CompletedPhase 2

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

United States, Netherlands20 participantsStarted 2020-03-31

Plain-language 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).

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)

Timeframe: Up to Week 221

Percentage of Participants With Clinically Meaningful Changes in Clinical Laboratory Assessments

Timeframe: Up to Week 221

Percentage of Participants With Clinically Meaningful Changes in Vital Signs

Timeframe: Up to Week 221

Percentage of Participants With Clinically Meaningful Changes in Electrocardiograms (ECGs) Parameters

Timeframe: Up to Week 221

Percentage of Participants Who Received At Least One Concomitant Medication

Timeframe: Up to Week 221

Trial details

NCT IDNCT04307381

SponsorIonis Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2025-01-24

Results submitted2026-01-24

View on ClinicalTrials.gov More Hereditary Angioedema trials

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

What they're measuring

Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)

Timeframe: Up to Week 221

Percentage of Participants With Clinically Meaningful Changes in Clinical Laboratory Assessments

Timeframe: Up to Week 221

Percentage of Participants With Clinically Meaningful Changes in Vital Signs

Timeframe: Up to Week 221

Percentage of Participants With Clinically Meaningful Changes in Electrocardiograms (ECGs) Parameters

Timeframe: Up to Week 221

Percentage of Participants Who Received At Least One Concomitant Medication

Timeframe: Up to Week 221