What side effects are associated with this type of intervention?

Common treatments for vitiligo that modulate the immune response, such as topical corticosteroids and calcineurin inhibitors (e.g., tacrolimus and pimecrolimus), can have several side effects. Topical corticosteroids may cause skin thinning, stretch marks, and increased susceptibility to skin infections. Calcineurin inhibitors can lead to burning or itching sensations at the application site, and there is a potential risk of systemic absorption leading to immunosuppression. Topical rapamycin, being studied for vitiligo, may similarly cause local irritation and potential systemic immunosuppressive effects, although specific side effects for this treatment are still under investigation.

What prior FDA approvals exist?

The FDA has approved several treatments for vitiligo, though options remain limited. Immunosuppressive agents like topical corticosteroids and calcineurin inhibitors (e.g., tacrolimus and pimecrolimus) are commonly used off-label to manage the condition by modulating the immune response. These treatments aim to reduce inflammation and promote repigmentation of the skin. While topical rapamycin is being studied for its efficacy in treating vitiligo, it is not yet FDA-approved for this indication.

What other types of research exist?

Promising novel alternatives to Topical Rapamycin for Vitiligo patients include: 1) Photodynamic therapy combined with microneedle treatment, which has shown potential in treating extensive alopecia areata and may be applicable to Vitiligo. 2) Simvastatin/ezetimibe therapy, which has demonstrated hair regrowth in alopecia areata and could be explored for its immunomodulatory effects in Vitiligo. 3) Low-dose oral minoxidil, which has been reviewed for non-scarring alopecia and may offer benefits in immune modulation and repigmentation.

← Back to Search

mTOR inhibitor

Topical Rapamycin for Vitiligo

Phase 2

Waitlist Available

Led By Ahmad Aleisa, MD

Research Sponsored by Medical University of South Carolina

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Patients 13 years of age and older

Must not have

Patients with a history of a serious or life-threatening infection

Patients taking strong CYP3A4 inducers

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 6 months

Awards & highlights

Approved for 5 Other Conditions

All Individual Drugs Already Approved

Summary

This trial tests if a daily cream with rapamycin can help treat vitiligo, a skin condition causing color loss. Participants will use the cream on one side of their body and a different cream on the other for several months. The study will also check patient satisfaction and any side effects. Rapamycin has been studied for its potential to stabilize cyst growth in ADPKD, but larger trials showed no benefit on kidney function.

Who is the study for?

This trial is for people aged 13 and older with nonsegmental vitiligo, which causes loss of skin color in blotches. It's not for those on certain medications affecting the immune system, have had transplants, serious infections, are pregnant or currently treating vitiligo.

What is being tested?

The study tests if daily use of a cream called rapamycin can help with vitiligo. Participants will apply two strengths of rapamycin to different body lesions and a placebo to another lesion for six months to compare effectiveness and satisfaction.

What are the potential side effects?

Possible side effects may include local skin reactions like redness or irritation where the cream is applied. Since rapamycin affects the immune system, there might be an increased risk of infection or other less common systemic effects.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I am 13 years old or older.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

I have had a serious or life-threatening infection in the past.

Select...

I am currently taking medication that strongly affects liver enzymes.

Select...

I am currently being treated for vitiligo.

Select...

I am currently taking medication that affects liver enzyme levels.

Select...

I cannot take rapamycin due to health reasons.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 6 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~6 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 6 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Number of lesions with no response

Number of lesions with partial response

Numbers of lesions with complete response

Secondary study objectives

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

Side effects data

From 2009 Phase 4 trial • 20 Patients • NCT00223678

31%

pneumonia

15%

acute kidney failure

dehydration

hip replacement

acute rejection

total abdominal hysterectomy and salpingectomy

anemia

100%

80%

60%

40%

20%

Study treatment Arm

Rapamycin Group

CNI Group

Awards & Highlights

Approved for 5 Other Conditions

This treatment demonstrated efficacy for 5 other conditions.

All Individual Drugs Already Approved

Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

Trial Design

3Treatment groups

Experimental Treatment

Active Control

Placebo Group

Group I: Topical rapamycin 0.001%Experimental Treatment1 Intervention

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 II: Topical rapamycin 0.1%Active Control1 Intervention

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 III: PlaceboPlacebo Group1 Intervention

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

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Sirolimus

FDA approved

0 / 6Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Vitiligo treatments often aim to modulate the immune response to prevent the immune system from attacking melanocytes, the cells responsible for skin pigmentation. Topical rapamycin, an immunosuppressive agent, works by inhibiting the mTOR pathway, which reduces the activity of T-cells that contribute to the destruction of melanocytes. This helps in stabilizing the disease and potentially promoting repigmentation. Other common treatments include corticosteroids and calcineurin inhibitors, which also suppress the immune response and reduce inflammation. These mechanisms are crucial for Vitiligo patients as they help in halting the progression of depigmentation and can lead to the restoration of skin color, improving both the physical appearance and psychological well-being of patients.