Daily Topical Rapamycin for Vitiligo

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE2

Total Enrollment

20 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-07-28

Study Completion Date

2026-08-01

Brief Summary

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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.

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Detailed Description

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Conditions

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Vitiligo

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

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.

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

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.

Study Groups

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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

Group Type EXPERIMENTAL

Rapamycin

Intervention Type 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.

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

Group Type ACTIVE_COMPARATOR

Rapamycin

Intervention Type DRUG

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 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

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

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

Interventions

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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.

Intervention Type 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.

Intervention Type DRUG

Placebo

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

Intervention Type DRUG

Other Intervention Names

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Sirolimus, rapamune Sirolimus, rapamune

Eligibility Criteria

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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

Minimum Eligible Age

13 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No