A Phase 3 Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of ISIS 721744 in Patients with Hereditary Angioedema (HAE)

Ionis Pharmaceuticals, Inc.0 个研究点目标入组 84 人开始时间: 2022年1月12日最近更新: 2022年4月19日

研究概览

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•Patients must be aged = 12 years at the time of informed consent, and, as applicable, assent
•Patients must have a documented diagnosis of HAE-1/HAE-2 based upon ALL of the following:
•a. Documented clinical history consistent with HAE (subcutaneous [SC] or mucosal, non-pruritic swelling episodes without accompanying urticaria)
•b. Diagnostic testing results that confirm HAE-1/HAE-2: C1-INH functional level < 40% normal level. Patients with a functional level of 40% to 50% of normal can be enrolled if their complement factor C4 level is below the lower limit of normal (LLN) or if a known pathogenic mutation in the SERPING1 gene has been demonstrated
•c. At least 1 of the following: age at reported HAE onset = 30 years; a family history consistent with HAE-1/HAE-2; or complement component 1q within the normal range
•Patients must:
•a. Experience a minimum of 2 HAE attacks (confirmed by the Investigator) during the Screening Period
•b. Be willing to complete the PRO assessments throughout the study
•Patients must have access to, and the ability to use, = 1 acute medication(s) (e.g., plasma-derived or recombinant C1-INH concentrate or a BK2-receptor antagonist) to treat angioedema attacks
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 10
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 69
•F.1.3 Elderly (>=65 years) yes
•F.1.3.1 Number of subjects for this age range 5

•Anticipated use of short-term prophylaxis for angioedema attacks for a pre-planned procedure during the Screening, Treatment or Post-Treatment Periods
•Concurrent diagnosis of any other type of recurrent angioedema, including acquired, idiopathic angioedema or HAE with normal C1-INH (also known as HAE Type III)
•Anticipated change in the use of concurrent androgen prophylaxis used to treat angioedema attacks
•Participation in a prior ISIS 721744 study
•Exposure to any of the following medications:
•a. Angiotensin-converting enzyme (ACE) inhibitors or any estrogen-containing medications with systemic absorption (such as oral contraceptive or hormonal replacement therapy) within 4 weeks prior to Screening
•b. Chronic prophylaxis with Takhzyro, Haegarda, Cinryze or Orladeyo within 5 half-lives prior to Screening (i.e., Takhzyro within 10 weeks prior to Screening, Haegarda/Cinryze within 2 weeks prior to screening, Orladeyo within 3 weeks prior to Screening)
•c. Oligonucleotides (including small interfering ribonucleic acid) within 4 months of
•Screening if single dose received, or within 12 months of Screening if multiple doses received. This exclusion does not apply to vaccines

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