Mepolizumab for Eosinophilic Fasciitis

Phase 1

Withdrawn

• Conditions: Eosinophilic Fasciitis
• Interventions: Drug: Mepolizumab

• Registration Number: NCT04305678
• Lead Sponsor: Mayo Clinic

Brief Summary

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

Detailed Description

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.

Study Timeline

• Study First Submitted: 2020-03-10
• Study First Submitted QC: 2020-03-10
• Study First Posted: 2020-03-12
• Study Start: 2023-02
• Primary Completion: 2023-02-15
• Study Completion: 2023-02-15
• Status Verified: 2024-11
• Last Update Submitted: 2024-11-18
• Last Update Posted: 2024-11-20

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: Not specified

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Single Arm	Mepolizumab	Patients with a biopsy proven diagnosis of eosinophilic fasciitis

Primary Outcome Measures

Name	Time	Method
Modified Rodnan Skin Score (mRSS)	24 weeks	Mean change before and after treatment. The mRSS is a standardized scored assessment of tissue sclerosis as assessed by palpitation.

Secondary Outcome Measures

Name	Time	Method
Localized Scleroderma Skin Severity Index (mLoSSI) score	24 weeks	Mean change before and after treatment. mLoSSI score quantifies disease activity.
Localized Scleroderma Cutaneous Assessment Tool (LoSCAT)	24 weeks	Mean change before and after treatment. LoSCAT is a composite score of disease activity (mLoSSI) and disease damage (LoSDI).
Dermatology Quality of Life Index (DLQI)	24 weeks.	Mean change before and after treatment. DLQI is a skin-specific health questionnaire with higher scores reflecting decreased quality of life
Physician's Global Assessment of Disease Activity (PhysGA-A)	24 weeks	Mean change before and after treatment. PhysGA-A is a 100-mm analog scale anchored by "inactive" at 0 and "markedly active" at 100.
Physician's Global Assessment of Disease Damage (PhysGA-D)	24 weeks.	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.
Localized Scleroderma Damage Index (LoSDI) score.	24 weeks	Mean change before and after treatment. LoSDI score quantifies disease damage.
Patient Global Assessment of Disease Severity (PtGA-S)	24 weeks.	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.