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

• ClinicalTrials.gov Identifier: NCT03351114
• Recruitment Status: Completed
• First Posted: November 22, 2017
• Results First Posted: April 1, 2021
• Last Update Posted: April 1, 2021
• Sponsor: Duke University
• Collaborator: Pfizer
• Information provided by (Responsible Party): Duke University

Study Description

Brief Summary:

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

Condition or disease	Intervention/treatment	Phase
Morphea	Drug: Crisaborole	Phase 2

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 Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 8 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Pilot Study Evaluating the Efficacy of a Topical PDE4 Inhibitor for Morphea
• Actual Study Start Date: September 1, 2018
• Actual Primary Completion Date: March 10, 2020
• Actual Study Completion Date: March 10, 2020

Arms and Interventions

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 Name: Eucrisa

Outcome Measures

Primary Outcome Measures :

1. Number of Participants Who Had a Reduction in Dermal Thickness of Sentinel Plaque [ Time Frame: Baseline, 12 weeks ]
   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.

Secondary Outcome Measures :

1. Percentage of Reduction in DIET Score of Sentinel Plaque [ Time Frame: Baseline and 12 weeks ]
   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.
2. Percentage of Reduction in LoSCAT Score [ Time Frame: Baseline,12 weeks ]
   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.
3. Percentage of Reduction in Skindex-29 Score [ Time Frame: Baseline and 12 weeks ]
   Skindex-29 is a health-related quality of life score; minimum score 30, maximum 150. Lower score indicates a better quality of life.
4. Change in Dermal Thickness of Sentinel Plaque by Ultrasonography [ Time Frame: Baseline, 4, 8 and 12 weeks, optional at 20 weeks ]
   B-Mode ultrasonographic imaging will be utilized to measure dermal thickness of the sentinel plaque

Eligibility Criteria

• Ages Eligible for Study: 18 Years and older (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

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.

Contacts and Locations

Locations

United States, North Carolina

Duke University

Durham, North Carolina, United States, 27710

Sponsors and Collaborators

Duke University

Pfizer

Investigators

• Principal Investigator: Adela Cardones, MD; Duke UMC

  Study Documents (Full-Text)

Documents provided by Duke University:

Study Protocol and Statistical Analysis Plan  [PDF] August 3, 2018

More Information

• Responsible Party: Duke University
• ClinicalTrials.gov Identifier: NCT03351114
• Other Study ID Numbers: Pro00086947
• First Posted: November 22, 2017
• Results First Posted: April 1, 2021
• Last Update Posted: April 1, 2021
• Last Verified: March 2021

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No

• 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

Keywords provided by Duke University:

Morphea, Localized Scleroderma

Additional relevant MeSH terms:

Scleroderma, Localized; Connective Tissue Diseases; Skin Diseases