VYN201 Gel for Vitiligo

Not currently recruiting at 50 trial locations

Overseen By Iain Stuart, PhD

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Vyne Therapeutics Inc.

Must not be taking: Biologics, Immune-modulating, Photosensitivity, others

Disqualifiers: Other vitiligo, Dermatologic conditions, Infections, others

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

Compensation: Varies

Number of Visits: Unknown

Duration: 24 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a gel called VYN201 (also known as repibresib) to determine its effectiveness for people with non-segmental vitiligo, a condition where patches of skin lose color. Researchers aim to assess the gel's efficacy, safety, and how the body processes it. Participants will apply either the gel or a placebo (a harmless gel with no active ingredient) daily on their vitiligo spots. The trial seeks individuals with non-segmental vitiligo affecting less than 10% of their body who are willing to stop other vitiligo treatments during the study. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to potential new therapies for vitiligo.

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

You will need to stop taking any medications used to treat vitiligo during the trial. However, if you are taking other medications for different conditions, you can continue them as long as your regimen is stable from the start of the trial to its completion.

Is there any evidence suggesting that VYN201 Gel is likely to be safe for humans?

Research has shown that VYN201 Gel was well-tolerated in earlier studies for treating vitiligo. In a previous study with patients who have non-segmental vitiligo, the treatment improved their condition without causing serious side effects. This suggests that the gel is generally safe for use.

Although no serious side effects were reported, this trial remains part of ongoing research to further evaluate the treatment's safety and effectiveness. Researchers closely monitor participants to ensure their safety throughout the study. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Researchers are excited about VYN201 Gel for vitiligo because it offers a novel approach compared to current treatments like corticosteroids and calcineurin inhibitors. VYN201 Gel is unique as it is applied directly to the skin, targeting affected areas with potentially fewer systemic side effects. Unlike the standard treatments, which can take longer to show results and may have more widespread effects on the body, VYN201 Gel could provide a more localized and efficient treatment option. Its specific formulation may lead to enhanced repigmentation and improved outcomes for vitiligo patients, making it a promising alternative in dermatological care.

What evidence suggests that VYN201 Gel might be an effective treatment for vitiligo?

Research on VYN201 Gel, also known as repibresib, has shown mixed results for treating non-segmental vitiligo. Recent studies did not achieve their primary goals, such as F-VASI50 and F-VASI75, which measure 50% and 75% skin repigmentation, respectively. However, some results were statistically significant, indicating promise in certain areas. While the gel's potential remains under investigation, these initial findings suggest it may have some beneficial effects, but further research is needed to confirm its efficacy. ^{3 4 6 7 8}

Are You a Good Fit for This Trial?

This trial is for individuals with non-segmental vitiligo, a condition where skin loses its pigment. Details about who can join or reasons why someone might not be eligible are not provided.

Inclusion Criteria

I have vitiligo affecting less than 10% of my body.

Has completed and signed an Informed Consent Form (ICF) prior to any study-related procedures

I understand and can follow the study's requirements.

Exclusion Criteria

I don't have skin injuries near the test site that could affect the study.

My hair in the treatment area is not dyed.

I have had treatments to change the color of my skin.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive VYN201 Gel or placebo once daily for 24 weeks in a double-blind, vehicle-controlled period

24 weeks

Treatment Extension

Participants continue treatment with VYN201 Gel for an additional 28 weeks, maintaining the blind

28 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• VYN201 Gel

Trial Overview The study is testing the effectiveness and safety of VYN201 Gel compared to a Vehicle Gel (a gel without the active drug) in treating non-segmental vitiligo. It also looks at how the body processes the gel.

How Is the Trial Designed?

Group I: Low dose VYN201 (1.0%) Active Control 1 Intervention

Subjects will apply VYN201 1.0% once daily on affected vitiligo areas. The first part of the study will be a 24-week, double-blind, vehicle controlled treatment period. Following, the second part will be a 28-week, double-blind treatment extension period, with a 4-week safety follow-up period after the last investigational medicinal product (IMP) dose. After completion of the Week 24 assessments, subjects randomized to 1.0% will remain and continue on the same dose until Week 52.

