MedDataX Logo

Donidalorsen Treatment in Children With Hereditary Angioedema

NCT ID: NCT07298447

Last Updated: 2025-12-23

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

NOT_YET_RECRUITING

Clinical Phase

PHASE3

Total Enrollment

20 participants

Study Classification

INTERVENTIONAL

Study Start Date

2026-01-31

Study Completion Date

2029-06-30

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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).

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Hereditary Angioedema (HAE)

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Donidalorsen: Group 1

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

Group Type EXPERIMENTAL

Donidalorsen

Intervention Type DRUG

Donidalorsen will be administered by subcutaneous (SC) injection.

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.

Group Type EXPERIMENTAL

Donidalorsen

Intervention Type DRUG

Donidalorsen will be administered by subcutaneous (SC) injection.

Donidalorsen: Group 3

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

Group Type EXPERIMENTAL

Donidalorsen

Intervention Type DRUG

Donidalorsen will be administered by subcutaneous (SC) injection.

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Donidalorsen

Donidalorsen will be administered by subcutaneous (SC) injection.

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

ISIS 721744 IONIS-PKK-LRx

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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.

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).
Minimum Eligible Age

2 Years

Maximum Eligible Age

11 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Ionis Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Central Contacts

Reach out to these primary contacts for questions about participation or study logistics.

Ionis Pharmaceuticals, Inc.

Role: CONTACT

Phone: (844) 201-3192

Email: [email protected]

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

ISIS 721744-CS8

Identifier Type: -

Identifier Source: org_study_id

U1111-1326-8613

Identifier Type: OTHER

Identifier Source: secondary_id

2025-523499-22-00

Identifier Type: OTHER

Identifier Source: secondary_id