Pilot Study Evaluating the Efficacy of a Topical PDE4 Inhibitor for Morphea

This is a pilot study to determine the safety and clinical efficacy of crisaborole 2% ointment in the treatment of morphea.

Study Overview

• Status: Completed
• Conditions: Morphea
• Intervention / Treatment: Drug: Crisaborole

Detailed Description

A Phase 2, pilot, proof-of-concept, open label, single arm prospective study to assess the safety, tolerability and efficacy of using crisaborole 2% ointment for adult morphea. Patients identified by their physician, nurse, or other member of their clinical care team as meeting criteria for the study will be asked to apply crisaborole 2% ointment, twice daily, on affected plaques for 12 weeks. A 4 mm skin punch biopsy will be performed at baseline and at 12 weeks, on the same affected plaque. Dermal thickness will be measured and compared before and at the end of treatment. Patients will have a clinical assessment at baseline and at weeks 4, 8 and 12. After week 12, patients who need further treatment will undergo standard of care treatment as determined by their primary dermatologist. Physician and patient reported assessment and skin imaging will be performed at baseline, week 4, 8 and 12, and at week 20. Photography will also be performed at baseline and week 12. An optional post treatment follow-up will be performed at week 20.

Medication will be provided free of charge by Pfizer, as part of the grant that is funding this study. These will be dispensed by the study group at baseline and during the subsequent clinic visits.

• Study Type: Interventional
• Enrollment (Actual): 8
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • North Carolina
    • Durham, North Carolina, United States, 27710
      • Duke University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. >= 18 years of age
2. Clinical diagnosis of morphea.
3. <20% Total body surface area involvement.
4. Does not require systemic immunosuppressive therapy for morphea.
5. No immunosuppressive systemic therapy 1 month prior to starting the study (methotrexate, mycophenolate mofetil, azathioprine, cyclosporine, cyclophosphamide, prednisone >=10 mg PO daily).
6. No immunomodulating topical therapy (topical steroids or topical calcineurin inhibitor), and no topical vitamin D analogue, 2 weeks prior to starting study.
7. No allergy to crisaborole or vehicle.
8. No known renal disease
9. Able to give informed consent.

Exclusion Criteria:Subjects fulfilling any of the following criteria are not eligible for inclusion in this study.

1. Clinical diagnosis of depression or history of suicidal ideation.
2. Pregnant or breastfeeding women, with pregnant women being defined as the state of a female after conception until the termination of gestation, confirmed by a positive urine human chorionic gonadotropin (hCG) laboratory test. Women with a positive urine hCG at any time during the study will be withdrawn from the study.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Crisaborole 2% ointment; Crisaborole 2% ointment applied to affected skin twice per day.	Drug: Crisaborole; Apply Crisaborole 2% ointment to affected skin twice per day.; Other Names: Eucrisa

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants Who Had a Reduction in Dermal Thickness of Sentinel Plaque	Change in dermal thickness on skin biopsy. A sentinel plaque will be selected at baseline. A 4 mm skin punch biopsy will be performed at baseline and at 12 weeks, on the same affected plaque.	Baseline, 12 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of Reduction in DIET Score of Sentinel Plaque	The DIET (Dyspigmentation, Induration, Erythema, and Telangiectasias) score is a physician-assessed, previously validated score. Range is 0-12, and a higher score denotes increased activity or severity.	Baseline and 12 weeks
Percentage of Reduction in LoSCAT Score	LoSCAT (Localized Scleroderma Cutaneous Assessment Tool ) score is a physician-assessed, previously validated score. It has 2 components, the LoSSI (localized scleroderma skin severity index, range 0-162), which measures disease activity, and LoSDI (localized scleroderma damage index, range 0-216). The higher the number, the higher the disease activity and damage, respectively.	Baseline,12 weeks
Percentage of Reduction in Skindex-29 Score	Skindex-29 is a health-related quality of life score; minimum score 30, maximum 150. Lower score indicates a better quality of life.	Baseline and 12 weeks
Change in Dermal Thickness of Sentinel Plaque by Ultrasonography	B-Mode ultrasonographic imaging will be utilized to measure dermal thickness of the sentinel plaque	Baseline, 4, 8 and 12 weeks, optional at 20 weeks

Collaborators and Investigators

• Sponsor: Duke University
• Collaborators: Pfizer
• Investigators: Principal Investigator: Adela Cardones, MD, Duke UMC

Study record dates

Study Major Dates

• Study Start (Actual): September 1, 2018
• Primary Completion (Actual): March 10, 2020
• Study Completion (Actual): March 10, 2020

Study Registration Dates

• First Submitted: November 20, 2017
• First Submitted That Met QC Criteria: November 20, 2017
• First Posted (Actual): November 22, 2017

Study Record Updates

• Last Update Posted (Actual): April 1, 2021
• Last Update Submitted That Met QC Criteria: March 10, 2021
• Last Verified: March 1, 2021

More Information

Terms related to this study

• Keywords: Morphea, Localized Scleroderma
• Additional Relevant MeSH Terms: Skin Diseases; Connective Tissue Diseases; Scleroderma, Localized
• Other Study ID Numbers: Pro00086947

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No