A Phase 3 Double Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of ISIS 721744 in Patients with Hereditary Angioedema (HAE) - ISIS 721744-CS5

Ionis Pharmaceuticals, Inc.0 个研究点目标入组 8 人开始时间: 待定最近更新: 2024年4月15日

适应症

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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

•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
•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 (lanadelumab), Haegarda (C1-esterase
•inhibitor SQ), Cinryze and Ruconest (C1 esterase inhibitor) or Orladeyo
•(berotralstat) within 5 half lives prior to Screening (i.e., Takhzyro within 10
•weeks prior to Screening, Haegarda/Cinryze/Ruconest 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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