Paediatric StrokePhase 3PASTANCT04873583
High Dose Steroids in Children With Stroke
A Phase 3 study testing Methylprednisolone and Prednisolone in people with Paediatric Stroke, run by Insel Gruppe AG, University Hospital Bern at 35 sites in 8 countries.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 6 months to 17 years
- Conditions the study is for
- Paediatric Stroke
- What is being tested?
- Methylprednisolone and Prednisolone
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 70 participants
- Where
- 35 sitesin 8 countries
- Study dates
- 16 Nov 2021to Jul 2026
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 months to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Informed consent of the legal representative of the trial participant documented by signature
- Age > 6 months \& < 18 years at time of stroke
- Randomisation possible within 48 hours of diagnosis and maximum 96 hours after stroke onset
- Unilateral arteriopathy according to the following criteria:
- Newly acquired neurologic deficits
- Specific neuroimaging (MRA) features of either
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- unilateral stenosis, or
- unilateral vessel irregularities within the Central Nervous System (CNS)
- Unless otherwise defined in the national addendum: Female participants age ≥ 13: Negative pregnancy test (blood or urine)
Who cannot join
- Previous stroke
- Known syndromal disorders, as e.g. Trisomy 21, Neurofibromatosis type 1
- Known genetic vasculopathies as e.g. posterior fossa anomalies, hemangioma, arterial anomalies, cardiac anomalies and eye anomalies syndrome (PHACES), actin alpha 2 (ACTA II)
- Moyamoya or sickle cell disease
- Small vessel cerebral vasculitis (primary CNS vasculitis)
- Bilateral arteriopathy
Show all 17Show fewer
- Arterial dissection(s)
- Evidence of underlying systemic disorders, as e.g. lupus, rheumatoid problems
- Secondary CNS angiitis due to infections (meningitis, endocarditis, borreliosis), or generalised angiitis due to rheumatic or other autoimmune problems
- Progressive large to medium childhood primary angiitis of the CNS (cPACNS ) with 2 of the following 3 criteria:
- pre-existing progressive neurocognitive dysfunction
- bilateral MRI lesions/vessel involvement
- small vessel arterial stenosis
- On steroid treatment at disease onset
- Contraindication to steroid treatment as e.g. a congenital or acquired immunodeficiency
- Inability to follow the procedures of the study, e.g. due to language problems
- Participation in another interventional study within the 30 days preceding the indication stroke and during the present study
Trial locations and contacts
35 sites in 8 countries. Contact the study team or a site near you to ask about taking part.
35 sites in 8 countries
Switzerland10 sites, 9 recruiting
- Recruiting
Universitätskinderklinik beider Basel
Basel, 4056
Alexandre Datta, PD Dr.
- Recruiting
Ospedale Regionale di Bellinzona e Valli
Bellinzona, Canton Ticino, 6500
Barbara Goeggel Simonetti, PD Dr.
- Recruiting
Inselspital Bern
Bern, 3010
Gabriela Oesch Nemeth, MD
- Recruiting
Kantonsspital Graubünden, Departement Kinder- und Jugendmedizin
Chur, Kanton Graubünden, 7000
Reta Malär, MD
Susi Strozzi, MD
- Recruiting
Hôpitale Universitaire de Genève, Neuropediatrie, Hôpital des Enfants
Geneva, 1211
Joel Fluss, PD Dr.
- Recruiting
Centre Hôpitalier Universitaire Vaud (CHUV), Unité de Neurologie
Lausanne, Canton of Vaud, 1011
Stéphane Darteyre, MD
- Active, not recruiting
Luzerner Kantonsspital, Kinderspital, Neuropädiatrie
Lucerne, 6000
- Recruiting
Stiftung ostschweizerisches Kinderspital
Sankt Gallen, 9006
Oliver Maier, Dr.
- Recruiting
Hôpital du Valais
Sion, Valais, 1950
Nicole Faignart, MD
Claudia Poloni, MD
- Recruiting
Kidnerspital Zürich
Zurich, 8032
Annette Hackenberg, Dr.
France6 sites, 6 recruiting
- Recruiting
Hôpital Femme Mère Enfant Lyon
Bron, Auvergne-Rhône-Alpes, 69677
Maryline Carneiro, Dr.
