DONIDALORSEN for Hereditary angioedema

Inclusion criteria

• {"criterion_text":"- 3. Must be between the ages of 2 and less than 12 years, inclusive, at the time of informed consent and, as applicable, assent.\n- 4. Must weigh at least 9 kg at the time of informed consent and, as applicable, assent.\n- 6. Documented diagnosis of HAE-1/HAE-2 based upon both of the following: a. Documented clinical history consistent with HAE b. Diagnostic testing results that confirm HAE-1/HAE-2"}

Exclusion criteria

• {"criterion_text":"- 2. Must not have any screening laboratory abnormalities or any other clinically significant abnormalities during screening that would render a participant unsuitable for inclusion\n- 12.\tConcurrent diagnosis of any other type of recurrent angioedema, including idiopathic angioedema or HAE with normal C1-INH (HAE-nC1-INH or Type III).\n- 8. 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."}

Primary endpoints

• {"endpoint_text":"- 1. Incidence and severity of treatment-emergent adverse events (TEAEs)","definition_or_measurement_approach":"Assessment of incidence and severity of treatment-emergent adverse events reported during the study (TEAEs)."}
• {"endpoint_text":"- 2. Plasma pharmacokinetics and concentrations of donidalorsen","definition_or_measurement_approach":"Measurement of plasma pharmacokinetics and concentrations of donidalorsen."}

Secondary endpoints

• {"endpoint_text":"- 1. The time-normalized number of investigator-confirmed HAE attacks (per month) over the period of 12 months.","definition_or_measurement_approach":"Time-normalized monthly rate of investigator-confirmed HAE attacks measured over 12 months."}
• {"endpoint_text":"- 3. The percentage of investigator-confirmed HAE attack-free participants over the period of 12 months.","definition_or_measurement_approach":"Proportion of participants without investigator-confirmed HAE attacks during 12 months."}
• {"endpoint_text":"- 4. The time-normalized number of moderate or severe investigatorconfirmed HAE attacks (per month) over the period of 12 months.","definition_or_measurement_approach":"Time-normalized monthly rate of moderate or severe investigator-confirmed HAE attacks measured over 12 months."}
• {"endpoint_text":"- 5. The number of participants with a clinical response defined as a ≥ 50%, ≥ 70%, or ≥ 90% reduction from Baseline in investigator-confirmed HAE attack rate over the period of 12 months.","definition_or_measurement_approach":"Number of participants achieving ≥50%, ≥70% or ≥90% reduction from Baseline in investigator-confirmed HAE attack rate over 12 months."}
• {"endpoint_text":"- 6. The number of investigator-confirmed HAE attacks requiring rescue treatment (i.e., acute therapy) over the period of 12 months.","definition_or_measurement_approach":"Count of investigator-confirmed HAE attacks requiring rescue/acute therapy over 12 months."}
• {"endpoint_text":"- 7. Change in PKK levels in plasma over the period of 12 months.","definition_or_measurement_approach":"Measurement of change in plasma prekallikrein (PKK) concentrations over 12 months (pharmacodynamic assessment)."}
• {"endpoint_text":"- 8. Changes in Pediatrics Quality of Life (PedsQL) scores for participants over the period of 12 months.","definition_or_measurement_approach":"Assessment of change in PedsQL score from baseline over 12 months."}

Italy

Earliest CTIS Part Ii Submission Date

02-02-2026

Latest Decision Or Authorization Date

25-02-2026

Processing Time Days

23

Number Of Sites

4

Number Of Participants

4

Poland

Earliest CTIS Part Ii Submission Date

02-02-2026

Latest Decision Or Authorization Date

26-02-2026

Processing Time Days

24

Number Of Sites

2

Number Of Participants

2

Spain

Earliest CTIS Part Ii Submission Date

04-02-2026

Latest Decision Or Authorization Date

05-05-2026

Processing Time Days

90

Number Of Sites

2

Number Of Participants

2

Primary sponsor

Full Name

Ionis Pharmaceuticals Inc.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Contract research organisations

Name

Medpace Finland Oy

Responsibilities

sponsorDuties codes: 1,10,12,2,4,6,8

Third parties

• {"country":"United Kingdom","full_name":"Cognizant Worldwide Limited","duties_or_roles":"sponsorDuties codes: 8","organisation_type":"Non-Pharmaceutical company"}
• {"country":"Finland","full_name":"Medpace Finland Oy","duties_or_roles":"sponsorDuties codes: 1,10,12,2,4,6,8","organisation_type":"Pharmaceutical company"}
• {"country":"Netherlands","full_name":"Xerimis B.V.","duties_or_roles":"sponsorDuties codes: 14","organisation_type":"Pharmaceutical company"}