Phase II/III Study of FXS5626 in Participants With Non-segmental Vitiligo
This is a Multicenter, Randomized, Double-blind, Placebo-controlled, Adaptive Seamless Design, Phase II/III Study Evaluating the Efficacy and Safety of FXS5626 in Participants With Non-segmental Vitiligo
This is a multicenter, randomized, double-blind, placebo-controlled, adaptive seamless design, Phase II/III study evaluating the efficacy and safety of FXS5626 in participants with non-segmental vitiligo.
研究概览
地位
条件
详细说明
This is a multicenter, randomized, double-blind, placebo-controlled, adaptive seamless design, Phase II/III study evaluating the efficacy and safety of FXS5626 in participants with non-segmental vitiligo. The study consisted 2 periods: a phase 2 period and a phase 3 period.
Phase 2 design: This period is planned to enroll 120 patients with non-segmental vitiligo. Eligible participants will be randomized in a 2:2:1:1 ratio to the FXS5626 75 mg twice daily (BID) group, the FXS5626 50 mg BID group, the FXS5626 25 mg BID group, or the placebo group, with 40, 40, 20, and 20 participants enrolled in each group, respectively. The overall study duration consists of a 4-week screening period, a 24-week double-blind treatment period, and a 4-week safety follow-up period.
Phase 3 design: This period is planned to enroll 360 patients with non-segmental vitiligo. Eligible participants will be randomized in a 2:1 ratio to the FXS5626 group and the placebo group. The overall study duration consists of a 4-week screening period, a 52-week double-blind treatment period, and a 4-week safety follow-up period.
研究类型
注册 (估计的)
阶段
- 阶段2
- 第三阶段
联系人和位置
学习联系方式
- 姓名:Wenyu Wu
- 电话号码:+86 021-52887783
- 邮箱:wwylcsy@163.com
研究联系人备份
- 姓名:Jian Li
- 电话号码:+86 021-52887783
- 邮箱:lil3579_nn@qq.com
学习地点
-
-
Shanghai Municipality
-
Shanghai、Shanghai Municipality、中国、200040
- Huashan Hospital, Fudan University
-
接触:
- Guoying Cao
- 电话号码:+86 021-52887892
- 邮箱:Sherley_76@163.com
-
接触:
- Yuran Cao
- 电话号码:+86 021-52888041
- 邮箱:yurancao@126.com
-
-
参与标准
资格标准
适合学习的年龄
接受健康志愿者
有资格学习的性别
描述
Participants must meet all of the following inclusion criteria:
- Understand the trial procedures and methods, voluntarily participate in this trial, be able to communicate well with the investigator, comply with the requirements of the entire study, and provide written informed consent;
- Screening age must meet the following criteria:
1) Age ≥18 years and ≤75 years, regardless sex (Phase 2); 2) Age ≥12 years and ≤75 years, regardless of sex (Phase 3); 3. At screening,participants must have a body mass index (BMI) ≥18 kg/m²; for adolescent participants in the Phase 3, baseline body weight must be ≥40 kg; 4. Clinically diagnosed with non-segmental vitiligo, either active or stable, with a disease history ≥3 months; 5. At the screening and baseline visits, vitiligo lesions must satisfy all of the following criteria:
- The percentage of vitiligo lesion area relative to the total body surface area (BSA) is ≥3%, excluding involvement of the palms, dorsa of the hands (fingers), soles, and dorsa of the feet;
- Facial involvement BSA (F-BSA) ≥0.5%;
- T-VASI ≥0.5 and 3 ≤ T-VASI ≤50; 6. Participants must ensure and agree that, from 28 days before signing the informed consent form to 28 days after the last dose, female participants of childbearing potential, male participants, and their spouses will use effective contraceptive methods other than oral medications (such as condoms or intrauterine devices), and will not donate sperm or ova during this study.
Exclusion Criteria
- Presence of vitiligo-related medical conditions and other skin diseases at screening, as assessed by the investigator;
- Presence of >33% leukotrichia in areas of vitiligo on the face or > 33% leukotrichia in areas of vitiligo on the body (including the face);
- As assessed by the investigator, accompanied by active infection or history of infection;
- Patients with a history of severe herpes simplex and herpes zoster infections, or with active/current herpes simplex or herpes zoster infection;
- Participants with any other clinically significant medical history, or other conditions that, as assessed by the investigator, may affect the participant's safety or compliance, or result in the participant's inability to complete the study.
