Multicenter, Single-arm, Open-label Phase III Study to Evaluate Efficacy and Safety of HRS-5965 Capsule in Patients With PNH Who Are Still Anemia After Anti-C5 Antibody Treatment

Chengdu Suncadia Medicine Co., Ltd.4 个研究点分布在 1 个国家目标入组 39 人2025年1月7日

适应症Paroxysmal Nocturnal Hemoglobinuria

干预措施HRS-5965 capsule

概览

阶段: 3 期
干预措施: HRS-5965 capsule
疾病 / 适应症: Paroxysmal Nocturnal Hemoglobinuria
发起方: Chengdu Suncadia Medicine Co., Ltd.
入组人数: 39
试验地点: 4
主要终点: In the absence of red blood cell infusion (defined as no red blood cell infusion after week 2 to week 24), the proportion of subjects with at least 3 times of ≥12 g/dL of hemoglobin level measured 4 times between weeks 18 and 24.
状态: 已完成
最后更新: 3个月前

概览研究者入排标准研究组 & 干预措施结局指标研究点相似试验

概览

简要总结

This trial is a multi-center, single-arm, open-label phase III clinical trial. A total of approximately 35 patients with paroxysmal nocturnal hemoglobinuria who remained anemic despite stable use of C5 complement inhibitor (eculizumab/Kevacumab) for the first 6 months before randomization were included in the study. Approximately 40% of the subjects had received at least one red blood cell (RBC) transfusion within the first 6 months before receiving the experimental intervention. Subjects who met the criteria were all treated with HRS-5965 capsules. This trial includes an 8-week screening period, a 24-week treatment period, a 2-week dose reduction period, and a 4-week safety follow-up period.

注册库

clinicaltrials.gov

开始日期

2025年1月7日

结束日期

2026年1月6日

最后更新

3个月前

研究类型

Interventional

研究设计

Single Group

性别

All

研究者

发起方

Chengdu Suncadia Medicine Co., Ltd.

责任方

Sponsor

入排标准

入选标准

•Understand the specific process of the experiment, voluntarily participate in this experiment, and sign a written informed consent form.
•Age ≥18 on the day of signing the informed consent, regardless of gender
•It was confirmed to be PNH during screening, and the clone size of red blood cells or granulocytes was detected by flow cytopy \>10%
•Stable use of C5 complement inhibitor ikuzumab/covalimab for the first 6 months of random treatment
•Have at least one blood transfusion record within the last 3 months, or sustain a hemoglobin level below 10g/dL during the eculizumab treatment within the last 3 months prior to screening.
•The average hemoglobin level from two tests conducted by the central laboratory at the time of screening is less than 10 g/dL, with each individual Hb value being less than 10.5 g/dL.
•Inoccution of Neisseris meningitis and Streptococcus pneumoniae vaccine at least 2 weeks before the first administration of HRS-5965; if HRS-5965 treatment must begin less than 2 weeks after vaccination, preventive antibiotic treatment must begin at least 2 weeks after vaccination.
•Male and female subjects with fertility must agree to adopt efficient contraceptive measures with their partners within 30 days from the signing of the informed consent form to the last administration, and have no family planning and no sperm/egg donation.

排除标准

•In addition to C5 complement inhibitors (ikuzuzumab/covalimamab), those who have participated in clinical trials of any other drug or medical device within 1 month before and are expected to have a residual effect of experimental treatment (judged by researchers), or those who were still in the follow-up period of a clinical trial or the 5 half-life of the experimental drug before screening Inside (whichever is longer)
•Known or suspected hereditary or acquired complement deficiency
•Currently active primary or secondary immunodeficiency
•Those who have a history of splenectomy or plan to perform surgery during the trial
•History of bone marrow/hematopoietic stem cells or solid organ transplantation
•Diagnosed malignant tumors in the past 5 years
•There is laboratory evidence for patients with bone marrow failure during screening
•History of infection with pod bacteria (such as Neisseris meningitis, Streptococcus pneumoniae, etc.)
•There is or is suspected of systemic active bacteria, virus or fungal infection 2 weeks before the first administration of HRS-5965 (according to the researcher's judgment)
•Fever occurs within 1 week before the first administration of HRS-5965 (body temperature ≥38 ℃)

研究组 & 干预措施

HRS-5965 capsule

干预措施: HRS-5965 capsule

结局指标

主要结局

In the absence of red blood cell infusion (defined as no red blood cell infusion after week 2 to week 24), the proportion of subjects with at least 3 times of ≥12 g/dL of hemoglobin level measured 4 times between weeks 18 and 24.

时间窗: From the 18th to the 24th week.

次要结局

The proportion of subjects who did not receive red blood cell infusion from week 2 to week 24(From the 18th to the 24th week)
The average change of hemoglobin compared with baseline during weeks 18 to 24(From the 18th to the 24th week)
The average percentage change of lactate dehydrogenase (LDH) level compared with the baseline during weeks 18 to 24(From the 18th to the 24th week)
The average change of reticular red blood cell count compared with the baseline during weeks 18 to 24(From the 18th to the 24th week)
Chronic Disease Treatment Function Assessment (FACIT) from weeks 18 to 24 - the average change of the score compared to the baseline(From the 18th to the 24th week)
The incidence of breakthrough hemolysis from week 1 to week 24(From the 18th to the 24th week)
The incidence of MAVEs (including thrombosis) from week 1 to week 24(From the 1st to the 24th week)
The incidence and severity of adverse events from week 1 to week 24(From the 1st to the 24th week)
The concentration of HRS-5965 in plasma(From the 1st to the 24th week)
The percentage of the change of complement bypass pathway (AP) activity at each point in time compared with the baseline(From the 1st to the 24th week)