A Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of VYN201 Gel in Subjects With Non-segmental Vitiligo.

A Randomized, Double-Blind, Vehicle-Controlled Phase 2b Trial Evaluating the Efficacy, Safety & Pharmacokinetics of VYN201 Gel in the Treatment of Non Segmental Vitiligo

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

Study Overview

• Status: Terminated
• Conditions: Non-segmental Vitiligo
• Intervention / Treatment: Drug: VYN201 Gel; Drug: Vehicle Gel

• Study Type: Interventional
• Enrollment (Actual): 205
• Phase: Phase 2

Contacts and Locations

Study Locations

• Canada
  • Alberta
    • Calgary, Alberta, Canada, T2J 7E1
      • Dermatology Research Institute
  • Ontario
    • Barrie, Ontario, Canada, L4M 7G1
      • SimcoDerm Medical and Surgical Dermatology Center
    • Markham, Ontario, Canada, L3P 1X3
      • Lynderm Research Inc.
    • Ottawa, Ontario, Canada, K1KL2
      • JRB Research Inc
    • Peterborough, Ontario, Canada, K9J 5K2
      • Skin Centre for Dermatology
    • Toronto, Ontario, Canada, M4W 2N4
      • Research Toronto
  • Quebec
    • Montreal, Quebec, Canada, H3Z 2S6
      • Siena Medical Research
• United States
  • Alabama
    • Birmingham, Alabama, United States, 35244
      • Cahaba Dermatology & Skin Health Center
  • Arizona
    • Phoenix, Arizona, United States, 85008
      • Saguaro Dermatology
    • Scottsdale, Arizona, United States, 85260
      • Center for Dermatology and Plastic Surgery/CCT Research
    • Tucson, Arizona, United States, 85704
      • Noble Clinical Research
  • Arkansas
    • Fayetteville, Arkansas, United States, 72703
      • Clinical Trial Institute of Northwest Arkansas, LLC
    • Hot Springs, Arkansas, United States, 71913
      • Burke Pharmaceutical Research
  • California
    • Encinitas, California, United States, 92024
      • California Dermatology & Clinical Research Institute
    • Fountain Valley, California, United States, 92708
      • First OC Dermatology Research, Inc
    • Fremont, California, United States, 94538
      • CENTER FOR DERMATOLOGY CLINICAL RESEARCH, Inc
    • Huntington Beach, California, United States, 92647
      • Marvel Clinical Research
    • Northridge, California, United States, 91325
      • Northridge Clinical Trials
    • Palm Springs, California, United States, 92262
      • Palmtree Clinical Research, Inc.
    • Sacramento, California, United States, 95815
      • Integrative Skin Science and Research
  • Colorado
    • Castle Rock, Colorado, United States, 80109
      • Clarity Dermatology
    • Denver, Colorado, United States, 80210
      • Colorado Medical Research Center
  • Florida
    • Boca Raton, Florida, United States, 33486
      • Skin Care Research
    • Coral Gables, Florida, United States, 33134
      • Driven Research LLC
    • Hollywood, Florida, United States, 33021
      • Skin Care Research
    • Sanford, Florida, United States, 32771
      • International Clinical Research - FL LLC
    • West Palm Beach, Florida, United States, 33401
      • Metabolic Research Institute, Inc
  • Illinois
    • Chicago, Illinois, United States, 60614
      • MetroMed Clinical Trials
  • Indiana
    • Clarksville, Indiana, United States, 47129
      • DS Research of Southern Indiana
    • Indianapolis, Indiana, United States, 46250
      • Dawes Fretzin Clinical Research Group, LLC
  • Kentucky
    • Louisville, Kentucky, United States, 40241
      • DS Research of Kentucky
  • Louisiana
    • New Orleans, Louisiana, United States, 70115
      • DelRicht Research at Audubon Dermatology
  • Maryland
    • Rockville, Maryland, United States, 20850
      • Lawrence J. Green, MD LLC
  • Nevada
    • Las Vegas, Nevada, United States, 89148
      • JDR Dermatology Research
  • New York
    • New City, New York, United States, 10956
      • The Skin Center Dermatology Group
    • New York, New York, United States, 10012
      • Bobby Buka MD, PC
  • North Carolina
    • Hickory, North Carolina, United States, 28602
      • Hickory Dermatology Research Center
    • Winston-Salem, North Carolina, United States, 27103
      • The Skin Surgery Center for Clinical Research
  • Ohio
    • Canal Winchester, Ohio, United States, 80109
      • Clarity Dermatology
  • Oklahoma
    • Oklahoma City, Oklahoma, United States, 73170
      • Central Sooner Research
  • Pennsylvania
    • Plymouth Meeting, Pennsylvania, United States, 19462
      • Dermatology Associates of Plymouth Meeting
  • Tennessee
    • Hermitage, Tennessee, United States, 37076
      • Cumberland Skin Center for Clinical Research
  • Texas
    • Arlington, Texas, United States, 76011
      • Arlington Research Center
    • Dallas, Texas, United States, 75230
      • Dermatology Treatment and Research Center
    • El Paso, Texas, United States, 79902
      • Newco 3A Research
    • Houston, Texas, United States, 77004
      • Center for Clinical Studies, LTD.LLP
    • Pflugerville, Texas, United States, 78660
      • Austin Insitute for Clinical Research
    • Prosper, Texas, United States, 75078
      • DelRicht Research at Lockhart Matter Dermatology
  • Utah
    • South Jordan, Utah, United States, 84095
      • Jordan Valley Dermatology Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Has completed and signed an Informed Consent Form (ICF) prior to any study-related procedures.
2. Able to understand and comply with study requirements.
3. Male or female aged 18 to 75 years, inclusive.
4. Clinical diagnosis of non-segmental vitiligo where the total affected BSA does not exceed 10%.
5. F-VASI score of ≥0.5 and ≤3.0.
6. T-VASI score of ≥3.0 and ≤10.0.
7. Agree to discontinue all agents used to treat vitiligo from Screening through the study completion. Over-the-counter preparations deemed acceptable by the investigator are permitted.
8. If receiving concomitant medication for any reason other than vitiligo, must be on a stable regimen at Screening, and anticipating staying on a stable regimen through the study completion.

