High Dose Steroids in Children With Stroke

NCT04873583 | Recruiting Phase 3

• 6 other identifiers: 1473_PASTA2022-500631-36-002021-005571-392021-00453305395HREC/78937/RCHM-2022
• Study Type: interventional
• Target: 70
• Locations: 7 countries
• Sites: 32

Brief 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, 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.

Conditions

Paediatric Stroke

Keywords

Ischemic; Stroke; Pediatric; Steroids; Unilateral; Focal; Arteriopathy; Neurology; Cerebral

Outcome Measures

Primary Outcomes (1)

• 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

  1 month (30 days)

Secondary Outcomes (13)

• Functional impairment outcome measured by Pediatric Stroke Outcome Measure (PSOM)

  1, 3, 6 and 12 months

• Recovery assessed by Recovery and Recurrence Questionnaire (RRQ)

  1, 3, 6, and 12 months

• Degree of disability or dependence by modified Rankin Scale (mRS)

  1, 3, 6, and 12 months

• Clinical outcome by Vineland adaptive behavior scale (VABS)

  6 and 12 months

• Change in FCASS (Focal Cerebral Arteriopathy Severity Score) from baseline

  6 months

Study Arms (2)

Steroids + Standard of care

EXPERIMENTAL

Standard of care (including aspirin) and intravenous steroids, followed by oral tapering.

Drug: Methylprednisolone; Drug: Prednisolone

Standard of care

NO INTERVENTION

Standard of care (including aspirin)

Interventions

Methylprednisolone DRUG

At the time of inclusion, intravenous Methylprednisolone for 3 days. Dose: 30 mg/kg/day (max. 1000 mg/dose)

Also known as: Solu-Medrol

Steroids + Standard of care

Prednisolone DRUG

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

Steroids + Standard of care

Eligibility Criteria

• Age: 6 Months - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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
• 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)

You may not qualify if:

• 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
• 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

Sponsors & Collaborators

• Insel Gruppe AG, University Hospital Bern: lead
• University of Bern: collaborator
• LUMIS International GmbH: collaborator

Study Sites (35)

Sydney Childrens Hospital Randwick

Randwick, New South Wales, 2031, Australia

RECRUITING

Sydney Childrens Hospital Network

Westmead, New South Wales, 2145, Australia

RECRUITING

Melbourne Childrens Hospital

Melbourne, Victoria, 3052, Australia

RECRUITING

Universitätsklinik für Pädiatrie 1 A.ö. Landeskrankenhaus/ Universitätskliniken Innsbruck

Innsbruck, Tyrol, 6020, Austria

NOT YET RECRUITING

Johannes Kepler University Linz, Med Campus IV, Univ.-Klinik für Kinder- und Jugendheilkunde

Linz, Upper Austria, 4020, Austria

NOT YET RECRUITING

Universitätsklinik für Kinder und Jugendheilkunde Wien

Vienna, 1090, Austria

RECRUITING

Børn og Unge - Aarhus Universitetshospital

Aarhus, 8200, Denmark

NOT YET RECRUITING

Department of Pediatric and Adolescence Medicine Copenhagen University Hospital

Copenhagen, 2100, Denmark

RECRUITING

L'ASSISTANCE PUBLIQUE-HOPITAUX DE MARSEILLE (AP-HM) - Hôpital de la Timone

Marseille, Aix-en-Provence, 13005, France

RECRUITING

Pediatric Neurology Strasbourg - Hautepierre University Hospital

Strasbourg, Alsace, 67098, France

RECRUITING

Hôpital Femme Mère Enfant Lyon

Bron, Auvergne-Rhône-Alpes, 69677, France

RECRUITING

Hôpital Roger Salengro, CHRU de Lille

Lille, 59037, France

RECRUITING

Hôpitaux Universitaires Paris Sud

Le Kremlin-Bicêtre, Île-de-France Region, 94275, France

RECRUITING

Hôpital Necker-Enfants Malades

Paris, Île-de-France Region, 75743, France

RECRUITING

Universitätsklinikum Freiburg Zentrum für Kinder- und Jugendmedizin Klinik für Neuropädiatrie und Muskelerkrankungen

