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Purpose

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).

Condition

Eligibility

Eligible Ages
Between 2 Years and 11 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

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.

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.

Study Design

Phase
Phase 3
Study Type
Interventional
Allocation
Non-Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Donidalorsen: Group 1 		Participant weighing 9 kilograms (kg) to less than (<)26 kg, will be administered donidalorsen over the period of one year.
  • Drug: Donidalorsen
    Donidalorsen will be administered by subcutaneous (SC) injection.
    Other names:
    • ISIS 721744
    • IONIS-PKK-LRx
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.
  • Drug: Donidalorsen
    Donidalorsen will be administered by subcutaneous (SC) injection.
    Other names:
    • ISIS 721744
    • IONIS-PKK-LRx
Experimental
Donidalorsen: Group 3 		Participant weighing ≥41kg, will receive donidalorsen over the period of one year.
  • Drug: Donidalorsen
    Donidalorsen will be administered by subcutaneous (SC) injection.
    Other names:
    • ISIS 721744
    • IONIS-PKK-LRx

Recruiting Locations

Ionis Investigative Site
Santa Monica, California 90404

Ionis Investigative Site
Miami, Florida 33136

Ionis Investigative Site
Baton Rouge, Louisiana 70809

Ionis Investigative Site
St Louis, Missouri 63141

Ionis Investigative Site
Cincinnati, Ohio 45236

Ionis Investigative Site
Cleveland, Ohio 44195

Ionis Investigative Site
Hershey, Pennsylvania 17033

Ionis Investigative Site
West Allis, Wisconsin 53214

More Details

Status
Recruiting
Sponsor
Ionis Pharmaceuticals, Inc.

Study Contact

Ionis Pharmaceuticals, Inc.
(844) 201-3192
IonisDonidalorsenPediatric@clinicaltrialmedia.com

Detailed Description

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.

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.