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Donidalorsen Treatment in Children With Hereditary Angioedema

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NCT07298447 · readout ≤ 1,046 d

Sponsored by Ionis Pharmaceuticals, Inc. (industry) · IONS — their whole pipeline →.

Phase
Phase 3
Status
Recruiting
Study type
Interventional
Design
Non randomized · None · Treatment
Enrollment
20estimated
Sites
13
Countries
Italy, Poland, Spain +1

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
DateFiledBasisWhat it is
Start2026-04-21actualWhen the study began enrolling
Primary completionJun 2029estimatedThe date the last participant is measured for the primary outcome — the readout window
Study completionJun 2029estimatedThe whole study's end, after follow-up
First posted2025-12-23actualWhen this record first appeared on the registry
Results postedWhen the sponsor posted results to the registry
Record updated2026-08-05actualThe sponsor's own last edit to this record — every projection above is as current as this date

What it studies

Condition

  • Hereditary Angioedema (HAE)

Intervention

  • Drug: Donidalorsen — also filed as ISIS 721744, IONIS-PKK-LRx

Primary outcomes

what the primary-completion date above is the date OF
Number of Participants with Treatment Emergent Adverse Events (TEAEs)
measured over the period of approximately 17 months
Maximum Plasma Concentration (Cmax) of Donidalorsen
measured over the period of approximately 17 months
Maximum Time to Reach Cmax (Tmax) of Donidalorsen
measured over the period of approximately 17 months
Trough Plasma Concentration (Ctrough) of Donidalorsen
measured over the period of approximately 17 months

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