Идёт набор NCT03582800

Subcutaneous Injection of Sodium Thiosulfate for Ectopic Calcifications or Ossifications. A Pilot Study

Фаза II С лечением Systemic Sclerosis Dermatomyositis iPPSD2

Ориентир для пациента и семьи

Простыми словами

Автоматическая сводка по структурированным данным реестра. Она помогает сориентироваться, но не заменяет официальный протокол или оценку врача.

Что изучают: В протоколе указаны: STS.
Кому может быть актуально: Состояния в реестре: Systemic Sclerosis, Dermatomyositis, iPPSD2. Базовые параметры: от 6 мес. · Все.
Что важно проверить: Возраст, диагноз и пол — только базовые ориентиры. Предыдущее лечение, анализы и другие обязательные условия указаны ниже в критериях участия.
Где проводится: Франция
Следующий шаг: Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →

Не всё понятно в терминах? Прочитайте наш гид для пациентов →

Обзор

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).

Вмешательства

Препарат STS
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.

Первичные конечные точки

Change of the percentage of volume of the treated calcifications / ossifications [Срок оценки: between Month 6 and Month 12]

Вторичные конечные точки (7)

Change of the volume of the treated calcifications / ossifications [Срок оценки: Month 0, Month 6 and Month 12]
Adverse events [Срок оценки: Month 12]
Change of the Hounsfield density of the treated ectopic calcifications/ossifications [Срок оценки: Month 0, Month 6 and Month 12]
Change of the percentage of patient with a clinically pertinent variation in pain [Срок оценки: 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 [Срок оценки: 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 [Срок оценки: 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 [Срок оценки: Between Month0 and Month 6 and Between Month 6 and Month 12]

Критерии участия

Критерии включения

Patient presenting with:

ectopic ossification secondary to iPPSD2 or
ectopic calcification secondary to dermatomyositis or
ectopic calcification secondary to systemic sclerosis
Patient aged 2 years or over
Indication of STS infusion 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)
Men with women of childbearing potential partners should use condoms during the whole treatment period and until 91 days after the last injection.
Informed consent signed by the patient / parents
Patient affiliated to the social security system