Donidalorsen for children with hereditary angioedema

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This open-label Phase 3 trial gives donidalorsen to pediatric participants aged 2 to less than 12 years with HAE type I or II and follows them for safety, efficacy, pharmacokinetics, and pharmacodynamics. The protocol includes a 3-month screening period, a one-year treatment period, and a 3-month post-treatment follow-up. Eligibility requires confirmed HAE by low C1‑INH functional level and low C4 (or a known SERPING1 mutation) and a minimum weight of 9 kg. Dosing and monitoring visits occur at Ionis investigational sites in Santa Monica, CA; St. Louis, MO; and Cincinnati, OH.

Why it matters

Who can join: the full rules

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.

Where it's running

• Ionis Investigative Site
  Santa Monica, California, United States Recruiting
• Ionis Investigative Site
  Miami, Florida, United States Recruiting
• Ionis Investigative Site
  Baton Rouge, Louisiana, United States Recruiting
• Ionis Investigative Site
  St Louis, Missouri, United States Recruiting
• Ionis Investigative Site
  Cincinnati, Ohio, United States Recruiting
• Ionis Investigative Site
  Cleveland, Ohio, United States Recruiting

Study contacts

• Study coordinator: Ionis Pharmaceuticals, Inc.
• Email: IonisDonidalorsenPediatric@clinicaltrialmedia.com
• Phone: (844) 201-3192

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Takhzyro use in teenagers and adults with hereditary angioedema in South Korea

  This study looks at how safe and useful Takhzyro is when used in real-world care for teenagers and adults with hereditary angioedema in South Korea.

  Recruiting at 3 sites Takeda · Suwon, Gyeonggi-do and 2 other locations · NCT07445087

• Long-term treatment with ADX-324 for people with hereditary angioedema

  This extension will try additional doses of ADX-324 (onvuzosiran) to see if continuing the drug is safe and helps people with Type I or II hereditary angioedema who finished the earlier Phase 3 trial.

  Recruiting at 3 sites Phase 3 ADARx Pharmaceuticals, Inc. · Litchfield Park, Arizona and 47 other locations · NCT07428499

• C1-Inhibitor Function Test to Diagnose HAE in Algeria

  A test that measures C1-inhibitor function is being tried to see if it can accurately diagnose hereditary angioedema in patients in Algeria.

  Recruiting Takeda · Algiers · NCT07293364

• Oral deucrictibant to prevent and treat hereditary angioedema attacks

  This trial tests whether daily oral deucrictibant XR can prevent angioedema attacks and whether deucrictibant capsules can quickly relieve acute attacks in adults with bradykinin-mediated hereditary angioedema.

  Recruiting Phase 1/2 Institute for Asthma and Allergy · Wheaton, Maryland · NCT07046806

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.