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).
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.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
20
Donidalorsen will be administered by subcutaneous (SC) injection.
Ionis Investigative Site
Santa Monica, California, United States
RECRUITINGIonis Investigative Site
St Louis, Missouri, United States
RECRUITINGIonis Investigative Site
Cincinnati, Ohio, United States
Number of Participants with Treatment Emergent Adverse Events (TEAEs)
Time frame: over the period of approximately 17 months
Maximum Plasma Concentration (Cmax) of Donidalorsen
Time frame: over the period of approximately 17 months
Maximum Time to Reach Cmax (Tmax) of Donidalorsen
Time frame: over the period of approximately 17 months
Trough Plasma Concentration (Ctrough) of Donidalorsen
Time frame: over the period of approximately 17 months
Time-Normalized Number of Investigator-Confirmed HAE Attacks (per Month)
Time frame: over the period of 12 months
Percentage of Investigator-Confirmed HAE Attack-free Participants
Time frame: over the period of 12 months
Time-Normalized Number of Moderate or Severe Investigator-Confirmed HAE Attacks (per Month)
Time frame: over the period of 12 months
Number of Participants with a Clinical Response Defined as a ≥ 50 Percent (%), ≥ 70% and ≥ 90% Reduction from Baseline in Investigator-Confirmed HAE Attack Rate
Time frame: over the period of 12 months
Time-Normalized Number of Investigator-Confirmed HAE Attacks Requiring Rescue Treatment
Time frame: over the period of 12 months
Change From Baseline in Prekallikrein (PKK) Levels in Plasma
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Ionis Investigative Site
Milan, Italy
RECRUITINGIonis Investigative Site
Barcelona, Spain
RECRUITINGTime frame: over the period of 12 months
Percent Change From Baseline in PKK Levels in Plasma
Time frame: over the period of 12 months
Changes in Pediatrics Quality of Life (PedsQL) Scores for Participants
Time frame: over the period of 12 months