A Phase 3 interventional study of Methylprednisolone and Prednisolone in Paediatric Stroke, sponsored by Insel Gruppe AG, University Hospital Bern. Recruiting at 35 sites in 8 countries. Open to participants aged 6 Months to 17 Years. Per ClinicalTrials.gov, last updated 2025-11-18.
Sponsored by Insel Gruppe AG, University Hospital Bern · Phase 3, Interventional, and Treatment
This clinical trial deals with focal cerebral arteriopathy and childhood stroke, a rare but devastating condition.
Focal cerebral arteriopathy (FCA) is an inflammatory vessel wall disease provoked by infection and there is increasing evidence that inflammatory processes play a crucial role in childhood stroke, influencing the outcome of the disease.
Analysis of existing data suggests that outcomes are improved and that there is less stroke recurrence in children treated with steroids to reduce the acute inflammatory processes. This clinical trial will be conducted in over 20 hospitals in several countries in order to investigate this.
Participants will be randomly separated into two groups. The first group will be treated with standard of care (including aspirin) combined with high dose steroids. The second group will be treated with standard of care (including aspirin) but without steroid treatment.
The objective is to investigate if children treated with a combination of high dose steroid and aspirin will have a better and quicker recovery of FCA, better clinical functional outcome, and less recurrence compared to children treated with aspirin alone.
This project has been identified by international pediatric stroke experts as the most important topic for a clinical trial in the field and is as well one of the most important research priorities identified by parents. The study results will also provide insight into the evolution of inflammatory vessel disease.
Background: Arterial ischemic stroke (AIS) is a rare but devastating condition affecting 2-5/100,000 children/year. Children do not recover better than adults with 2/3 suffering long term neurological, cognitive and behavioural problems. The economic cost of stroke is substantial. Arteriopathy is identified as AIS aetiology in 60-80% of previously healthy children and is the strongest predictor of recurrent events. 30-40% of these children will have a focal cerebral arteriopathy (FCA). FCA in childhood is shown to be an inflammatory vessel wall pathology provoked by infections. This encourages treatment with steroids, despite lack of evidence.
Rationale: There is increasing evidence that etiologically inflammatory processes play a crucial role in childhood stroke, and influence outcome. Retrospective analyses suggest improved outcome and less recurrence with steroid treatment. With the exception of sickle cell disease, this study will be the first randomized clinical trial in children with arterial ischemic stroke. It will provide high-level evidence for the most appropriate treatment for children with AIS due to FCA. Alignment of interventions and outcome as well as pooled analysis with the planned Focal Cerebral Arteriopathy Steroid (FOCAS) study in North America will allow pooled analysis results.This is very important in view of the marked neurological, social and economic burden of childhood AIS for patients and families. This project has been identified as the most important AIS treatment trial by a Delphi survey of international paediatric stroke experts and is one of the most important research priorities identified by parents. In addition, the study will provide insights into the pathogenesis of inflammatory vasculopathies.The objective of this trial is to show that children with first stroke event due to unilateral FCA treated with a combination of high dose steroid and aspirin will have better and quicker recovery of arteriopathy, better clinical functional outcome, and less recurrence compared to children treated with aspirin alone.
The proposed study is a prospective multicentre, parallel group, two-arm, randomized controlled, open-label clinical trial with blinded outcome assessment, comparing a high dose course of methylprednisolone / prednisolone plus standard of care with standard of care alone in children with unilateral arteriopathy and acute ischemic stroke.
Measurements and procedures: Participants will be randomized within 48 hours after diagnosis (maximum 96 hours after stroke onset) to standard of care (SC) alone (control group) or SC plus steroids (experimental group). SC will be harmonized among the study centres to include aspirin treatment. Patients will be assessed at 1, 3, 6 and 12 months. Magnetic imaging and angiography (MRI/MRA) will be done at 1, (3) and 6 months.
Number of Participants: 70 participants in total, 35 per treatment arm
Study duration: 48 months
Study Centre(s): International multi-centre study with approximately 20 to 30 centres
Participating countries:Switzerland, Germany, France, Austria, Great Britain \& Australia
Centres in additional countries might be considered.
