NCT04307381

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

An Open-Label Extension Study of ISIS 721744 in Patients With Hereditary Angioedema

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 20 (actual)
• Sponsor: Ionis Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is 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).

Detailed description

This is an open-label extension study of donidalorsen in up to 24 participants with HAE. The length of participation in the study is approximately 68 weeks, which includes an up to 4-week qualification period, a 52-week treatment period, and a 12-week post-treatment period. Following the Week 53 treatment period visit, participants will receive donidalorsen in an extended treatment period for up to an additional 156 weeks. Participants taking part in the extended treatment period will enter the 12-week post-treatment period after completion of, or early termination from, the extended treatment period. This study was extended to allow participants to receive donidalorsen for an additional 156 weeks following the initial 53-week treatment period.

Conditions

• Hereditary Angioedema

Interventions

Type	Name	Description
DRUG	Donidalorsen	Donidalorsen administered SC

Timeline

• Start date: 2020-04-01
• Primary completion: 2025-01-24
• Completion: 2025-01-24
• First posted: 2020-03-13
• Last updated: 2025-03-24

Locations

7 sites across 2 countries: United States, Netherlands

Regulatory

• FDA-regulated drug study