Health TrialsbyHealthPanel

Donidalorsen Treatment in Children With Hereditary Angioedema

ConditionHereditary Angioedema (HAE)
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age2-11
SponsorIonis Pharmaceuticals, Inc.

About this trial

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 criteria

This trial does not accept healthy volunteers

Qualifiers

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 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.

Disqualifiers

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).

Trial design

Design model

Parallel

Treatments tested in this trial

  • Donidalorsen

    Drug

    Donidalorsen will be administered by subcutaneous (SC) injection.

Treatment groups

20 Participants
are divided into 3 treatment groups
Group A: Donidalorsen: Group 1Experimental treatment 1 intervention
Group B: Donidalorsen: Group 2Experimental treatment 1 intervention
Group C: Donidalorsen: Group 3Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Number of Participants with Treatment Emergent Adverse Events (TEAEs)

Time frame
over the period of approximately 17 months
2

Maximum Plasma Concentration (Cmax) of Donidalorsen

Time frame
over the period of approximately 17 months
3

Maximum Time to Reach Cmax (Tmax) of Donidalorsen

Time frame
over the period of approximately 17 months
4

Trough Plasma Concentration (Ctrough) of Donidalorsen

Time frame
over the period of approximately 17 months

Secondary outcomes

1

Time-Normalized Number of Investigator-Confirmed HAE Attacks (per Month)

Time frame
over the period of 12 months
2

Percentage of Investigator-Confirmed HAE Attack-free Participants

Time frame
over the period of 12 months
3

Time-Normalized Number of Moderate or Severe Investigator-Confirmed HAE Attacks (per Month)

Time frame
over the period of 12 months
4

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

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Ionis Pharmaceuticals, Inc.

Lead sponsor

Source ClinicalTrials.gov
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