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Donidalorsen Treatment in Children With Hereditary Angioedema
NCT07298447 · View on ClinicalTrials.gov ↗
Study 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 Studied
Interventions
- DRUG Donidalorsen
Study Locations (3)
California
- Ionis Investigative Site — Santa Monica
Missouri
- Ionis Investigative Site — St Louis
Ohio
- Ionis Investigative Site — Cincinnati
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 20 participants |
| Start Date | 2026-07 |
| Est. Completion | 2029-06 |
| Phase | Phase 3 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT07298447
The ClinicalTrials.gov registry entry for NCT07298447 describes a study currently listed as recruiting. It is categorized as Phase 3, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 20 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Ionis Pharmaceuticals, which has 55 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Hereditary Angioedema (HAE) appearing as the primary indexed condition, and to 1 intervention — of which Donidalorsen is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT07298447 reports 3 study locations spanning 3 distinct geographic areas — top geographies include California, Missouri, Ohio. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT07298447 about?
NCT07298447 is a clinical study titled "Donidalorsen Treatment in Children With Hereditary Angioedema". 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).
What is the current status of trial NCT07298447?
This trial is currently recruiting. It is a Phase 3 study. The enrollment target is 20 participants. The study started on 2026-07. Estimated completion is 2029-06.
What conditions does trial NCT07298447 study?
This clinical trial studies the following conditions: Hereditary Angioedema (HAE). These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT07298447?
The interventions under investigation include: Donidalorsen (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT07298447?
This trial is sponsored by Ionis Pharmaceuticals, which has 55 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT07298447 being conducted?
This trial has 3 study locations across California, Missouri, Ohio. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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