Comparative Effectiveness Trial in the Treatment of Pediatric Plaque Morphea

Comparative Effectiveness Trial of Topical Calcipotriene, Clobetasol, and Tacrolimus in the Treatment of Pediatric Plaque Morphea

This is a study designed to evaluate the comparative effectiveness of topical medications for the treatment of circumscribed morphea.

Study Overview

• Status: Withdrawn
• Conditions: Scleroderma
• Intervention / Treatment: Drug: Calcipotriene; Drug: Clobetasol; Drug: Tacrolimus

Detailed Description

Morphea, also known as localized scleroderma, is a rare autoimmune fibrosing disorder that affects children and adults. Morphea causes significant morbidity through atrophy and fibrosis of the skin and underlying structures. Treatment can halt the progression of disease but cannot reverse disease damage, leaving children with disfiguring scars and sometimes functional impairment. Consequently, it is of extreme importance to intervene early in the disease course with effective therapies to limit morbidity.

Circumscribed morphea is the second most common subtype in children, comprising approximately 1/3 of pediatric morphea cases. It is a disfiguring disease that can cause pain and pruritus. There are no FDA-approved treatments and no consensus exists on the ideal therapy for circumscribed morphea, either in adults or children. Most physicians use topical medications for circumscribed morphea yet there is little evidence to support these therapies. Topical immune modulators such as tacrolimus, imiquimod, and calcipotriene have been investigated in small studies, though these trials were limited by very small sample sizes of 9-19 patients and lack of a control in most. Surveys have demonstrated that topical steroids are the most commonly prescribed medication, even though the only evidence to support their use comes from case reports. With insufficient evidence regarding topical therapies, providers have no guidance when making treatment decisions, practice patterns vary widely even amongst pediatric dermatologists, and children continue with preventable scars.

The investigators specific aim is to identify effective topical therapy for circumscribed morphea. The investigators will utilize a comparative cohort study design to evaluate the comparative effectiveness of three topical medications: clobetasol ointment, calcipotriene ointment, and tacrolimus 0.1% ointment. The investigators will enroll subjects at CHW and other institutions to achieve the study enrollment necessary to power this study.

• Study Type: Interventional
• Phase: Phase 1

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years to 18 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• presence of at least 1 active lesion of morphea on the neck, trunk, or extremities with no prior treatment to that lesion for at least 4 weeks

Exclusion Criteria:

• children receiving phototherapy or systemic therapy for their morphea
• children with linear or generalized morphea
• lesions on the face
• greater than 3% body surface area involvement
• open or ulcerated skin within morphea lesions

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: Treatment 1; Calcipotriene 0.005% ointment	Drug: Calcipotriene; ointment, twice daily
Active Comparator: Treatment 2; Clobetasol 0.05% ointment	Drug: Clobetasol; ointment, twice daily (alternating weeks)
Active Comparator: Treatment 3; Tacrolimus 0.1% ointment	Drug: Tacrolimus; ointment, 0.1%, twice daily

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Visual Analog Scale	This is a 100 mm scale with 3 anchors; each 5 mm of the scale is equivalent to 10% change.	2 months
Visual Analog Scale	This is a 100 mm scale with 3 anchors; each 5 mm of the scale is equivalent to 10% change.	4 months

Collaborators and Investigators

• Sponsor: Medical College of Wisconsin
• Collaborators: Mayo Clinic; Seattle Children's Hospital; University of Toronto
• Investigators: Principal Investigator: Yvonne Chiu, MD, Medical College of Wisconsin

Study record dates

Study Major Dates

• Study Start: March 1, 2016
• Primary Completion (Anticipated): May 1, 2017
• Study Completion (Anticipated): May 1, 2017

Study Registration Dates

• First Submitted: February 9, 2016
• First Submitted That Met QC Criteria: February 10, 2016
• First Posted (Estimate): February 11, 2016

Study Record Updates

• Last Update Posted (Actual): July 18, 2018
• Last Update Submitted That Met QC Criteria: July 17, 2018
• Last Verified: July 1, 2018

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Skin Diseases; Connective Tissue Diseases; Scleroderma, Localized; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Anti-Inflammatory Agents; Immunosuppressive Agents; Immunologic Factors; Glucocorticoids; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Dermatologic Agents; Calcineurin Inhibitors; Calcipotriene; Tacrolimus; Clobetasol
• Other Study ID Numbers: 730382-2