Lichen Planopilaris
Conditions
Keywords
Valchor
Brief summary
The primary objective of this study is to assess the potential effectiveness of once daily application of Valchlor in decreasing disease activity in patients with Lichen Planopilaris. The primary measurement of efficacy will be with the Lichen Planopilaris Activity Index (LPPAI) before and after 6 months of treatment. Secondary measures of efficacy will be the mean follicular density, Physician Global assessment (PGA) score, and the Dermatology Quality of Life Index (DQLI) score before and after six months of therapy.
Detailed description
This is a single arm, open label, exploratory study to evaluate the efficacy of Valchlor in the treatment of LPP. Subjects will be screened by the Department of Dermatology at the Mayo Clinic in Florida outpatient clinic and interested qualified subjects will be consented and offered participation. This study is designed to establish feasibility and proof of concept and will not include randomization or crossover components. Patients with biopsy proven LPP who have failed one prior topical or systemic therapy with evidence of active disease will be eligible to participate. The presence of active disease will be based on a baseline clinical exam showing perifollicular erythema with scaling. Patients with predominance of end stage scarring hair loss but without significant active erythema will be excluded. Involvement restricted to the frontal scalp is a recognized clinical variant of LPP and is known as frontal fibrosing alopecia (FAA). As the histological features of LPP and FAA are identical, patients with FAA subtype of LPP would also be eligible to participate in the study. Eligible participants using high-potency topical corticosteroids, intralesional corticosteroids, or oral hydroxychloroquine may enroll but will be required to discontinue use during the study period. All study participants will apply Valchlor 0.016% gel to involved areas at night. Patients will be instructed to first part the hair away from involved area as needed, limit application to areas with alopecia and erythema, apply 30 minutes after showering or washing, and allow treated areas to dry for 5 to 10 minutes before covering with clothing or going to bed. Participants will be instructed to wash their hands with soap and water after applying Valchlor. Caregivers who assist in application will be instruction to wear disposable nitrile gloves when applying Valchlor and dispose with the household trash. Patients will be instructed to store Valchlor in the refrigerator away from foods at 36°F - 46°F and apply within 30 minutes after removing from refrigeration.
Interventions
DRUGMechlorethamine 0.016% Top Gel
Valchlor, a 0.016% gel formulation of mechlorethamine. Once daily application of Valchlor in decreasing disease activity in patients with Lichen Planopilaris.
Sponsors
Actelion
Mayo Clinic
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Adults patient with active Lichen Planopilaris or Frontal Fibrosing Alopecia
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Male and female patients 18 years or older. * Biopsy proven diagnosis of Lichen Planopilaris * Biopsy proven diagnosis of Fontal Fibrosing Alopecia (a clinical variant of LPP restricted to frontal scalp) * Good general health as confirmed by medical history * Patients who are willing and capable of cooperating to the extent and degree required by the protocol; and * Patients who read and sign an approved informed consent for this study
Exclusion criteria
* Vulnerable study population * Pregnant or nursing women * Women planning a pregnancy within the study period * Active smokers * Known history of adverse reaction to mechlorethamine * Use of systemic immunosuppressive * Presence of ulcerated scalp lesions
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in Lichen Planopilaris Activity Index (LLPAI) | baseline, up to 24 weeks | The Lichen Planopilaris Activity Index is a standardized validated quantitative measure of disease activity. LPPAI score (0-10) is calculated as follows: (pruritus + pain + burning)/3 + (scalp erythema + perifollicular erythema + perifollicular scale)/3 + 2.5 (pull test) + 1.5 (spreading/2). Symptoms and signs are graded on a 4-point scale with 0 = absent, 1 = mild, 2 = moderate, and 3 =severe. Clinical progression and a positive hair pull test are graded 1=yes; 0=no. The percent change of LLPAI score from before and after treatment. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in Dermatology Quality of Life Index (DQLI) | baseline, 24 weeks | The DQLI is 10 standardized items measuring impact of skin disease rated as 0=not at all/not relevant; 1=a little; 2=a lot; 3=very much. The absolute change of total DQLI score from before and after treatment |
| Change in Follicular Units | baseline, 24 weeks | Phototrichograms of all subjects scalps performed using FotoFinder video-epiluminescence microscopy (FotoFinder Systems GmbH) in combination with TrichoScan digital image analysis (TRICHOLOG GmbH and DatInf mbH). The percent change in the total number of hairs per square cm was measured before and after treatment. |
| Change in Mean Follicular Density | baseline, 24 weeks | Phototrichograms of all subjects scalps performed using FotoFinder video-epiluminescence microscopy (FotoFinder Systems GmbH) in combination with TrichoScan digital image analysis (TRICHOLOG GmbH and DatInf mbH). The percent change in the mean number of follicular units per square cm was measured before and after treatment. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Valchor Treatment of Lichen Planopilaris Once daily application of Valchlor in decreasing disease activity in patients with Lichen Planopilaris.