- Recruiting
Hôpitaux Universitaires Paris Sud
Le Kremlin-Bicêtre, Île-de-France Region, 94275
Kumaran Deiva, Dr.
- Recruiting
Hôpital Roger Salengro, CHRU de Lille
Lille, 59037
Laure Lacan, MD
- Recruiting
L'ASSISTANCE PUBLIQUE-HOPITAUX DE MARSEILLE (AP-HM) - Hôpital de la Timone
Marseille, Aix-en-Provence, 13005
Beatrice Desnous, MD
- Recruiting
Hôpital Necker-Enfants Malades
Paris, Île-de-France Region, 75743
Manoëlle Kossorotoff, Dr.
- Recruiting
Pediatric Neurology Strasbourg - Hautepierre University Hospital
Strasbourg, Alsace, 67098
Vincent Laugel, Pr, MD, PhD
Germany6 sites, 6 recruiting
- Recruiting
Charité-Universitätsmedizin Berlin
Berlin, 13353
Marc Nikolaus, MD
Bernhard Weschke, MD
- Recruiting
Universitätsklinikum Düsseldorf
Düsseldorf, Nordrhein-Westfahlen, 40225
Stefani Harmsen, MD
Annette Horn, MD
- Recruiting
Universitätsklinikum Freiburg Zentrum für Kinder- und Jugendmedizin Klinik für Neuropädiatrie und Muskelerkrankungen
Freiburg im Breisgau, Baden-Wurttemberg, 79106
Janbernd Kirschner, Pr., MD
Matthias Eckenweiler, MD
- Recruiting
Medizinische Hochschule Hannover OE 6720
Hanover, 30625
Christian Menke, MD
- Recruiting
LMU Klinikum
München, Bavaria, 80337
Lucia Gerstl, PD Dr.
Martin Olivieri, MD
- Recruiting
Universitäts Kinderklinik Münster
Münster, Nordrhein-Westfahlen, 48129
Timo Deba, MD
Roland Straeter, MD
United Kingdom4 sites, 4 recruiting
- Recruiting
University Hospital Bristol
Bristol, BS13NU
Adrew Mallick, Dr.
- Recruiting
Addenbrookes Hospital - Cambridge University Hospitals NHS Foundation Trust
Cambridge, Cambridgeshire, CB22QQ
Manali Chitre, MD
- Recruiting
Royal Manchester Children's Hospital
Manchester, Lancashire, M139WL
Dipak Ram, Dr.
- Recruiting
University Hospital Southampton
Southampton, Hampshire, SO166YD
Jaspal Singh, Dr.
Australia3 sites, 3 recruiting
- Recruiting
Melbourne Childrens Hospital
Melbourne, Victoria, 3052
Mark MacKay, Prof. Dr.
- Recruiting
Sydney Childrens Hospital Randwick
Randwick, New South Wales, 2031
Russel Dale, Dr.
- Recruiting
Sydney Childrens Hospital Network
Westmead, New South Wales, 2145
Russell Dale, Dr.
Austria3 sites, 3 recruiting
- Not yet recruiting
Universitätsklinik für Pädiatrie 1 A.ö. Landeskrankenhaus/ Universitätskliniken Innsbruck
Innsbruck, Tyrol, 6020
Christian Lechner, OA Mag. MD
- Not yet recruiting
Johannes Kepler University Linz, Med Campus IV, Univ.-Klinik für Kinder- und Jugendheilkunde
Linz, Upper Austria, 4020
Biebl Ariane, PD MD
- Recruiting
Universitätsklinik für Kinder und Jugendheilkunde Wien
Vienna, 1090
Rainer Seidl, Prof. Dr.
Denmark2 sites, 2 recruiting
- Not yet recruiting
Børn og Unge - Aarhus Universitetshospital
Aarhus, 8200
Karen Markussen Linnet, PhD
- Recruiting
Department of Pediatric and Adolescence Medicine Copenhagen University Hospital
Copenhagen, 2100
Malene Landbo Børresen, MD PhD
Sweden1 site, 1 recruiting
- Not yet recruiting
Centrum för Kliniska Barnstudier, Astrid Lindgrens Bansjukhus, Kaolinska Universitetssjukhuset
Stockholm, 171 76
Helena Marell Hessla, MD
Anna Gunnerbeck, MD
About this study
Official title: High Dose Steroids in Children With Stroke and Unilateral Focal Arteriopathy: A Multicentre Randomized Controlled Trial PASTA (Paediatric Arteriopathy Steroid Aspirin) Trial
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.
