A Multicenter, Randomized, Double-Blind, Placebo-Controlled Dose-Ranging Study to Evaluate the Safety and Efficacy of Upadacitinib in Subjects With Non-Segmental Vitiligo
Trial Snapshot
- Phase
- Phase 2
- Status
- Completed
- Sponsor
- Enrollment
- 185
- Locations
- 35
- Primary Endpoint
- Percent Change From Baseline in Facial-Vitiligo Area Scoring Index (F-VASI) at Week 24
Study Overview
Brief Summary
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. Adverse effects and change in disease activity will be assessed.
Upadacitinib is being evaluated for the treatment of non-segmental vitiligo. The study will enroll approximately 160 participants aged 18-65 with non-segmental vitiligo in 5 treatment arms across 35 sites worldwide.
Participants will either receive study drug vs placebo oral tablets once daily (QD) for 24 weeks (Period A). In Period B (up to 52 weeks), participants who received placebo during the first 24 weeks will switch to study drug. Participants who received study drug during the first 24 weeks, will continue to receive study drug.
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 Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Eligibility Criteria
- Ages
- 18 Years to 65 Years (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Clinical diagnosis of non-segmental vitiligo (NSV) and no segmental or localized vitiligo.
- •Participants with all of the following at Screening and Baseline.
- •Visits: ≥ 0.5 F-VASI and ≥ 5 total vitiligo area scoring index (T-VASI).
- •Participants who have had prior exposure to immunomodulatory biologic therapy, for any indications, but discontinued the biologic therapy prior to the first dose of study drug. Recommended washout periods for biologic therapies include ≥ 4 weeks for etanercept; ≥ 8 weeks for adalimumab, infliximab, certolizumab, golimumab, abatacept, tocilizumab, and ixekizumab; ≥ 16 weeks for secukinumab; and ≥ 12 weeks for ustekinumab. For biologic therapies not specified, therapies must be discontinued at least 5 times the mean terminal elimination half-life of a drug or 3 months prior to Baseline, whichever is longer.
Exclusion Criteria
- •Participants with segmental or localized vitiligo.
- •Participants with other skin conditions that would interfere with evaluation of vitiligo, participants with uncontrolled thyroid disease, and participants with > 33% leukotrichia on the face or > 33% leukotrichia on the body (including face).
- •Participants previously treated with any topical or systemic janus kinase (JAK) inhibitor or permanent skin bleaching agents.
- •Participants treated with any systemic vitiligo therapy (e.g., methotrexate, mycophenolate mofetil, corticosteroids), supplemental vitiligo therapy (antioxidants/vitamins/herbal medicine/traditional Chinese medicine), and/or topical vitiligo therapy including permanent or temporary tattoos within a minimum of 30 days prior to the first dose of study drug (Note: Camouflage and makeup may be used).
- •Participants treated with any phototherapy, including excimer (or other forms of laser therapy), within a minimum of 12 weeks prior to the first dose of study drug.
- •Participants have history of malignancy other than successfully treated non-melanoma skin cancer (NMSC) or localized carcinoma in situ of the cervix.
- •Recent (within past 6 months) cerebrovascular accident, myocardial infarction, coronary stenting, and aorto-coronary bypass surgery;
- •History of an organ transplant which requires continued immunosuppression;
- •History of gastrointestinal (GI) perforation (other than due to appendicitis or mechanical injury), diverticulitis, or significantly increased risk for GI perforation per investigator judgment;
- •Conditions that could interfere with drug absorption including but not limited to short bowel syndrome or gastric bypass surgery; subjects with a history of gastric banding/segmentation are not excluded;
- •Uncontrolled thyroid disease;
Arms & Interventions
Upa 22 mg Period 1, then Upa 22 mg Period 2
Participants received upadacitinib 22 mg administered orally once a day (QD) as tablets for 24 weeks during Period 1. Participants then received upadacitinib 22 mg administered orally once a day (QD) as tablets for 28 weeks during Period 2.
Intervention: Upadacitinib (Drug)
Upa 11 mg Period 1, then Upa 11 mg Period 2
Participants received upadacitinib 11 mg administered orally once a day (QD) as tablets for 24 weeks during Period 1. Participants then received upadacitinib 11 mg administered orally once a day (QD) as tablets for 28 weeks during Period 2.
Intervention: Upadacitinib (Drug)
Upa 6 mg Period 1, then Upa 6 mg Period 2
Participants received upadacitinib 6 mg administered orally once a day (QD) as tablets for 24 weeks during Period 1. Participants then received upadacitinib 6 mg administered orally once a day (QD) as tablets for 28 weeks during Period 2.
Intervention: Upadacitinib (Drug)
Placebo Period 1, then Upa 22 mg Period 2
Participants received Placebo for upadacitinib administered orally once a day (QD) as tablets for 24 weeks during Period 1. Participants then received upadacitinib 22 mg administered orally once a day (QD) as tablets for 28 weeks during Period 2.
Intervention: Upadacitinib (Drug)
Placebo Period 1, then Upa 22 mg Period 2
Participants received Placebo for upadacitinib administered orally once a day (QD) as tablets for 24 weeks during Period 1. Participants then received upadacitinib 22 mg administered orally once a day (QD) as tablets for 28 weeks during Period 2.
Intervention: Placebo (Drug)
Placebo Period 1, then Upa 11 mg Period 2
Participants received Placebo for upadacitinib administered orally once a day (QD) as tablets for 24 weeks during Period 1. Participants then received upadacitinib 11 mg administered orally once a day (QD) as tablets for 28 weeks during Period 2.
Intervention: Upadacitinib (Drug)
Placebo Period 1, then Upa 11 mg Period 2
Participants received Placebo for upadacitinib administered orally once a day (QD) as tablets for 24 weeks during Period 1. Participants then received upadacitinib 11 mg administered orally once a day (QD) as tablets for 28 weeks during Period 2.
Intervention: Placebo (Drug)
Outcomes
Primary Outcomes
Percent Change From Baseline in Facial-Vitiligo Area Scoring Index (F-VASI) at Week 24
Time Frame: Baseline, Week 24
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, with higher scores indicating more severe disease. Negative changes from baseline indicate improvement.
Secondary Outcomes
- Percentage of Participants Achieving F-VASI 75 (≥ 75% Improvement in F-VASI From Baseline) at Week 24(Baseline, Week 24)
- Percentage of Participants Achieving F-VASI 50 (≥ 50% Improvement in F-VASI From Baseline) at Week 24(Baseline, Week 24)
- Percentage of Participants Achieving Total Vitiligo Area Scoring Index (T-VASI) 50 (≥ 50% Improvement in T-VASI From Baseline) at Week 24(Baseline, Week 24)
- Percent Change From Baseline in T-VASI at Week 24(Baseline, Week 24)
- Change From Baseline in the Vitiligo Quality-of-Life (VitiQoL) Instrument Total Score at Week 24(Baseline, Week 24)