Donidalorsen for Hereditary Angioedema

Recruiting in Palo Alto (17 mi)

+13 other locations

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Ionis Pharmaceuticals, Inc.

No Placebo Group

Prior Safety Data

Trial Summary

What is the purpose of this trial?This trial uses donidalorsen, given as an injection under the skin, to help people with hereditary angioedema. The goal is to reduce the frequency and severity of their swelling episodes.

Eligibility Criteria

This trial is for individuals who have hereditary angioedema and completed a previous study (ISIS 721744-CS2) without safety concerns. Participants must be able to commit to a 64-week study, use effective contraception if of childbearing potential, and have access to acute medications for angioedema attacks.

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

I am a male either surgically sterile, abstinent, or using contraception if my partner can bear children.

+2 more

Exclusion Criteria

Have any new condition or worsening of an existing condition or change or anticipated change in medication, which in the opinion of the Investigator would make the participant unsuitable for enrollment, or could interfere with the participant participating in or completing the study

Participant Groups

The trial is testing the extended use of donidalorsen administered subcutaneously in various dosing patterns or frequencies. It aims to assess the long-term safety and effectiveness of this treatment approach in people with hereditary angioedema.

1Treatment groups

Experimental Treatment

Group I: DonidalorsenExperimental Treatment1 Intervention

Participants will be administered donidalorsen SC for up to 53 weeks. Participants will also be administered donidalorsen in the extended treatment period for an additional 156 weeks, up to week 209.

Donidalorsen is already approved in European Union, United States, Canada for the following indications:

🇪🇺 Approved in European Union as Donidalorsen for: