Clinical trial NCT03351114: Pilot Study Evaluating the Efficacy of a Topical PDE4 Inhibitor for Morphea
Official title: Pilot Study Evaluating the Efficacy of a Topical PDE4 Inhibitor for Morphea
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Morphea
Keywords: Morphea, Localized Scleroderma
Lead sponsor: Duke University
Sponsor class: OTHER
Collaborators: name: Pfizer
First posted: 2017-11-22
Enrollment: 8
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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

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.

Eligibility 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.

Primary outcomes:
measure: Number of Participants Who Had a Reduction in Dermal Thickness of Sentinel Plaque
timeFrame: Baseline, 12 weeks
Secondary outcomes:
measure: Percentage of Reduction in DIET Score of Sentinel Plaque
timeFrame: Baseline and 12 weeks
measure: Percentage of Reduction in LoSCAT Score
timeFrame: Baseline,12 weeks
measure: Percentage of Reduction in Skindex-29 Score
timeFrame: Baseline and 12 weeks
measure: Change in Dermal Thickness of Sentinel Plaque by Ultrasonography
timeFrame: Baseline, 4, 8 and 12 weeks, optional at 20 weeks
Other outcomes:
Locations:
city: Durham
country: United States
facility: Duke University
state: North Carolina
Study officials:
affiliation: Duke UMC
name: Adela Cardones, MD
role: PRINCIPAL_INVESTIGATOR
References: