Daily Topical Rapamycin for Vitiligo

Daily Topical Rapamycin Therapy for the Treatment of Vitiligo

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.

Study Overview

• Status: Active, not recruiting
• Conditions: Vitiligo
• Intervention / Treatment: Drug: Rapamycin; Drug: Rapamycin; Drug: Placebo

• Study Type: Interventional
• Enrollment (Estimated): 20
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • South Carolina
    • Charleston, South Carolina, United States, 29425
      • Medical University of South Carolina

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 13 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Topical rapamycin 0.001%; Patients randomized to this arm of the study will apply 2 finger tip units (FTU) or 0.5 cc topical rapamycin 0.001% cream daily for 6 months	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.; Other Names: Sirolimus, rapamune; Drug: 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.; Other Names: Sirolimus, rapamune
Active Comparator: Topical rapamycin 0.1%; Patients randomized to this arm of the study will apply 2 FTU (0.5 cc) topical rapamycin 0.1% cream daily for 6 months	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.; Other Names: Sirolimus, rapamune; Drug: 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.; Other Names: Sirolimus, rapamune
Placebo Comparator: Placebo; All patients will apply 2 FTU (0.5 cc) topical placebo cream daily for 6 months to the corresponding anatomic location on the opposite side of the body where the experimental drug is not being applied	Drug: Placebo; All patients will be assigned to received topical placebo cream to the lesion not being treated with the active study drug.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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
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
Numbers of lesions with complete response	Targeted lesion is no longer grossly visible.	6 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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

Collaborators and Investigators

• Sponsor: Medical University of South Carolina
• Collaborators: American Skin Association
• Investigators: Principal Investigator: Ahmad Aleisa, MD, Medical University of South Carolina

Study record dates

Study Major Dates

• Study Start (Actual): July 28, 2022
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): December 1, 2027

Study Registration Dates

• First Submitted: April 19, 2022
• First Submitted That Met QC Criteria: April 19, 2022
• First Posted (Actual): April 22, 2022

Study Record Updates

• Last Update Posted (Actual): March 5, 2026
• Last Update Submitted That Met QC Criteria: March 3, 2026
• Last Verified: March 1, 2026

More Information

Terms related to this study

• Keywords: Pediatrics; Rapamycin; Dermatology
• Additional Relevant MeSH Terms: Skin Diseases; Hypopigmentation; Pigmentation Disorders; Skin and Connective Tissue Diseases; Vitiligo; Organic Chemicals; Macrolides; Lactones; Sirolimus
• Other Study ID Numbers: 115924

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes