Phase 3 study for people with Hereditary Angioedema Type 2

Recruiting Phase 3

Sources: ClinicalTrials.gov Checked: 11 October 2026 Not medical advice

This trial at a glance

Status Recruiting
Phase Phase 3
Sponsor Ionis Pharmaceuticals, Inc.
Registry number NCT07298447
Started 21 April 2026 Today 16 October 2026 Estimated completion 1 June 2029

Where this trial is running

Trial sites and the status recorded for each
SiteCityState or countryStatus at this site
Ionis Investigative Site South Brisbane QLD Recruiting
Ionis Investigative Site Clayton VIC Recruiting

voxsanity.com.au · trial sites from public registries · registry record synced 11 October 2026 · not medical advice

Who can join

Age 2 Years to 11 Years
Sex Anyone, regardless of sex
Healthy volunteers Not accepted
Enrolment target 20

The official eligibility criteria

Registry text, reproduced exactly. These are the words the trial's sponsor published on ClinicalTrials.gov. They have not been edited, shortened or rewritten, and they are written for researchers rather than for patients.

Key Inclusion Criteria:

1. Must be between the ages of 2 and less than 12 years, inclusive, at the time of informed consent and, as applicable, assent.

2. Must weigh at least 9 kg at the time of informed consent and, as applicable, assent.

3. Documented diagnosis of HAE-1/HAE-2 based upon both of the following:

1. Documented clinical history consistent with HAE (SC or mucosal, non-pruritic swelling episodes without accompanying urticaria).

2. Diagnostic testing results that confirm HAE-1/HAE-2: C1-inhibitor (C1-INH) functional level \<50% normal level AND complement factor C4 level below the lower limit of normal (LLN); OR a known pathogenic mutation in the SERPING1 gene.

Key Exclusion Criteria:

1. Must not have any screening laboratory abnormalities or any other clinically significant abnormalities during screening that would render a participant unsuitable for inclusion.

2. Must not have been treated with another investigational drug, biological agent, or device within 1 month of Screening, or 5 half-lives of investigational agent, whichever is longer.

3. Concurrent diagnosis of any other type of recurrent angioedema, including idiopathic angioedema or HAE with normal C1-INH (HAE-nC1-INH or Type III).

Note: Other protocol-specified inclusion/exclusion criteria may apply.

Dates on the record

Started 21 April 2026
Estimated completion 1 June 2029
Registry last updated 2 September 2026
Checked by Voxsanity 11 October 2026

Results, publications and the official record

Sources and dates

  • ClinicalTrials.gov – the record this page reformats
  • ClinicalTrials.gov processing date: 2 September 2026.
  • This page last checked by Voxsanity: 11 October 2026.

Voxsanity reformats data from public registries into this layout. It does not edit, interpret or add to what the registry published.

Not medical advice.

Individual trial records are refreshed on a rolling basis rather than all at once, so a record may be up to several weeks old. Each trial page and trial card shows the date its own record was last synced. Where a record has lagged, its status, sites and enrolment can differ from the current registry state in either direction, so check the registry link on the trial page before acting on them.

Letter for your doctor

A one-page summary of this trial to print or email to your GP. It carries the registry's own record of the trial and its eligibility criteria, and nothing Voxsanity has written about whether it suits you.