A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of the Efficacy and Safety of Parsaclisib in Participants With Primary Warm Autoimmune Hemolytic Anemia
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 入组人数
- 13
- 试验地点
- 56
- 主要终点
- Percentage of Participants Attaining a Durable Hemoglobin Response
研究概览
简要总结
The purpose of this study is to evaluate the efficacy and safety of parsaclisib compared with placebo in participants with Primary Warm Autoimmune Hemolytic Anemia (wAIHA),
详细描述
Prospective participants must have primary wAIHA as well as other protocol-defined criteria. After participants have been determined to be eligible for the study, they will be randomized to 2:1, with stratification factor of corticosteroid dose and hemoglobin (Hgb <9 g/dL or ≥ 9 g/dL). Once a participant has completed the week 24 assessments in the double-blind period, the participant will have the opportunity to receive parsaclisib in the open-label treatment which will last up to another 24 weeks. Participants may then continue to receive parsaclisib in a long-term extension period.
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
- Triple (Participant, Care Provider, Investigator)
盲法说明
Study will be a 24 week double-blind period followed by a 24 week open-label period, followed by a long term extension period.
入排标准
- 年龄范围
- 18 Years 至 99 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of primary warm AIHA.
- •Participants who have at least 1 unsuccessful prior therapy for warm AIHA or unable to receive or tolerate other therapies.
- •Hemoglobin ≥ 6.5 to < 10 g/dL with symptoms of anemia at screening.
- •FACIT-F score ≤ 43 at screening.
- •Willingness to avoid pregnancy or fathering children.
- •Willingness to receive PJP prophylaxis.
- •Further inclusion criteria apply.
排除标准
- •Women who are pregnant, breastfeeding or who are planning a pregnancy.
- •Diagnosis of other types of AIHA (CAD, cold agglutinin syndrome, mixed-type AIHA or paroxysmal cold hemoglobinuria).
- •Secondary warm AIHA from any cause or diagnosis of Evans syndrome.
- •Splenectomy less than 3 months before randomization.
- •Participants with a history or ongoing significant illness as assessed by the investigator.
- •Participants with a current of medical history of a malignancy within the past 5 years except basal or squamous cell skin cancer that has been removed and considered cured, or superficial bladder cancer, prostate intraepithelial neoplasm, carcinoma in situ of the cervix, or other noninvasive or indolent malignancy.
- •Participants know to be infected with HIV, Hepatitis B, or hepatitis C.
- •Chronic or current active infectious disease requiring systemic antibiotics, antifungal, or antiviral treatment or exposure to a live vaccine.
- •Participants with laboratory values outside of the protocol defined ranges.
- •Further exclusion criteria apply.
研究组 & 干预措施
Group A: Parsaclisib
Participants will receive parsaclisib for 24 weeks (double-blind period). Participant who completed the double-blind period and tolerating the study treatment upon investigator's opinion will continue into open-label period for an additional 24 weeks. Participants may then continue to receive parsaclisib in a long-term extension period.
干预措施: parsaclisinib (Drug)
Group B: Placebo followed by Parsaclisib
Participants will receive placebo for 24 weeks (double-blind period). Participants who completed the double-blind period will receive parsaclisib in the 24 week open-label period. Participants may then continue to receive parsaclisib in a long-term extension period.
干预措施: placebo (Drug)
结局指标
主要结局
Percentage of Participants Attaining a Durable Hemoglobin Response
时间窗: up to Week 24
A durable hemoglobin response was defined as hemoglobin ≥10 grams per deciliter (g/dL) with an increase from Baseline of ≥2 g/dL not attributed to rescue therapy at ≥3 of the 4 available visits at Week 12 and/or later during the 24-week double-blind treatment period.
次要结局
- Percentage of Participants With a ≥3-point Increase From Baseline in Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-F) Score at Week 24(Baseline; Week 24)
- Percentage of Participants With a 50 Meter Increase From Baseline to Week 24 in a 6-minute Walk Test (6MWT)(Baseline; Week 24)
- Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit(Baseline; Day 1; Weeks 8, 12, 16, 20, 24, 28, 32, 40, 48, 56, and every 16 weeks post-Week 56)
- Percentage Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit(Baseline; Day 1; Weeks 8, 12, 16, 20, 24, 28, 32, 40, 48, 56, and every 16 weeks post-Week 56)
- Change From Baseline in Hemoglobin at Each Post-Baseline Visit(Baseline; Day 1; Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, and every 8 weeks post-Week 56)
- Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit(Baseline; Day 1; Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, and every 8 weeks post-Week 56)
- Percentage of Participants Who Received Transfusions From Week 6 to Week 24 and From Week 24 to Week 48(Week 6 to Week 24; Week 24 to Week 48)
- Change From Baseline in Daily Corticosteroid Dose at Week 24(Baseline; Week 24)
- Percentage Change From Baseline in Daily Corticosteroid Dose at Week 24(Baseline; Week 24)
- Percentage of Participants Who Required Rescue Therapy at Any Visit From Week 6 Through Week 24, and From Week 24 to Week 48(Week 6 to Week 24; Week 24 to Week 48)
- Number of Participants With Any Treatment-emergent Adverse Event (TEAE)(up to 446 days)
- Number of Participants With Any ≥Grade 3 TEAE(up to 446 days)