Donidalorsen Treatment in Children With Hereditary Angioedema

Part of paid clinical trials in Santa Monica, California.

Sponsor
Ionis Pharmaceuticals, Inc.
Study ID
NCT07298447
Phase
PHASE3
Status
Recruiting
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Conditions

  • Hereditary Angioedema (HAE)

Eligibility Criteria

Sex
ALL
Age
2 Years - 11 Years
Healthy Volunteers
Not accepted

Interventions

  • Donidalorsen — DRUG
    Donidalorsen will be administered by subcutaneous (SC) injection.

Study Details

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).

Key Dates

Start date
Apr 21, 2026
Status verified
May 2026
Primary completion
Jun 30, 2029
Completion
Jun 30, 2029

Study Design

Enrollment
20 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • 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.

Primary Outcome Measure

Number of Participants with Treatment Emergent Adverse Events (TEAEs) [ Time Frame: over the period of approximately 17 months ]

Central Contacts

Locations (5)

FacilityCityStateZIPSite coordinators
Ionis Investigative SiteSanta MonicaCalifornia90404-
Ionis Investigative SiteMiamiFlorida33136-
Ionis Investigative SiteSt LouisMissouri63141-
Ionis Investigative SiteCincinnatiOhio45236-
Ionis Investigative SiteHersheyPennsylvania17033-

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