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Recruiting NCT07298447

Donidalorsen Treatment in Children With Hereditary Angioedema

Phase III Interventional Hereditary Angioedema (HAE)

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Donidalorsen.
Who it may be relevant to
Registry conditions: Hereditary Angioedema (HAE). Basic parameters: 2 years — 11 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States, Italy, Poland, Spain
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

An Open-Label Study of Donidalorsen in Pediatric Patients Age 2 to Less Than 12 Years Old With Hereditary Angioedema

Overview

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).

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.

Interventions

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

Primary outcome measures

  • 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]
Secondary outcome measures (8)
  • 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 [Time 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]

Eligibility criteria

Inclusion criteria

  • 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.

Exclusion criteria

  • Must not have any screening laboratory abnormalities or any other clinically significant abnormalities during screening that would render a participant unsuitable for inclusion.
  • 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.
  • 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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Non-randomized
Model
Parallel assignment
Masking
Open label
Primary purpose
Treatment

Study locations

United States · 7 centers
  • Ionis Investigative Site — Santa Monica
  • Ionis Investigative Site — Miami
  • Ionis Investigative Site — Baton Rouge
  • Ionis Investigative Site — St Louis
  • Ionis Investigative Site — Cincinnati
  • Ionis Investigative Site — Cleveland
  • Ionis Investigative Site — Hershey
Italy · 4 centers
  • Ionis Investigative Site — Milan
  • Ionis Investigative Site — Padova
  • Ionis Investigative Site — Roma
  • Ionis Investigative Site — San Donato Milanese
Poland · 1 center
  • Ionis Investigative Site — Warsaw
Spain · 1 center
  • Ionis Investigative Site — Barcelona

Identifiers

NCT: NCT07298447 · ISIS 721744-CS8 · U1111-1326-8613 · 2025-523499-22-00

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