NCT05139810

OASIS-HAE: A Study to Evaluate the Safety and Efficacy of Donidalorsen (ISIS 721744 or IONIS-PKK-LRx) in Participants With Hereditary Angioedema (HAE)

A Phase 3 Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of ISIS 721744 in Patients With Hereditary Angioedema (HAE)

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 91 (actual)
• Sponsor: Ionis Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study was to evaluate the safety and efficacy of donidalorsen in participants with HAE and effect of donidalorsen on the quality and pattern of HAE attacks and their impact on quality of life (QoL).

Detailed description

This was a Phase 3, multi-center, double-blind, randomized, placebo-controlled study of donidalorsen in 91 participants. Participants were randomly assigned in a 2:1 ratio to Cohort A (donidalorsen or placebo every 4 weeks) or Cohort B (donidalorsen or placebo every 8 weeks), respectively. Within each Cohort, participants were randomized in a 3:1 ratio to receive donidalorsen or matching-placebo. The study included an up to 8-week Screening Period, a 24-week Treatment Period, and an up to 13-week Post-treatment Period.

Conditions

• Hereditary Angioedema

Interventions

Type	Name	Description
DRUG	Donidalorsen	Donidalorsen was administered by SC injection.
DRUG	Placebo	Donidalorsen-matching placebo was administered by SC injection.

Timeline

• Start date: 2021-12-03
• Primary completion: 2023-11-09
• Completion: 2023-11-09
• First posted: 2021-12-01
• Last updated: 2025-03-06
• Results posted: 2025-03-06

Locations

51 sites across 14 countries: United States, Belgium, Bulgaria, Canada, Denmark, France, Germany, Israel, Italy, Netherlands, Poland, Spain, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study