Subcutaneous Injection of Sodium Thiosulfate for Ectopic Calcifications or Ossifications. A Pilot Study
Status:
Recruiting
Trial end date:
2023-03-01
Target enrollment:
Participant gender:
Summary
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).