A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)
← catalyst calendarNCT05392114 · readout ≤ 133 d
Sponsored by Ionis Pharmaceuticals, Inc. (industry) · IONS — their whole pipeline →.
Phase
Phase 3
Status
Active, not recruiting
Study type
Interventional
Design
Na · None · Treatment
Enrollment
154actual
Sites
50
Countries
Belgium, Bulgaria, Canada +11
Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →
Source: ClinicalTrials.gov, retrieved 2026-08-19
Dates
each axis at the precision it was filed, with the registry's own basis| Date | Filed | Basis | What it is |
|---|---|---|---|
| Start | 2022-07-13 | actual | When the study began enrolling |
| Primary completion | Dec 2026 | estimated | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | Mar 2027 | estimated | The whole study's end, after follow-up |
| First posted | 2022-05-26 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2025-12-12 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Condition
- Hereditary Angioedema
Intervention
- Drug: Donidalorsen — also filed as ISIS 721744, IONIS-PKK-LRx
Primary outcome
what the primary-completion date above is the date OFPercentage of Participants with at Least One Treatment-emergent Adverse Event (TEAE), Graded by Severity
measured Up to approximately 70 weeks, plus 104 weeks for Group 1; up to approximately 76 weeks, plus 104 weeks for Group 2
Publications
- PMID 41767175 — linked by the registry
- PMID 40673861 — linked by the registry
Linked on the registry record. A “linked by the registry” entry is NLM's own automated PubMed match, not a claim by the sponsor that the paper reports this study.
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