Statistical Considerations: The sample size is based on the comparison of the primary outcome - the change in FCASS from baseline to 1 month - between the two treatment groups. The standard deviation from 13 patients of a retrospective study was calculated. The standard deviation of the baseline and follow-up FCASS was 3.0 and 3.3, respectively. The standard deviation of the change in FCASS from baseline was 2.8. Based on the standard deviation of 2.8 and a two-sample means test, 64 patients (32 in each group) are required to detect a difference of 2.0 with a power of 80% at a two-sided alpha-level of 0.05. To account for dropouts (8%), we enlarge the sample size to 70 patients (35 in each group). The primary analysis will follow the intention-to-treat (ITT) principle, i.e. all patients will be analysed in the allocated group regardless of any protocol violations such as cross-overs. The primary outcome (change in FCASS from baseline to 1 month) as well as other secondary continuous score outcomes that are measured multiple times during follow-up (RRQ, mRS, Pediatric Stroke Outcome Measure (PSOM), VABS, modAspect) will be assessed in a repeated-measure, mixed-effects linear model.
Good Clinical Practice (GCP) Statement: This study will be conducted in compliance with the protocol, the current version of the Declaration of Helsinki, International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH-GCP) as well as all national legal and regulatory requirements.
545 studies on the registry are indexed under Ischemia; 72 are open to participants now.
This study's planned enrollment of 70 is close to the median of 74 across 356 interventional studies indexed under Ischemia.
Browse Ischemia studies →Insel Gruppe AG, University Hospital Bern is the lead sponsor of 741 studies on the registry; 180 are open to participants now.
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Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Unilateral arteriopathy according to the following criteria:
Specific neuroimaging (MRA) features of either
Exclusion Criteria:
Progressive large to medium childhood primary angiitis of the CNS (cPACNS ) with 2 of the following 3 criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Autoimmune disease
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Standard of care (including aspirin) and intravenous steroids, followed by oral tapering.
Drug: Methylprednisolone · Drug: Prednisolone
Standard of care (including aspirin)
At the time of inclusion, intravenous Methylprednisolone for 3 days. Dose: 30 mg/kg/day (max. 1000 mg/dose)
Also known as: Solu-Medrol
Intravenous treatment will be immediately followed by oral tapering with Prednisolone. Oral Prednisolone, 2 weeks (week 1 and 2) Dose: 1 mg/kg/day (max 40 mg/day) Oral Prednisolone, 2 weeks (week 3 and 4) Dose: 0.5 mg/kg/day (max 20 mg/day)
Also known as: Spiricort
Change in Focal Cerebral Arteriopathy Severity Score (FCASS) from baseline
The FCASS is a scaling system that has been developed to improve diagnostic criteria and to better document the typical course of initial worsening followed by improvement in FCA-i (focal cerebral arteriopathy of inflammatory type). FCASS Minimum score (best outcome): 0 FCASS Maximum baseline score (worst outcome): 20 FCASS Maximum follow up score (worst outcome): 21
Time frame: 1 month (30 days)
Functional impairment outcome measured by Pediatric Stroke Outcome Measure (PSOM)
The PSOM is a measure that has been specifically developed and validated for pediatric stroke patients and addresses pediatric specific domains such as development, behavior and cognition in addition to sensory-motor and language function. PSOM Minimum score (best outcome): 0 PSOM Maximum baseline score (worst outcome): 10
Time frame: 1, 3, 6 and 12 months
Recovery assessed by Recovery and Recurrence Questionnaire (RRQ)
The Pediatric Recovery and Recurrence Questionnaire was specifically developed and validated for pediatric stroke patients and addresses pediatric- specific problems of manifestation of stroke and difficulties in reliable clinical examination. Minimum score (best outcome): 0 Maximum baseline score (worst outcome): 10
Time frame: 1, 3, 6, and 12 months
Degree of disability or dependence by modified Rankin Scale (mRS)
Minimum score (best outcome): 0 Maximum baseline score (worst outcome): 6
Time frame: 1, 3, 6, and 12 months
Clinical outcome by Vineland adaptive behavior scale (VABS)
The vineland adaptive behavior scale is a validated instrument to monitor cognitive and behavior problems of children by interview. (range=40-160, the higher the score the better the performance)
Time frame: 6 and 12 months
Change in FCASS (Focal Cerebral Arteriopathy Severity Score) from baseline
The FCASS is a scaling system that has been developed to improve diagnostic criteria and to better document the typical course of initial worsening followed by improvement in FCA-i (focal cerebral arteriopathy of inflammatory type). FCASS Minimum score (best outcome): 0 FCASS Maximum baseline score (worst outcome): 20 FCASS Maximum follow up score (worst outcome): 21
Time frame: 6 months
Volume of stroke
Volume of stroke will be measured by modASPECTS in diffusion-weighted MRI (DWI) and fluid-attenuated inversion recovery (FLAIR) images. Higher scores represent greater volumes, with a range of 0-30 (15 per hemisphere). modASPECTS: Modified pediatric ASPECTS ASPECTS: Alberta stroke program early CT score
Time frame: baseline, 1, 3 (if imaging is available) and 6 months
Residual vasculopathy
Residual vasculopathy measured by FCASS (Focal Cerebral Arteriopathy Severity Score) The FCASS is a scaling system that has been developed to improve diagnostic criteria and to better document the typical course of initial worsening followed by improvement in FCA-i (focal cerebral arteriopathy of inflammatory type). FCASS Minimum score (best outcome): 0 FCASS Maximum baseline score (worst outcome): 20 FCASS Maximum follow up score (worst outcome): 21
Time frame: 6 months
Stroke recurrence after index stroke
Stroke recurrence is defined as (i) new focal neurological deficit(s) (ii) worsening of the neurological deficits by \> 4 pedNIHSS points lasting for more than 24 hours with new or increased diffusion restriction at the time of recurrence (with or without FLAIR/T2 lesions) in the corresponding vascular territory, or (iii) new areas of clinically silent infarction, remote from the initial infarct (at 1 and 6 months)
Time frame: 1, 6 and 12 months
Stroke recurrence after index stroke in relation to the initial degree of vessel stenosis
Degree of vessel stenosis measured by change in FCASS from baseline to follow-up. Stroke recurrence will be measured as proportion in each category of vessel stenosis.
Time frame: 6 and 12 months
Stroke Quality of Life Measure (PSQLM)
For children between 2 -18 years quality of life will be assessed with the Pediatric Stroke Quality of Life Measure (PSQLM) (range=-10 to 10, the higher the score the better the performance)
Time frame: 12 month
Preschool Wechsler Intelligence Scale for Children (WISC V) / Wechsler Preschool and Primary Scale of Intelligence (WIPPSI IV)
For Children between 2 -18 years intelligence will be assessed by Preschool Wechsler Intelligence Scale for Children (WISC V) / Wechsler Preschool and Primary Scale of Intelligence (WIPPSI IV, as age appropriate) (range=40-160, the higher the score the better the performance)
Time frame: 12 month
Delis-Kaplan Executive Function System (D-KEFS)
Children \> 8 years will undergo specific evaluation of executive function (EF). They will be assessed with the Delis-Kaplan Executive Function System (D-KEFS) Trail Making Test, Delis-Kaplan Executive Function System (D-KEFS) Color-Word-Interference Test (range=1-19, the higher the score the better the performance)
Time frame: 12 month
Continuous performance task (CPT-III)
Children \> 8 years will undergo specific evaluation of attention. They will be assessed with the Continuous performance task (CPT-III). (range=20-80, the higher the score the better the performance)
Time frame: 12 month
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Plan to share: No — It is not planned to share individual participant data to other researchers.
Started Nov 2021 (actual). Primary completion is estimated for Jul 2026, 56 months after the start.
Of 12 phase 3 interventional studies under Ischemia, half reached primary completion within 40 months of starting (the median), counting those still running; a quarter within 21 and three quarters within 51. This study's planned 56 months is above that.
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Studies of the drugs tested here: Methylprednisolone · Prednisolone
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