An Open-Label Study of Donidalorsen in Pediatric Patients Age 2 to Less Than 12 Years Old With Hereditary Angioedema
Status: Recruiting
Location: See all (4) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 3
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
Participation Requirements
Sex: All
Minimum Age: 2
Maximum Age: 11
Healthy Volunteers: f
View:
• Must be between the ages of 2 and less than 12 years, inclusive, at the time of informed consent and, as applicable, assent.
• Must weigh at least 9 kg at the time of informed consent and, as applicable, assent.
• Documented diagnosis of HAE-1/HAE-2 based upon both of the following:
‣ Documented clinical history consistent with HAE (SC or mucosal, non-pruritic swelling episodes without accompanying urticaria).
⁃ 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.
Locations
United States
California
Ionis Investigative Site
RECRUITING
Santa Monica
Missouri
Ionis Investigative Site
RECRUITING
St Louis
Ohio
Ionis Investigative Site
RECRUITING
Cincinnati
Other Locations
Spain
Ionis Investigative Site
RECRUITING
Barcelona
Contact Information
Primary
Ionis Pharmaceuticals, Inc.
IonisDonidalorsenPediatric@clinicaltrialmedia.com
(844) 201-3192
Time Frame
Start Date:2026-07
Estimated Completion Date:2029-06
Participants
Target number of participants:20
Treatments
Experimental: Donidalorsen: Group 1
Participant weighing 9 kilograms (kg) to less than (\<)26 kg, will be administered donidalorsen over the period of one year.
Experimental: 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.
Experimental: Donidalorsen: Group 3
Participant weighing ≥41kg, will receive donidalorsen over the period of one year.