A Phase III Clinical Study to Evaluate the Efficacy and Safety of MH004 Ointment in Non-segmental Vitiligo

Phase 3RecruitingNCT07682506

Minghui Pharmaceutical (Hangzhou) Ltd405 enrolled

Overview

This is a randomized, double-blind, multicenter, placebo-controlled clinical study intended to evaluate the efficacy, safety and population pharmacokinetic profiles of MH004 ointment in eligible participants with NSV.

The study consists of a screening period, a treatment period and a follow-up period, with the treatment period divided into two phases. In Double-blind Treatment Phase (24 weeks, D1 to W24), participants will be randomized at a 2:1 ratio to receive either MH004 ointment or placebo. And in Extended Treatment Phase (28 weeks, W25 to W52), after all participants complete all assessments prior to dosing at W24, they will enter the extended treatment phase and receive 1.0% MH004 ointment with the same dosing regimen as that in the double-blind treatment phase, administered twice daily (BID) until W52. Upon completion of study treatment, all participants will enter a 4-week safety follow-up period. The primary objective of this trial is the proportion of participants achieving at least a 75% improvement from baseline in the Facial Vitiligo Area Scoring Index at Week 24 (F-VASI75).

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

TRIPLE

Enrollment

405

Conditions

Vitiligo

Interventions

MH004 1.0% OintmentDRUG

MH004 1% ointment applied topically to the affected area as a thin film twice a day (BID).

MH004 Placebo OintmentDRUG

MH004 Placebo ointment applied topically to the affected area as a thin film twice a day (BID).

Eligibility

Sex: ALLMin age: 12 YearsMax age: 75 Years

Medical Language ↔ Plain English

Inclusion Criteria: 1. Subjects aged 12 to 75 years inclusive at the time of signing the Informed Consent Form (ICF), with no restriction on gender. 2. Clinically diagnosed with non-segmental vitiligo. 3. Prior to study drug administration, participants with vitiligo shall meet the following criteria regarding vitiligo lesion area: Facial lesion area ≥ 0.3% body surface area (BSA), and Facial Vitiligo Area Scoring Index (F-VASI) score ≥ 0.3. 4. Agree to discontinue all vitiligo therapeutic medications and interventions from the time of ICF signature until the end of the last study visit. Over-the-counter (OTC) drugs shall not exert therapeutic effects on vitiligo or interfere with skin pigmentation, including corticosteroids and other immunomodulators. Final eligibility of such OTC drugs shall be determined by the Investigator. Camouflaging makeup is permitted. 5. Women of Childbearing Potential (WOCBP) and male participants whose partners are WOCBP must agree to use reliable contraceptive methods throughout the trial period and for 28 days after the last study drug administration (abstinence, prior sterilization, oral contraceptives, and/or barrier methods \[condoms, diaphragms, cervical caps, etc.\]). For WOCBP, the human chorionic gonadotropin (hCG) pregnancy test result at the screening visit and prior to the first drug administration at the baseline visit must be negative. Male participants shall not donate sperm during the trial and for 3 months after study completion or drug discontinuation. 6. The participant and/or their legal guardian shall fully understand the trial content, requirements and procedures, voluntarily participate in this clinical trial and sign the ICF, and be willing and able to comply with scheduled visits, treatment regimens, laboratory tests and other study procedures throughout the trial. Exclusion Criteria: 1. All terminal hairs (i.e., beard, eyebrows, eyelashes) within facial vitiligo areas are depigmented white. 2. Other subtypes of vitiligo or hypopigmentary disorders 1. Segmental vitiligo, unclassified vitiligo, or mixed vitiligo; 2. Other differential diagnoses of vitiligo or other cutaneous hypopigmentary diseases (e.g., piebaldism, pityriasis alba, nevus anemicus, post-inflammatory hypopigmentation, chemical leukoderma, tinea versicolor, etc.). 3. Specific prior treatment history: 1. Prior use of any JAK inhibitor (systemic or topical) for vitiligo treatment at any time; 2. Prior depigmentation therapy for vitiligo (e.g., monobenzone), excluding hydroquinone; 3. Prior melanocyte-keratinocyte transplantation or other surgical procedures for vitiligo on the face. 4. Treatments administered within the required washout period 1. Use of biologic agents within 12 weeks or 5 half-lives (whichever is longer) prior to the first study drug administration; 2. Receipt of laser therapy or any form of phototherapy (including tanning beds) within 8 weeks prior to the first study drug administration; 3. Within 4 weeks prior to the first study drug administration: Systemic immunomodulators (e.g., corticosteroids, methotrexate, cyclosporine, etc.); Systemic medications that may affect vitiligo (e.g., melanocyte-stimulating agents, tetracyclines, methoxsalen, etc.); Administration of live or live-attenuated vaccines; 4. Oral traditional Chinese medicines for vitiligo within 2 weeks prior to the first study drug administration; 5. Topical agents applied to vitiligo lesions within 1 week prior to the first study drug administration (e.g., corticosteroids, calcineurin inhibitors, PDE4 inhibitors, retinoids, vitamin D3 analogues, or topical Chinese herbal preparations); 5. Temporary tattoos within vitiligo lesions within 30 days before the first dose (excluding vinyl adhesive tattoos), or any permanent tattoos previously placed within vitiligo lesions. 6. Concomitant diseases or medical history that may interfere with the study 7. Specific risks of cardiovascular and thromboembolic events 8. Active or latent infections 9. Positive virology screening results at screening 10. History of malignancy 11. Clinically significant abnormal laboratory values 12. Subjects planning major invasive procedures during the study, or with prior or planned organ transplantation requiring long-term immunosuppressants (e.g., kidney or liver transplantation). 13. Planned administration of live or live-attenuated vaccines during the study period. 14. Female subjects who are pregnant or breastfeeding. 15. Participation in another interventional drug clinical trial within 3 months or at least 5 half-lives (whichever is longer) prior to randomization; or participation in a medical device clinical trial within 3 months prior to randomization. 16. Hypersensitivity and intolerance Known hypersensitivity to the investigational product or any excipient components. 17. Other exclusion factors 1. History of alcohol abuse or substance misuse; 2. Subjects shall avoid intentional excessive sun exposure during the study (e.g., prolonged sunbathing, sun exposure for tanning purposes); 3. Body mass index (BMI) \< 16 kg/m² or \> 40 kg/m², where BMI = weight (kg) / height² (m²); 4. Any other conditions deemed inappropriate for study participation by the Investigator.

