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

Phase 2

Recruiting

• Conditions: Non-segmental Vitiligo
• Interventions: Drug: VYN201 Gel; Drug: Vehicle Gel

• Registration Number: NCT06493578
• Lead Sponsor: Vyne Therapeutics Inc.

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2024-03-18
• Study Start: 2024-06-04
• Study First Submitted QC: 2024-07-01
• Study First Posted: 2024-07-10
• Status Verified: 2024-08
• Last Update Submitted: 2024-08-06
• Last Update Posted: 2024-08-09
• Primary Completion: 2025-06-30
• Study Completion: 2026-01-30

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 160

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.

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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.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Mid dose VYN201 (2.0%)	VYN201 Gel	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.
High dose VYN201 (3.0%)	VYN201 Gel	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.
Low dose VYN201 (1.0%)	VYN201 Gel	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.
Vehicle	VYN201 Gel	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.
Vehicle	Vehicle Gel	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.

Primary Outcome Measures

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

Secondary Outcome Measures

Name	Time	Method
To evaluate the efficacy of VYN201 as measured by T-VASI assessment over time in subjects with NSV.	Week 52	Proportion of subjects who achieve a ≥50% improvement and mean and percent changes from baseline in T-VASI at Week 52.
To evaluate the efficacy of VYN201 as measured by F-VASI over time in subjects with NSV.	Week 24 and Week 52	Mean and percent changes from Baseline in F-VASI at Week 24 and Week 52.
To determine the PK of VYN201 in subjects with NSV (Dose Proportionality)	Week 24 and Week 52	Dose response relationship with VYN201
To evaluate the efficacy of VYN201 as measured by Vitiligo Noticeability Scale over time in subjects with NSV.	Week 52	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: 1. More noticeable; 2. As noticeable; 3. Slightly less noticeable; 4. A lot less noticeable; 5. No longer noticeable
To evaluate the safety and tolerability of VYN201 as measured by Treatment-Emergent Adverse Events (TEAEs) over time in subjects with NSV.	Baseline through Week 56	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.
To evaluate the safety and tolerability of VYN201 as measured by Physical Examinations over time in subjects with NSV.	Screening through Week 56	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.
To evaluate the safety and tolerability of VYN201 as measured by Local Skin Tolerability assessment over time in subjects with NSV.	Baseline through Week 56	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; 1. = Mild; 2. = Moderate; 3. = Severe
To evaluate the safety and tolerability of VYN201 as measured by ECGs over time in subjects with NSV.	Screening through Week 56	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).
To evaluate the efficacy of VYN201 as measured by vital signs over time in subjects with NSV.	Screening through Week 56	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)
To evaluate the efficacy of VYN201 as measured by Laboratory Tests over time in subjects with NSV.	Screening through Week 56	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.
To determine the PK of VYN201 in subjects with NSV (Cmax)	Baseline through Week 52	Maximum observed plasma concentration of VYN201
To determine the PK of VYN201 in subjects with NSV (Tmax)	Baseline through Week 52	Time to reach Maximum observed plasma concentration of VYN201
To determine the PK of VYN201 in subjects with NSV (Time to Steady State Ratio)	Week 24 and Week 52	Time to Steady State Ratio
To determine the PK of VYN201 in subjects with NSV (Accumulation Ratio)	Week 24 and Week 52	Ratio of VYN201 accumulation under steady state conditions

Trial Locations

Locations (47)

Saguaro Dermatology; 🇺🇸 Phoenix, Arizona, United States

Clinical Trial Institute of Northwest Arkansas, LLC; 🇺🇸 Fayetteville, Arkansas, United States

Palmtree Clinical Research, Inc.; 🇺🇸 Palm Springs, California, United States

MetroMed Clinical Trials; 🇺🇸 Chicago, Illinois, United States

Dawes Fretzin Clinical Research Group, LLC; 🇺🇸 Indianapolis, Indiana, United States

DelRicht Research at Audubon Dermatology; 🇺🇸 New Orleans, Louisiana, United States

DOCS Dermatology Research - Canal Winchester; 🇺🇸 Canal Winchester, Ohio, United States

Clarity Dermatology; 🇺🇸 Castle Rock, Ohio, United States

Newco 3A Research; 🇺🇸 El Paso, Texas, United States

Center for Clinical Studies, LTD.LLP; 🇺🇸 Houston, Texas, United States

DelRicht Research at Lockhart Matter Dermatology; 🇺🇸 Prosper, Texas, United States

Dermatology Research Institute; 🇨🇦 Calgary, Alberta, Canada

JRB Research Inc; 🇨🇦 Ottawa, Ontario, Canada

SKiN Centre for Dermatology; 🇨🇦 Peterborough, Ontario, Canada

Research Toronto; 🇨🇦 Toronto, Ontario, Canada

Cahaba Dermatology & Skin Health Center; 🇺🇸 Birmingham, Alabama, United States

Center for Dermatology and Plastic Surgery/CCT Research; 🇺🇸 Scottsdale, Arizona, United States

Noble Clinical Research; 🇺🇸 Tucson, Arizona, United States

Burke Pharmaceutical Research; 🇺🇸 Hot Springs, Arkansas, United States

California Dermatology & Clinical Research Institute; 🇺🇸 Encinitas, California, United States

First OC Dermatology Research, Inc; 🇺🇸 Fountain Valley, California, United States

Center for Dermatology Clinical Research, Inc; 🇺🇸 Fremont, California, United States

Marvel Clinical Research; 🇺🇸 Huntington Beach, California, United States

Northridge Clinical Trials; 🇺🇸 Northridge, California, United States

Integrative Skin Science and Research; 🇺🇸 Sacramento, California, United States

Colorado Medical Research Center; 🇺🇸 Denver, Colorado, United States

Skin Care Research; 🇺🇸 Hollywood, Florida, United States

Driven Research LLC; 🇺🇸 Coral Gables, Florida, United States

International Clinical Research - FL LLC; 🇺🇸 Sanford, Florida, United States

Metabolic Research Institute, Inc; 🇺🇸 West Palm Beach, Florida, United States

DS Research of Southern Indiana; 🇺🇸 Clarksville, Indiana, United States

DS Research of Kentucky; 🇺🇸 Louisville, Kentucky, United States

Lawrence J. Green, MD LLC; 🇺🇸 Rockville, Maryland, United States

JDR Dermatology Research; 🇺🇸 Las Vegas, Nevada, United States

The Skin Center Dermatology Group; 🇺🇸 New City, New York, United States

Bobby Buka MD, PC; 🇺🇸 New York, New York, United States

Hickory Dermatology Research Center; 🇺🇸 Hickory, North Carolina, United States

The Skin Surgery Center for Clinical Research; 🇺🇸 Winston-Salem, North Carolina, United States

Central Sooner Research; 🇺🇸 Oklahoma City, Oklahoma, United States

Dermatology Associates of Plymouth Meeting; 🇺🇸 Plymouth Meeting, Pennsylvania, United States

Cumberland Skin Center for Clinical Research; 🇺🇸 Hermitage, Tennessee, United States

Dermatology Treatment and Research Center; 🇺🇸 Dallas, Texas, United States

Austin Insitute for Clinical Research; 🇺🇸 Pflugerville, Texas, United States

Jordan Valley Dermatology Center; 🇺🇸 South Jordan, Utah, United States

SimcoDerm Medical and Surgical Dermatology Center; 🇨🇦 Barrie, Ontario, Canada

Lynderm Research Inc.; 🇨🇦 Markham, Ontario, Canada

Siena Medical Research; 🇨🇦 Montréal, Quebec, Canada