An Open-Label Study to Evaluate Efficacy and Safety of Long-Term Treatment With ACH-0144471 in Participants Who Completed Clinical Study ACH471-100
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 8
- 试验地点
- 1
- 主要终点
- Change From Baseline in Hgb Level in the Absence of RBC Transfusion at Week 25
研究概览
简要总结
The purpose of this study is to evaluate the long-term safety and efficacy of ACH-0144471 in participants with paroxysmal nocturnal hemoglobinuria (PNH) who have demonstrated clinical benefit from ACH-0144471 in Study ACH471-100. This study is designed to include up to 12 participants.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Study designed to include up to 12 participants who completed treatment in Study ACH471-100 and demonstrated clinical benefit from ACH-0144471 with no significant safety or tolerability concerns.
- •Negative pregnancy test for females prior to dosing and throughout the study.
排除标准
- •Have developed any clinically relevant co-morbidities while participating in Study ACH471-100 that would make the participant inappropriate for the continuation of treatment with ACH-0144471, in the opinion of the Investigator.
- •Have developed any clinically significant laboratory abnormalities while participating in Study ACH471-100 that, in the opinion of the Investigator, would make the participant inappropriate for the study or put the participant at undue risk.
- •Females who are pregnant, nursing, or planning to become pregnant during the study or within 90 days of study drug administration or participants with a female partner who is pregnant, nursing, or planning to become pregnant during the study or within 90 days of study drug administration.
研究组 & 干预措施
ACH-0144471
All participants will receive ACH-0144471 during the treatment period.
干预措施: ACH-0144471 (Drug)
结局指标
主要结局
Change From Baseline in Hgb Level in the Absence of RBC Transfusion at Week 25
时间窗: Baseline, Week 25
Change from Baseline = Hgb levels at Week 25 - Baseline Hgb levels. Baseline was the baseline value from the primary Study ACH471-100.
Number of RBC Units Transfused
时间窗: Baseline up to Week 169
Number of RBC Transfusion Instances
时间窗: Baseline up to Week 169
Change From Baseline in PNH Clone Size at Week 25
时间窗: Baseline, Week 25
The PNH clone size refers to the percentage of PNH-affected cells versus normal cells within the total cell population. Change from Baseline = PNH clone size at Week 25 - Baseline PNH clone size. Baseline was the baseline value from the primary Study ACH471-100.
Change From Baseline in Free Hgb at Week 25
时间窗: Baseline, Week 25
Change from Baseline = free Hgb at Week 25 - Baseline free Hgb. Baseline was the baseline value from the primary Study ACH471-100.
Change From Baseline in LDH Level at Week 25
时间窗: Baseline, Week 25
Change from Baseline = Serum LDH levels at Week 25 - Baseline Serum LDH levels. Baseline was the baseline value from the primary Study ACH471-100.
Change From Baseline in Reticulocyte Counts at Week 25
时间窗: Baseline, Week 25
Change from Baseline = reticulocyte count at Week 25 - Baseline reticulocyte count. Baseline was the baseline value from the primary Study ACH471-100.
Change From Baseline in AP Complement Functional Activity at Week 25
时间窗: Baseline, Week 25
Serum AP functional activity was measured by the Wieslab functional immunoassay method. Change from Baseline = Serum AP functional activity at Week 25 - Baseline Serum AP functional activity. Baseline was the baseline value from the primary Study ACH471-100.
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Grade 3 and Grade 4 Adverse Events (AEs), And AEs Leading To Discontinuation
时间窗: Baseline up to 4.5 years
An AE was as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An SAE was an AE that met at least 1 of the following criteria: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization for the AE, persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect (in the child of a participant who was exposed to the study drug), important medical event or reaction. The intensity of an AE was graded according to the Common Terminology Criteria for Adverse Events (CTCAE) Adverse Event Severity Grading Table. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.
次要结局
- Change From Baseline in Reticulocyte Counts at Weeks 49 and 169(Baseline, Weeks 49 and 169)
- Change From Baseline in PNH Clone Size at Weeks 49 and 73(Baseline, Weeks 49 and 73)
- Change From Baseline in AP Complement Functional Activity at Weeks 49 and 145(Baseline, Weeks 49 and 145)
- Change From Baseline in European Organisation for Research and Treatment of Cancer, Quality of Life Questionnaire-Core 30 Scale (EORTC-QLQ-C30): Global Health Status/Qol Score at Weeks 21, 41, and 153(Baseline, Weeks 21, 41, and 153)
- Change From Baseline in LDH Level at Weeks 49 and 169(Baseline, Weeks 49 and 169)
- Change From Baseline in Free Hgb at Weeks 49 and 169(Baseline, Weeks 49 and 169)
- Change From Baseline in Hgb Level in the Absence of RBC Transfusion at Weeks 49 and 169(Baseline, Weeks 49 and 169)
- Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score at Weeks 21, 41, and 153(Baseline, Weeks 21, 41, and 153)