An Open-Label, Long Term Safety and Efficacy Study of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)

IONIS PHARMACEUTICALS, INC.0 个研究点目标入组 144 人开始时间: 2022年6月24日最近更新: 2024年7月8日

研究概览

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•1. Participants and, as applicable, legally authorized representatives (i.e., parent(s)/legal guardian), must provide written and signed informed consent form (ICF).
•2. Participants must have access to, and the ability to use, = 1 acute medication(s) (e.g., plasma-derived or recombinant C1-INH concentrate or a bradykinin receptor (BK) 2-receptor antagonist) to treat angioedema attacks
•Open-Label Extension Participants ONLY:
•3. Satisfactory completion of ISIS 721744-CS5 (randomized placebo-controlled index study) through Week 25 or participants who are allowed to exit ISIS 721744-CS5 study per protocol with an acceptable safety and tolerability profile
•New (not previously on donidalorsen) Participants ONLY
•4. Participants must be aged = 12 years at the time of informed consent and, as applicable, assent
•5. Participants must have a documented diagnosis of HAE-1/HAE-2
•6. Participants must be on a stable dose (= 12 weeks) of prophylaxis treatment with lanadelumab or berotralstat or C1-esterase inhibitor prior to the Screening Period
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 13
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 125
•F.1.3 Elderly (>=65 years) yes
•F.1.3.1 Number of subjects for this age range 6

•1. Have any new condition or worsening of an existing condition or change or anticipated change in medication
•De-novo Participants:
•2. 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)
•3. Anticipated change in the use of concurrent androgen or tranexamic acid prophylaxis used to prevent angioedema attacks
•Any clinically-significant abnormalities in screening laboratory values
•4. Malignancy within 5 years of Screening, except for non-melanoma skin cancers, cervical in situ carcinoma, breast ductal carcinoma in situ, or stage 1 prostate carcinoma that has been successfully treated.
•5. Hypersensitivity to the active substance (donidalorsen) or to any of the excipients
•6. Treatment with another investigational drug (non-oligonucleotide) or biological agent within 1 month of Screening or 5 half-lives of investigational agent, whichever is longer
•7. Recent history of, or current drug or alcohol abuse
•8. Participated in a prior donidalorsen study
•9. Exposure to any of the following medications:
•Angiotensin-converting enzyme (ACE) inhibitors or any estrogen containing medications with systemic absorption
•Oligonucleotides (including small interfering ribonucleic acid [siRNA]) 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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