学习计划
研究是如何设计的?
设计细节
- 主要用途:治疗
- 分配:随机化
- 介入模型:并行分配
- 屏蔽:四人间
武器和干预
参与者组/臂 |
干预/治疗 |
|---|---|
|
实验性的:Arm1: FXS5626 25 mg (Phase 2)
FXS5626 25 mg |
FXS5626 tablets administered orally twice daily (BID) for 24 weeks. |
|
实验性的:Arm2: FXS5626 50 mg (Phase 2)
FXS5626 50 mg |
FXS5626 tablets administered orally twice daily (BID) for 24 weeks. |
|
实验性的:Arm3: FXS5626 75 mg (Phase 2)
FXS5626 75 mg |
FXS5626 tablets administered orally twice daily (BID) for 24 weeks. |
|
实验性的:Arm4: FXS5626 group (Phase 3)
FXS5626 group (Dosage will be confirmed after Phase 2) |
FXS5626 tablets administered orally twice daily (BID) for 52 weeks. |
|
安慰剂比较:Arm5: Placebo (Phase 2)
Placebo |
FXS5626 placebo tablets administered orally twice daily (BID) for 24 weeks. |
|
安慰剂比较:Arm6: Placebo (Phase 3)
Placebo |
FXS5626 placebo tablets administered orally twice daily (BID) for 52 weeks. |
研究衡量的是什么?
主要结果指标
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Percent change from baseline in facial vitiligo area scoring index (F-VASI) at week 24 in Phase 2 study.
大体时间:24 weeks
|
The vitiligo area scoring index (VASI) is a validated scoring method used to assess the areas of depigmentation due to vitiligo. The F-VASI includes contributions from the face, with a possible range from 0 to 3. Higher scores of F-VASI mean a worse outcome. |
24 weeks
|
|
Proportion of participants achieving Facial Vitiligo Area Scoring Index 75 (F-VASI 75, at least 75% improvement from baseline) at Week 52 in Phase 3 study.
大体时间:52 weeks
|
The vitiligo area scoring index (VASI) is a validated scoring method used to assess the areas of depigmentation due to vitiligo. The F-VASI includes contributions from the face, with a possible range from 0 to 3. Higher scores of F-VASI mean a worse outcome. F-VASI 75 denotes a at least 75% improvement from baseline in the Facial Vitiligo Area Scoring Index (F-VASI). |
52 weeks
|
次要结果测量
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Percent change from baseline in Facial Vitiligo Area Scoring Index (F-VASI) at planned visits other than Week 24(Phase 2)
大体时间:24 weeks
|
The vitiligo area scoring index (VASI) is a validated scoring method used to assess the areas of depigmentation due to vitiligo. The F-VASI includes contributions from the face, with a possible range from 0 to 3. Higher scores of F-VASI mean a worse outcome. |
24 weeks
|
|
Proportion of participants achieving Facial Vitiligo Area Scoring Index 75 (F-VASI 75, at least 75% improvement from baseline) at planned visits (Phase 2)
大体时间:24 weeks
|
At least 75% improvement from baseline in facial Vitiligo Area Scoring Index. The F-VASI includes contributions from the face, with a possible range from 0 to 3. Higher scores of F-VASI mean a worse outcome. |
24 weeks
|
|
Proportion of participants achieving Facial Vitiligo Area Scoring Index 50 (F-VASI 50, at least 50% improvement from baseline) at planned visits (Phase 2)
大体时间:24 weeks
|
At least 50% improvement from baseline in facial Vitiligo Area Scoring Index. The F-VASI includes contributions from the face, with a possible range from 0 to 3. Higher scores of F-VASI mean a worse outcome. |
24 weeks
|
|
Proportion of participants achieving Facial Vitiligo Area Scoring Index 90 (F-VASI 90, at least 90% improvement from baseline) at planned visits (Phase 2)
大体时间:24 weeks
|
At least 90% improvement from baseline in facial Vitiligo Area Scoring Index. The F-VASI includes contributions from the face, with a possible range from 0 to 3. Higher scores of F-VASI mean a worse outcome. |
24 weeks
|
|
Percent change from baseline in Total Vitiligo Area Scoring Index (T-VASI) at planned visits (Phase 2)
大体时间:24 weeks
|
The vitiligo area scoring index (VASI) is a validated scoring method used to assess the areas of depigmentation due to vitiligo. It is based on a composite estimate of the overall area of vitiligo patches, measured by the number of hand units (palm plus 5 digits = 1% body surface area [BSA]) multiplied by the degree of depigmentation within each affected area (0%, 10%, 25%, 50%, 75%, 90%, or 100%). The T-VASI is calculated using a formula that includes contributions from all body regions, with a possible range from 0 to 100. Higher scores of T-VASI mean a worse outcome. |
