VitiligoPhase 2NCT05342519
Daily Topical Rapamycin for Vitiligo
A Phase 2 study testing Rapamycin in people with Vitiligo, run by Medical University of South Carolina at 1 site in United States.
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. Read more about this study
- Who is it for?
- Ages 13 years and older
- Conditions the study is for
- Vitiligo
- What is being tested?
- Rapamycin
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- 28 Jul 2022to 1 Dec 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 13 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients 13 years of age and older
- Patients with nonsegmental vitiligo
Who cannot join
- 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
Show all 10Show fewer
- Patients taking CYP3A4 inhibiting medications
- Patients taking strong CYP3A4 inducers
- Patients undergoing current treatment for vitiligo
- Women that are or may become pregnant
Trial locations and contacts
1 site in 1 country.
United States1 site
Medical University of South Carolina
Charleston, South Carolina, 29425
About this study
Official title: 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.
- Conditions
- Vitiligo
- Being tested
- Rapamycin, Placebo
- Keywords
- Dermatology, Pediatrics, Rapamycin
- Medical subject headings
- Vitiligo
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RapamycinDrug
Also called Sirolimus, rapamune
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.
RapamycinDrug
Also called Sirolimus, rapamune
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.
PlaceboDrug
All patients will be assigned to received topical placebo cream to the lesion not being treated with the active study drug.
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: 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 Receives: 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 Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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.
Measured: 6 months
Numbers of lesions with complete response
Targeted lesion is no longer grossly visible.
Measured: 6 months
Secondary outcomes (1)
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.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Apr 2022
- Study start28 Jul 2022
- Primary completion1 Dec 2027 (estimated)
- Study completion1 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for VitiligoStudies of the same condition, open and under way.
- More trials run by Medical University of South CarolinaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- 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.
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Medical University of South Carolina
- Sponsor type
- University, hospital or other
- Collaborators
- American Skin Association
- Responsible party
- Principal investigator, Chelsea Shope, Medical University of South Carolina
- Investigators
- Ahmad Aleisa, MD, Principal investigator, Medical University of South Carolina
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05342519
- Sponsor's protocol ID
- 115924
- Status last verified
- Mar 2026
- First submitted
- 19 Apr 2022
- Last updated
- 5 Mar 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Daily Topical Rapamycin Therapy for the Treatment of Vitiligo
- ClinicalTrials.gov ID
- NCT05342519
- Acronym / sponsor ID
- 115924
- Conditions
- Vitiligo
- Keywords
- Dermatology, Pediatrics, Rapamycin
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Medical University of South CarolinaUniversity, hospital or other
- Collaborators
- American Skin Association
- Study start
- 28 Jul 2022 (actual)
- Primary completion
- 1 Dec 2027 (estimated)
- Study completion
- 1 Dec 2027 (estimated)
- First posted
- 22 Apr 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
- Model description
- 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.
| Arm | Type | Receives |
|---|---|---|
| Topical rapamycin 0.001% | Experimental | Drug: RapamycinPatients 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 |
| Topical rapamycin 0.1% | Active comparator | Drug: RapamycinPatients randomized to this arm of the study will apply 2 FTU (0.5 cc) topical rapamycin 0.1% cream daily for 6 months |
| Placebo | Placebo comparator | Drug: PlaceboAll 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 |
Who may qualify
- Ages
- 13 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- Patients 13 years of age and older
- Patients with nonsegmental vitiligo
Key exclusion criteria (10 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RapamycinAlso: Sirolimus, rapamuneWhile 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. | Drug | Topical rapamycin 0.001% |
| RapamycinAlso: Sirolimus, rapamuneRapamycin 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. | Drug | Topical rapamycin 0.1% |
| PlaceboAll patients will be assigned to received topical placebo cream to the lesion not being treated with the active study drug. | Drug | Placebo |
Primary outcome
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
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.
Numbers of lesions with complete response
Time frame: 6 months
Targeted lesion is no longer grossly visible.
1 secondary outcome
Average score of Dermatology Quality of Life questionnaire (or Teenager Quality of Life Index)
Time frame: 6 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Mar 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Ahmad Aleisa, MD principal investigator
Medical University of South Carolina
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Rapamycin in people with Vitiligo, run by Medical University of South Carolina at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 5 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of lesions with no response (time frame: 6 months). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Mar 2026.