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

• Register: ClinicalTrials.gov
• Last refreshed on: 4 March 2024
• Main ID: NCT03267277
• Date of registration: 29/08/2017
• Prospective Registration: Yes
• Primary sponsor: National Institute of Environmental Health Sciences (NIEHS)
• Public title: Sodium Thiosulfate for Treatment of Calcinosis Associated With Juvenile and Adult Dermatomyositis
• Scientific title: An Open-label Study of Sodium Thiosulfate for Treatment of Calcinosis Associated With Juvenile and Adult Dermatomyositis
• Date of first enrolment: October 5, 2017
• Target sample size: 15
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT03267277
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2/Phase 3

Countries of recruitment

United States

Contacts

• Name: Adam I Schiffenbauer, M.D.
• Affiliation: National Institute of Environmental Health Sciences (NIEHS)

Key inclusion & exclusion criteria

- INCLUSION CRITERIA:

1. At least 7 years of age

2. Meets Bohan and Peter criteria, as modified by the International Myositis
Assessment and Clinical Studies Group (IMACS), for probable or definite DM or JDM

3. Has extensive calcinosis, defined as calcinosis involving at least 2 extremities
or the torso

4. Has moderate to severe calcinosis, defined as having a calcinosis activity visual
analogue scale score of greater than or equal to 3.5 cm out of 10 cm

5. Is willing and able to comply with the requirements of the protocol and to
undergo all testing

6. Can have IV access established to receive study infusions

7. Myositis disease activity is stable*

8. Medications for myositis are stable for at least 6 weeks prior to study entry**

9. Men and women of reproductive potential must agree to use a reliable form of
birth control during the 62-week duration of the study

10. Subjects or their legal guardian must sign a written informed consent

- Stable myositis disease activity will be defined by physician global and
patient/parent global VAS that are <4 cm, as well as creatine kinase (CK),
lactate dehydrogenase (LDH), aldolase, aspartate aminotransferase (AST), and
alanine aminotransferase (ALT) that are less than or equal to 2X upper limit
of normal (ULN).

- If a patient has a medication for myositis changed in this window for
reasons besides

their myositis activity and has returned to their baseline medication use prior to

enrollment they will still be eligible.

EXCLUSION CRITERIA:

1. Is pregnant or breastfeeding

2. Has known allergies to sodium thiosulfate, any of its components, or dextrose

3. Has severe myositis disease activity as defined by patient/parent or physician global
activity visual analogue scale score >4 cm out of 10 cm

4. Has had an escalation of immunosuppressive therapy in the 2 months prior to enrollment
for the purpose of treating active myositis disease activity, including the addition
of a new agent to treat the patients underlying disease or an increase in dose of an
existing medication used to treat the patient s disease (other than an adjustment for
weight or body surface area in children)

5. Has a malignancy or had a malignancy within 5 years of diagnosis of their DM (except
for benign skin lesions or basal cell carcinoma)

6. Known or suspected history of alcohol or drug abuse in the 6 months prior to study
enrollment

7. Has systemic lupus erythematosus, scleroderma, or a condition other than DM that is
associated with calcinosis as a complication

8. Has had a change in medications used specifically for calcinosis in the 2 months prior
to enrollment, including but not limited to alendronate, etidronate, pamidronate,
probenecid, colchicine, diltiazem, thalidomide, and aluminum hydroxide

9. Has used probenecid, diltiazem, aluminum hydroxide, or hydrochlorothiazide in the 2
months prior to enrollment

10. Has currently or has a history of any of the following: heart failure, renal
impairment (GFR less than 30 representing severe renal disease), liver disease
(Child-Pugh class C), arrhythmias (that are symptomatic or are concerning for
progression to symptomatic arrhythmias), or recurrent kidney stones (more than one
episode of symptomatic kidney stones separated by at least 1 month), or QT
prolongation, or hypocalcemia, or metabolic acidosis, or hypotension

11. Has severe osteoporosis or has had a bone fracture within a year prior to enrollment.
For adults, severe osteoporosis as defined by the World Health Organization (WHO) as
bone mineral density (BMD) 2.5 standard deviations below that of a young, normal adult
(T-score at or below -2.5 and one or more fractures). For individuals, less than age
18, severe osteoporosis as defined by the First Pediatric Consensus Development
Conference as a Z-score below -2 and one or more fractures.

12. Has a psychiatric illness or medical non-compliance that the study team feels will
make the patient unlikely to complete the study

13. Has dysphagia where non-oral feeding alternatives are needed.

14. Requires supplemental oxygen therapy

15. Has >3 episodes of cellulitis requiring IV antibiotics related to calcinosis within a
year prior to enrollment or cellulitis within 1 month of enrollment

16. Previously received or currently receiving sodium thiosulfate by any route

17. Is on an oral prednisone dose of more than 1mg/kg/day or other oral corticosteroid
equivalent.

18. Is taking any concomitant medications that are thought to alter sodium thiosulfate s
effects or pharmacokinetics. Once patients have met all other inclusion criteria and
no other exclusion criteria this criteria will be checked. A PharmD will evaluate the
patient's current medication list for medications with the potential for interaction
with sodium thiosulfate. Methodology is as follows: He or she will perform a search in
two individual validated medication interaction software programs. He or she will also
perform a literature search via PubMed for case reports of interactions with sodium
thiosulfate. As an additional safeguard, the PharmD will evaluate the medication list
utilizing principles of pharmacology and pharmacokinetics to attempt to identify any
potential interactions not yet documented in the literature.

19. Has any health conditions that, in the opinion of the investigator, significantly
increase the risk of taking sodium thiosulfate or participating in any of the study
procedures

20. Weighs less than 26 kilograms.**

21. Has a regimen of pulse steroids or intravenous immunoglobulin (IVIG) that is at an
interval besides every 1, 2, or 5 weeks.

22. Has a chronic infection that makes assessment of muscle disease difficult including,
but not limited to, hepatitis, HIV, HTLV 1, and HTLV 2.

23. Has had a severe complication of diabetes in the past year prior to enrollment.

24. Anemia with a hemoglobin (HgB) less than 10 at time of screening or deemed to be too
low to safely complete study by hematology consult team.

- We will attempt to enroll patients at a weight greater than 28 kg as these
patients will be able to obtain all lab work for the study. Patients weighing 26
to 28 kg will only be able to obtain some of the research blood work. Patients
less than


Age minimum: 7 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Idiopathic Inflammatory Myopathies

Dermatomyositis

Intervention(s)

Drug: Sodium Thiosulfate

Primary Outcome(s)

The Change in Calcinosis Activity Visual Analogue Scale Score [Time Frame: Week 10 minus week 0 (on therapy) and week 0 minus week -10 (baseline)]

Secondary ID(s)

17-E-0161

170161

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 26/02/2024
• URL: https://clinicaltrials.gov/ct2/show/results/NCT03267277