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

IDENTIFIERS

Trial Protocol: SHP643-301

Clinicaltrials.gov: NCT04070326

EudraCT: 2018-002093-42

PURPOSE

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.

Recruitment Complete

Trial at a glance

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

What is the trial testing? Lanadelumab

How many participants are being enrolled? 21

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

How long is participation in the trial? Participants will stay in the study for up to 72 weeks.

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 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 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: Day 0 (after start of study drug administration) through Day 364 (Week 52)]

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]

Plasma Kallikrein (pKal) Activity [Time Frame: Day 0 (Pre-dose), Day 4, 14, 28, 56, 84, 112, 140, 168, 182 196, 252, 308, 364 and 392]

Number of Participants with Positive Immunogenicity [Time Frame: Day 0 (Pre-dose), Day 28, 84, 140, 182, 196, 252, 308, 364 and 392]

Entry Criteria:

- Boys and girls can take part. - Must be children from 2 to under 12 years old. - Must have been diagnosed with HAE type I or II. - Must have had at least 1 HAE attack within 3 months. Additional entry criteria will be discussed with the study doctor.

Trial Locations

RESULTS

AIRE Medical of Los Angeles

1301 20th St #380, Santa Monica, CA 90404, USA

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Allergy & Asthma Clinical Research

370 N Wiget Ln #210, Walnut Creek, CA 94598, USA

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IMMUNOe Research Centers

6801 S Yosemite St, Centennial, CO 80112, USA

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Institute Asthma and Allergy

2 Wisconsin Cir #250, Chevy Chase, MD 20815, USA

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Hudson-Essex Allergy

5 Franklin Ave #102, Belleville, NJ 07109, USA

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Clinical Research Center of Charlotte

600 Towne Centre Blvd, Pineville, NC 28134, USA

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Bernstein Clinical Research Center

8444 Winton Rd, Cincinnati, OH 45231, USA

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Toledo Institute of Clinical Research Asthma & Allergy Center

7247 Central Ave suite a, Toledo, OH 43617, USA

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AARA Research Center

10100 N Central Expy # 100, Dallas, TX 75231, USA

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Icahn School of Medicine at Mount Sinai

1 Gustave L. Levy Pl, New York, NY 10029, USA

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Yang Medicine

2935 Conroy Rd #110, Ottawa, ON K1G 6C6, Canada

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Charité - Universitätsmedizin Berlin.

Charitépl. 1, 10117 Berlin, Germany

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Klinikum der Johann-Wolfgang Goethe-Universitat.

Theodor-Stern-Kai 7, 60590 Frankfurt am Main, Germany

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Hämophilie Zentrum Rhein Main GmbH

Hessenring 13a/Geb G, 64546 Mörfelden-Walldorf, Germany

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Semmelweis Egyetem.

Budapest, Üllői út 26, 1085 Hungary

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Hospital Universitario La Paz. Paseo de la Castellana

Paseo de la Castellana, 261, 28046 Madrid, Spain

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Washington University School of Medicine

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

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Trial Protocol ID: SHP643-301

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