24 weeks
|
|
Proportion of participants achieving Total Vitiligo Area Scoring Index 50 (T-VASI 50, at least 50% improvement from baseline) at planned visits (Phase 2)
大体时间:24 weeks
|
At least 50% improvement from baseline in Total Vitiligo Area Scoring Index. The T-VASI is calculated using a formula that includes contributions from all body regions, with a possible range from 0 to 100. Higher scores of T-VASI mean a worse outcome. |
24 weeks
|
|
Proportion of participants achieving Total Vitiligo Area Scoring Index 75 (T-VASI 75, at least 75% improvement from baseline) at planned visits (Phase 2)
大体时间:24 weeks
|
At least 75% improvement from baseline in Total Vitiligo Area Scoring Index. The T-VASI is calculated using a formula that includes contributions from all body regions, with a possible range from 0 to 100. Higher scores of T-VASI mean a worse outcome. |
24 weeks
|
|
Change from baseline in the Facial Body Surface Area (F-BSA) affected by vitiligo at planned visits (Phase 2)
大体时间:24 weeks
|
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. |
24 weeks
|
|
Change from baseline in the Total Body Surface Area (T-BSA) affected by vitiligo at planned visits (Phase 2)
大体时间:24 weeks
|
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. |
24 weeks
|
|
Proportion of participants achieving a score of 0 or 1 on the Facial Physician's Global Vitiligo Assessment (F-PhGVA) at planned visits (Phase 2)
大体时间:24 weeks
|
The F-PhGVA is an assessment tool used by physicians to evaluate the severity of facial vitiligo. It is rated on a 4-point scale: overall improvement: (0) no change, (1)limited extent, (2)moderate extent, (3)extensive, (4)very extensive. Higher score corresponds to worse evaluation. |
24 weeks
|
|
Proportion of participants achieving a score of 0 or 1 on the Total Physician's Global Vitiligo Assessment (T-PhGVA) at planned visits (Phase 2)
大体时间:24 weeks
|
The T-PhGVA is a assessment tool used by physicians to evaluate the severity of total body vitiligo. It is rated on a 4-point scale: overall improvement: (0) no change, (1)limited extent, (2)moderate extent, (3)extensive, (4)very extensive. Higher score corresponds to worse evaluation. |
24 weeks
|
|
Proportion of participants achieving a score of 1 or 2 on the Facial Patient's Global Impression of Change-Vitiligo (F-PaGIC-V) at planned visits (Phase 2)
大体时间:24 weeks
|
F-PaGIC-V is an assessment of improvement of vitiligo on the face by the participant. It is a 7-point scale ((1) Very much improved, (2) Much improved, (3) Minimally improved, (4) No change, (5) Minimally worse, (6) Much worse, or (7) Very much worse). |
24 weeks
|
|
Proportion of participants achieving a score of 1 or 2 on the Total Body Patient's Global Impression of Change-Vitiligo (T-PaGIC-V) at planned visits (Phase 2)
大体时间:24 weeks
|
T-PaGIC-V is an assessment of improvement of vitiligo on the total body by the participant. It is a 7-point scale ((1) Very much improved, (2) Much improved, (3) Minimally improved, (4) No change, (5) Minimally worse, (6) Much worse, or (7) Very much worse). |
24 weeks
|
|
Change from baseline in the Vitiligo-Specific Quality of Life (VitiQoL) at planned visits (Phase 2)
大体时间:24 weeks
|
VitiQoL is a 15-item QoL assessment that asks participants to rate various aspects of their condition during the past month using a 7-point scale ("Not at all" to "All of the time") |
24 weeks
|
|
Number of Participants with Adverse Events (AEs) (Phase 2)
大体时间:24 weeks and 4 weeks
|
An AE is defined as any untoward medical occurrence in a patient or clinical investigation in which a participant is administered a pharmaceutical product which does not necessarily have a causal relationship with this treatment. AE severity was assessed as mild, moderate and severe. Mild: Transient discomfort, no interference with daily activities, and/or no medical intervention required. Moderate: Mild to moderate limitation of daily activities, and/or minimal medical intervention required. Severe: Marked limitation of daily activities, requiring assistance with activities of daily living, and/or medical intervention required, possibly hospitalization. |
