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

NCT03582800 | Recruiting Phase 2

• 1 other identifier: I17004 (ITS-PILOT)
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 8

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

Keywords

Sodium thiosulfate; Ectopic calcification; Ectopic Ossification

Outcome Measures

Primary Outcomes (1)

• Change 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.

  between Month 6 and Month 12

Secondary Outcomes (7)

• Change of the volume of the treated calcifications / ossifications

  Month 0, Month 6 and Month 12

• Adverse events

  Month 12

• Change of the Hounsfield density of the treated ectopic calcifications/ossifications

  Month 0, Month 6 and Month 12

• Change of the percentage of patient with a clinically pertinent variation in pain

  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

  Between Month0 and Month 6 and Between Month 6 and Month 12

Study Arms (1)

Treated

EXPERIMENTAL

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

Drug: STS

Interventions

STS 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.

Treated

Eligibility Criteria

• Age: 6 Months+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• University Hospital, Limoges: lead

Study Sites (8)

CHU de BORDEAUX

Bordeaux, 33000, France

RECRUITING

Hospice Civil de Lyon

Bron, 69500, France

NOT YET RECRUITING

ApHp - Hôpital Bicêtre

Le Kremlin-Bicêtre, 94270, France

RECRUITING

CHU de Limoges

Limoges, 87042, France

RECRUITING

ApHp - hôpital Lariboisière

Paris, 75000, France

RECRUITING

Hôpital de la Pitié Salpétriêre

Paris, 75651, France

NOT YET RECRUITING

CHU de ROUEN

Rouen, 76031, France

NOT YET RECRUITING

CHU de Toulouse

Toulouse, 31000, France

RECRUITING

MeSH Terms

Conditions

Scleroderma, Systemic; Dermatomyositis; Ossification, Heterotopic

Condition Hierarchy (Ancestors)

Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases; Polymyositis; Myositis; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Vincent GUIGONIS, MD

  University Hospital, Limoges

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Vincent GUIGONIS, MD

CONTACT

555056358; vincent.guigonis@unilim.fr

Claire BAHANS

CONTACT

claire.bahans@chu-limoges.fr

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 13, 2018
• First Posted: July 11, 2018
• Study Start: January 6, 2020
• Primary Completion (Estimated): June 6, 2029
• Study Completion (Estimated): June 6, 2029
• Last Updated: July 20, 2026

Record last verified: 2026-07

Locations

FR (8)