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NCT03267277

Sodium Thiosulfate for Treatment of Calcinosis Associated With Juvenile and Adult Dermatomyositis

Completed Phase 2, PHASE3 Results posted Last updated 26 November 2024
What this trial tests

Phase 2, PHASE3 trial testing Sodium Thiosulfate in Dermatomyositis in 15 participants. Completed in 9 November 2023.

Timeline
5 October 2017
Primary endpoint
4 November 2022
9 November 2023

Quick facts

Lead sponsorNational Institute of Environmental Health Sciences (NIEHS)
PhasePhase 2, PHASE3
StatusCompleted
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment15
Start date5 October 2017
Primary completion4 November 2022
Estimated completion9 November 2023
Sites1 location across United States

Drugs / interventions tested

Conditions studied

Sponsor

National Institute of Environmental Health Sciences (NIEHS)

Who can join

7 and older, any sex, with Dermatomyositis or Idiopathic Inflammatory Myopathies. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Change in Calcinosis Activity Visual Analogue Scale Score Primary · Week 10 minus week 0 (on therapy) and week 0 minus week -10 (baseline)

Calcinosis activity is defined by the metabolic activity and the inflammation associated with calcinosis. In evaluating calcinosis activity, the study physicians take into account the change in extent of calcinosis and the location of calcinosis lesions, the consistency and texture of calcinosis lesions, the presence of erythema surrounding calcinosis lesion, and any pain associated with the calcinosis lesions. A 10 cm visual analogue scale (VAS) was scored by a physician with a vertical line on the scale marking calcinosis activity where 0 cm indicates no evidence of calcinosis, and 10 cm mar

GroupValue95% CI
Treatment-3.45± 0.89
Change in Quality of Life Measured by the Child Health Questionnaire-Parent Form 50 (CHQ-PF50): Physical Function Domain Secondary · Week -10 to 0 (pre-treatment); Week 0 to 10 (on treatment)

The Child Health Questionnaire-Parent Form 50 (CHQ-PF50) is a 50-item survey with 14 domains that parents complete to assess their child's physical and mental well-being. The change in quality of life was measured by the physical functioning domain score on the CHQ-PF50. The CHQ-PF50 physical functioning domain scale was transformed to 0 to 100 score with higher score indicating better health or more positive functioning. The change in quality of life using the CHQ-PF50 score was measured as the mean difference in scores between time points.

Pre-treatment
GroupValue95% CI
Treatment-1.00± 6.15
On treatment
GroupValue95% CI
Treatment-8.00± 11.60
Change in Quality of Life Measured by the 36-Item Short Form Health Survey (SF-36) Score: General Health Domain Secondary · Week -10 to 0 (pre-treatment); Week 0 to 10 (on treatment); Week 24 to 62 (post-treatment)

36-Item Short Form Health Survey (SF-36) is a 36-item patient-reported survey of patient health status that consists of eight scaled scores, which are the weighted sums of the questions in their section. Each scale is directly transformed into a 0-100 scale. The Change in quality of life measured was assessed using the general health domain of SF-36. The higher the score the less disability. The change in quality of life using the SF-36 general health domain was measured as the mean difference in scores between time points.

Pre-treatment
GroupValue95% CI
Treatment2.50± 7.17
On treatment
GroupValue95% CI
Treatment-1.00± 6.15
Post treatment
GroupValue95% CI
Treatment-8.00± 11.60
Improvement of Calcinosis Lesions Measured by the Change in Mawdsley Calcinosis Questionnaire (MCQ) Score Secondary · Week -10 to 0 (pre-treatment); Week 0 to 10 (on treatment); Week 24 to 62 (post-treatment)

The Mawdsley Calcinosis Questionnaire (MCQ) is a 17-item questionnaire scale that measures the severity and impact of calcinosis. Each item is scored on 11-point scale of 0 to 10. Total score is the the average of the cumulative measure ranging from 0-10. Higher score indicates worse severity and impact of calcinosis. Improvement of calcinosis lesions was measured by the MCQ. The change in score was measured as the mean difference between time points.

Pre-treatment
GroupValue95% CI
Treatment0.33± 1.54
On treatment
GroupValue95% CI
Treatment-0.74± 2.18
Post treatment
GroupValue95% CI
Treatment-0.17± 0.86
Change in Quality of Life Measured by Skindex-29 Score Secondary · Week -10 to 0 (pre-treatment); Week 0 to 10 (on treatment); Week 24 to 62 (post-treatment)

The Skindex-29 scale is 29-item questionnaire that measures the quality of life. Each item is scored 0 (Never) to 4 (All the time). All responses are transformed to a linear scale of 100, varying from 0 (no effect) to 100 (effect experienced all the time) and overall score is the mean of the responses. Higher score indicates more severe impact on quality of life. The change in quality of life was measured as the mean difference in Skindex-29 scores between time points.

