Recruiting
Phase 3

Donidalorsen

Sponsor:

Ionis Pharmaceuticals, Inc.

Code:

NCT07298447

Conditions

Hereditary Angioedema (HAE)

Eligibility Criteria

Sex: All

Age: 2 - 11

Healthy Volunteers: Not accepted

Interventions

Donidalorsen

Study Details

Brief summary:

The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of donidalorsen in pediatric participants with hereditary angioedema (HAE) Type I (HAE-1) or Type II (HAE-2).

Conditions

Hereditary Angioedema (HAE)

Study ID

NCT07298447

Start date

Apr 21, 2026

Status verified date

Sep, 2026

Completion date

Jun, 2029

Anticipated

Primary completion date

Jun, 2029

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 2 - 11

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

20 participants

Anticipated

Allocation

Non randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Donidalorsen: Group 1

Participant weighing 9 kilograms (kg) to less than (<)26 kg, will be administered donidalorsen over the period of one year.

experimental: Donidalorsen: Group 2

Participant weighing greater than or equal to (≥)26 kg to <41 kg, will be administered donidalorsen over the period of one year.

experimental: Donidalorsen: Group 3

Participant weighing ≥41kg, will receive donidalorsen over the period of one year.

Interventions

Donidalorsen

Donidalorsen will be administered by subcutaneous (SC) injection.

Primary outcome measure

  • Number of Participants with Treatment Emergent Adverse Events (TEAEs) [ Time Frame: over the period of approximately 17 months ]
  • Maximum Plasma Concentration (Cmax) of Donidalorsen [ Time Frame: over the period of approximately 17 months ]
  • Maximum Time to Reach Cmax (Tmax) of Donidalorsen [ Time Frame: over the period of approximately 17 months ]
  • Trough Plasma Concentration (Ctrough) of Donidalorsen [ Time Frame: over the period of approximately 17 months ]

Central Contacts and Locations

Central contacts

Locations

Ionis Investigative Site

Recruiting

Santa Monica, California, United States, 90404

Ionis Investigative Site

Recruiting

Miami, Florida, United States, 33136

Ionis Investigative Site

Recruiting

Baton Rouge, Louisiana, United States, 70809

Ionis Investigative Site

Recruiting

St Louis, Missouri, United States, 63141

Ionis Investigative Site

Recruiting

Cincinnati, Ohio, United States, 45236

Ionis Investigative Site

Recruiting

Cleveland, Ohio, United States, 44195

Ionis Investigative Site

Recruiting

Hershey, Pennsylvania, United States, 17033

Ionis Investigative Site

Recruiting

West Allis, Wisconsin, United States, 53214

More Information

Sponsor

Ionis Pharmaceuticals, Inc.

Last update posted

Sep 2, 2026

Last verified

Sep, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by Ionis Pharmaceuticals, Inc. on 2026-09-02.