Valchlor: Valchlor, a 0.016% gel formulation of mechlorethamine. Once daily application of Valchlor in decreasing disease activity in patients with Lichen Planopilaris. | 12 |
| Total | 12 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Protocol Violation | 3 |
| Overall Study | Withdrawal by Subject | 1 |
Baseline characteristics
| Characteristic | Valchor Treatment of Lichen Planopilaris |
|---|---|
| Age, Continuous | 66 years |
| LICHEN PLANOPILARIS ACTIVITY INDEX (LPPAI) | 5 Integer |
| 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 | 1 Participants |
| Race (NIH/OMB) White | 11 Participants |
| Region of Enrollment United States | 12 participants |
| Sex: Female, Male Female | 12 Participants |
| Sex: Female, Male Male | 0 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 12 |
| other Total, other adverse events | 11 / 12 |
| serious Total, serious adverse events | 0 / 12 |
Outcome results
Primary
Change in Lichen Planopilaris Activity Index (LLPAI)
The Lichen Planopilaris Activity Index is a standardized validated quantitative measure of disease activity. LPPAI score (0-10) is calculated as follows: (pruritus + pain + burning)/3 + (scalp erythema + perifollicular erythema + perifollicular scale)/3 + 2.5 (pull test) + 1.5 (spreading/2). Symptoms and signs are graded on a 4-point scale with 0 = absent, 1 = mild, 2 = moderate, and 3 =severe. Clinical progression and a positive hair pull test are graded 1=yes; 0=no. The percent change of LLPAI score from before and after treatment.
Time frame: baseline, up to 24 weeks
Population: One subject withdrew and data was not analyzed for that subject
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Valchor Treatment of Lichen Planopilaris | Change in Lichen Planopilaris Activity Index (LLPAI) | Week 12 | -42.9 percentage of change from baseline |
| Valchor Treatment of Lichen Planopilaris | Change in Lichen Planopilaris Activity Index (LLPAI) | Week 24 | -66.7 percentage of change from baseline |
Comparison: Week 12p-value: 0.014Wilcoxon (Mann-Whitney)
Comparison: Week 24p-value: 0.006Wilcoxon (Mann-Whitney)
Secondary
Change in Dermatology Quality of Life Index (DQLI)
The DQLI is 10 standardized items measuring impact of skin disease rated as 0=not at all/not relevant; 1=a little; 2=a lot; 3=very much. The absolute change of total DQLI score from before and after treatment
Time frame: baseline, 24 weeks
Population: One subject withdrew and three subjects were non-compliant with study procedures. Data was not analyzed for a total of four subjects.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Valchor Treatment of Lichen Planopilaris | Change in Dermatology Quality of Life Index (DQLI) | -2 score on a scale |
p-value: 0.093Wilcoxon (Mann-Whitney)
Secondary
Change in Follicular Units
Phototrichograms of all subjects scalps performed using FotoFinder video-epiluminescence microscopy (FotoFinder Systems GmbH) in combination with TrichoScan digital image analysis (TRICHOLOG GmbH and DatInf mbH). The percent change in the total number of hairs per square cm was measured before and after treatment.
Time frame: baseline, 24 weeks
Population: One subject withdrew, and three subjects were non-compliant with study procedures. Data was not was not analyzed for a total of four subjects.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Valchor Treatment of Lichen Planopilaris | Change in Follicular Units | -9.1 percent change |
p-value: 0.57Wilcoxon (Mann-Whitney)
Secondary
Change in Mean Follicular Density
Phototrichograms of all subjects scalps performed using FotoFinder video-epiluminescence microscopy (FotoFinder Systems GmbH) in combination with TrichoScan digital image analysis (TRICHOLOG GmbH and DatInf mbH). The percent change in the mean number of follicular units per square cm was measured before and after treatment.
Time frame: baseline, 24 weeks
Population: One subject withdrew and three subjects were non-compliant with study procedures. Data was not analyzed for a total of four subjects.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Valchor Treatment of Lichen Planopilaris | Change in Mean Follicular Density | -9.0 percent change |
p-value: 0.55Wilcoxon (Mann-Whitney)