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

Radboud University Medical Center

Status and phase: Unknown; Early Phase 1

Conditions: Raynaud Phenomena; Systemic Sclerosis

Treatments: Other: sodium chloride; Drug: Methylprednisolone

Study type: Interventional

Funder types: Other

Identifiers

NCT03059979

HHaE

Details and patient eligibility

About

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

Full 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

Enrollment: 30 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No 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

Exclusion criteria

• Presence of acroosclerosis, acrosteolysis and digital ulcers
• Presence of anti-RNA polymerase III auto antibodies

Previous systemic treatment for SSc, namely:

• methotrexate,
• prednisone (> 14 days in previous 6 months),
• mofetil mycophenolate
• cyclophosphamide.

Clinically significant internal organ involvement:

• diffusion capacity of lung for carbon monoxide (DLCO) < 80% predicted,
• vital capacity (VC) < 70% predicted
• renal dysfunction with glomerular filtration rate (GFR) < 60 ml/min
• diastolic dysfunction > grade 1 on echocardiography
• pulmonary hypertension
• weight loss >10% in the last 6 months with unknown cause

Contra-indications for methylprednisolone, such as:

• pregnancy, lactation
• psychotic or depressive disorder
• ulcus duodeni or ventriculi
• untreated hypertension (> 160-90 mmHg)
• acute infections

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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30 participants in 2 patient groups, including a placebo group

Methylprednisolone 1000 mg

Active Comparator group

Description:

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

Treatment:

Drug: Methylprednisolone

sodium chloride

Placebo Comparator group

Description:

The placebo intervention with physiologic salt solution is identical in appearance

Treatment:

Other: sodium chloride