Pre-treatment
GroupValue95% CI
Treatment1.77± 6.30
On treatment
GroupValue95% CI
Treatment-8.15± 10.63
Post treatment
GroupValue95% CI
Treatment-0.38± 6.53
Change in Muscle Strength Over Time Measured by Manual Muscle Test-8 (MMT-8) Score Secondary · Week -10 to 0 (pre-treatment); Week 0 to 10 (on treatment); Week 24 to 62 (post-treatment)

Manual Muscle Test-8 (MMT-8) is a measure of muscle strength. The MMT-8 uses a 10-point scale to score each muscle group, with 0 indicating extreme weakness and 10 indicating normal strength. The scores for each muscle group are then added together to get a total score ranging from 0 to 80, with higher scores indicating greater muscle strength. Change in muscle strength over time was measured as the mean difference in MMT-8 scores between time points.

Pre-treatment
GroupValue95% CI
Treatment-0.08± 3.40
On treatment
GroupValue95% CI
Treatment1.38± 2.79
Post treatment
GroupValue95% CI
Treatment-0.27± 3.29
Change in Muscle Strength Over Time Measured by Quantitative Muscle Assessment (QMA): Hip Abductor Muscle Secondary · Week -10 to 0 (pre-treatment); Week 0 to 10 (on treatment); Week 24 to 62 (post-treatment)

Quantitative muscle assessment (QMA) tests how much force participant can be exerted by the hip abductor muscle. It is measured as the kilograms of force produced. Higher score indicates more force. Change in hip abductor muscle strength over time was measured by the quantitative muscle assessment (QMA) as the mean difference in QMA scores between time points.

Pre-treatment
GroupValue95% CI
Treatment0.16± 1.26
On treatment
GroupValue95% CI
Treatment0.74± 1.75
Post treatment
GroupValue95% CI
Treatment1.35± 1.77
Change in Myositis Activity Measured by Physician Global Activity (PGA) Secondary · Week -10 to 0 (pre-treatment); Week 0 to 10 (on treatment); Week 24 to 62 (post-treatment)

The Physician Global Activity (PGA) is an index that uses a 10-cm Visual Analog Scale (VAS) to score a patient's disease activity. Higher score indicates more activity. Change in myositis activity was measured as the mean difference in disease activity scores between time points.

Pre-treatment
GroupValue95% CI
Treatment0.36± 1.54
On treatment
GroupValue95% CI
Treatment-0.87± 1.27
Post treatment
GroupValue95% CI
Treatment-0.32± 0.47
Change in Myositis Damage Measured by Physician Global Damage (PGD) Secondary · Week -10 to 0 (pre-treatment); Week 0 to 10 (on treatment); Week 24 to 62 (post-treatment)

The Physician Global Damage (PGD) is an index that uses a 10-cm Visual Analog Scale (VAS) to score a patient's disease damage. Higher score indicates more global damage. Change in myositis damage was measured as the mean difference in disease damage scores between time points.

Pre-treatment
GroupValue95% CI
Treatment-0.02± 1.09
On treatment
GroupValue95% CI
Treatment-1.07± 1.09
Post treatment
GroupValue95% CI
Treatment-0.18± 0.53
Change in Total Percent Body Surface Area (BSA) Involved With Calcinosis Secondary · Week -10 to 0 (pre-treatment); Week 0 to 10 (on treatment); Week 24 to 62 (post-treatment)

Total percent of body surface area (BSA) involved with calcinosis was measured by physician assessment. Improvement of calcinosis lesions was measured as the mean difference in total percent of BSA scores between time points

Pre-treatment
GroupValue95% CI
Treatment2.79± 4.06
On treatment
GroupValue95% CI
Treatment-10.18± 5.01
Post treatment
GroupValue95% CI
Treatment-0.45± 2.24

Adverse events — posted to ClinicalTrials.gov

Time frame: 62 weeks. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Treatment
Serious: 6/14 (43%)
Deaths: 0/14

Serious adverse events (10 terms)

ReactionSystemTreatment
AnemiaBlood and lymphatic system disorders
Catheter site infectionInfections and infestations
Device related infectionInfections and infestations
Foreign bodyInjury, poisoning and procedural complications
Lymphocyte count decreasedInvestigations
HypokalemiaMetabolism and nutrition disorders
HypomagnesemiaMetabolism and nutrition disorders
Blood calcium decreasedInvestigations
Calcium ionized decreasedInvestigations
Red blood cell count decreasedInvestigations
Other adverse events (237 terms — click to expand)