24 weeks and 4 weeks
|
|
Plasma concentrations of FXS5626 and its metabolite TT-190 (Phase 2)
大体时间:24 weeks
|
24 weeks
|
|
|
Proportion of participants achieving Total Vitiligo Area Scoring Index 50 (T-VASI 50) at Week 52 (Phase 3)
大体时间:52 weeks
|
At least 50% improvement from baseline in total body Vitiligo Area Scoring Index. The T-VASI is calculated using a formula that includes contributions from all body regions, with a possible range from 0 to 100. Higher scores of T-VASI mean a worse outcome. |
52 weeks
|
|
Percent change from baseline in Facial Vitiligo Area Scoring Index (F-VASI) at planned visits (Phase 3)
大体时间:52 weeks
|
The vitiligo area scoring index (VASI) is a validated scoring method used to assess the areas of depigmentation due to vitiligo. The F-VASI includes contributions from the face, with a possible range from 0 to 3. Higher scores of F-VASI mean a worse outcome. |
52 weeks
|
|
Proportion of participants achieving Facial Vitiligo Area Scoring Index 50 (F-VASI 50) at planned visits (Phase 3)
大体时间:52 weeks
|
At least 50% improvement from baseline in facial Vitiligo Area Scoring Index. The F-VASI includes contributions from the face, with a possible range from 0 to 3. Higher scores of F-VASI mean a worse outcome. |
52 weeks
|
|
Proportion of participants achieving Facial Vitiligo Area Scoring Index 75 (F-VASI 75) at planned visits other than Week 52 (Phase 3)
大体时间:52 weeks
|
At least 75% improvement from baseline in facial Vitiligo Area Scoring Index. The F-VASI includes contributions from the face, with a possible range from 0 to 3. Higher scores of F-VASI mean a worse outcome. |
52 weeks
|
|
Proportion of participants achieving Facial Vitiligo Area Scoring Index 90 (F-VASI 90) at planned visits (Phase 3)
大体时间:52 weeks
|
At least 90% improvement from baseline in facial Vitiligo Area Scoring Index. The F-VASI includes contributions from the face, with a possible range from 0 to 3. Higher scores of F-VASI mean a worse outcome. |
52 weeks
|
|
Percent change from baseline in Total Vitiligo Area Scoring Index (T-VASI) at planned visits (Phase 3)
大体时间:52 weeks
|
The vitiligo area scoring index (VASI) is a validated scoring method used to assess the areas of depigmentation due to vitiligo. It is based on a composite estimate of the overall area of vitiligo patches, measured by the number of hand units (palm plus 5 digits = 1% body surface area [BSA]) multiplied by the degree of depigmentation within each affected area (0%, 10%, 25%, 50%, 75%, 90%, or 100%). The T-VASI is calculated using a formula that includes contributions from all body regions, with a possible range from 0 to 100. Higher scores of T-VASI mean a worse outcome. |
52 weeks
|
|
Proportion of participants achieving Total Vitiligo Area Scoring Index 50 (T-VASI 50) at planned visits other than Week 52 (Phase 3)
大体时间:52 weeks
|
At least 50% improvement from baseline in total body Vitiligo Area Scoring Index. The T-VASI is calculated using a formula that includes contributions from all body regions, with a possible range from 0 to 100. Higher scores of T-VASI mean a worse outcome. |
52 weeks
|
|
Proportion of participants achieving Total Vitiligo Area Scoring Index 75 (T-VASI 75) at planned visits (Phase 3)
大体时间:52 weeks
|
At least 75% improvement from baseline in total body Vitiligo Area Scoring Index. The T-VASI is calculated using a formula that includes contributions from all body regions, with a possible range from 0 to 100. Higher scores of T-VASI mean a worse outcome. |
52 weeks
|
|
Proportion of participants achieving Total Vitiligo Area Scoring Index 90 (T-VASI 90) at planned visits (Phase 3)
大体时间:52 weeks
|
At least 90% improvement from baseline in facial Vitiligo Area Scoring Index.The T-VASI is calculated using a formula that includes contributions from all body regions, with a possible range from 0 to 100. Higher scores of T-VASI mean a worse outcome. |
52 weeks
|
|
Change from baseline in the F-BSA affected by vitiligo at planned visits (Phase 3)
大体时间:52 weeks
|
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. |
52 weeks
|
