Corticosteroids for Pediatric Stroke

(FOCAS Trial)

Overseen By Maria A Kuchherzki, BA

Age: < 65

Sex: Any

Trial Phase: Phase 4

Sponsor: University of California, San Francisco

Must be taking: Aspirin

Must not be taking: Corticosteroids

Disqualifiers: Prior stroke, Connective tissue disorder, Moyamoya, Autoimmune disorder, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 1

Duration: 1 month

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores how corticosteroids (such as Methylprednisolone, Prednisolone, and Prednisone) might benefit children who have experienced a stroke or mini-stroke (TIA) due to a specific condition affecting brain blood vessels. The study compares two approaches: administering corticosteroids immediately after diagnosis or delaying use until the condition worsens. All participating children will also receive standard treatments like aspirin. Children who have had a stroke or mini-stroke in the past four days and show certain brain imaging results qualify as good candidates for this trial. As a Phase 4 trial, this research involves an FDA-approved treatment and aims to understand its benefits for more patients.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but it does mention that all participants will receive standard care, including aspirin. If you are currently on corticosteroids, you may need to stop, as recent corticosteroid treatment is an exclusion criterion.

What is the safety track record for these treatments?

In a previous study, children who had a stroke and received high doses of steroids like methylprednisolone, prednisolone, and prednisone showed better recovery and fewer repeat strokes. This suggests these treatments might be safe and helpful. A European study also found that steroid treatment improved recovery for children with a specific type of brain artery problem called focal cerebral arteriopathy (FCA).

While steroids are usually safe for children, they can have some side effects, such as increased hunger, mood changes, and trouble sleeping. However, these side effects are typically mild and temporary.

In this Phase 4 trial, researchers are closely examining the safety of these treatments. Extensive prior use and study of these treatments help ensure their safety for children who have had a stroke. ^{1 2 3 4 5}

Why are researchers enthusiastic about this study treatment?

Researchers are excited about using corticosteroids for pediatric stroke because these treatments might offer faster and more direct ways to reduce brain inflammation compared to current options. While most treatments focus on managing symptoms after a stroke, corticosteroids like Methylprednisolone, Prednisolone, and Prednisone work by actively reducing inflammation, potentially preventing further damage. Additionally, the trial explores whether administering these drugs early or selectively can make a significant difference in outcomes, which could lead to more personalized and effective care for young patients.

What evidence suggests that this trial's treatments could be effective for pediatric stroke?

Research has shown that medications called corticosteroids, such as methylprednisolone, prednisolone, and prednisone, can help children recover from a stroke. These treatments lower the risk of another stroke and aid in healing from the first one. In this trial, participants in Arm A will receive corticosteroids as soon as doctors diagnose focal cerebral arteriopathy (FCA), while those in Arm B will receive corticosteroids only if there is evidence of FCA disease progression. A study in Europe found that corticosteroids might be especially useful for children with FCA, a condition where blood vessels in the brain become narrow. Other research found that children who took steroids often recovered better and had fewer long-term problems. This suggests that starting corticosteroid treatment early could be beneficial for children who have had a stroke. ^{1 2 3 6 7}

Who Is on the Research Team?

Heather J Fullerton, MD, MAS

Principal Investigator

University of California, San Francisco

Are You a Good Fit for This Trial?

This trial is for children aged 1-18 who've had a stroke or TIA within the last 4 days, with imaging showing signs of focal cerebral arteriopathy. They must be able to return for follow-up MRI/MRA scans. Kids on recent steroids, pregnant/post-partum/nursing, with prior strokes or other specific risk factors are excluded.

Inclusion Criteria

You may need to have an additional MRI/MRA scan within 24 hours of joining the study.

Your initial imaging shows specific irregularities or damage in the blood vessels in your brain, or specific types of stroke in certain areas of your brain.

You can come back for a follow-up MRI/MRA scan within a month after the stroke.

Exclusion Criteria

My stroke or TIA is not caused by FCA, but intracranial dissection is considered.

I don't have conditions like lupus or recent drug use that could cause blood vessel problems.

I have heart conditions that could increase my risk of blood clots.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

Up to 4 days

1 visit (in-person)

Treatment

Participants receive corticosteroid treatment based on randomization to Arm A or Arm B

1 month

Weekly visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

12 months

Monthly visits (in-person or virtual)

What Are the Treatments Tested in This Trial?

Interventions

• Methylprednisolone
• Prednisolone
• Prednisone

Trial Overview

The study compares early versus delayed/no steroid treatment in kids after a stroke/TIA due to suspected focal cerebral arteriopathy. Participants will either receive immediate corticosteroids (methylprednisolone/prednisolone/prednisone) or delayed treatment if their condition worsens.

How Is the Trial Designed?

Group I: Arm A Active Control 1 Intervention

Treat all children with suspected FCA with corticosteroids as soon as the diagnosis is made.

Group II: Arm B Active Control 1 Intervention

Treat only the subset of children that develop evidence of FCA disease progression.

Methylprednisolone is already approved in United States, European Union, Canada for the following indications:

Approved in United States as Medrol for:

• Allergic reactions
• Blood disorders
• Cancer
• Eye diseases
• Immune system disorders
• Inflammatory diseases
• Respiratory diseases
• Skin diseases

Approved in European Union as Depo-Medrol for:

• Allergic reactions
• Blood disorders
• Cancer
• Eye diseases
• Immune system disorders
• Inflammatory diseases
• Respiratory diseases
• Skin diseases

Approved in Canada as Solu-Medrol for:

• Allergic reactions
• Blood disorders
• Cancer
• Eye diseases
• Immune system disorders
• Inflammatory diseases
• Respiratory diseases
• Skin diseases

Who Is Running the Clinical Trial?

University of California, San Francisco

Lead Sponsor

Trials

2,636

Recruited

19,080,000+

Medical University of South Carolina

Collaborator

Trials

994

Recruited

7,408,000+

American Heart Association

Collaborator

Trials

352

Recruited

6,196,000+

University of Cincinnati

Collaborator

Trials

442

Recruited

639,000+

University of Iowa

Collaborator

Trials

486

Recruited

934,000+

M.D. Anderson Cancer Center

Collaborator

Trials

3,107

Recruited

1,813,000+

National Institute of Neurological Disorders and Stroke (NINDS)

Collaborator

Trials

1,403

Recruited

655,000+

University of Colorado, Denver

Collaborator

Trials

1,842

Recruited

3,028,000+

Published Research Related to This Trial

In a study of 15 patients previously diagnosed with corticosteroid-induced arachnoiditis, only 3 exhibited the clinical symptoms of arachnoiditis, suggesting a low incidence of the condition among those affected.

The findings indicate that the severity of radiological changes did not correlate with the severity of symptoms, leading to the conclusion that methylprednisolone acetate (Depo-Medrol) should be avoided in procedures involving the thecal sac due to potential risks.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Depo-Medrol and myelographic arachnoiditis. Johnson, A., Ryan, MD., Roche, J.[2021]

Inadvertent injection of methylprednisolone acetate into the eye during vitrectomy can lead to severe retinal toxicity, resulting in significant vision loss and complications such as retinal necrosis and optic nerve damage.

This case highlights the critical need for careful identification of medications during eye surgeries to prevent serious adverse effects associated with incorrect injections.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Retinal necrosis secondary to inadvertent intravitreal methylprednisolone acetate (depo-medrol) injection during pars plana vitrectomy. Yonekawa, Y., Sun, G., D'Amico, DJ., et al.[2014]

In a study of 901 patients with relapsing-remitting multiple sclerosis, methylprednisolone (MP) pulse therapy was administered with a mean frequency of 4 times, showing that the majority of patients received treatment multiple times, indicating its common use in managing acute exacerbations.

The study found that female patients were approximately 3.7 times more likely to seek MP pulse therapy compared to male patients, suggesting a gender difference in treatment-seeking behavior for MS management.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Preliminary study related the incidence of methylprednisolone pulse therapy in patients visited multiple sclerosis clinic located at the isfahan kashani hospital. Tolou-Ghamari, Z., Shaygannejad, V., Ashtari, F.[2022]

A survey of sports medicine physicians revealed that 47% prescribe methylprednisolone taper (Medrol Dosepak) for sports injuries, particularly in patients aged 40 and younger, with postinjury disease being the most common reason for its use.

Concerns about complications, especially the risk of osteonecrosis (reported in 8.5% of prescribers), and a lack of proven efficacy were significant reasons for the 52% of physicians who chose not to prescribe Medrol Dosepak.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Survey of orthopaedic and sports medicine physicians regarding use of medrol dosepak for sports injuries. Langer, P., Fadale, P., Hulstyn, M., et al.[2018]

Citations

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clinicaltrials.gov

clinicaltrials.gov/study/NCT04873583

NCT04873583 | High Dose Steroids in Children With Stroke

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 ...

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pediatricstrokejournal.com

pediatricstrokejournal.com/high-dose-steroids-in-children-with-stroke-and-unilateral-focal-arteriopathy-a-multicenter-randomized-controlled-trial/

High Dose Steroids in Children with Stroke and Unilateral ...

The objectives of this study are to evaluate if early anti-inflammatory treatment may influence the course of arteriopathy, improve clinical outcomes, and ...

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pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC11877790/

Efficacy and safety of corticosteroids for stroke and traumatic ...

Corticosteroids reduced the risk of death after in some patients with stroke, such as those with large artery occlusion after thrombectomy, but ...

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clinicaltrials.ucsf.edu

clinicaltrials.ucsf.edu/trial/NCT06040255

UCSF Stroke Trial → Focal Cerebral Arteriopathy Steroid Trial

A 2017 European retrospective cohort study suggested that corticosteroid treatment may improve outcomes for FCA (Steinlin M, Stroke 2017).

5.

centerwatch.com

centerwatch.com/clinical-trials/listings/NCT04873583/high-dose-steroids-in-children-with-stroke

High Dose Steroids in Children With Stroke

Analysis of existing data suggests that outcomes are improved and that there is less stroke recurrence in children treated with steroids to ...

6.

clinicaltrials.gov

clinicaltrials.gov/study/NCT06040255

NCT06040255 | Focal Cerebral Arteriopathy Steroid Trial

A 2017 European retrospective cohort study suggested that corticosteroid treatment may improve outcomes for FCA (Steinlin M, Stroke 2017). Europeans have begun ...

7.

onlinelibrary.wiley.com

onlinelibrary.wiley.com/doi/full/10.1111/dmcn.13393

Planning interventional trials in childhood arterial ...

The third and fourth surveys reached consensus among 43 out of 44 respondents on design of the highest ranked trial, and allowed agreement on ...