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NCT05392114Phase 3ACTIVE NOT RECRUITING

A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)

Study Summary

The purpose of this study is to evaluate the long-term safety and efficacy of donidalorsen in people with HAE and the effects of donidalorsen on the number of HAE attacks and their impact on quality of life (QoL).

Conditions
Hereditary Angioedema
Interventions / Drugs
Donidalorsen
View on ClinicalTrials.gov ↗
Sponsor
IONS
Ionis Pharmaceuticals
Antisense Therapeutics
D50/100
Study Details
Enrollment154 participants
Primary Completion—
Lead SponsorIonis Pharmaceuticals, Inc.
Data Retrieved2026-04-11 04:17:53.139354+00:00
What This Trial Means for Biotech Investors
Trial Phase Context

Phase 3 trials are pivotal studies with large patient populations (hundreds to thousands) designed to prove efficacy and safety for FDA submission. A successful Phase 3 readout is typically the last major hurdle before a New Drug Application (NDA) or BLA filing.

Status & Expected Data

Enrollment is complete. The trial is in its follow-up or data collection phase — a readout may occur on or near the primary completion date.

Enrolled: 154 participants
Sponsor Catalyst Profile

Ionis Pharmaceuticals (IONS) is the sponsoring company for this trial. BiotechSign currently grades this company D (50/100) based on composite catalyst signals across its full pipeline. This trial is one data point in that overall catalyst picture.

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