Group II: Mid dose VYN201 (2.0%) Active Control 1 Intervention

Subjects will apply VYN201 2.0% once daily on affected vitiligo areas. The first part of the study will be a 24-week, double-blind, vehicle controlled treatment period. Following, the second part will be a 28-week, double-blind treatment extension period, with a 4-week safety follow-up period after the last investigational medicinal product (IMP) dose. After completion of the Week 24 assessments, subjects randomized to 2.0% will remain and continue on the same dose until Week 52.

Group III: High dose VYN201 (3.0%) Active Control 1 Intervention

Subjects will apply VYN201 3.0% once daily on affected vitiligo areas. The first part of the study will be a 24-week, double-blind, vehicle controlled treatment period. Following, the second part will be a 28-week, double-blind treatment extension period, with a 4-week safety follow-up period after the last investigational medicinal product (IMP) dose. After completion of the Week 24 assessments, subjects randomized to 3.0% will remain and continue on the same dose until Week 52.

Group IV: Vehicle Placebo Group 2 Interventions

Subjects will apply VYN201 vehicle (placebo) once daily on affected vitiligo areas. The first part of the study will be a 24-week, double-blind, vehicle controlled treatment period. Following, the second part will be a 28-week, double-blind treatment extension period, with a 4-week safety follow-up period after the last investigational medicinal product (IMP) dose. After completion of the Week 24 assessments, subjects randomized to vehicle (placebo) will be re-randomized to 1 of the 3 higher VYN201 (active) dose groups (1.0%, 2.0% or 3.0% once daily) in a 1:1:1 ratio while maintaining the blind until Week 52.

Who Is Running the Clinical Trial?

Vyne Therapeutics Inc.

Lead Sponsor

Trials

37

Recruited

9,700+

Published Research Related to This Trial

Photodynamic therapy using verteporfin effectively occluded bilateral corneal neovascularization and significantly reduced lipid keratopathy in a 36-year-old patient, leading to improved visual acuity from 20/80 and 20/100 to 20/30 in both eyes over a 4-year follow-up period.

The treatment was safe, with no observed recurrences or complications during the follow-up, suggesting that verteporfin laser therapy is a reliable option for managing corneal neovascularization.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Resolution of bilateral corneal neovascularization and lipid keratopathy after photodynamic therapy with verteporfin. Al-Abdullah, AA., Al-Assiri, A.[2018]

Excimer light (EL) therapy showed superior repigmenting efficacy in vitiligo patients compared to targeted UVB (TUVB), with 82.6% of EL patients achieving at least 50% repigmentation versus 76.3% in the TUVB group.

Patients receiving EL required fewer treatment sessions and lower cumulative doses to achieve complete or near-complete repigmentation, indicating it may be a more efficient option for vitiligo treatment, with negligible adverse effects reported in both groups.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Is targeted UVB as effective as excimer light phototherapy in treatment of vitiligo? Tabassum, H., Majid, I., Imran, S.[2021]

In a study involving five patients with non-segmental vitiligo, the addition of heterologous type I collagen (HTIC) to narrowband ultraviolet B (NB-UVB) therapy resulted in a significantly higher mean repigmentation rate of 70.5% after 12 weeks, compared to just 16.5% with NB-UVB alone.

Repigmentation was observed to begin as early as the first treatment session for some patients, indicating that HTIC may provide a more rapid onset of repigmentation when used alongside NB-UVB therapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Heterologous Type I Collagen as an Add-on Therapy to Narrowband Ultraviolet B for the Treatment of Vitiligo: A Pilot Study. Gkouvi, A., Nicolaidou, E., Corbo, A., et al.[2022]

Vitiligo affects 0.5 to 2% of the global population and currently has no FDA-approved treatments specifically for repigmentation, highlighting a significant gap in effective therapies.

Topical medications, particularly steroid formulations and immunomodulatory agents like tacrolimus, are the primary treatment options available, while new therapies such as Janus Kinase inhibitors are still under development.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A Potential Alternative Treatment for Vitiligo: An Observational Study on Tacrolimus 0.3% Lotion. Deverapalli, S., Kahn, J., Rosmarin, D.[2022]

In a study involving 10 patients with stable vitiligo, the 308-nm Excilite mu phototherapy showed promising results, with 60% of patients experiencing repigmentation within 2 weeks, as reported by patients, and 50% confirmed by physicians.