Read more
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.
Read the detailed description
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.
- Conditions
- Paediatric Stroke
- Being tested
- Methylprednisolone, Prednisolone
- Keywords
- Ischemic, Stroke, Pediatric, Steroids, Unilateral, Focal, Arteriopathy, Neurology, Cerebral
- Medical subject headings
- Ischemia, Stroke
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Also called Solu-Medrol
At the time of inclusion, intravenous Methylprednisolone for 3 days. Dose: 30 mg/kg/day (max. 1000 mg/dose)
PrednisoloneDrug
Also called Spiricort
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)
| Study group | Role | What they receive |
|---|---|---|
| Steroids + Standard of care | Experimental | Standard of care (including aspirin) and intravenous steroids, followed by oral tapering. Receives: Drug: Methylprednisolone; Drug: Prednisolone |
| Standard of care | No intervention | Standard of care (including aspirin) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 1 month (30 days)
Secondary outcomes (13)
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
Measured: 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
Measured: 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
Measured: 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)
Measured: 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
Measured: 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
Measured: 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
Measured: 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)
Measured: 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.
Measured: 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)
Measured: 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)
Measured: 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)
Measured: 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)
Measured: 12 month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 May 2021
- Study start16 Nov 2021
- Primary completionJul 2026 (estimated)
- Study completionJul 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Paediatric StrokeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Multi-center, randomized, controlled, non-blinded trial
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 70 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Insel Gruppe AG, University Hospital Bern
- Sponsor type
- University, hospital or other
- Collaborators
- University of Bern, LUMIS International GmbH
- Responsible party
- Sponsor
- Investigators
- Maja Steinlin, Prof. em. Dr. med., Study director, Bern university hospital, Inselspital Bern, Kinderklinik
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04873583
- Sponsor's protocol ID
- 1473_PASTA
- Other IDs
- 2022-500631-36-00, 2021-005571-39, 2021-00453, 305395, HREC/78937/RCHM-2022
- Acronym
- PASTA
- Status last verified
- Nov 2025
- First submitted
- 15 Apr 2021
- Last updated
- 18 Nov 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: It is not planned to share individual participant data to other researchers.
Publications
Steinlin M, O'callaghan F, Mackay MT. Planning interventional trials in childhood arterial ischaemic stroke using a Delphi consensus process. Dev Med Child Neurol. 2017 Jul;59(7):713-718. doi: 10.1111/dmcn.13393. Epub 2017 Jan 25.
PubMed 28121022Fullerton HJ, Hills NK, Chen H, Dlamini N, Stence NV, Wintermark M; VIPS II Investigators. Changing Management of Focal Cerebral Arteriopathy of Childhood From 2010 to 2022. Stroke. 2025 Jun;56(6):1460-1468. doi: 10.1161/STROKEAHA.124.050550. Epub 2025 May 12.
PubMed 40351190
Study at a glance
From the registry record, last updated by the sponsor on 18 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- High Dose Steroids in Children With Stroke and Unilateral Focal Arteriopathy: A Multicentre Randomized Controlled Trial PASTA (Paediatric Arteriopathy Steroid Aspirin) Trial
- ClinicalTrials.gov ID
- NCT04873583
- Acronym / sponsor ID
- PASTA / 1473_PASTA
- Conditions
- Paediatric Stroke
- Keywords
- Ischemic, Stroke, Pediatric, Steroids, Unilateral, Focal, Arteriopathy, Neurology
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 70 participants (estimated)
- Lead sponsor
- Insel Gruppe AG, University Hospital BernUniversity, hospital or other
- Collaborators
- University of Bern, LUMIS International GmbH
- Study start
- 16 Nov 2021 (actual)
- Primary completion
- Jul 2026 (estimated)
- Study completion
- Jul 2026 (estimated)
- First posted
- 5 May 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 2
- Model description
- Multi-center, randomized, controlled, non-blinded trial
