Prospective, multicenter, randomized, double-blind, parallel group, placebo-controlled, efficacy and safety phase 3 study of an intravenous human plasma-derived C1 esterase inhibitor (C1-INH) concentrate in participants with congenital C1-INH deficiency for the treatment and pre-procedure prevention of acute hereditary angioedema attacks - CONE-02

Octapharma Pharmazeutika Produktionsgesellschaft mbH0 个研究点目标入组 124 人开始时间: 2023年10月5日最近更新: 2024年8月21日

适应症

试验速览

暂无简介。

•Is at least 18 years of age (applicable for 1st study phase) or is at least 2 years of age (applicable for 2nd study phase), Has confirmed diagnosis of HAE type I or II, Has had at least 3 moderate or severe HAE attacks (excluding extremity attacks) in the last 3 months before the Screening Visit. For participants =2 and =12 years of age, has had at least 1 moderate or severe HAE attack (excluding extremity attacks) in the last 6 months before Screening Visit, Has a documented congenital C1-INH functional activity <50% with or without C1-INH deficiency and C4 antigen level below the laboratory reference range, Participant or the participant’s legally authorized representative(s) has signed informed consent (as required by local law), with the assent of participants legally capable of providing it, as applicable, States willingness to comply with all study procedures and availability for the duration of the study, If the participant is of childbearing potential (CBP), has a negative pregnancy test and must have been using a highly effective method of contraception and continue to do so until at least 2 weeks after their last dose (for both blinded and open-label doses of IMP). Not of CBP is defined as surgically sterilized (hysterectomy, bilateral oophorectomy) or who are postmenopausal (defined as women with no menses for 12 months without an alternative medical cause)

•Has a history of clinically relevant antibody development against C1-INH, (applicable until IDMC review of the interim preliminary safety and efficacy data): has clinically significant derangement in measurements of cardiovascular status (i.e. uncontrolled arterial hypertension, cardiac insufficiency New York Heart Association (NYHA) class III-IV), pulmonary status (i.e., COPD GOLD classification 3 and 4, severe asthma) and renal status (i.e., eGFR below 90 ml/min per 1.73 m2), Has a medical history consistent with Type 3 HAE (i.e., onset at age above 40 year, no family history, no known HAE mutation, low C1q level in plasma), Has a history of allergic reaction to C1-INH or other blood/plasma product, Has a history of B-cell malignancy that was unresolved in the past 5 years, Has a narcotic and/or alcoholic addiction, Has participated in any other investigational drug evaluation within 30 days before screening, Is pregnant or breastfeeding, Has any clinically significant medical or psychiatric condition that, in the investigator’s opinion would interfere with the participant’s ability to participate in the study, Has a history of thromboembolic events (TEEs), myocardial infarction, unstable angina pectoris, critical aortic stenosis, cerebrovascular accident, transient ischemic attack, severe peripheral vascular disease, or disseminated intravascular coagulation within one year before screening

发起方