The Effect of High Dose Methylprednisolone on Nailfold in Early Systemic Sclerosis ( SSc )

Sponsor

Radboud University Medical Center (Other)

Overall Status

Unknown status

CT.gov ID

NCT03059979

Collaborator

(none)

Enrollment

Location

Arms

53.9

Duration (Months)

0.6

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This is a 12 week double-blind randomized placebo controlled trial in which 30 patients with very early SSc, fulfilling the Very Early Diagnosis Of Systemic Sclerosis (VEDOSS) criteria (9) will be randomized in a 2:1 fashion to receive intravenous methylprednisolone or placebo. Three-day treatment courses are given at week 0, week 4 and week 8. The final assessment is at week 12, and patients will be followed up to one year after baseline

Condition or Disease	Intervention/Treatment	Phase
Systemic Sclerosis Raynaud Phenomena	Drug: Methylprednisolone Other: sodium chloride	Early Phase 1

Detailed Description

Systemic sclerosis is a disease with a high burden caused by morbidity and increased mortality. To date a cure for SSc is not available. In this trial, patients are treated very early in the disease which could change the long term outcome of SSc in these patients.

In daily practice, patients so early in the disease course are not treated although they might be at risk for early escalation and internal organ involvement, reducing their prognosis. A trial to investigate the efficacy of a relative save, inexpensive and easy accessible treatment will provide us with the opportunity to change the disease course and reducing the disease burden of a portion of the SSc patients

Study Design

Study Type:

Interventional

Anticipated Enrollment :

30 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

Triple (Participant, Care Provider, Investigator)

Primary Purpose:

Treatment

Official Title:

Hit Hard and Early. The Effect of High Dose Methylprednisolone on Nailfold Capillary Changes and Biomarkers in Early SSc: a 12-week Randomised Explorative Double-blind Placebo-controlled Trial.

Study Start Date :

Jan 1, 2017

Anticipated Primary Completion Date :

Jul 1, 2021

Anticipated Study Completion Date :

Jul 1, 2021

Arms and Interventions

Arm	Intervention/Treatment
Active Comparator: Methylprednisolone 1000 mg the methylprednisolone is dissolved in 100 cc of sodium chloride (NaCl 0.9%) by intravenous infusion in 30 minutes on three consecutive days	Drug: Methylprednisolone methylprednisolone 1000 mg dissolved in 100 cc of NaCl 0.9%, by intravenous infusion in 30 minutes on three consecutive days Three-day treatment courses are given at week 0, week 4 and week 8. Other Names: Solu-Medrol
Placebo Comparator: sodium chloride The placebo intervention with physiologic salt solution is identical in appearance	Other: sodium chloride 100 cc of NaCl 0,9% , administered by intravenous infusion in 30 minutes on three consecutive days Three-day treatment courses are given at week 0, week 4 and week 8. Other Names: NaCl 0,9%

Arm

Intervention/Treatment

Active Comparator: Methylprednisolone 1000 mg

the methylprednisolone is dissolved in 100 cc of sodium chloride (NaCl 0.9%) by intravenous infusion in 30 minutes on three consecutive days

Drug: Methylprednisolone

methylprednisolone 1000 mg dissolved in 100 cc of NaCl 0.9%, by intravenous infusion in 30 minutes on three consecutive days Three-day treatment courses are given at week 0, week 4 and week 8.

Other Names:

Solu-Medrol

Placebo Comparator: sodium chloride

The placebo intervention with physiologic salt solution is identical in appearance

Other: sodium chloride

100 cc of NaCl 0,9% , administered by intravenous infusion in 30 minutes on three consecutive days Three-day treatment courses are given at week 0, week 4 and week 8.

Other Names:

NaCl 0,9%

Outcome Measures

Primary Outcome Measures

the change in capillary density from baseline [12 weeks]
presence of enlarged and giant capillaries, hemorrhages, loss of capillaries, disorganization of the micro vascular array, and capillary ramifications.

Secondary Outcome Measures

change in selected biomarkers: the interferon signature in peripheral blood from baseline [1 year]
Plasma biomarkers consist soluble inflammatory mediators platelet factor 4, interleukin-1β, interleukin-6, tumor necrosis factor-α, endothelin-1, intercellular adhesion molecule-1 and vascular endothelial growth factor
change in nail fold capillary changes other than capillary density and giant capillaries from baseline [1 year]
changes in nail fold capillary pattern (early, active, late, normal
change in modified Rodnan skin score (mRSS) from baseline [1 year]
presence of puffy fingers from baseline [1 year]
presence of synovitis from baseline [1 year]
presence of tendon friction rubs from baseline [1 year]
fulfilling EULAR/ACR ( American College of Rheumatology )classification from baseline criteria for SSc from baseline [1 year]
pulmonary function tests from baseline [1 year]
presence of interstitial lung disease from baseline [1 year]
suspicion of pulmonary hypertension from baseline [1 year]
Change in physical function from baseline [1 year]
general health score from baseline [1 year]
Change in 36-Item Short Form Survey (SF-36) total score from baseline [1 year]
Change in Scleroderma Health Assessment Questionnaire (SHAQ)total score from baseline [1 year]
Change in EQ-5D is a standardised instrument for use as a measure of health outcome. (EQ5D) total score from baseline [1 year]
Change in gastrointestinal tract ( GIT ) total score from baseline [1 year]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Written informed consent
Age over 18 years
Fulfilling VEDOSS criteria (9):
Raynauds' Phenomenon and
Positive for disease specific auto antibodies (anti-centromere or anti-topoisomerase antibodies) and
typical nail fold capillaroscopic findings
Puffy fingers < 3 years
Modified Rodnan skin score = 0