and the safety of intralesional administration of STS for the treatment of calcifications secondary to dermatomyositis or systemic sclerosis and ectopic ossifications secondary to pseudo-hypoparathyroidism 1a type (PHP1A/iPPSD2) (inactivating parathyroid hormone / parathyroid-hormone-related peptid (PTH/PTHrP) signalling disorder).
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
40
M0-M6 (run-in phase): medical care and follow-up as usual M6-M12 (STS phase): * Patients with iPPSD2 will be treated with subcutaneous infusion using a portable pump. * Patients with dermatomyositis or systemic sclerosis will be treated with repeated injections every two weeks. Patients will receive a maximal total number of 11 STS injections. M12: final visit (V5): clinical evaluation, photograph and CT scan of the treated lesion, pain and quality of life evaluation.
CHU de BORDEAUX
Bordeaux, France
RECRUITINGHospice Civil de Lyon
Bron, France
NOT_YET_RECRUITINGApHp - Hôpital Bicêtre
Le Kremlin-Bicêtre, France
RECRUITINGCHU de Limoges
Limoges, France
RECRUITINGApHp - hôpital Lariboisière
Paris, France
RECRUITINGHôpital de la Pitié Salpétriêre
Paris, France
NOT_YET_RECRUITINGCHU de ROUEN
Rouen, France
NOT_YET_RECRUITINGCHU de Toulouse
Toulouse, France
RECRUITINGChange of the percentage of volume of the treated calcifications / ossifications
Calculation of percentage of volume evolution of the treated calcification / ossification between the beginning and the end of STS treatment, in each of the three diseases (dermatomyositis, systemic sclerosis and iPPSD2), evaluated on CT-scan measurements.
Time frame: between Month 6 and Month 12
Change of the volume of the treated calcifications / ossifications
Calculation of volume of the treated calcification / ossification at (i) inclusion, the end of the run-in period (6 month) and after 6 months of local injections of STS in each disease (12 month), evaluated on CT-scan measurements.
Time frame: Month 0, Month 6 and Month 12
Adverse events
Collection of adverse events (clinical and biological): causality, severity, and seriousness during the STS treatment.
Time frame: Month 12
Change of the Hounsfield density of the treated ectopic calcifications/ossifications
Hounsfield density analysis of the treated ectopic calcifications/ossifications at inclusion, the end of the run-in period (6 month) and after 6 months of local injections of STS (12 month), evaluated on CT-scan measurements.
Time frame: Month 0, Month 6 and Month 12
Change of the percentage of patient with a clinically pertinent variation in pain
Calculation of Percentage of patients with a clinically pertinent variation in pain evaluated with pain scales: difference in hetero-evaluation scale of pain in children (HEDEN) ≥ 2 (2-7 years old) between M0-M6 and M6-M12
Time frame: Between Month0 and Month 6 and Between Month 6 and Month 12
Change of the percentage of patient with a clinically pertinent variation in pain
Calculation of Percentage of patients with a clinically pertinent variation in pain evaluated with pain scales: difference in visual analogue pain intensity scale (VAS) score ≥ 2 (\> 7 years old) between M0-M6 and M6-M12
Time frame: Between Month0 and Month 6 and Between Month 6 and Month 12
Change of the percentage of patients with a clinically pertinent variation in quality of life
Calculation of percentage of patients with a clinically pertinent variation in quality of life : difference in PedsQL ≥ 5 (2-18 years old, using appropriates reports) between M0-M6 and M6-M12
Time frame: Between Month0 and Month 6 and Between Month 6 and Month 12
Change of the percentage of patients with a clinically pertinent variation in quality of life
Calculation of percentage of patients with a clinically pertinent variation in quality of life : difference in Short Form 36 (SF36) score ≥ 20 (\> 18 years old) between M0-M6 and M6-M12
Time frame: Between Month0 and Month 6 and Between Month 6 and Month 12
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