Mepolizumab for Eosinophilic Fasciitis

Study Overview
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Brief Summary
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This is a proof of concept pilot study to investigate the efficacy of mepolizumab in the treatment of eosinophilic fasciitis.

Detailed Description
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This is an exploratory study designed to generate preliminary data in evaluating the efficacy of mepolizumab (Nucala) in the treatment of EF using mean change in the Rodnan Skin score (mRSS) before and after treatment. The study is powered at 90% to detect a 4 point change in the mRSS at a 5% significant level with a minimum target accrual of 6 patients.

Official Title
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Mepolizumab for the Treatment of Eosinophilic Fasciitis; An Open-Label, Single-Arm, Exploration Study

Conditions
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Eosinophilic Fasciitis

Intervention / Treatment
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* Drug: Mepolizumab


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Male and female patients 18 years or older. History and physical examination consistent with EF Full thickness skin biopsy characteristic of eosinophilic fascitis and/or MRI radiographic findings characteristic of EF Documented peripheral eosinophilia (≥500 microliter) Patients who are willing and capable of cooperating to the extent and degree required by the protocol; and Patients who read and sign an approved informed consent for this study Exclusion Criteria: Eosinophilic fascitis disease duration > 5 years Known history of adverse reaction to mepolizumab (Nucala) Pregnant females Females actively trying to conceive Vulnerable study population Asthma requiring inhaled cortiosteroids

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Interventional Model Description: Mepolizumab for the treatment of eosinophilic faciitis.
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Single Arm<br>Patients with a biopsy proven diagnosis of eosinophilic fasciitis | Drug: Mepolizumab<br>* Mepolizumab (Nucala) 400 mg SC every 4 weeks x 24 weeks<br>* Other names: Nucala;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Modified Rodnan Skin Score (mRSS) | Mean change before and after treatment. The mRSS is a standardized scored assessment of tissue sclerosis as assessed by palpitation. | 24 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Localized Scleroderma Skin Severity Index (mLoSSI) score | Mean change before and after treatment. mLoSSI score quantifies disease activity. | 24 weeks | 
| Localized Scleroderma Damage Index (LoSDI) score. | Mean change before and after treatment. LoSDI score quantifies disease damage. | 24 weeks | 
| Localized Scleroderma Cutaneous Assessment Tool (LoSCAT) | Mean change before and after treatment. LoSCAT is a composite score of disease activity (mLoSSI) and disease damage (LoSDI). | 24 weeks | 
| Physician's Global Assessment of Disease Activity (PhysGA-A) | Mean change before and after treatment. PhysGA-A is a 100-mm analog scale anchored by inactive at 0 and markedly active at 100. | 24 weeks | 
| Physician's Global Assessment of Disease Damage (PhysGA-D) | Mean change before and after treatment. PhysGA-D is a 100-mm analog scale anchored by no damage at 0 and markedly damaged at 100. | 24 weeks. | 
| Dermatology Quality of Life Index (DLQI) | Mean change before and after treatment. DLQI is a skin-specific health questionnaire with higher scores reflecting decreased quality of life | 24 weeks. | 
| Patient Global Assessment of Disease Severity (PtGA-S) | Mean change before and after treatment.PhysGA-A is a 100-mm analog scale anchored by not severe at 0 and very severe at 100. | 24 weeks. | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Mepolizumab