Trial Outcomes & Findings for CSL312_3003 Safety and Pharmacokinetic Study in Subjects 2 to 11 Years of Age With Hereditary Angioedema (NCT NCT05819775)

This study was conducted at 9 sites in the United States, Australia, Canada, Germany, and Israel.

A total of 24 participants were screened, of whom 22 were enrolled and 2 were screen failures.

CSL312_3003 Safety and Pharmacokinetic Study in Subjects 2 to 11 Years of Age With Hereditary Angioedema

The percentage of participants was rounded to one place of decimal.

The TEAE rate per injection was calculated as the number of TEAE/ number of injections. The number of injections was defined as the total injections a participant received during the Safety Evaluation Period under the dosing regimen to which the TEAE was assigned.

The TEAE rate per participant year was calculated as number of TEAEs/ participant years. Participant-years of exposure were calculated as the sum of each participant's exposure duration (in years) under the specified dosing regimen or overall. For the time assigned to a dosing regimen, each study day was counted under the corresponding regimen.

Time-normalized number of HAE attacks per month during treatment was calculated per participant as: \[Number of HAE attacks / Length of participant treatment in days\] \* 30.4375.

Time-normalized number of HAE attacks per year during treatment was calculated per participant as: \[Number of HAE attacks / Length of participant treatment in days\] \* 365.25.

The time-normalized number of HAE attacks per month treated with on-demand treatment were calculated as follows: \[(Number of HAE attacks treated with on - demand treatment during treatment period)/ Length of participant treatment in days\] ∗ 30.4375.

The time-normalized number of HAE attacks per year treated with on-demand treatment were calculated as follows: \[(Number of HAE attacks treated with on - demand treatment during treatment period)/ Length of participant treatment in days\] ∗ 365.25.

Time-normalized number of moderate or severe HAE attacks per month during treatment period was calculated per participant as: \[number of moderate or severe HAE attacks / length of participant treatment in days\] \* 30.4375.

Time-normalized number of moderate or severe HAE attacks per month during treatment period was calculated per participant as: \[number of moderate or severe HAE attacks / length of participant treatment in days\] \* 365.25.

The percentage reduction in the time-normalized number of HAE attacks was calculated within a participant as follows: 100\*\[ 1 - (Time-normalized number of HAE attacks per month during treatment period/Time-normalized number of HAE attacks per month from historical data)\].

A participant was classified as a responder if the percentage reduction in the time-normalized number of HAE attacks under treatment compared to the time-normalized number of HAE attacks documented in the medical records was \>= 50%. Percent Reduction = 100 \* \[1 - (time-normalized number of HAE attacks during corresponding time window / time-normalized number of HAE attacks based on historical data)\]. Here number of participants experiencing at least \>= 50%, \>= 70%, \>= 90%, or equal to 100% (Attack-free) reduction in the time-normalized number of HAE attacks are reported. The number of responders at each reduction category have been reported.

The percentage of participants was rounded to one decimal place.

Severity of AE was assessed by the investigator and categorized as mild, moderate and severe where: Mild: AE that is usually transient and may require only minimal treatment or therapeutic intervention. The event does not generally interfere with usual activities of daily living. Moderate: AE that is usually alleviated with additional specific therapeutic intervention. The event interferes with usual activities of daily living, causing discomfort but poses no significant or permanent risk of harm to the research participant. Severe: AE that interrupts usual activities of daily living, significantly affects clinical status, or may require intensive therapeutic intervention.

Severity of AE was assessed by the investigator and categorized as mild, moderate and severe where: Mild: AE that is usually transient and may require only minimal treatment or therapeutic intervention. The event does not generally interfere with usual activities of daily living. Moderate: AE that is usually alleviated with additional specific therapeutic intervention. The event interferes with usual activities of daily living, causing discomfort but poses no significant or permanent risk of harm to the research participant. Severe: AE that interrupts usual activities of daily living, significantly affects clinical status, or may require intensive therapeutic intervention. The percentage of participants was rounded to one place of decimal.

The percentage of participants was rounded to one place of decimal.

AESI included severe hypersensitivity including anaphylaxis. The AESI reported have been identified by investigators and suggestive events were independently identified for further review with a Standardized MedDRA Query (SMQ).

AESI included severe hypersensitivity including anaphylaxis. The AESI reported have been identified by investigators and suggestive events were independently identified for further review with an SMQ. The percentage of participants was rounded to one place of decimal.

Percent of Baseline at Visit \[i\] = 100 \* (actual value at Visit \[i\] / Baseline value), where Baseline is defined as the most recent, non-missing value before the first IP administration (including unscheduled visits). Here unit of measure is Percent (%) of FXIIa-mediated Kallikrein Activity.

The participant data were rounded to one decimal place.