The treatment was found to be safe, with all patients maintaining their repigmentation during a 5-week follow-up period, indicating its potential as a rapid and effective option for patients with skin types 3 through 6.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A pilot study to determine the safety and efficacy of monochromatic excimer light in the treatment of vitiligo. Chimento, SM., Newland, M., Ricotti, C., et al.[2022]

Verteporfin therapy leads to short-lived skin photosensitivity, peaking at 1.5 hours post-treatment and decreasing rapidly, with a duration of 2 to 6.7 days depending on the dose.

In clinical trials, only 2.2% of patients experienced photosensitivity reactions, indicating a low incidence of adverse effects, which could be minimized with proper skin protection following treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Duration of skin photosensitivity and incidence of photosensitivity reactions after administration of verteporfin. Houle, JM., Strong, HA.[2019]

Vemurafenib, a BRAF inhibitor used for treating late-stage melanoma with BRAF V600E mutations, can cause various skin reactions, including a rare case of vitiligo, as reported in a 63-year-old patient after 4 weeks of treatment.

The development of vitiligo in this patient was confirmed through clinical examination and advanced imaging techniques, raising questions about whether this adverse reaction could be linked to positive treatment outcomes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Vitiligo of the Face in a Patient Treated With Vemurafenib for Metastatic Melanoma. Nasca, MR., Lacarrubba, F., Ferraù, F., et al.[2018]

The study identified a significant association between vitiligo and several drugs, particularly immune checkpoint inhibitors like pembrolizumab and nivolumab, with reporting odds ratios indicating a strong link to vitiligo occurrence.

New associations were also found with selective immunosuppressants such as alemtuzumab and interleukin inhibitors, suggesting that these classes of drugs may contribute to the development of vitiligo.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Drug-induced vitiligo: a case/non-case study in Vigibase® , the WHO pharmacovigilance database. Anthony, N., Bourneau-Martin, D., Ghamrawi, S., et al.[2021]

A new treatment for vitiligo using hydrogel microneedles to deliver alpha-melanocyte-stimulating hormone (α-MSH) and tofacitinib shows promise in promoting skin and hair pigmentation.

The microneedles effectively penetrate the skin barrier to deliver these agents directly to the epidermis and dermis, resulting in significant melanin deposition and accelerated pigmentation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Tofacitinib combined with melanocyte protector α-MSH to treat vitiligo through dextran based hydrogel microneedles. Liang, J., Yu, Y., Li, C., et al.[2023]

In a phase 2 trial involving patients with vitiligo, those using 1.5% ruxolitinib cream showed a significant improvement in facial Vitiligo Area Scoring Index scores, with a mean improvement of 79.9% at week 24 compared to only 1.1% in those who did not respond.

Proteomic analysis revealed distinct inflammatory biomarker profiles between patients who achieved at least a 50% improvement and those who did not, suggesting that these biomarkers could help identify which patients are more likely to benefit from ruxolitinib treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Baseline Levels of Circulating Inflammatory Biomarkers Stratify Patients with Vitiligo Who Significantly Repigment after Treatment with Ruxolitinib Cream. Howell, MD., Kuo, FI., Rumberger, B., et al.[2023]

The combination of apremilast and narrowband UVB therapy significantly reduces immune cell markers (like CD8+ T cells) and enhances melanogenesis markers in vitiligo skin, suggesting a more effective treatment approach than narrowband UVB alone.

This study supports the clinical observation that apremilast can enhance the repigmentation effects of narrowband UVB therapy in patients with generalized vitiligo, particularly in skin types IV-VI, although it was limited by a small sample size and high dropout rate.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Apremilast and narrowband ultraviolet B combination therapy suppresses Th17 axis and promotes melanogenesis in vitiligo skin: a randomized, split-body, pilot study in skin types IV-VI. Kim, HJ., Del Duca, E., Pavel, AB., et al.[2023]

In a study of 12 patients receiving subretinal voretigene neparvovec (VN) gene therapy for retinal disease, 39% of eyes developed vitritis, an inflammation of the eye, which typically resolved within 89 days with appropriate treatment.

