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

Vyne Therapeutics

Status and phase: Enrolling; Phase 2

Conditions: Non-segmental Vitiligo

Treatments: Drug: Vehicle Gel; Drug: VYN201 Gel

Study type: Interventional

Funder types: Industry

Identifiers

NCT06493578

VY2024-02

Details and patient eligibility

About

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

Enrollment: 160 estimated patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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160 participants in 4 patient groups, including a placebo group

Low dose VYN201 (1.0%)

Active Comparator group

Description:

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.

Treatment:

Drug: VYN201 Gel

Mid dose VYN201 (2.0%)

Active Comparator group

Description:

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.

Treatment:

Drug: VYN201 Gel

High dose VYN201 (3.0%)

Active Comparator group

Description:

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.

Treatment:

Drug: VYN201 Gel

Vehicle

Placebo Comparator group

Description:

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.

Treatment:

Drug: VYN201 Gel

Drug: Vehicle Gel