9. Female participants must:

   • Be of non-childbearing potential (i.e., surgically sterilized [hysterectomy, bilateral salpingectomy, bilateral oophorectomy, tubal ligation/occlusion at least 6 weeks before the Screening]) or postmenopausal (where postmenopausal is defined as no menses for 12 months without an alternative medical cause) OR

   • If of childbearing potential, must agree not to donate ova, not to attempt to become pregnant and, if engaging in sexual intercourse with a male partner, must agree to use an acceptable method of contraception from signing the ICF until at least 1 month after the last dose of IMP. An acceptable method of contraception includes one of the following:

     1. Hormonal contraception (for at least 3 months prior to Screening) in combination with a barrier method.
     2. Intrauterine device (placement at least 3 months prior to Screening).
     3. Diaphragm with spermicide.
     4. Cervical cap with spermicide.
     5. Condoms with spermicide.
     6. Vasectomized partner (6 months minimum since vasectomy).
10. Male participants, if not biologically or surgically sterilized, must agree not to donate sperm and, if engaging in sexual intercourse, must agree to use a condom from signing the ICF until at least 1 month after the last dose of study drug. If engaging with a female partner who could become pregnant, the female partner must additionally use an acceptable method of contraception for this same period.

Exclusion Criteria:

1. Clinical diagnosis of other forms of vitiligo (e.g., segmental) or other hypo- or de-pigmentation skin diseases (e.g., piebaldism, leukoderma, Vogt-Koyanagi-Harada disease, malignancy induced hypopigmentation, etc.).
2. Concomitant dermatologic conditions or other medical condition(s) which may, in the opinion of the investigator, interfere with IMP application or study assessments.
3. History of melanocyte transplantation procedure or depigmentation treatment [e.g. Monobenzyl ether of hydroquinone (Monobenzone)].
4. Visible test site skin injury, damage, or observations in or around the application site which, in the opinion of the investigator, will interfere with study assessments or increase participation risk.
5. Dyed hair in the treatment area that could interfere with any clinical assessments.
6. Significant facial hair or are unable to maintain very short cropped facial hair (<5mm) during course of the study.
7. Leukotrichia in >33% of vitiligo lesional surface of the face or the body.
8. History or presence of any clinically significant condition(s) which, in the opinion of the investigator, could interfere with the course of the study or expose the participant to undue risk by participating in this study, including, but not limited to: metabolic, allergic, cardiovascular, pulmonary, hepatic, renal, hematologic (including bleeding disorders), gastrointestinal (including peptic ulcer disease, gastritis or bleeding diathesis, excluding appendectomy or hernia repair), endocrine, immunologic, dermatologic, muscular, neurological, psychiatric, neoplastic, or other disease(s).
9. Major surgery within 3 months of randomization or with planned major surgery during trial.
10. Current or recent history (<30 days before Screening and/or <45 days before randomization) of a clinically meaningful bacterial, fungal, parasitic, or mycobacterial infection.
11. Any known or suspected premalignant or malignant disease within 5 years prior to Screening (excluding successfully treated basal or squamous cell carcinomas, actinic keratoses, melanoma in situ, cervical dysplasias, cervical cancer).
