CompletedPhase 2

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

United States20 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 range18 Years

SexALL

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

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 range18 Years

SexALL

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