Morphea
Conditions
Keywords
Morphea, Localized Scleroderma
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.
Interventions
DRUGCrisaborole
Apply Crisaborole 2% ointment to affected skin twice per day.
Sponsors
Pfizer
Duke University
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants Who Had a Reduction in Dermal Thickness of Sentinel Plaque | 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
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Reduction in DIET Score of Sentinel Plaque | 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. |
| Percentage of Reduction in LoSCAT Score | 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. |
| Percentage of Reduction in Skindex-29 Score | 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. |
| Change in Dermal Thickness of Sentinel Plaque by Ultrasonography | 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 |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Crisaborole 2% Ointment Crisaborole 2% ointment applied to affected skin twice per day.
Crisaborole: Apply Crisaborole 2% ointment to affected skin twice per day. | 8 |
| Total | 8 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Withdrawal by Subject | 1 |
Baseline characteristics
| Characteristic | Crisaborole 2% Ointment |
|---|---|
| Age, Continuous | 51 years |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 8 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 8 Participants |
| Region of Enrollment United States | 8 Participants |
| Sex: Female, Male Female | 7 Participants |
| Sex: Female, Male Male | 1 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 8 |
| other Total, other adverse events | 0 / 8 |
| serious Total, serious adverse events | 0 / 8 |
Outcome results
Primary
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.
Time frame: Baseline, 12 weeks
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Crisaborole 2% Ointment | Number of Participants Who Had a Reduction in Dermal Thickness of Sentinel Plaque | 5 Participants |
Secondary
Change in Dermal Thickness of Sentinel Plaque by Ultrasonography
B-Mode ultrasonographic imaging will be utilized to measure dermal thickness of the sentinel plaque
Time frame: Baseline, 4, 8 and 12 weeks, optional at 20 weeks
Population: Data not collected.
Secondary
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.
Time frame: Baseline and 12 weeks
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Crisaborole 2% Ointment | Percentage of Reduction in DIET Score of Sentinel Plaque | 33 percentage of reduction | Standard Deviation 26 |
Secondary
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.
Time frame: Baseline,12 weeks
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Crisaborole 2% Ointment | Percentage of Reduction in LoSCAT Score | LoSSI (Activity) | 37.5 percentage of reduction | Standard Deviation 29 |
| Crisaborole 2% Ointment | Percentage of Reduction in LoSCAT Score | LoSDI (Damage) | 5 percentage of reduction | Standard Deviation 36 |
Secondary
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.
Time frame: Baseline and 12 weeks
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Crisaborole 2% Ointment | Percentage of Reduction in Skindex-29 Score | 25.7 percentage of reduction | Standard Deviation 16.4 |