Efficacy of Betamethasone Sodium Phosphate in Pediatric Acute Facial Nerve Palsy: A Randomized, Double-Blind, Placebo-Controlled Multicenter Trial

The primary objective of this study is to evaluate the **efficacy** of cortisone versus placebo treatment in children with acute **facial nerve palsy**. This is measured as total recovery using the House-Brackmann scale at a 12-month follow-up. The clinical relevance of this objective lies in determining the potential benefits of cortisone in improving recovery outcomes for pediatric patients suffering from this condition, which can significantly impact facial function and quality of life.

Secondary objectives include:

• Evaluating the efficacy of cortisone versus placebo treatment in children with acute facial nerve palsy, measured as total recovery with the Sunnybrook scale at 12 months follow-up.
• Assessing the total recovery rate with both Sunnybrook and House-Brackmann scales at 1 month compared to the total recovery rate at 12 months, to determine if prediction of recovery at 1 month is feasible and/or useful.
• Evaluating subjective symptoms and their influence on daily life using the Facial Clinimetric Evaluation (FaCE) scale, the Facial Disability Index (FDI), and the Synkinesis Assessment Questionnaire (SAQ) at 1 and 12 months follow-up.
• Assessing the safety profile of cortisone in children with acute facial nerve palsy.

The clinical trial involves a study population of children aged **1-17 years** diagnosed with acute **facial nerve palsy**, commonly known as Bell's Palsy. The trial includes both male and female participants, and the population is considered vulnerable due to the age range. Participants were selected based on the acute onset of peripheral facial nerve palsy with symptoms lasting less than 72 hours, and all participants provided signed informed consent. The sponsor has not provided the total number of participants involved in the study. The trial does not specify any particular lifestyle considerations such as diet or physical activity. The study aims to assess the efficacy of cortisone compared to a placebo in achieving total recovery, as measured by the House-Brackmann scale, at a 12-month follow-up.

The clinical trial is designed as a **randomized**, **double-blind**, placebo-controlled study to evaluate the efficacy of cortisone treatment in children with acute **facial nerve palsy**. The trial aims to assess the total recovery of facial nerve function using the House-Brackmann scale at a 12-month follow-up. The study involves two groups: one receiving **betamethasone sodium phosphate** and the other receiving placebo capsules corresponding to Prednisolone 5 mg for oral administration. The trial is expected to run from May 2019 to December 2026, with participant involvement lasting up to 12 months.

Participants will undergo a series of study visits, beginning with an inclusion (screening) visit to confirm eligibility based on criteria such as age (1-17 years), acute peripheral facial nerve palsy, symptom duration of less than 72 hours, and signed informed consent. Following randomization, participants will attend follow-up visits at 1 month and 12 months to evaluate recovery using both the House-Brackmann and Sunnybrook grading scales. Additional assessments will include subjective symptoms and daily life impact using the Facial Clinimetric Evaluation (FaCE) scale, the Facial Disability Index (FDI), and the Synkinesis Assessment Questionnaire (SAQ). The end-of-study visit will coincide with the 12-month follow-up, marking the conclusion of participant involvement.

Participants may be withdrawn from the study if they experience adverse events that compromise safety or if they fail to adhere to the study protocol. The primary endpoint is the number of patients achieving total recovery at 12 months, while secondary endpoints include recovery rates at 1 month, subgroup analyses, and safety profiles. The trial is not classified as a low-intervention study and is categorized as a Phase 4 trial, focusing on the post-marketing evaluation of the treatment's efficacy and safety.

The clinical trial involves the administration of **betamethasone sodium phosphate**, a chemical compound used as the experimental medication. This active substance is provided in the form of **PREDNISOLONE** tablets, intended for **oral use**. The maximum daily dose is set at 50 mg, with a total maximum dose of 500 mg over the course of the treatment. The treatment period is limited to a maximum of 10 days. The pharmaceutical form is identified by the code PHF00059MIG. The administration of the medication is monitored to ensure compliance with the dosing schedule, which is crucial for evaluating the efficacy of the treatment in children with acute facial nerve palsy.

The study also includes a **placebo** group, which receives placebo capsules corresponding to Prednisolone 5 mg for oral administration. These placebo capsules are designed to match the experimental medication in appearance and administration route, ensuring the double-blind nature of the trial. The placebo serves as a comparator treatment to evaluate the efficacy of the experimental medication against a non-active substance. Participant compliance with the placebo administration is similarly monitored to maintain the integrity of the trial results.

Efficacy in the clinical trial titled "The FACE (Facial nerve palsy And Cortisone Evaluation) study in children" will be assessed by evaluating the treatment outcomes of cortisone versus placebo in children with acute facial nerve palsy. The primary endpoint for efficacy is the number of patients achieving total recovery of facial nerve palsy, as measured by the House-Brackmann grading scale at a 12-month follow-up. This endpoint is selected for its robustness and prior use in similar studies involving both pediatric and adult populations.

Secondary endpoints include the total number of patients with total recovery measured by the Sunnybrook grading scale at 12 months, specifically in subgroups with idiopathic or Borrelia-associated facial nerve palsy. Additionally, the trial will assess the total recovery rate using both the Sunnybrook and House-Brackmann scales at 1 month compared to 12 months. Subjective symptoms and their impact on daily life will be evaluated using the Facial Clinimetric Evaluation (FaCE) scale, the Facial Disability Index (FDI), and the Synkinesis Assessment Questionnaire (SAQ) at both 1 and 12 months follow-up. The safety profile will be monitored through adverse event reporting.