Valchlor in the Treatment of Lichen Planopilaris

Phase 2

Completed

Conditions: Lichen Planopilaris

Interventions: Drug: Mechlorethamine 0.016% Top Gel

Registration Number: NCT03417141

Lead Sponsor: Mayo Clinic

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.

Recruitment & Eligibility

Status: COMPLETED

Sex: All

Target Recruitment: 12

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

Study & Design

Study Type: INTERVENTIONAL

Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Valchor treatment of Lichen Planopilaris	Mechlorethamine 0.016% Top Gel	Once daily application of Valchlor in decreasing disease activity in patients with Lichen Planopilaris.

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
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 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 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.