CompletedPhase 3

A Study of Lanadelumab to Prevent Hereditary Angioedema (HAE) Attacks in Children

United States21 participantsStarted 2019-08-19

Plain-language summary

The main aims of this study are to learn how lanadelumab moves through a child's body and if the children have any medical problems from lanadelumab. Other aims are to learn if prophylactic treatment with lanadelumab reduces the number and severity of HAE attacks in children, how lanadelumab affects the child's body, and if the children develop antibodies to lanadelumab. The study doctors will treat acute HAE attacks according to their standard practice. Participants will receive lanadelumab for up to 52 weeks. When they start treatment, participants will visit their clinic every week for the first 4 weeks. Then, they will visit their clinic every 4 weeks during treatment.

Who can participate

Age range2 Years – 11 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Documented clinical history consistent with HAE (SC or mucosal, nonpruritic swelling episodes without accompanying urticarial).
✓. Diagnostic testing results obtained during screening from a sponsor- approved central laboratory that confirm C1-INH functional level \< 40 percent (%) of the normal level. Participants with functional C1 esterase inhibitor (C1-INH) level 40-50% of the normal level may be enrolled if they also have a complement4 (C4) level below the normal range. With prior sponsor approval, participants may be retested during the baseline observation period if results are incongruent with clinical history or believed by the investigator to be confounded by recent complement1 (C1) inhibitor use.

What they're measuring

Number of Participants With Adverse Events Including Serious Adverse Events (SAEs) and Adverse Events of Special Interest (AESIs)

Timeframe: Up to approximately 115 weeks

Number of Participants With Clinically Significant Laboratory Assessment Abnormalities

Timeframe: Up to approximately 115 weeks

Number of Participants With Clinically Significant Vital Signs Measurements

Timeframe: Up to approximately 115 weeks

Plasma Concentrations of Lanadelumab Over The Treatment Period

Timeframe: Day 0 (Pre-dose), Day 4, 14, 28, 56, 84, 112, 140, 168, 182 196, 252, 308, 364 and 392

Maximum Observed Concentration at Steady State (Cmax,ss) of Lanadelumab in Plasma

Timeframe: Day 0 (Pre-dose), at any time pre-dose on Day 4, 14, 28, 56, 84, 112, 140, 168, 182 196, 252, 308, 364 and 392

Average Concentration Over Dosing Interval at Steady State (Cavg,ss) of Lanadelumab in Plasma

Timeframe: Day 0 (Pre-dose), at any time pre-dose on Day 4, 14, 28, 56, 84, 112, 140, 168, 182 196, 252, 308, 364 and 392

Minimum Concentration at Steady State (Cmin,ss) of Lanadelumab in Plasma

Trial details

NCT IDNCT04070326

SponsorShire

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2021-10-30

Results submitted2022-04-29

View on ClinicalTrials.gov More Hereditary Angioedema trials

Plain-language summary

Who can participate

Age range2 Years – 11 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Documented clinical history consistent with HAE (SC or mucosal, nonpruritic swelling episodes without accompanying urticarial).
✓. Diagnostic testing results obtained during screening from a sponsor- approved central laboratory that confirm C1-INH functional level \< 40 percent (%) of the normal level. Participants with functional C1 esterase inhibitor (C1-INH) level 40-50% of the normal level may be enrolled if they also have a complement4 (C4) level below the normal range. With prior sponsor approval, participants may be retested during the baseline observation period if results are incongruent with clinical history or believed by the investigator to be confounded by recent complement1 (C1) inhibitor use.

What they're measuring

Number of Participants With Adverse Events Including Serious Adverse Events (SAEs) and Adverse Events of Special Interest (AESIs)

Timeframe: Up to approximately 115 weeks

Number of Participants With Clinically Significant Laboratory Assessment Abnormalities

Timeframe: Up to approximately 115 weeks

Number of Participants With Clinically Significant Vital Signs Measurements

Timeframe: Up to approximately 115 weeks

Plasma Concentrations of Lanadelumab Over The Treatment Period

Timeframe: Day 0 (Pre-dose), Day 4, 14, 28, 56, 84, 112, 140, 168, 182 196, 252, 308, 364 and 392

Maximum Observed Concentration at Steady State (Cmax,ss) of Lanadelumab in Plasma

Timeframe: Day 0 (Pre-dose), at any time pre-dose on Day 4, 14, 28, 56, 84, 112, 140, 168, 182 196, 252, 308, 364 and 392

Average Concentration Over Dosing Interval at Steady State (Cavg,ss) of Lanadelumab in Plasma

Timeframe: Day 0 (Pre-dose), at any time pre-dose on Day 4, 14, 28, 56, 84, 112, 140, 168, 182 196, 252, 308, 364 and 392

Minimum Concentration at Steady State (Cmin,ss) of Lanadelumab in Plasma