Donidalorsen Expanded Access Program for Patients With Hereditary Angioedema

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NCT06415448

Sponsored by Ionis Pharmaceuticals, Inc. (industry) · IONS — their whole pipeline →.

Phase

—

Status

Approved for marketing

Study type

Expanded access

Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →

Source: ClinicalTrials.gov, retrieved 2026-09-04

Dates

each axis at the precision it was filed, with the registry's own basis

Date

Filed

Basis

What it is

Start

—

When the study began enrolling

Primary completion

—

The date the last participant is measured for the primary outcome — the readout window

Study completion

—

The whole study's end, after follow-up

First posted

2024-05-16

actual

When this record first appeared on the registry

Results posted

—

When the sponsor posted results to the registry

Record updated

2026-09-04

actual

The sponsor's own last edit to this record — every projection above is as current as this date

What it studies

Condition

Hereditary Angioedema

Intervention

Drug: Donidalorsen — also filed as IONIS PKK-LRx, ISIS 721744

IONS · their whole pipeline

IONS's other studiesmost recently updated first

CARDIO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Participants With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM) Phase 3 · Completed
ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet Syndrome Phase 1/2 · Recruiting
A Study to Assess the Safety, Tolerability and Efficacy of ION775 Phase 2 · Recruiting
Donidalorsen Treatment in Children With Hereditary Angioedema Phase 3 · Recruiting
REVEAL: A Phase 3 Study of Obudanersen (ION582) in Angelman Syndrome Phase 3 · Recruiting

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