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

  • End date
    Oct 24, 2023
  • participants needed
  • sponsor
    Ionis Pharmaceuticals, Inc.
Updated on 24 October 2022
Ionis Pharmaceuticals
Primary Contact
Ionis Investigative Site (5.7 mi away) Contact
+28 other location


The purpose of this study is to evaluate the safety and efficacy of donidalorsen in participants with HAE and effect of donidalorsen on the quality and pattern of HAE attacks and their impact on quality of life (QoL).


This is a Phase 3, multi-center, double-blind, randomized, placebo-controlled study of donidalorsen in up to 84 participants. Participants will be randomized in a 2:1 ratio to Cohort A (donidalorsen or placebo every 4 weeks) or Cohort B (donidalorsen or placebo every 8 weeks), respectively. Within each Cohort, participants will be randomized in a 3:1 ratio to receive donidalorsen or matching-placebo. The length of participation in the study is approximately 11 months, which includes an up to 8-week Screening Period, a 25-week Treatment Period, and an up to 13-week Post-treatment Period.

Condition Hereditary Angioedema
Treatment Placebo, donidalorsen
Clinical Study IdentifierNCT05139810
SponsorIonis Pharmaceuticals, Inc.
Last Modified on24 October 2022


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Inclusion Criteria

Participants, or their legally appointed and authorized representatives, must provide written and signed informed consent form (ICF)/assent
Participants must be aged ≥ 12 years at the time of informed consent and, as applicable, assent
Participants must have a documented diagnosis of hereditary angioedema type 1 (HAE-1)/hereditary angioedema type 2 (HAE-2)
Participants must
Experience a minimum of 2 HAE attacks (confirmed by the Investigator) during the Screening Period
Be willing to complete the participant reported outcomes (PRO) assessments throughout the study
Participants must have access to, and the ability to use acute medication(s) to treat
angioedema attacks

Exclusion Criteria

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)
Any clinically-significant abnormalities in screening laboratory values that would render a participant unsuitable for inclusion in the study
Treatment with another investigational drug or biological agent within 1 month or 5 half-lives, whichever is longer, of Screening
Participated in a prior ISIS 721744 study
Exposure to any of the following medications
Angiotensin-converting enzyme (ACE) inhibitors or any estrogen containing medications with systemic absorption within 4 weeks prior to Screening
Chronic prophylaxis with Takhzyro, Haegarda, Cinryze and Ruconest or Orladeyo within 5 half-lives prior to Screening
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
Recent history (3 years) of, or current drug or alcohol abuse
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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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