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OpenTrials
Active, Not Recruiting

NCT Number: NCT05342519

Daily Topical Rapamycin for 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.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Conditions

Vitiligo Hypopigmentation Pigmentation Disorders Skin Diseases Skin and Connective Tissue Diseases

Age range

13 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Rapamycin

Drug

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

Placebo

Drug

All patients will be assigned to received topical placebo cream to the lesion not being treated with the active study drug.

Primary outcomes

  1. Number of lesions with no response

    Time frame: 6 months

    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

  2. Number of lesions with partial response

    Time frame: 6 months

    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.

  3. Numbers of lesions with complete response

    Time frame: 6 months

    Targeted lesion is no longer grossly visible.

Secondary outcomes

  1. Average score of Dermatology Quality of Life questionnaire (or Teenager Quality of Life Index)

    Time frame: 6 months

    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.

Sponsors and collaborators

Lead sponsor

Medical University of South Carolina

Other

Collaborators

  • American Skin Association

Registry information

Official study title

Daily Topical Rapamycin Therapy for the Treatment of Vitiligo

Important dates

Study start
2022
Primary completion
2027
Study completion
2027
First posted
Apr 22, 2022
Registry last updated
Mar 5, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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