Daily Topical Rapamycin for Vitiligo

Vitiligo Clinical Trial

Official title:

Daily Topical Rapamycin Therapy for the Treatment of Vitiligo

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT05342519
• Other study ID #: 115924
• Status: Active, not recruiting
• Phase: Phase 2
• Start date: July 28, 2022
• Est. completion date: August 1, 2024

Study information

• Verified date: August 2023
• Source: Medical University of South Carolina
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

In current Dermatology practice, options for vitiligo remain limited. The purpose of this study is to determine if once daily dosed topical rapamycin is effective for the treatment of patients with vitiligo. Participants will apply either 0.1% topical rapamycin or 0.001% topical rapamycin for six months to a lesion on one side of the body, and topical placebo to a corresponding lesion on the opposite side of the body. The study also aims to evaluate patient satisfaction and identify any adverse effects on these dosing regimens.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 20
• Est. completion date: August 1, 2024
• Est. primary completion date: August 1, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 13 Years and older

Eligibility

Inclusion Criteria:

• Patients 13 years of age and older
• Patients with nonsegmental vitiligo

Exclusion Criteria:

• Patients with segmental or mucosal vitiligo
• Patients with contraindication to use of rapamycin
• Patients with history of transplant
• Patients with a history of natural immunodeficiency
• Patients with a history of artificially induced immunodeficiency
• Patients with a history of a serious or life-threatening infection
• Patients taking CYP3A4 inhibiting medications
• Patients taking strong CYP3A4 inducers
• Patients undergoing current treatment for vitiligo
• Women that are or may become pregnant

Related Conditions & MeSH terms

• Vitiligo

Intervention

Drug:
• Rapamycin
While rapamycin is not approved for the treatment of vitiligo, it has been found to be efficacious in stopping disease progression in animal models. Another recent study in humans showed that doses of 0.5cc daily of 0.001% of the topical formulation are able to achieve improvement in hypopigmented lesions of the skin at both the clinical and histological level. At this dose, improvement in cell function was observed regarding maintenance of proliferative potential and prevention of senescence, with avoidance of total inhibition of cell growth as has been seen at higher potencies used for immunosuppression.
• Rapamycin
Rapamycin is an immunosuppressant that works by inhibiting the mammalian target of rapamycin (mTOR) leading to inhibition of the cell cycle and antibody production. It has also been shown to promote expansion of Treg populations. Although studies evaluating alternative dosing for vitiligo are limited, topical rapamycin has previously been used to treat angiofibromas related to Tuberous Sclerosis, vascular malformations such as port-wine stains, and inflammatory lesions such as plaque psoriasis. In a 2019 meta-analysis, 38 out of 40 reports included used topical formulations of 1% or lower potency rapamycin; the majority of publications were focused on the treatment of Tuberous Sclerosis, where the median concentration of mTOR inhibition was 0.1% dosed twice daily.
• Placebo
All patients will be assigned to received topical placebo cream to the lesion not being treated with the active study drug.

Locations

Country	Name	City	State
United States	Medical University of South Carolina	Charleston	South Carolina

Sponsors (2)

Lead Sponsor	Collaborator
Medical University of South Carolina	American Skin Association

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of lesions with no response	Absence of change in appearance of targeted lesion. Vitiligo is a depigmenting disease and treatment responses are graded by percentage of repigmentation on a 5-point scale	6 months
Primary	Number of lesions with partial response	There is change in the size of dyspigmentation as compared to its appearance and symptoms at the initial visit, but the lesion remains grossly visible. Vitiligo is a depigmenting disease and treatment responses are graded by percentage of repigmentation on a 5-point scale. Changes in the characteristics of the targeted lesions will be evaluated by a dermatologist and documented via photographs at follow-up visits at 2, 4, and 6-months, with the final outcome measured at the 6-month visit.	6 months
Primary	Numbers of lesions with complete response	Targeted lesion is no longer grossly visible.	6 months
Secondary	Average score of Dermatology Quality of Life questionnaire (or Teenager Quality of Life Index)	The Dermatology Life Quality Index (DLQI) is designed to measure the health-related quality of life of adult (or teenage) patients suffering from a skin disease. Both physical and psychological symptoms are ranked on a 4-point scale from "not at all" to "very much" to provide a cumulative patient score.	6 months