Locations (1)

Peking University People's Hospital

Beijing, Beijing Municipality, China

RECRUITING

Outcomes

Primary Outcomes

Proportion of participants achieving at least a 75% improvement from baseline in Facial Vitiligo Area Scoring Index (F-VASI75) at Week 24 (W24)

An F-VASI75 responder achieved at least 75% improvement from Baseline in F-VASI, measured by the percentage of vitiligo involvement (percentage of body surface area \[BSA\]) and the degree of depigmentation: 0% (no depigmentation), 10% (only specks of depigmentation), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment), or 100% (no pigment).

Time frame: Baseline; Week 24

Secondary Outcomes

Proportion of participants achieving at least a 50% improvement from baseline in Facial Vitiligo Area Scoring Index (F-VASI50) at Week 24 (W24)

An F-VASI50 responder achieved at least 50% improvement from Baseline in F-VASI, measured by the percentage of vitiligo involvement (percentage of body surface area \[BSA\]) and the degree of depigmentation: 0% (no depigmentation), 10% (only specks of depigmentation), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment), or 100% (no pigment). The percentage of BSA (hand unit) vitiligo involvement was estimated to the nearest 0.1% by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant's hand size to evaluate the percentage of BSA vitiligo involvement. F-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site on the face and summing the values of all sites (possible range: 0-3; lower scores indicate increased improvement).

Time frame: Baseline; Week 24

Proportion of participants achieving at least a 90% improvement from baseline in Facial Vitiligo Area Scoring Index (F-VASI90) at Week 24 (W24)

An F-VASI90 responder achieved at least 90% improvement from Baseline in F-VASI, measured by the percentage of vitiligo involvement (percentage of body surface area \[BSA\]) and the degree of depigmentation: 0% (no depigmentation), 10% (only specks of depigmentation), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment), or 100% (no pigment).

Central Contacts

CMO/ Senior Vice President of R&D

CONTACT

+ 86 0571-86963293 jwshi@minghuipharma.com

Data from ClinicalTrials.gov

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Time frame: Baseline; Week 24

Proportion of participants achieving at least a 50% improvement from baseline in Total Vitiligo Area Scoring Index (T-VASI50) at Week 24 (W24)

A T-VASI50 responder achieved at least 50% improvement from Baseline in T-VASI, calculated with contributions from 6 sites. The percentage of vitiligo involvement was estimated in hand units (percentage of BSA estimated to the nearest 0.1%) by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant's hand size to evaluate percent BSA vitiligo involvement. The degree of depigmentation for each site was estimated to the nearest percentage: 0% (no depigmentation present), 10% (only specks of depigmentation present), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment present), 100% (no pigment present). T-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site and summing the values (range: 0-100; lower scores indicate increased improvement).

Time frame: Baseline; Week 24

Proportion of participants achieving at least a 75% improvement from baseline in Total Vitiligo Area Scoring Index (T-VASI75) at Week 24 (W24)

A T-VASI75 responder achieved at least 75% improvement from Baseline in T-VASI, calculated with contributions from 6 sites. The percentage of vitiligo involvement was estimated in hand units (percentage of BSA estimated to the nearest 0.1%) by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant's hand size to evaluate percent BSA vitiligo involvement. The degree of depigmentation for each site was estimated to the nearest percentage: 0% (no depigmentation present), 10% (only specks of depigmentation present), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment present), 100% (no pigment present).

