RecruitingPhase 3

Donidalorsen Treatment in Children With Hereditary Angioedema

United States, Italy, Poland20 participantsStarted 2026-07

Plain-language 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).

Who can participate

Age range2 Years – 11 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Must be between the ages of 2 and less than 12 years, inclusive, at the time of informed consent and, as applicable, assent.
✓. Must weigh at least 9 kg at the time of informed consent and, as applicable, assent.
✓. Documented diagnosis of HAE-1/HAE-2 based upon both of the following:
✓. Documented clinical history consistent with HAE (SC or mucosal, non-pruritic swelling episodes without accompanying urticaria).
✓. 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.

Exclusion criteria

✕. Must not have any screening laboratory abnormalities or any other clinically significant abnormalities during screening that would render a participant unsuitable for inclusion.
✕. 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.
✕. Concurrent diagnosis of any other type of recurrent angioedema, including idiopathic angioedema or HAE with normal C1-INH (HAE-nC1-INH or Type III).

What they're measuring

Number of Participants with Treatment Emergent Adverse Events (TEAEs)

Timeframe: over the period of approximately 17 months

Maximum Plasma Concentration (Cmax) of Donidalorsen

Timeframe: over the period of approximately 17 months

Maximum Time to Reach Cmax (Tmax) of Donidalorsen

Timeframe: over the period of approximately 17 months

Trough Plasma Concentration (Ctrough) of Donidalorsen

Timeframe: over the period of approximately 17 months

Trial details

NCT IDNCT07298447

SponsorIonis Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2029-06

Contact for this trial

Ionis Pharmaceuticals, Inc.

(844) 201-3192 IonisDonidalorsenPediatric@clinicaltrialmedia.com

View on ClinicalTrials.gov More Hereditary Angioedema (HAE) trials

Who can participate

Age range2 Years – 11 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Must be between the ages of 2 and less than 12 years, inclusive, at the time of informed consent and, as applicable, assent.
✓. Must weigh at least 9 kg at the time of informed consent and, as applicable, assent.
✓. Documented diagnosis of HAE-1/HAE-2 based upon both of the following:
✓. Documented clinical history consistent with HAE (SC or mucosal, non-pruritic swelling episodes without accompanying urticaria).
✓. 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.

Exclusion criteria

✕. Must not have any screening laboratory abnormalities or any other clinically significant abnormalities during screening that would render a participant unsuitable for inclusion.
✕. 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.
✕. Concurrent diagnosis of any other type of recurrent angioedema, including idiopathic angioedema or HAE with normal C1-INH (HAE-nC1-INH or Type III).

What they're measuring

Number of Participants with Treatment Emergent Adverse Events (TEAEs)

Timeframe: over the period of approximately 17 months

Maximum Plasma Concentration (Cmax) of Donidalorsen

Timeframe: over the period of approximately 17 months

Maximum Time to Reach Cmax (Tmax) of Donidalorsen

Timeframe: over the period of approximately 17 months

Trough Plasma Concentration (Ctrough) of Donidalorsen

Timeframe: over the period of approximately 17 months