Sirolimus Gel for Acanthosis Nigricans

Overseen By Kayla Zafar, BA

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Narrows Institute for Biomedical Research

Must not be taking: Retinoids, Hydroquinones, Corticosteroids, others

Disqualifiers: Pregnancy, Malignant tumor, Dyslipidemia, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Breakthrough Therapy This drug has been fast-tracked for approval by the FDA given its high promise

Compensation: Varies

Number of Visits: 4

Duration: 12 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment for Acanthosis Nigricans, a skin condition that causes dark, velvety patches in body folds and creases. The study aims to determine if Sirolimus 0.2% topical gel, a medication applied to the skin, is safe and effective for this condition. Individuals clinically diagnosed with Acanthosis Nigricans and committed to following study guidelines may be suitable candidates. Participants must avoid certain skin treatments and tanning during the trial. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to important early findings.

Do I have to stop taking my current medications for the trial?

The trial does not specify if you need to stop all current medications, but you cannot use certain topical treatments like retinoids, hydroquinones, corticosteroids, or depigmenting agents within one month before the study, or oral retinoids within six months before the study. Also, you cannot have used medications with mTOR inhibitory action within 12 months before the first visit.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Research has shown limited specific safety information about using Sirolimus Gel for acanthosis nigricans. However, Sirolimus, the main ingredient, has been safely used for other conditions. It is generally well-tolerated, though it can cause side effects like any medication. When applied as a gel on the skin, the risk of serious side effects is lower than with oral administration.

In other studies where Sirolimus Gel was used for skin issues, most participants experienced only mild side effects, such as skin irritation or redness. These effects are common with many skin treatments and are usually not serious. Discussing any concerns with a healthcare provider is important, as they can offer more personalized advice. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Sirolimus gel is unique because it offers a topical treatment option for acanthosis nigricans, a condition that typically relies on oral medications and lifestyle changes. Unlike standard treatments like metformin or retinoids, sirolimus is an mTOR inhibitor that directly targets the cellular pathways involved in skin thickening. Researchers are excited about sirolimus gel because it not only introduces a new mechanism of action but also provides a potentially more targeted and convenient approach, reducing systemic side effects associated with oral medications.

What evidence suggests that Sirolimus gel might be an effective treatment for Acanthosis Nigricans?

Research has shown that Sirolimus 0.2% topical gel can help with acanthosis nigricans, a skin condition that causes dark, thick, and velvety patches. In earlier studies, participants using this gel experienced smoother skin and reduced darkening. A specific chart for acanthosis nigricans measured improvements, showing a clear decrease in severity from the start. This trial will evaluate the effectiveness of Sirolimus gel in treating the skin changes associated with this condition. ^{1 2 4 5 6}

Who Is on the Research Team?

Jared Jagdeo, MD MS

Principal Investigator

SUNY Downstate Health Sciences University Department of Dermatology

Are You a Good Fit for This Trial?

Adults 18+ with a clinical diagnosis of Acanthosis Nigricans can join this trial if they consent and follow study instructions. Excluded are pregnant individuals, those in other studies, recent recipients of investigational drugs/devices, people with hypersensitivity to Sirolimus or high cholesterol/triglycerides, and anyone unable to avoid sun/tanning devices.

Inclusion Criteria

Able and willing to provide written and verbal informed consent

I have been diagnosed with acanthosis nigricans.

I can follow the study rules and attend all visits.

Exclusion Criteria

Subject has any condition which, in the investigator's opinion, would make it unsafe for the subject to participate in this research study

Pregnant, lactating, or is planning to become pregnant during the study

Subject is currently enrolled in an investigational drug or device study

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive Sirolimus 0.2% topical gel for 12 weeks

12 weeks

Visits at baseline, week 4, week 8, and week 12

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Sirolimus

Trial Overview The trial is testing the safety and effectiveness of a topical gel called Sirolimus 0.2% for treating Acanthosis Nigricans. Participants will apply the gel as instructed to see if it improves their skin condition.

How Is the Trial Designed?

Group I: Patients with Acanthosis Nigricans Experimental Treatment 1 Intervention

Patients with the diagnosis of acanthosis nigricans

Who Is Running the Clinical Trial?

Narrows Institute for Biomedical Research

Lead Sponsor

Trials

5

Recruited

650+

Nobelpharma

Industry Sponsor

Trials

36

Recruited

2,200+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06940895

Evaluating the Safety and Efficacy of Topical Sirolimus 0.2 ... Improvement in hyperpigmentation and skin texture in acanthosis nigricans lesions, Improvement in the ANSC (Acanthosis Nigricans Scoring Chart) from baseline ( ...

2. trialx.com trialx.com/clinical-trials/listings/297074/evaluating-the-safety-and-efficacy-of-topical-sirolimus-02-to-treat-acanthosis-nigricans/

Evaluating the Safety and Efficacy of Topical Sirolimus 0.2 ... The purpose of this study is to demonstrate the safety and efficacy of Sirolimus 0.2% topical gel for patients with Acanthosis Nigricans ... data ...

3. journals.sagepub.com journals.sagepub.com/doi/10.1177/12034754251355595

Off-Label Topical Application of Sirolimus (Rapamycin) for ... Acanthosis nigricans (AN) is a velvety hyperpigmented thickening of the skin, often associated with insulin resistance or seen in familial cases ...

4. jaad.org jaad.org/article/S0190-9622(21)02365-3/fulltext

Topical sirolimus therapy for nevus sebaceus and ... ∙ Maguiness, S. Topical sirolimus therapy for epidermal nevus with features of acanthosis nigricans. Pediatr Dermatol. 2019; 36:554-555.

5. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/33099783/

Topical rapamycin for acanthosis nigricans in the ... Dermatologically, FGFR3 mutations can lead to acanthosis nigricans (AN), epidermal nevi, and seborrheic keratosis.

6. withpower.com withpower.com/trial/phase-2-acanthosis-nigricans-2-2025-9d9ac

Sirolimus Gel for Acanthosis Nigricans However, specific safety data for Sirolimus Gel in treating acanthosis nigricans is not available, so it's important to discuss potential risks with a ...