Time frame: Baseline; Week 24

Proportion of participants achieving at least a 90% improvement from baseline in Total Vitiligo Area Scoring Index (T-VASI90) at Week 24 (W24)

A T-VASI90 responder achieved at least 90% improvement from Baseline in T-VASI, calculated with contributions from 6 sites. The percentage of vitiligo involvement was estimated in hand units (percentage of BSA estimated to the nearest 0.1%) by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant's hand size to evaluate percent BSA vitiligo involvement. The degree of depigmentation for each site was estimated to the nearest percentage: 0% (no depigmentation present), 10% (only specks of depigmentation present), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment present), 100% (no pigment present).

Time frame: Baseline; Week 24

Proportion of participants achieving F-VASI75 at Week 52 (W52)

Time frame: Baseline; Week 52

Proportion of participants achieving T-VASI75 at Week 52 (W52)

Time frame: Basline; Week 52

Percentage change from baseline in F-VASI score at Week 24

F-VASI was measured by the percentage of vitiligo involvement (percentage of BSA) and the degree of depigmentation: 0% (no depigmentation), 10% (only specks of depigmentation), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment), or 100% (no pigment). The percentage of BSA (hand unit) vitiligo involvement was estimated to the nearest 0.1% by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant's hand size to evaluate the percentage of BSA vitiligo involvement. F-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site on the face and summing the values of all sites (possible range: 0-3; lower scores indicate increased improvement). Percentage change = (\[post-BL value minus BL value\]/BL value) X 100.

Time frame: Baseline; Week 24

Percentage change from baseline in F-VASI score at Week 52

Time frame: Baseline; Week 52

Percentage change from baseline in T-VASI score at Week 24

T-VASI was calculated with contributions from 6 sites. The percentage of vitiligo involvement was estimated in hand units (percentage of BSA estimated to nearest 0.1%) by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant's hand size to evaluate percent BSA vitiligo involvement. The degree of depigmentation for each site was estimated to the nearest percentage: 0% (no depigmentation present), 10% (only specks of depigmentation present), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment present), 100% (no pigment present). T-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site and summing the values (range: 0-100; lower scores indicate increased improvement). Percentage change = (\[post-BL value minus BL value\]/BL value) X 100.

Time frame: Baseline; Week 24

Percentage change from baseline in T-VASI score at Week 52

Time frame: Baseline; Week 52

Percentage change from baseline in facial body surface area (F-BSA) score at Week 24 (W24)

F-BSA involvement was the proportion of the facial body surface area with vitiligo. The area "Face" was defined as including the area on the forehead to the original hairline, on the cheek to the jawline vertically to the jawline and laterally from the corner of the mouth to the tragus. The area "Face" did not include surface area of the lips, scalp, ears, or neck, but included the nose and eyelids. Body surface area assessment was performed by the Palmar Method. Body surface area was estimated to the nearest 0.1%. The approximate size of the participant's entire palmar surface (i.e., the palm plus 5 digits) was considered as 1% BSA, and the approximate size of the participant's thumb was considered as 0.1% BSA. Percentage change = (\[post-Baseline (BL) value minus BL value\]/BL value) X 100.

Time frame: Baseline; Week 24

Percentage change from baseline in total body surface area (T-BSA) score at Week 24 (W24)

T-BSA involvement was the proportion of the body surface area with vitiligo. Body surface area assessment was performed by the Palmar Method. Body surface area was estimated to the nearest 0.1%. The approximate size of the participant's entire palmar surface (i.e., the palm plus 5 digits) was considered as 1% BSA, and the approximate size of the participant's thumb was considered as 0.1% BSA. Percentage change = (\[post-BL value minus BL value\]/BL value) X 100.

Time frame: Baseline; Week 24

Proportion of participants achieving F-VASI50 at Week 52 (W52)

Time frame: Baseline; Week 52

Proportion of participants achieving F-VASI90 at Week 52 (W52)

Time frame: Baseline; Week 52

Proportion of participants achieving T-VASI50 at Week 52 (W52)

Time frame: Basline; Week 52

Proportion of participants achieving T-VASI90 at Week 52 (W52)

Time frame: Basline; Week 52

Percentage change from baseline in facial body surface area (F-BSA) score at Week 52 (W52)

Time frame: Baseline; Week 52

Percentage change from baseline in total body surface area (T-BSA) score at Week 52 (W52)

Time frame: Baseline; Week 52

Incidence, Frequency, Duration and Severity of Treatment-Emergent Adverse Event (TEAE)

An AE is any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study treatment. A SAE is defined as any untoward medical occurrence that, at any dose results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect or an important medical event may be considered serious when, based on appropriate medical judgment, the event may jeopardize the participant and may require medical or surgical intervention to prevent one of the outcomes listed above. A TEAE or treatment emergent SAE is any AE or SAE either reported for first time or worsening of a pre-existing event after first dose of study drug.

Time frame: From the time of Informed Consent Form signing until at least 30 days after the last application of study drug (up to Week 56)

Incidence of Treatment-Emergent Serious Adverse Event (SAE) and Incidence of AEs resulting discontinue medication

Time frame: From the time of Informed Consent Form signing until at least 30 days after the last application of study drug (up to Week 56)