Daily Topical Rapamycin for Vitiligo

NCT05342519 | Active Not Recruiting Phase 2 FDA Drug

• 1 other identifier: 115924
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Vitiligo

Keywords

Dermatology; Pediatrics; Rapamycin

Outcome Measures

Primary Outcomes (3)

• 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 Outcomes (1)

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

  6 months

Study Arms (3)

Topical rapamycin 0.001%

EXPERIMENTAL

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

Topical rapamycin 0.1%

ACTIVE COMPARATOR

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

Placebo

PLACEBO COMPARATOR

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

Interventions

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.

Also known as: Sirolimus, rapamune

Topical rapamycin 0.001%

Placebo DRUG

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

Placebo

Eligibility Criteria

• Age: 13 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• 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

Sponsors & Collaborators

• American Skin Association: collaborator
• Medical University of South Carolina: lead

Study Sites (1)

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

MeSH Terms

Conditions

Vitiligo

Interventions

Sirolimus

Condition Hierarchy (Ancestors)

Hypopigmentation; Pigmentation Disorders; Skin Diseases; Skin and Connective Tissue Diseases

Intervention Hierarchy (Ancestors)

Macrolides; Lactones; Organic Chemicals

Study Officials

• Ahmad Aleisa, MD

  Medical University of South Carolina

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: The pharmacy will distribute medications in unlabeled, 60-gram tubes following patient enrollment. Tubes will be marked as "Left" and "Right." The association between tube label and treatment type will only be known to pharmacy staff. The pharmacy staff will allocate 10 patients to receive 0.001% rapamycin and 10 patients to receive 0.1% rapamycin.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Medical/Dental Resident

Model Details: During a clinical care visit, if a patient is eligible and agrees to participate, one lesion occupying less than 2% body surface area (BSA) will be identified for treatment with either 0.1% or 0.001% topical rapamycin cream to be applied daily for 6 months. Ten patients will be randomized to each treatment arm, however, if it is clear that one treatment arm is less effective, that treatment arm will be dropped, and study participants will be reallocated to the more effective treatment arm. A second, symmetric lesion about the same size and evolution time occupying less than 2% BSA will be identified for treatment with topical placebo cream to be applied daily for 6 months. The second lesion must be at the corresponding anatomic location on the other half of the body, i.e., if topical rapamycin is applied to the left arm, topical placebo will be applied to the right arm.

Study Record Dates

• First Submitted: April 19, 2022
• First Posted: April 22, 2022
• Study Start: July 28, 2022
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): December 1, 2027
• Last Updated: March 5, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)