| Arm | Type | Receives |
|---|---|---|
| Steroids + Standard of care | Experimental | Drug: Methylprednisolone, Drug: PrednisoloneStandard of care (including aspirin) and intravenous steroids, followed by oral tapering. |
| Standard of care | No intervention | –Standard of care (including aspirin) |
Who may qualify
- Ages
- 6 months to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (9 listed)
- Informed consent of the legal representative of the trial participant documented by signature
- Age > 6 months \& < 18 years at time of stroke
- Randomisation possible within 48 hours of diagnosis and maximum 96 hours after stroke onset
- Unilateral arteriopathy according to the following criteria:
- Unless otherwise defined in the national addendum: Female participants age ≥ 13: Negative pregnancy test (blood or urine)
Key exclusion criteria (17 listed)
- Previous stroke
- Known syndromal disorders, as e.g. Trisomy 21, Neurofibromatosis type 1
- Known genetic vasculopathies as e.g. posterior fossa anomalies, hemangioma, arterial anomalies, cardiac anomalies and eye anomalies syndrome (PHACES), actin alpha 2 (ACTA II)
- Moyamoya or sickle cell disease
- Small vessel cerebral vasculitis (primary CNS vasculitis)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MethylprednisoloneAlso: Solu-MedrolAt the time of inclusion, intravenous Methylprednisolone for 3 days. Dose: 30 mg/kg/day (max. 1000 mg/dose) | Drug | Steroids + Standard of care |
| PrednisoloneAlso: SpiricortIntravenous 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) | Drug | Steroids + Standard of care |
Primary outcome
Change in Focal Cerebral Arteriopathy Severity Score (FCASS) from baseline
Time frame: 1 month (30 days)
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
13 secondary outcomes
Functional impairment outcome measured by Pediatric Stroke Outcome Measure (PSOM)
Time frame: 1, 3, 6 and 12 months
Recovery assessed by Recovery and Recurrence Questionnaire (RRQ)
Time frame: 1, 3, 6, and 12 months
Degree of disability or dependence by modified Rankin Scale (mRS)
Time frame: 1, 3, 6, and 12 months
Clinical outcome by Vineland adaptive behavior scale (VABS)
Time frame: 6 and 12 months
Change in FCASS (Focal Cerebral Arteriopathy Severity Score) from baseline
Time frame: 6 months
Volume of stroke
Time frame: baseline, 1, 3 (if imaging is available) and 6 months
Residual vasculopathy
Time frame: 6 months
Stroke recurrence after index stroke
Time frame: 1, 6 and 12 months
Stroke recurrence after index stroke in relation to the initial degree of vessel stenosis
Time frame: 6 and 12 months
Stroke Quality of Life Measure (PSQLM)
Time frame: 12 month
Preschool Wechsler Intelligence Scale for Children (WISC V) / Wechsler Preschool and Primary Scale of Intelligence (WIPPSI IV)
Time frame: 12 month
Delis-Kaplan Executive Function System (D-KEFS)
Time frame: 12 month
Continuous performance task (CPT-III)
Time frame: 12 month
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Nov 2025.
- Background: PubMed 28121022Steinlin M, O'callaghan F, Mackay MT. Planning interventional trials in childhood arterial ischaemic stroke using a Delphi consensus process. Dev Med Child Neurol. 2017 Jul;59(7):713-718. doi: 10.1111/dmcn.13393. Epub 2017 Jan 25.
- Derived from this study: PubMed 40351190Fullerton HJ, Hills NK, Chen H, Dlamini N, Stence NV, Wintermark M; VIPS II Investigators. Changing Management of Focal Cerebral Arteriopathy of Childhood From 2010 to 2022. Stroke. 2025 Jun;56(6):1460-1468. doi: 10.1161/STROKEAHA.124.050550. Epub 2025 May 12.
- Protocol PublicationLink provided by the sponsor.
- Individual participant dataSharing planned: No. It is not planned to share individual participant data to other researchers.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Maja Steinlin, Prof. em. Dr. med. study director
Bern university hospital, Inselspital Bern, Kinderklinik
35 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Methylprednisolone and Prednisolone in people with Paediatric Stroke, run by Insel Gruppe AG, University Hospital Bern at 35 sites in 8 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 18 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 35 sites in Switzerland, France, Germany, United Kingdom, Australia and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Maja Steinlin, Dr. med. (+41 31 6329424, maja.steinlin@insel.ch), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Focal Cerebral Arteriopathy Severity Score (FCASS) from baseline (time frame: 1 month (30 days)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04873583, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Nov 2025.