Focal Cerebral Arteriopathy Steroid (FOCAS) Trial

Overview

The Focal Cerebral Arteriopathy Steroid (FOCAS) trial investigates whether early corticosteroid treatment improves outcomes in children with focal cerebral arteriopathy (FCA) presenting with arterial ischemic stroke (AIS) or transient ischemic attack (TIA). FCA is an inflammatory disease that can rapidly progress, causing new or worsening brain injury. Despite increasing use of corticosteroids in clinical practice, there are no randomized data to guide timing of therapy. FOCAS directly compares two common strategies — early steroid treatment for all suspected cases versus delayed treatment only if disease progression occurs — to determine which leads to better vascular and neurological outcomes.

Population of the study: Children aged 1 to 18 years presenting with acute AIS or TIA within 96 hours of symptom onset and imaging evidence of suspected focal cerebral arteriopathy
Intervention: High-dose intravenous methylprednisolone (30 mg/kg/day, max 1 g/day × 3 days), followed by an oral prednisolone taper over four weeks
Control: All participants receive standard stroke management, including aspirin and supportive care.

• Early-treatment arm: Steroids started immediately after randomization.
• Delayed-treatment arm: Steroids started only if clinical or imaging evidence of disease progression occurs (usual practice in many centers).

Primary Outcome: Change in Focal Cerebral Arteriopathy Severity Score (FCASS) from baseline to 1 month, measured on MR angiography by blinded central reviewers.

Secondary Outcomes:

• FCASS at 1 and 12 months
• Relative infarct volume (as % of brain volume) at 1 and 12 months
• Pediatric Stroke Outcome Measure (PSOM) at 6 months
• Safety and tolerability of corticosteroid therapy (rates of infection, hemorrhage, and adverse events)

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Trial Information

Target Enrollment: 80 children (randomized 1:1 to early vs delayed treatment)
Funder: National Institutes of Health (NIH), National Institute of Neurological Disorders and Stroke (NINDS) through the StrokeNet clinical trials network
Duration of Study: Enrollment period is 5.5 years. Total study duration is approximately 6.5 years (including follow-up through 12 months)

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UTHealth Houston Principal Investigator

Stuart Fraser, MD

 

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