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

NCT ID: NCT03582800

Last Updated: 2025-04-04

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 40 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-01-06
• Study Completion Date: 2027-06-06

Brief 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).

Conditions

Systemic Sclerosis; Dermatomyositis; iPPSD2

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Treated

M0-M6: run-in phase (control) M6-M12: STS treatment phase

Group Type: EXPERIMENTAL

STS

Intervention Type: DRUG

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.

Interventions

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.

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• 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

Exclusion Criteria

• Allergy to STS 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

• Minimum Eligible Age: 6 Months
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

University Hospital, Limoges

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Vincent GUIGONIS, MD

Role: PRINCIPAL_INVESTIGATOR

University Hospital, Limoges

Locations

CHU de BORDEAUX

Bordeaux, , France

Site Status: RECRUITING

Hospice Civil de Lyon

Bron, , France

Site Status: NOT_YET_RECRUITING

ApHp - Hôpital Bicêtre

Le Kremlin-Bicêtre, , France

Site Status: RECRUITING

CHU de Limoges

Limoges, , France

Site Status: RECRUITING

ApHp - hôpital Lariboisière

Paris, , France

Site Status: RECRUITING

Hôpital de la Pitié Salpétriêre

Paris, , France

Site Status: NOT_YET_RECRUITING

CHU de ROUEN

Rouen, , France

Site Status: NOT_YET_RECRUITING

CHU de Toulouse

Toulouse, , France

Site Status: RECRUITING

Countries

France

Central Contacts

Vincent GUIGONIS, MD

Role: CONTACT

vincent.guigonis@unilim.fr

555056358 ext. +33

Claire BAHANS

Role: CONTACT

claire.bahans@chu-limoges.fr

Facility Contacts

Marie-Elise TRUCHETET, MD

Role: primary

marie-elise.truchetet@chu-bordeaux.fr

Justine BACCHETTA, MD

Role: primary

justine.bacchetta@chu-lyon.fr

Agnès LINGLART, MD

Role: primary

agnes.linglart@aphp.fr

Vincent GUIGONIS, MD

Role: primary

vincent.guigonis@unilim.fr

Claire BAHANS

Role: backup

claire.bahans@chu-limoges.fr

Korng EA, MD

Role: primary

korngea@yahoo.fr

Olivier BENVENISTE, MD

Role: primary

olivier.benveniste@aphp.fr

+33142161088

Mireille CASTANET, MD

Role: primary

mireille.castanet@chu-rouen.fr

232880250 ext. +33

Valérie PORQUET-BORDES, MD

Role: primary

porquet-bordes.v@chu-toulouse.fr

Other Identifiers

I17004 (ITS-PILOT)

Identifier Type: -

Identifier Source: org_study_id