Freiburg im Breisgau, Baden-Wurttemberg, 79106, Germany

RECRUITING

LMU Klinikum

München, Bavaria, 80337, Germany

RECRUITING

Universitätsklinikum Düsseldorf

Düsseldorf, Nordrhein-Westfahlen, 40225, Germany

RECRUITING

Universitäts Kinderklinik Münster

Münster, Nordrhein-Westfahlen, 48129, Germany

RECRUITING

Charité-Universitätsmedizin Berlin

Berlin, 13353, Germany

RECRUITING

Medizinische Hochschule Hannover OE 6720

Hanover, 30625, Germany

RECRUITING

Centrum för Kliniska Barnstudier, Astrid Lindgrens Bansjukhus, Kaolinska Universitetssjukhuset

Stockholm, 171 76, Sweden

NOT YET RECRUITING

Centre Hôpitalier Universitaire Vaud (CHUV), Unité de Neurologie

Lausanne, Canton of Vaud, 1011, Switzerland

RECRUITING

Ospedale Regionale di Bellinzona e Valli

Bellinzona, Canton Ticino, 6500, Switzerland

RECRUITING

Kantonsspital Graubünden, Departement Kinder- und Jugendmedizin

Chur, Kanton Graubünden, 7000, Switzerland

RECRUITING

Hôpital du Valais

Sion, Valais, 1950, Switzerland

RECRUITING

Universitätskinderklinik beider Basel

Basel, 4056, Switzerland

RECRUITING

Inselspital Bern

Bern, 3010, Switzerland

RECRUITING

Hôpitale Universitaire de Genève, Neuropediatrie, Hôpital des Enfants

Geneva, 1211, Switzerland

RECRUITING

Luzerner Kantonsspital, Kinderspital, Neuropädiatrie

Lucerne, 6000, Switzerland

ACTIVE NOT RECRUITING

Stiftung ostschweizerisches Kinderspital

Sankt Gallen, 9006, Switzerland

RECRUITING

Kidnerspital Zürich

Zurich, 8032, Switzerland

RECRUITING

Addenbrookes Hospital - Cambridge University Hospitals NHS Foundation Trust

Cambridge, Cambridgeshire, CB22QQ, United Kingdom

RECRUITING

University Hospital Southampton

Southampton, Hampshire, SO166YD, United Kingdom

RECRUITING

Royal Manchester Children's Hospital

Manchester, Lancashire, M139WL, United Kingdom

RECRUITING

University Hospital Bristol

Bristol, BS13NU, United Kingdom

RECRUITING

Related Publications (2)

• 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.

  PMID: 28121022 BACKGROUND

• Fullerton 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.

  PMID: 40351190 DERIVED

Related Links

• Protocol Publication

MeSH Terms

Conditions

Ischemia; Stroke

Interventions

Methylprednisolone; Methylprednisolone Hemisuccinate; Prednisolone

Condition Hierarchy (Ancestors)

Pathologic Processes; Pathological Conditions, Signs and Symptoms; Cerebrovascular Disorders; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Vascular Diseases; Cardiovascular Diseases

Intervention Hierarchy (Ancestors)

Pregnadienetriols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds

Study Officials

• Maja Steinlin, Prof. em. Dr. med.

  Bern university hospital, Inselspital Bern, Kinderklinik

  STUDY DIRECTOR

Central Study Contacts

Maja Steinlin, Dr. med.

CONTACT

+41 31 6329424; maja.steinlin@insel.ch

Kathrin Bochud, PhD

CONTACT

+41316643472; kathrin.zuercher@insel.ch

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Multi-center, randomized, controlled, non-blinded trial

Study Record Dates

• First Submitted: April 15, 2021
• First Posted: May 5, 2021
• Study Start: November 16, 2021
• Primary Completion (Estimated): July 1, 2026
• Study Completion (Estimated): July 1, 2026
• Last Updated: November 18, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

AU (3); CH (10); DK (2); SE (1); DE (6); FR (6); GB (4)