12. Systemic biologic or immune-modulating treatment within 12 weeks prior to Screening and through study completion or topical treatment in the vitiligo areas within 2 weeks prior to Screening and through study completion.
13. Received any investigational therapy, device or procedure within 30 days or 5 half-lives (whichever is longer) prior to Screening. Investigational biologics should be discussed with the sponsor to determine if a longer period of discontinuation is required.
14. Relevant (in the investigator opinion) ultraviolet light exposure including phototherapy within 8 weeks prior to Screening.
15. Use of any other prior and concomitant therapy not listed above that, in the opinion of the investigator, may interfere with the evaluation of study outcomes, including drugs that cause photosensitivity or skin pigmentation (e.g. antibiotics such as tetracyclines, antifungals). These medications should be discontinued within 4 weeks of randomization.
16. Known or suspected history of alcohol or drug abuse within 12 months of Screening or in the opinion of the investigator, will interfere with the subject's ability to comply with the administration schedule and study assessments (alcohol abuse is defined as regular consumption of >10 standard units of alcohol per week).
17. Subjects who are pregnant or breastfeeding.

18. Clinically significant abnormal laboratory values at Screening:

    1. Neutrophils < lower limit of normal.
    2. Hemoglobin < 10 g/dL.
    3. Platelets < 100 × 109/L.
    4. Alanine transaminase (ALT) or aspartate transaminase (AST) ≥ 2.0 × upper limit of normal.
    5. Estimated creatinine clearance < 30 mL/min or renal disease requiring dialysis as determined by Cockcroft-Gault.
    6. Positive serology tests for Hepatitis B surface antigen (HBsAg), hepatitis C virus antibody (anti-HCV), or human immunodeficiency virus (HIV)-1 and HIV-2 antibody.
19. Subjects who received a live vaccine within 4 weeks prior to Screening.
20. Subjects with known allergy or reaction to any component of the study formulation.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: Low dose VYN201 (1.0%); 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.	Drug: VYN201 Gel; VYN201 Gel is a topical formulation applied as a thin layer to affected areas.
Active Comparator: Mid dose VYN201 (2.0%); 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.	Drug: VYN201 Gel; VYN201 Gel is a topical formulation applied as a thin layer to affected areas.
Active Comparator: High dose VYN201 (3.0%); 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.	Drug: VYN201 Gel; VYN201 Gel is a topical formulation applied as a thin layer to affected areas.
Placebo Comparator: Vehicle; 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.	Drug: VYN201 Gel; VYN201 Gel is a topical formulation applied as a thin layer to affected areas.; Drug: Vehicle Gel; Vehicle gel (placebo) is matching in appearance to VYN201 gel and is to be applied in the same manner as VYN201 gel.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
To evaluate the efficacy of VYN201 compared to vehicle at Week 24 in subjects with NSV.	Proportion of subjects who achieve a ≥50% improvement from Baseline in F-VASI score at Week 24.	Week 24

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
To evaluate the efficacy of VYN201 as measured by F-VASI over time in subjects with NSV.	Mean and percent changes from Baseline in F-VASI at Week 24 and Week 52.	Week 24 and Week 52
To evaluate the efficacy of VYN201 as measured by T-VASI assessment over time in subjects with NSV.	Proportion of subjects who achieve a ≥50% improvement from baseline in T-VASI at Week 52	Week 52