|
Change from baseline in the T-BSA affected by vitiligo at planned visits (Phase 3)
大体时间:52 weeks
|
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. |
52 weeks
|
|
Proportion of participants achieving a score of 0 or 1 on the F-PhGVA at planned visits (Phase 3)
大体时间:52 weeks
|
The F-PhGVA is an assessment tool used by physicians to evaluate the severity of facial vitiligo. It is rated on a 4-point scale: overall improvement: (0) no change, (1)limited extent, (2)moderate extent, (3)extensive, (4)very extensive. Higher score corresponds to worse evaluation. |
52 weeks
|
|
Proportion of participants achieving a score of 0 or 1 on the T-PhGVA at planned visits (Phase 3)
大体时间:52 weeks
|
The T-PhGVA is a assessment tool used by physicians to evaluate the severity of total body vitiligo. It is rated on a 4-point scale: overall improvement: (0) no change, (1)limited extent, (2)moderate extent, (3)extensive, (4)very extensive. Higher score corresponds to worse evaluation. |
52 weeks
|
|
Proportion of participants achieving score of 1 or 2 on the a F-PaGIC-V at planned visits (Phase 3)
大体时间:52 weeks
|
F-PaGIC-V is an assessment of improvement of vitiligo on the face by the participant. It is a 7-point scale ((1) Very much improved, (2) Much improved, (3) Minimally improved, (4) No change, (5) Minimally worse, (6) Much worse, or (7) Very much worse). |
52 weeks
|
|
Proportion of participants achieving a score of 1 or 2 on the T-PaGIC-V at planned visits (Phase 3)
大体时间:52 weeks
|
T-PaGIC-V is an assessment of improvement of vitiligo on the total body by the participant. It is a 7-point scale ((1) Very much improved, (2) Much improved, (3) Minimally improved, (4) No change, (5) Minimally worse, (6) Much worse, or (7) Very much worse). |
52 weeks
|
|
Change from baseline in the VitiQoL at planned visits (Phase 3)
大体时间:52 weeks
|
VitiQoL is a 15-item QoL assessment that asks participants to rate various aspects of their condition during the past month using a 7-point scale ("Not at all" to "All of the time") |
52 weeks
|
|
Change from baseline in the Dermatology Life Quality Index (DLQI)) at planned visits (Phase 3)
大体时间:52 weeks
|
DLQI is a 10-item instrument, each item scored from 0 to 3 where higher scores correspond to worse symptom impact, full range from 0 to 30. |
52 weeks
|
|
Proportion of participants achieving a DLQI total score of "0" or "1"at planned visits (Phase 3)
大体时间:52 weeks
|
DLQI is a 10-item instrument, each item scored from 0 to 3 where higher scores correspond to worse symptom impact, full range from 0 to 30. |
52 weeks
|
|
Proportion of participants achieving a score of 4 or 5 on the Vitiligo Noticeability Scale (VNS) at planned visits (Phase 3)
大体时间:52 weeks
|
The VNS is a patient-reported measure of vitiligo treatment success that is rated on a 5-point scale. The participant was asked to respond to the following query: Compared with before treatment, how noticeable is the vitiligo now? Responses: (1) more noticeable, (2) as noticeable, (3) slightly less noticeable, (4) a lot less noticeable, and (5) no longer noticeable. higher score corresponds to worse symptom impact. |
52 weeks
|
|
Number of Participants with Adverse Events (AEs) (Phase 3)
大体时间:52 weeks and 4 weeks
|
An AE is defined as any untoward medical occurrence in a patient or clinical investigation in which a participant is administered a pharmaceutical product which does not necessarily have a causal relationship with this treatment. AE severity was assessed as mild, moderate and severe. Mild: Transient discomfort, no interference with daily activities, and/or no medical intervention required. Moderate: Mild to moderate limitation of daily activities, and/or minimal medical intervention required. Severe: Marked limitation of daily activities, requiring assistance with activities of daily living, and/or medical intervention required, possibly hospitalization. |
52 weeks and 4 weeks
|
合作者和调查者
研究记录日期
研究主要日期
学习开始 (估计的)
初级完成 (估计的)
研究完成 (估计的)
研究注册日期
首次提交
首先提交符合 QC 标准的
首次发布 (实际的)
研究记录更新
最后更新发布 (实际的)
上次提交的符合 QC 标准的更新
最后验证
更多信息
与本研究相关的术语
其他研究编号
- FXS5626-VIT-301
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