NCT04307381Phase 2Completed

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

Study sponsor: Ionis Pharmaceuticals, Inc. · IONS

Compare the available registry edits below. Primary completion is the date data collection ends for the main study measures; it does not tell you when results will be published.

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Primary completion2025-01-24Actual

Enrollment · participants20Actual

Source updated2026-05-08Last update listed in the registry record

Version history

Changes to this trial

Last 12 months

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Posted 2026-02-11 · 1 field changed

Main study measure wording changed

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− Before

Measure: Percentage of Participants ~~with at Least One~~ Treatment-emergent Adverse ~~Event~~ (~~TEAE~~), ~~Graded~~ by ~~Severity~~

+ After

Measure: Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) Measure: Percentage of Participants With Clinically Meaningful Changes in Clinical Laboratory Assessments Measure: Percentage of Participants With Clinically Meaningful Changes in Vital Signs Measure: Percentage of Participants With Clinically Meaningful Changes in Electrocardiograms (ECGs) Parameters Measure: Percentage of Participants Who Received At Least One Concomitant Medication

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Study details

Conditions studied

Hereditary Angioedema

Treatments or interventions studied

DonidalorsenDrugAlso known as ISIS 721744, IONIS-PKK-LRx

Primary outcomes · main study measures

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measure: Percentage of Participants With Treatment-emergent Adverse Events (TEAEs); description: An adverse event (AE) was defined as any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not the AE is considered related to the medicinal (investigational) product. TEAEs were defined as those AEs that either started or worsened in severity on or after the date/time of first administration of study drug in this OLE Study. TEAEs included both serious and non-serious TEAEs.; timeFrame: Up to Week 221; measure: Percentage of Participants With Clinically Meaningful Changes in Clinical Laboratory Assessments; description: Clinical laboratory tests including clinical chemistry, hematology, coagulation, complement, inflammatory, urinalysis were performed. The percentage of participants with clinically meaningful changes were reported. Clinical meaningfulness was determined by the investigator.; timeFrame: Up to Week 221; measure: Percentage of Participants With Clinically Meaningful Changes in Vital Signs; description: Vital sign measurements including heart rate, respiratory rate, body temperature, systolic and diastolic blood pressure and pulse pressure were assessed. Percentage of participants with clinically meaningful changes in the vital signs were reported. Clinically meaningfulness was determined by the investigator.; timeFrame: Up to Week 221; measure: Percentage of Participants With Clinically Meaningful Changes in Electrocardiograms (ECGs) Parameters; description: The ECG parameters included ventricular rate, PR interval, QRS duration, QTc, QT corrected using the Fridericia's formula (QTcF), QT corrected using the Bazett's formula (QTcB), and overall interpretation. Percentage of participants with clinically meaningful changes in the ECG parameters were reported. Clinical meaningfulness was determined by the investigator.; timeFrame: Up to Week 221; measure: Percentage of Participants Who Received At Least One Concomitant Medication; description: Concomitant medications include medications that participants were exposed to on or after the first dose of ISIS 721744 in the OLE study. Percentage of participants who received at least one concomitant medication were reported.; timeFrame: Up to Week 221

History coverage

Research sample: selected versions from May 2023 onward plus a preceding baseline where available. Only selected fields were retained; endpoint measures were truncated to 300 characters. This is not complete trial or sponsor history. Daily snapshots begin when collection starts.

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