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
To evaluate the efficacy of VYN201 as measured by Vitiligo Noticeability Scale over time in subjects with NSV.	Vitiligo Noticeability Scale (VNS) is the subjects' perception of their vitiligo. A rating of 'More noticeable' would indicate the subject perceived the treatment as ineffective and a rating of 'No longer noticeable' would indicate the subjects' perceived the treatment to be highly effective. The assessment is rated by the following measures: More noticeable; As noticeable; Slightly less noticeable; A lot less noticeable; No longer noticeable	Week 52
To evaluate the safety and tolerability of VYN201 as measured by Treatment-Emergent Adverse Events (TEAEs) over time in subjects with NSV.	Frequency and severity of TEAEs. TEAEs will be defined as events that emerge after the first application of IMP having been absent pre-treatment, or worsens relative to the pre-treatment state.	Baseline through Week 56
To evaluate the safety and tolerability of VYN201 as measured by Local Skin Tolerability assessment over time in subjects with NSV.	Changes from baseline in Local Skin Tolerability. Assessment of the dryness, scaling, erythema, hyperpigmentation, burning/stinging and pruritus of the affected area. Incidence of severity grades in skin tolerability are assessed with the following severity scoring: 0 = None; = Mild; = Moderate; = Severe	Baseline through Week 56
To determine the PK of VYN201 in subjects with NSV (Cmax)	Maximum observed plasma concentration of VYN201	Baseline through Week 52
To determine the PK of VYN201 in subjects with NSV (Tmax)	Time to reach Maximum observed plasma concentration of VYN201	Baseline through Week 52
To determine the PK of VYN201 in subjects with NSV (Dose Proportionality)	Dose response relationship with VYN201	Week 24 and Week 52
To determine the PK of VYN201 in subjects with NSV (Accumulation Ratio)	Ratio of VYN201 accumulation under steady state conditions	Week 24 and Week 52
To determine the PK of VYN201 in subjects with NSV (Time to Steady State Ratio)	Time to Steady State Ratio	Week 24 and Week 52
To evaluate the efficacy of VYN201 as measured by T-VASI assessment over time in subjects with NSV.	Mean and percent changes from Baseline in T-VASI at Week 24 and Week 52.	Week 24 and Week 52
To evaluate the efficacy of VYN201 as measured by T-VASI assessment over time in subjects with NSV.	Proportion of subjects who achieve a ≥50% improvement from baseline in T-VASI at Week 24.	Week 24
To evaluate the safety and tolerability of VYN201 as measured by Physical Examinations over time in subjects with NSV.	Changes from baseline in physical examination findings. General physical examinations will include: height (cm) and weight (kg), head and neck, eyes, ears, nose and throat, lymph nodes, and the cardiovascular, respiratory systems, abdominal, urologic, and musculoskeletal systems. Targeted physical examinations will be a symptom-directed evaluation and will include body weight and assessment(s) of the body systems or organs, as indicated by subject's symptoms, AEs, or other findings.	Baseline through Week 56
To evaluate the safety and tolerability of VYN201 as measured by ECGs over time in subjects with NSV.	Changes from baseline in ECG assessments. A standard 12-lead ECG will be used to obtain RR interval (ms), PR interval (ms), QRS interval (ms), QT interval (ms), QTcF (ms), and QTcB (ms).	Baseline through Week 56
To evaluate the efficacy of VYN201 as measured by vital signs over time in subjects with NSV.	Changes from baseline of vital signs. The following will be monitored as safety variables: Systolic and diastolic blood pressure (mmHg); Pulse rate (beats per minute); Respiration rate (breaths per minute); Arterial oxygen saturation (SpO2); Body temperature (°C)	Baseline through Week 56
To evaluate the efficacy of VYN201 as measured by Laboratory Tests over time in subjects with NSV.	Changes from baseline in Laboratory Safety assessments. Assessed laboratory values include the following: clinical chemistry/biochemistry, hematology, coagulation parameters, urinalysis, urine drug screen, and viral serology.	Baseline through Week 56

Collaborators and Investigators

• Sponsor: Vyne Therapeutics Inc.

Study record dates

Study Major Dates

• Study Start (Actual): June 4, 2024
• Primary Completion (Actual): July 10, 2025
• Study Completion (Actual): September 30, 2025

Study Registration Dates

• First Submitted: March 18, 2024
• First Submitted That Met QC Criteria: July 1, 2024
• First Posted (Actual): July 10, 2024

Study Record Updates

• Last Update Posted (Actual): April 21, 2026
• Last Update Submitted That Met QC Criteria: April 16, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Keywords: vitiligo; NSV; VYN201 Gel
• Additional Relevant MeSH Terms: Skin Diseases; Hypopigmentation; Pigmentation Disorders; Skin and Connective Tissue Diseases; Vitiligo
• Other Study ID Numbers: VY2024-02

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No