A Safety, Pharmacokinetics (PK), and Pharmacodynamics (PD) Study of Lanadelumab to Prevent Hereditary Angioedema (HAE) Attacks in Pediatric Participants of 2 Years to Less Than (<) 12 Years of Age

IDENTIFIERS

Trial Protocol: SHP643-301

Clinicaltrials.gov: NCT04070326

EudraCT: 2018-002093-42

PURPOSE

This Phase 3, Open-label clinical study conducted in North America and Europe evaluates the safety andpharmacokinetics of lanadelumab in children aged 2 to <12 years with HAE. Participants will be treated within a 52-week period.

Recruitment Complete

Trial at a glance

What medical condition is being studied? Hereditary Angioedema (HAE)

What is the trial testing? Lanadelumab

Are placebos part of the trial? No

How many participants are being enrolled? 20

When is the trial being conducted? Aug 2019 - Dec 2021

How long is participation in the trial? A participant’s maximum duration of participation from screening through the completion of the safety follow-up visit is expected to be approximately 72 weeks, comprising the following: up to 2 weeks screening period, up to 12 weeks baseline observation period, 52 weeks study treatment period (26 weeks for Treatment Period A and 26 weeks for Treatment Period B) and 4 weeks of follow-up period. Participants must discontinue long-term prophylaxis before entering the baseline observation period. Participants who complete the first 26-week treatment period (Treatment Period A) will immediately continue into the second treatment period (Treatment Period B). After completion of Treatment Period B, participants will be followed for an additional 4 weeks. Participants aged 6 - <12 years will receive a total of 27 doses of lanadelumab over the 52-week treatment period; participants aged 2 - <6 years will receive a total of 14 lanadelumab doses over the 52-week treatment period. Except for the first 4 weeks of the 52-week treatment period, in which site visits are scheduled on a weekly basis, participants must return to the site every 4 weeks throughout the treatment period. An additional site visit is scheduled at the end of Treatment Period A (week 26).

Key requirements

Sex

All

Age

2 - 11 Years

Accepts Healthy Volunteers?

Trial Details / Participation Requirements

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Trial phase: Phase 3

Trial Type: Interventional

Trial Arms/Groups or Cohorts:

Lanadelumab: Participants aged 2 years to < 6 years will receive lanadelumab at a dose of 150 milligrams (mg) for every 4 weeks (q4wks) with a total of 14 doses over 52-week treatment period and participants aged 6 years to <12 years will receive lanadelumab at a dose of 150 mg for every 2 weeks (q2wks) with a total of 27 doses over 52-week treatment period.

Intervention: Lanadelumab

Primary Outcome Measure(s):

Number of Participants with Adverse Events Including Serious Adverse Events (SAE) and Adverse Events of Special Interest (AESI) [Time Frame: From start of study treatment up to Follow up (Day 392)]

Plasma Concentrations of Lanadelumab Over the Treatment Period [Time Frame: 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 [Time Frame: Day 0 (Pre-dose), 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 [Time Frame: Day 0 (Pre-dose), Day 4, 14, 28, 56, 84, 112, 140, 168, 182 196, 252, 308, 364 and 392]

Predose Concentration at Steady State (Ctrough,ss) of Lanadelumab in Plasma [Time Frame: Day 0 (Pre-dose), Day 4, 14, 28, 56, 84, 112, 140, 168, 182 196, 252, 308, 364 and 392]

Time to Reach Maximum Observed Concentration (Cmax) (tmax)of Lanadelumab in Plasma [Time Frame: Day 0 (Pre-dose), Day 4, 14, 28, 56, 84, 112, 140, 168, 182 196, 252, 308, 364 and 392]

Area Under the Concentration-Time Curve Over the Dosing Interval at Steady State (AUCtau,ss) of Lanadelumab in Plasma [Time Frame: Day 0 (Pre-dose), Day 4, 14, 28, 56, 84, 112, 140, 168, 182 196, 252, 308, 364 and 392]

Terminal half-life (t1/2) of Lanadelumab in Plasma [Time Frame: Day 0 (Pre-dose), Day 4, 14, 28, 56, 84, 112, 140, 168, 182 196, 252, 308, 364 and 392]

Apparent clearance (CL/F) of Lanadelumab [Time Frame: Day 0 (Pre-dose), Day 4, 14, 28, 56, 84, 112, 140, 168, 182 196, 252, 308, 364 and 392]

Apparent Volume of Distribution (V/F) of Lanadelumab [Time Frame: Day 0 (Pre-dose), Day 4, 14, 28, 56, 84, 112, 140, 168, 182 196, 252, 308, 364 and 392]

Secondary Outcome Measure(s):

Normalized Number of Investigator-Confirmed Hereditary Angioedema (HAE) Attacks During Overall Treatment Period [Time Frame: From start of study treatment up to Follow up (Day 392)]

Normalized Number of Investigator-Confirmed HAE Attacks For Each Efficacy Evaluation Period Other Than the Overall Treatment Period [Time Frame: Day 0 through Day 182, Day 70 through Day 182, Day 183 through Day 364, Day 70 through Day 364]

Time to the First Investigator-Confirmed Hereditary Angioedema (HAE) Attack for Each Evaluation Period [Time Frame: Day 0 through Day 364, Day 0 through Day 182, Day 70 through Day 182, Day 183 through Day 364, Day 70 through Day 364]

Normalized Number of Hereditary Angioedema (HAE) Attacks Requiring Acute Treatment for Each Efficacy Evaluation Period [Time Frame: Day 0 through Day 364, Day 0 through Day 182, Day 70 through Day 182, Day 183 through Day 364, Day 70 through Day 364]

Normalized Number of Moderate or Severe Investigator-Confirmed Hereditary Angioedema (HAE) Attacks for Each Efficacy Evaluation Period [Time Frame: Day 0 through Day 364, Day 0 through Day 182, Day 70 through Day 182, Day 183 through Day 364, Day 70 through Day 364]

Normalized Number of High Morbidity Investigator-Confirmed Hereditary Angioedema (HAE) Attacks for Each Efficacy Evaluation Period [Time Frame: Day 0 through Day 364, Day 0 through Day 182, Day 70 through Day 182, Day 183 through Day 364, Day 70 through Day 364]

Number of Participants with Characteristics of Investigator-Confirmed Hereditary Angioedema (HAE) Attacks for Each Efficacy Evaluation Period [Time Frame: Day 0 through Day 364, Day 0 through Day 182, Day 70 through Day 182, Day 183 through Day 364, Day 70 through Day 364]

Number of Participants with Hereditary Angioedema (HAE) Attack-Free Status for Each Evaluation Period [Time Frame: Day 0 through Day 364, Day 0 through Day 182, Day 70 through Day 182, Day 183 through Day 364, Day 70 through Day 364]

660 S Euclid Ave, St. Louis, MO 63110, USA

663 miles (1,067 km) awayfrom
Toronto, Ontario

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Trial Protocol ID: SHP643-301

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