An Extension Study of Donidalorsen (IONIS-PKK-LRx) in Participants With Hereditary Angioedema

Completed · Phase 2

Conditions studied: Hereditary Angioedema

In brief

The purpose of this study was to evaluate the safety and efficacy of extended dosing of donidalorsen administered subcutaneously (SC), with alternative dosing and/or dose frequency with donidalorsen in participants with hereditary angioedema (HAE).

Key facts

Study ID

NCT04307381

Run by

Ionis Pharmaceuticals, Inc.

People needed

20

Starts

2020-03-31

Expected to finish

2025-01-24

Last updated by the study team

2026-05-08

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Satisfactory completion of ISIS 721744-CS2 (index study) through Week 17 with an acceptable safety and tolerability profile, per Sponsor and Investigator judgement
• Able and willing to participate in a 64-week study
• Females must be non-pregnant, non-lactating and either surgically sterile or post-menopausal
• Males must be surgically sterile or abstinent* or if engaged in sexual relations with a female of child-bearing potential, participant is utilizing an acceptable contraceptive method
• Participants must have access to, and the ability to use, ≥ 1 acute medication(s) (e.g., plasma-derived or recombinant C1- inhibitor (C1-INH) concentrate or a bradykinin-2 [BK-2] antagonist) to treat angioedema attacks

You may not qualify if…

• Have any new condition or worsening of an existing condition or change or anticipated change in medication, which in the opinion of the Investigator would make the participant unsuitable for enrollment, or could interfere with the participant participating in or completing the study

Where it is running

• Ionis Investigative Site — Scottsdale, Arizona, United States
• Ionis Investigative Site — Santa Monica, California, United States
• Ionis Investigative Site — Plymouth, Minnesota, United States
• Ionis Investigative Site — Cincinnati, Ohio, United States
• Ionis Investigative Site — Hershey, Pennsylvania, United States
• Ionis Investigative Site — Dallas, Texas, United States
• Ionis Investigative Site — Amsterdam, Netherlands

Full record on ClinicalTrials.gov

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