ReactionSystemTreatment
Flow cytometryInvestigations
Venous oxygen saturation decreasedInvestigations
Venous oxygen saturation increasedInvestigations
HypertensionVascular disorders
Systolic hypertensionVascular disorders
AnemiaBlood and lymphatic system disorders
Blood chloride increasedInvestigations
Blood lactate dehydrogenase increasedInvestigations
Creatinine urine decreasedInvestigations
Red blood cell count decreasedInvestigations
Urine sodium increasedInvestigations
HyperglycemiaMetabolism and nutrition disorders
HypokalemiaMetabolism and nutrition disorders
HyponatremiaMetabolism and nutrition disorders
Immature granulocyte percentage increasedInvestigations
Diastolic hypotensionVascular disorders
Urine protein, quantitative increasedInvestigations
Urine analysis abnormalBlood and lymphatic system disorders
Coagulation factor VIII level increasedInvestigations
Red cell distribution width increasedInvestigations
Urine magnesium decreasedInvestigations
Urine protein/creatinine ratio increasedInvestigations
White blood cells urine increasedInvestigations
Blood bicarbonate decreasedInvestigations
Blood bicarbonate increasedInvestigations
Carbon dioxide decreasedInvestigations
PCO2 decreasedInvestigations
PO2 increasedInvestigations
Mean cell hemoglobin concentration decreasedInvestigations
Blood albumin decreasedInvestigations
Blood immunoglobulin G increasedInvestigations
Immature granulocyte count increasedInvestigations
Red blood cells urine increasedInvestigations
Urine ketone body presentInvestigations
Urine phosphorus decreasedInvestigations
Vitamin D decreasedInvestigations
HypocalcemiaMetabolism and nutrition disorders
Creatinine renal clearance decreasedInvestigations
Venous blood pH decreasedInvestigations
Sinus tachycardiaCardiac disorders

Most-reported serious reactions: Anemia, Catheter site infection, Device related infection, Foreign body, Lymphocyte count decreased, Hypokalemia, Hypomagnesemia, Blood calcium decreased.

Data from ClinicalTrials.gov NCT03267277 adverse events section.

Sponsor's own description

Background: Dermatomyositis (DM) and juvenile dermatomyositis (JDM) cause inflammation in the muscles. People with DM and JDM can develop calcium deposits in places they should not, known as calcinosis. Calcinosis can be painful and cause disabilities and other problems. Researchers want to learn more about calcinosis to find treatments for it. Objective: To test if sodium thiosulfate (STS) can treat people with DM with calcinosis. Eligibility: People ages 7 and older who have moderate or severe calcinosis. They must have stable DM and calcium deposits in the torso or at least 2 limbs. Design: Participants will be screened with: * Medical history * Physical exam * Muscle strength and function tests * Blood and urine tests Participants will have several visits: * 7-day pre-treatment visit about 10 weeks before starting STS * Treatment visits over 10 weeks. They will get STS 3 times a week through IV infusion. They may be hospitalized the whole time. If they tolerate the drug, they may be discharged at certain times. During these times, they will return for the infusions. * 3- to 5-day post-treatment visits 24 weeks and 62 weeks after starting STS. Visits may include repeats of screening tests and: * Questionnaires * Scans: They lie in a machine that takes pictures of the body. They may be injected with a radioactive agent. * Durometry: A small instrument applies pressure on the skin or exposed calcinosis. * Measurements of blood flow in the arms and fingernail blood vessels * Photographs of the skin * Kidney ultrasound * Tests of kidney function * Calcinosis aspiration: A needle placed into areas of calcinosis removes liquid.

Publications & conference data

2 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Calcinosis in juvenile dermatomyositis: Updates on pathogenesis and treatment.
    Pinotti CS, Cannon L, Dvergsten JA, Wu EY. · · 2023 · cited 9× · PMID 36936211 · DOI 10.3389/fmed.2023.1155839
  2. A glance into the future of myositis therapy.
    Chiapparoli I, Galluzzo C, Salvarani C, Pipitone N. · · 2022 · cited 4× · PMID 35634354 · DOI 10.1177/1759720x221100299

Verify or expand the search:

Other trials of Sodium Thiosulfate

Trials testing the same drug.

Other recruiting trials for Dermatomyositis

Currently open trials in the same condition.

Other National Institute of Environmental Health Sciences (NIEHS) trials

Trials by the same sponsor.

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Data sources for this page

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Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing