Phase 2
N=20
An Extension Study of Donidalorsen (IONIS-PKK-LRx) in Participants With Hereditary Angioedema
Hereditary Angioedema
Bottom Line
View on ClinicalTrials.gov: NCT04307381 ↗Enrolled (actual)
20
Serious AEs
2.2%
Results posted
May 2026
Primary outcome: Primary: Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) — 70.6; 66.7; 84.6; 62.5 percentage of participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Donidalorsen (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Ionis Pharmaceuticals, Inc.
- Primary completion
- Jan 2025
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) |
70.6; 66.7; 84.6; 62.5; 100; 100 | — |
| PRIMARY Percentage of Participants With Clinically Meaningful Changes in Clinical Laboratory Assessments |
0; 0; 0; 0; 0; 0 | — |
| PRIMARY Percentage of Participants With Clinically Meaningful Changes in Vital Signs |
0; 0; 0; 0; 0; 0 | — |
| PRIMARY Percentage of Participants With Clinically Meaningful Changes in Electrocardiograms (ECGs) Parameters |
0; 0; 0; 0; 0; 0 | — |
| PRIMARY Percentage of Participants Who Received At Least One Concomitant Medication |
100; 100 | — |
| SECONDARY Percent Change From Baseline in Time-normalized HAE Attack (Per 4 Weeks) Rate |
-94.41; -60.82; -97.21; -82.70; -95.16; -87.21 | — |
| SECONDARY Percentage of Participants Who Used On-demand Medications |
11.8; 66.7; 38.5; 62.5; 100; 100 | — |
Summary
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).
Eligibility Criteria
Inclusion Criteria
- 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
Exclusion Criteria
- 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
Data sourced from ClinicalTrials.gov (NCT04307381). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.