Donidalorsen Treatment in Children With Hereditary Angioedema

Ionis Pharmaceuticals

Status and phase: Not yet enrolling; Phase 3

Conditions: Hereditary Angioedema (HAE)

Treatments: Drug: Donidalorsen

Study type: Interventional

Funder types: Industry

Identifiers

NCT07298447

2025-523499-22-00 (Other Identifier)

U1111-1326-8613 (Other Identifier)

ISIS 721744-CS8

Details and patient eligibility

About

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).

Full description

This is an open-label study to evaluate the safety, efficacy, and pharmacokinetics (PK) and pharmacodynamics (PD) of donidalorsen in pediatric participants age 2 to less than 12 years old with HAE Type I (HAE-1) or Type II (HAE-2). The study consists of 3 parts: 1) a 3-month Screening Period, 2) a one-year Treatment Period, and 3) a 3-month Post-Treatment Period.

Enrollment: 20 estimated patients

Sex: All

Ages: 2 to 11 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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20 participants in 3 patient groups

Donidalorsen: Group 1

Experimental group

Description:

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

Treatment:

Drug: Donidalorsen

Donidalorsen: Group 2

Experimental group

Description:

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

Treatment:

Drug: Donidalorsen

Donidalorsen: Group 3

Experimental group

Description:

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

Treatment:

Drug: Donidalorsen