Vitiligo is a common chronic autoimmune disease that causes the body's immune system to attack its own pigment producing skin cells. This study is to evaluate how safe and effective upadacitinib is in participants with non-segmental vitiligo (NSV). Adverse effects and change in disease activity will be assessed. Upadacitinib is currently being investigated for the treatment of NSV. There will be 2 replicate studies running at the same time (Study 1 and Study 2 with periods A and B), an optional exploratory NB-UVB phototherapy study (Study 3), and a re-randomized dose-escalation study (Study 4). In Period A, participants are placed in 1 of 2 groups called treatment arms. Each group receives a different treatment. There is a 1 in 3 chance that participants will receive placebo. In Period B, all participants will receive upadacitinib. Approximately 540 adult and adolescent participants with NSV will be enrolled between each main study at around 180 sites worldwide with an option for adult participants who completed Period A of either study and did not achieve T-VASI90 at week 48 while on study drug, to enter Study 3. Participants who failed to achieve T-VASI75 response at week 118 of Study 1 or 2 or at week 70 of Study 3 will be considered for entry into Study 4. In Studies 1 and 2: Period A, participants will receive oral tablets of upadacitinib or placebo once a day for 48 weeks. In Period B, participants will receive oral tablets of upadacitinib 15 mg once a day for 112 weeks. Participants will be followed up for 30 days. Study 3 participants will receive upadacitinib monotherapy or upadacitinib with Narrow-Band Ultraviolet B (NB-UBV) phototherapy for at least 24 weeks followed by upadacitinib alone. In Study 4, participants will be re-randomized to receive oral tablets of upadacitinib 15 mg or 30 mg once a day for 56 weeks. There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, blood tests, checking for side effects and completing questionnaires.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
614
Oral Tablets
Oral Tablets
NB-UVB phototherapy is a commonly used treatment modality in participants with vitiligo and can be administered in an office setting or at home 2 times per week
Advanced Research Associates - Glendale /ID# 259915
Glendale, Arizona, United States
Alliance Dermatology and Mohs Center /ID# 259926
Phoenix, Arizona, United States
Private Practice - Dr. Tooraj Raoof /ID# 260055
Encino, California, United States
University of California Irvine /ID# 260080
Irvine, California, United States
Vitiligo & Pigmentation Institute of Southern California /ID# 259970
Los Angeles, California, United States
Percentage of Participants Achieving Total Vitiligo Area Scoring Index (T-VASI) 50 (≥ 50% Improvement in T-VASI From Baseline)
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.
Time frame: Week 48
Percentage of Participants Achieving Facial-Vitiligo Area Scoring Index (F-VASI) 75 (≥ 75% Improvement in F-VASI From Baseline)
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.
Time frame: Week 48
Percentage of Participants Achieving T-VASI 75 (≥ 75% Improvement in T-VASI From Baseline)
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.
Time frame: Study 4 Week 28
Number of Participants with Adverse Events (AEs)
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.
Time frame: Up to Approximately Week 175
Percentage of Participants Achieving F-VASI 50 (≥ 50% Improvement in F-VASI From Baseline)
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.
Time frame: Week 48
Percentage of Participants Achieving F-VASI 75 (≥ 75% Improvement in F-VASI From Baseline)
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.
Time frame: Week 24
Percent Change from Baseline in F-VASI
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.
Time frame: Week 24
Percent Change from Baseline in T-VASI
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.
Time frame: Week 48
Percentage of Participants Achieving F-VASI 90 (≥ 90% Improvement in F-VASI From Baseline)
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.
Time frame: Week 48
Percentage of Participants Achieving T-VASI 75 (≥ 75% Improvement in T-VASI From Baseline)
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.
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Dermatology Research Associates /ID# 260056
Los Angeles, California, United States
Duplicate_Stanford University /ID# 260081
Redwood City, California, United States
Integrative Skin Science and Research /ID# 260060
Sacramento, California, United States
University of California Davis Health /ID# 260062
Sacramento, California, United States
Clinical Trials Research Institute /ID# 259910
Thousand Oaks, California, United States
...and 138 more locations
Time frame: Week 48
Percentage of Participants Achieving 3D Imaging Facial Vitiligo 40 (≥ 40% Reduction in Facial Vitiligo Area Measured by 3D Digital Imaging from Baseline) (Study 1 Only)
3D imaging will be used to objectively quantify the facial vitiligo area.
Time frame: Week 48
Percentage of Participants Achieving No Increase from Baseline in T-VASI Among Participants with Actively Progressing Vitiligo at Baseline
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.
Time frame: Up to Approximately Week 12
EU/EMA Only: Percentage of Participants Achieving a Physician's Global Impression of Change - Vitiligo (PhGIC-V) of "Much better (1)"
The physician rates the overall change in the patients vitiligo by comparing the severity of vitiligo right now with the severity of vitiligo since the subject started the study treatment. Responses range from 1 = "Much better" to 5 = "Much worse."
Time frame: Week 48
EU/EMA Only: Percentage of Participants Achieving a Vitiligo Noticeability Scale (VNS) score of "A lot less noticeable (4)" or "No longer noticeable (5)"
The VNS is a single-item, validated questionnaire used in clinical trials to assess the noticeability of vitiligo lesions following therapy. The item is scored on a 5-point scale: 1 = more noticeable; 2 = as noticeable; 3 = slightly less noticeable; 4 = a lot less noticeable; and 5 = no longer noticeable. A score of 4 or 5 represents treatment success
Time frame: Week 48
EU/EMA Only: Percentage of Participants Achieving a Patient's Global Impression of Change-Vitiligo (PaGIC-V) of "Much better (1)"
The PaGIC-V asks participants to rate the overall change in their vitiligo by comparing the severity of their vitiligo right now with the severity of their vitiligo since they started the study treatment. Responses range from 1 = "Much better" to 5 = "Much worse."
Time frame: Week 48