An Open-Label, Long Term Safety and Efficacy Study of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE) - ISIS 721744-CS7

Ionis Pharmaceuticals, Inc.0 个研究点目标入组 11 人开始时间: 待定最近更新: 2024年4月29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

•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
•3. Anticipated change in the use of concurrent androgen or tranexamic acid
•prophylaxis used to prevent angioedema attacks
•4. Any clinically-significant abnormalities in screening laboratory values
•5. 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.
•6. Hypersensitivity to the active substance (donidalorsen) or to any of the
•7. 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
•8. Recent history of, or current drug or alcohol abuse
•9. Participated in a prior donidalorsen study
•10. 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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