A Phase 3 Randomized, Placebo-Controlled, Double-Blind Study to Evaluate Efficacy and Safety of Upadacitinib in Adult and Adolescent Subjects With Alopecia Areata and at Least 25% Scalp Hair Loss
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 125
- 试验地点
- 29
- 主要终点
- Percentage of Participants with the Achievement of Severity of Alopecia Tool (SALT) Score <= 10
研究概览
简要总结
Alopecia areata (AA) is a disease that happens when the immune system attacks hair follicles and causes hair loss. AA usually affects the head and face, but hair loss can happen on any part of the body. The purpose of this study is to assess how safe, effective, and tolerable upadacitinib is in adolescent and adult participants in Japan with severe AA.
Upadacitinib is an approved drug being investigated for the treatment of AA. In Period A, participants are placed in 1 of 3 groups, called treatment arms. Each group receives a different treatment. There is a 1 in 3 chance that participants will be assigned to placebo. In Period B, participants originally randomized to a upadacitinib dose group in Period A will continue their same treatment in Period B. Participants originally randomized to Placebo in Period A will be re-randomized in 1 of 2 groups receiving upadacitinib. Participants who complete Period B can join Period C and will receive 1 of 2 doses of upadacitinib for up to 52 weeks based on their SALT score. Around 123 adolescent and adult participants with severe AA will be enrolled in the study at approximately 20 sites in Japan.
Participants will receive oral tablets of either upadacitinib or placebo once daily for up to 104 weeks with the potential of being re-randomized into a different treatment group at Weeks 24 and 52. Participants will be followed up for up to 30 days after their last study drug dose.
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 12 Years 至 63 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participant is judged to be in good health as determined by the Principal Investigator, based upon the results of the Screening assessments and medical history.
- •Diagnosis of severe alopecia areata (AA) with Severity of Alopecia Tool (SALT) score >= 25 (>= 25% scalp hair loss) at Screening and Baseline.
- •Current episode of AA of less than 8 years.
排除标准
- •Current diagnosis of primarily diffuse type of AA.
- •Current diagnosis of other types of alopecia that would interfere with evaluation of AA, including but not limited to female pattern hair loss, male pattern hair loss (androgenetic alopecia) Stage III or greater based on Hamilton-Norwood classification, traction alopecia, lichen planopilaris (LPP), discoid lupus, frontal fibrosing alopecia (FFA), central centrifugal cicatricial alopecia (CCCA), folliculitis decalvans, trichotillomania, and telogen effluvium.
- •Diagnosis of other types of inflammatory scalp, eyebrow, or eyelash disorders that would interfere with evaluation of AA as determined by the investigator, including but not limited to seborrheic dermatitis, scalp psoriasis, atopic dermatitis (AD), and tinea capitis.
- •Active infection(s) requiring treatment with intravenous anti-infectives within 30 days, or oral/intramuscular anti-infectives within 14 days prior to the Baseline Visit.
- •Chronic recurring infection and/or active viral infection that, based on the investigator's clinical assessment, makes the participant an unsuitable candidate for the study.
研究组 & 干预措施
Period C: Open-Label Upadacitinib Dose B
Participants with no improvement or worsening from Baseline in their SALT score at the Week 40 visit or any scheduled visit thereafter will receive open-label upadacitinib dose B once daily for 52 weeks.
干预措施: Upadacitinib (Drug)
Group 1A: Upadacitinib Dose A
Participants will receive upadacitinib dose A once daily for 52 weeks in Period A and Period B.
干预措施: Upadacitinib (Drug)
Group 2B: Upadacitinib Dose B
Participants initially randomized to placebo (Period A) will be re-randomized to receive upadacitinib dose B once daily for 28 weeks in Period B.
干预措施: Upadacitinib (Drug)
Group 1B: Upadacitinib Dose A
Participants initially randomized to placebo (Period A) will be re-randomized to receive upadacitinib dose A once daily for 28 weeks in Period B.
干预措施: Upadacitinib (Drug)
Group 3A: Upadacitinib Placebo
Participants will receive upadacitinib placebo once daily for 24 weeks in Period A.
干预措施: Upadacitinib Placebo (Drug)
Group 2A: Upadacitinib Dose B
Participants will receive upadacitinib dose B once daily for 52 weeks in Period A and Period B.
干预措施: Upadacitinib (Drug)
Period C: Upadacitinib Dose B Non-Sustained Responders
For participants initially randomized to dose B in Periods A and B and participants initially randomized to placebo who switched to dose B in Period B: participants with a SALT score > 10 at Week 40 or Week 52 will remain on blinded upadacitinib dose B once daily in Period C for 52 weeks.
干预措施: Upadacitinib (Drug)
Period C: Upadacitinib Dose A Remains Dose A
For participants initially randomized to dose A in Periods A and B and participants initially randomized to placebo who switched to dose A in Period B: participants with a Severity of Alopecia Tool (SALT) score <= 10 at Week 52 (end of Period B) will remain on blinded upadacitinib dose A once daily in Period C for 52 weeks.
干预措施: Upadacitinib (Drug)
Period C: Upadacitinib Dose A to Dose B
For participants initially randomized to dose A in Periods A and B and participants initially randomized to placebo who switched to dose A in Period B: participants with a SALT score > 10 at Week 52 (end of Period B) will dose escalate to blinded upadacitinib dose B once daily in Period C for 52 weeks.
干预措施: Upadacitinib (Drug)
Period C: Upadacitinib Dose B Sustained Responders
For participants initially randomized to dose B in Periods A and B and participants initially randomized to placebo who switched to dose B in Period B: participants with a SALT score <= 10 at Week 40 and Week 52 will receive blinded upadacitinib dose A once daily in Period C for 52 weeks.
干预措施: Upadacitinib (Drug)
结局指标
主要结局
Percentage of Participants with the Achievement of Severity of Alopecia Tool (SALT) Score <= 10
时间窗: Week 24
The SALT is a global alopecia areata (AA) severity score based on the combination of extent and density of scalp hair loss. The score is determined by visually defining the amount of terminal hair loss in each of the 4 views of the scalp (left and right side each accounting for 18% of scalp area, the top for 40%, and the back for 24%) and adding these together with a maximum score of 100%. A SALT score of \<= 10 is defined as less than or equal to 10% hair loss.
Number of Participants with Adverse Events (AEs)
时间窗: Up to approximately 108 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.
次要结局
- Percentage of Participants with the Achievement of Patient-Reported Outcome (PRO) for Scalp Hair Assessment of 0 or 1(Week 24)
- Percentage of Participants with the Achievement of Patients' Global Impression of Change of Alopecia Areata (PaGIC-AA) Score of 1 "Much Better" or 2 "Moderately Better"(Week 24)