2025-524747-13-00招募中2 期
AN OPEN LABEL, SINGLE ARM, EXPLORATIVE TRIAL TO EVALUATE EFFICACY AND SAFETY OF BEROTRALSTAT IN PREVENTING ANGIOEDEMA ATTACKS IN SUBJECTS WITH ACQUIRED ANGIOEDEMA DUE TO C1 INHIBITOR DEFICIENCY (AAE)
Policlinico San Donato S.p.A.7 个研究点 分布在 1 个国家目标入组 16 人开始时间: 2026年9月1日最近更新:
适应症
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 16
- 试验地点
- 7
- 主要终点
- the change in the monthly HAE attacks rate, from week 1 to week 24 month of treatment compared to attack rate during the run-in period
研究概览
简要总结
Primary Efficacy Objective The primary objective of the study is to explore the effectiveness of Berotralstat in preventing angioedema attacks in patients with AAE. Safety Objectives. The safety objective is to evaluate the proportion of patients with adverse events (AEs) including treatment‐emergent serious adverse events (TESAEs).
入排标准
- 年龄范围
- 18 years 至 64 years（18-64 Years）
- 接受健康志愿者
- 否
入选标准
- •Males and non-pregnant, non-lactating females ≥ 18 years of age
- •Able to provide written, informed consent.
- •Subject weight of ≥ 40 kg.
- •A clinical diagnosis of AAE is defined from the following criteria: • Recurrent angioedema without wheals • No family history of angioedema and/or C1-INH deficiency • C1-inhibitor activity level of <50% of normal) and at least 1 of the following: ▪ Age at least 40 years at reported onset of first angioedema symptoms ▪ C1q level below the lower limit of normal. ▪ Absence of a pathogenic variant in the SERPING1 gene encoding for the C1-inhibitor protein. ▪ Serologic confirmation of anti–C1-inhibitor antibodies.
- •Access to and ability to use one or more acute medications approved by the relevant competent authority for the treatment of acute attacks of HAE (icatibant, plasma-derived C1-INH).
- •The subject must have at least 2 AAE attacks which meet all the requirements below during the run-in period of 28 days. In case of not recurrence of 2 attacks in 28 days or attacks do not meet all the requirements below, the run-in period will be extended to a maximum of 56 days from the Screening visit: • The attacks are unique, which is defined as an attack that does not begin within 48 hours of the end of a previous attack. • The attacks must include symptoms of swelling. Symptoms of swelling, in addition to visible swelling, may also include symptoms in the oropharyngeal or abdominal regions which are indicative of internal swelling. • The attacks are confirmed by the Investigator to be AAE attacks. Subjects will be contacted within 2 business days of the attack to discuss the attack, any queries on the entered data in the diary, as applicable.
- •In the opinion of the Investigator, the subject is expected to adequately comply with all required study procedures for the duration of the study. The subject must demonstrate adequate compliance with all study procedures required from the screening visit, including diary recording of HAE attacks beginning at the screening visit.
排除标准
- •Any clinically significant medical or psychiatric condition or medical history that, in the opinion of the Investigator or Sponsor, would interfere with the subject’s ability to participate in the study or increases the risk to the subject by participating in the study.
- •Concurrent diagnosis of any other type of recurrent angioedema.
- •Any abnormal clinical, laboratory or urinalysis parameter at screening that, in the opinion of the Investigator or any contraindication describe in berotralstat SmPC
- •Known hypersensitivity to the active substance of berotralstat or to any of its excipients
结局指标
主要结局
the change in the monthly HAE attacks rate, from week 1 to week 24 month of treatment compared to attack rate during the run-in period
the change in the monthly HAE attacks rate, from week 1 to week 24 month of treatment compared to attack rate during the run-in period
次要结局
- - monthly HAE attacks rate over 6 months of treatment - rate of attacks requiring rescue treatment - attack free period - the proportion of days without angioedema symptoms - proportion of attack rated mile, moderate or severe
研究者
PI
Scientific
Policlinico San Donato S.p.A.
研究点 (7)
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