Subcutaneous Injections of Sodium Thiosulfate for ectopic calcifications or ossifications. A pilot study.

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Centre Hospitalier Et Universitaire De Limoges

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Skin and Connective Tissue Diseases [C17]

Trial duration

5 Jun 2020 → ongoing

Decision date (initial)

2024-03-22

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

DGOS

External identifiers

EU CT number

2024-511620-13-00

EudraCT number

2018-001978-22

WHO UTN

U1111-1304-1541

ClinicalTrials.gov

NCT03582800

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy

To evaluate, after a run-in period of 6 months, the efficacy of a 6-month treatment of 10% STS solution when locally injected for the treatment of calcifications (secondary to dermatomyositis and systemic sclerosis) and ectopic ossifications (secondary to iPPSD2)

Secondary objectives 5

1. the evolution of ectopic calcification / ossification volume in between (i) time of inclusion, (ii) the end of the run-in period (6 months after inclusion) and (iii) after 6 months of local injections of STS
2. the safety of local injections of STS during 6 months of treatment
3. the evolution of the ectopic calcification/ossification density in between (i) time of inclusion, (ii) the end of the run-in period (6 months after inclusion) and (iii) after 6 months of local injections of STS
4. the percentage of clinically pertinent variation of pain after the 6-month run-in period and after the 6 months of local injections of STS
5. the percentage of clinically pertinent variation of quality of life after the 6 month run-in period and after the 6 months of local injections of STS

Conditions and MedDRA coding

Patient presenting with: - ectopic ossification secondary to iPPSD2 or - ectopic calcification secondary to dermatomyositis or - ectopic calcification secondary to systemic sclerosis

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

1. Patient presenting with ectopic calcification secondary to dermatomyositis or ectopic calcification secondary to systemic sclerosis And aged 18 years old or higher or ectopic ossification secondary to iPPSD2 And aged ≥ 2 years old and ≤ 30 years old
2. Indication of STS infusion validated by a multidisciplinary committee, based on the significant morbidity and/or functional impact of the targeted calcification/ossification
3. Patient with no planned surgery of the calcifications/ossifications for the twelve coming months
4. 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). Contraception will be extended up to one month after the last injection. Men with women of childbearing potential partners should use condoms during the whole treatment period and until 91 days after the last injection
5. Informed consent signed by the patient / parents
6. Patient affiliated to (or beneficiary of) the social security system

Exclusion criteria 10

1. Allergy to STS, sulfites or one of the excipients used
2. Contraindication to local injection of STS
3. Anticoagulant therapy
4. Patient deprived of freedom by a court judgment or an administrative decision
5. Patient undergoing psychiatric care under coercion
6. Legally protected adult patients (guardianship / curatorship)
7. Adult patient unable to give consent
8. Patient placed under judicial protection
9. Ongoing treatment by STS, including topical forms
10. Pregnant, parturient or breastfeeding woman

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Percentage of volume evolution of the treated calcifications / ossifications between the beginning (M6) and the end (M12) of STS treatment, in each of the three diseases (dermatomyositis, systemic sclerosis and iPPSD2), evaluated on CT-scan measurements

Secondary endpoints 5

1. Volume of the treated calcifications / ossifications at (i) inclusion (M0), the end of the run-in period (M6) and after 6 months of local injections of STS (M12) in each disease, evaluated on CT-scan measurements
2. (i) Adverse events (clinical and biological): causality, severity, and seriousness (outcome, drug discontinuation or drug reduction due to AE) during the 6 months of STS treatment. (ii) Trabecular bone density calculated through measurements of Hounsfield units level within the bone facing the treated area on M0, M6 and M12 CT evaluations. (iii) pain associated with STS infusion or injections
3. Hounsfield density of the treated ectopic calcifications/ossifications at (i) inclusion (M0), the end of the run-in period (M6) and after 6 months of local injections of STS (M12), evaluated on CT-scan measurements
4. Percentage of patients with a clinically pertinent variation in pain evaluated with pain scales:, difference in HEDEN ≥ 2 (2-7 years old), difference in VAS score ≥ 2 (> 7 years old) between M0-M6 and M6-M12.
5. Percentage of patients with a clinically pertinent variation in quality of life evaluated with quality of life scales: difference in PedsQL scale ≥ 5 (2 years old -18 years old, using appropriates reports), difference in SF36 score ≥ 20 (> 18 years old) between M0-M6 and M6-M12

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Centre Hospitalier Et Universitaire De Limoges

Sponsor organisation

Centre Hospitalier Et Universitaire De Limoges

Address

2 Avenue Martin Luther King

City

Limoges

Postcode

87000

Country

France

Scientific contact point

Organisation

Centre Hospitalier Et Universitaire De Limoges

Contact name

Coordinating Investigator

Public contact point

Organisation

Centre Hospitalier Et Universitaire De Limoges

Contact name

project manager

Locations

1 EU/EEA country · 12 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 12 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

7 submissions — initial application plus substantial / non-substantial modifications