NCT07298447 Donidalorsen Treatment in Children With Hereditary Angioedema
| NCT ID | NCT07298447 |
| Status | Recruiting |
| Phase | Phase 3 |
| Sponsor | Ionis Pharmaceuticals, Inc. |
| Condition | Hereditary Angioedema (HAE) |
| Study Type | INTERVENTIONAL |
| Enrollment | 20 participants |
| Start Date | 2026-04-21 |
| Primary Completion | 2029-06 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 3 trials are large pivotal studies comparing the treatment to current standard of care or placebo. Your participation directly contributes to the evidence needed for regulatory approval.
This trial targets 20 participants in total. It began in 2026-04-21 with a primary completion date of 2029-06.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
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).
Eligibility 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.
Contact & Investigator
Frequently Asked Questions
Who can join the NCT07298447 clinical trial?
This trial is open to participants of all sexes, aged 2 Years or older, up to 11 Years, studying Hereditary Angioedema (HAE). Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT07298447 trial and what does that mean for participants?
Phase 3 trials are large-scale studies comparing the new treatment to existing standards of care or a placebo. They provide the evidence needed for regulatory approval. This trial targets 20 participants.
Is NCT07298447 currently recruiting?
Yes, NCT07298447 is actively recruiting participants. Contact the research team at IonisDonidalorsenPediatric@clinicaltrialmedia.com for enrollment information.
Where is the NCT07298447 trial being conducted?
This trial is being conducted at Santa Monica, United States, Miami, United States, St Louis, United States, Cincinnati, United States and 6 additional locations.
Who is sponsoring the NCT07298447 clinical trial?
NCT07298447 is sponsored by Ionis Pharmaceuticals, Inc.. The trial plans to enroll 20 participants.