Despite the occurrence of inflammation, it did not negatively impact visual outcomes, although monitoring and treatment are essential to prevent potential long-term damage to the retina.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Inflammation after Voretigene Neparvovec Administration in Patients with RPE65-Related Retinal Dystrophy. Kessel, L., Christensen, UC., Klemp, K.[2022]

Ranibizumab (Lucentis®) significantly improves visual acuity in patients with macular oedema following retinal vein occlusion, as shown in two phase III trials with monthly injections leading to better outcomes than sham injections at 6 months.

The treatment was generally well tolerated, with adverse events aligning with those seen in other conditions treated with ranibizumab, indicating a favorable safety profile.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Ranibizumab: in macular oedema following retinal vein occlusion. Garnock-Jones, KP.[2021]

Improvements in functional vision, including ambulatory navigation and light sensitivity, were maintained for 3 to 4 years after voretigene neparvovec (VN) treatment in patients with biallelic RPE65 mutation-associated inherited retinal disease, indicating long-term efficacy.

The safety profile of VN was favorable, with no serious adverse events related to the treatment reported, although one patient experienced retinal detachment, which is consistent with risks associated with the injection procedure.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Durability of Voretigene Neparvovec for Biallelic RPE65-Mediated Inherited Retinal Disease: Phase 3 Results at 3 and 4 Years. Maguire, AM., Russell, S., Chung, DC., et al.[2021]

Citations

1. vynetherapeutics.com vynetherapeutics.com/press-releases/vyne-therapeutics-announces-topline-results-from-phase-2b-trial-with-repibresib-gel-in-nonsegmental-vitiligo/

VYNE Therapeutics Announces Topline Results from ... Trial Did Not Meet Primary Endpoint or Key Secondary Endpoint of F-VASI50 and F-VASI75. Nominally Statistically Significant Effects Observed ...

2. dermatologytimes.com dermatologytimes.com/view/phase-2b-repibresib-study-falls-short-of-key-goals

Phase 2b Repibresib Study Falls Short of Key Goals VYNE Therapeutics reveals disappointing phase 2b trial results for repibresib gel in nonsegmental vitiligo, missing key efficacy endpoints.

3. docs.publicnow.com docs.publicnow.com/viewDoc.aspx?filename=177175%5CEXT%5CEA0EFB4DB3DF4B7E607EA2183B9E6332B6715A1C_44AB107D5E38999A4F887D136CCEBAA6CF99936B.PDF

Repibresib (VYN201) Phase 2b Topline Results Repibresib (VYN201) Phase 2b Topline Results. Repibresib in Subjects with Non-segmental Vitiligo. July 2025. Page 2. Forward Looking Statements ...

4. clinicaltrialsarena.com clinicaltrialsarena.com/news/vyne-vitiligo-gel-trial/

VYNE Therapeutics completes enrolment in vitiligo gel trial VYNE Therapeutics has completed enrolment in the randomised Phase IIb clinical trial of VYN201 gel, known as repibresib, for treating non-segmental vitiligo.

5. sec.gov sec.gov/Archives/edgar/data/1566044/000162828025010777/vyne-20250306q42024exx991.htm

Repibresib gel (VYN201) Phase 2b vitiligo top-line results ... Top-line results from the Phase 2b trial are expected in mid-2025. ... Subjects have been randomized equally (1:1:1:1 ratio) across the active ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT06493578

NCT06493578 | A Study to Evaluate the Efficacy, Safety, ... The purpose of this study is to evaluate the efficacy, safety, and pharmacokinetics of VYN201 Gel in subjects with non-segmental vitiligo. Official Title. A ...

7. dermatologytimes.com dermatologytimes.com/view/vyn201-for-vitiligo-phase-1b-study-confirms-safety

VYN201 for Vitiligo: Phase 1b Study Confirms Safety VYNE Therapies announces that aNSV patients saw disease improvement without serious adverse effects.

8. ctv.veeva.com ctv.veeva.com/study/a-study-to-evaluate-the-efficacy-safety-and-pharmacokinetics-of-vyn201-gel-in-subjects-with-non-se

A Study to Evaluate the Efficacy, Safety, and Pharmacokinetics ... The purpose of this study is to evaluate the efficacy, safety, and pharmacokinetics of VYN201 Gel in subjects with non-segmental vitiligo.