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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
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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?    - 
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?   
No

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]
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:
Must be a child (male or female) 2 to <12 years of age at the time screening Must have a documented diagnosis of HAE (Type I or II) Must have a historical baseline HAE attack rate of at least 1 attack per 3 months Cannot have a concomitant diagnosis of another form of chronic, recurrent angioedema, such as acquired angioedema (AAE), HAE with normal C1-INH, idiopathic angioedema, or recurrent angioedema associated with urticaria Within 4 weeks prior to screening, - cannot have received an investigational drug or have been exposed to an investigational device - cannot have been exposed to angiotensin-converting enzyme (ACE) inhibitors or any estrogen-containing medications with systemic absorption Within 2 weeks prior to entering the observation period, cannot have initiated androgen treatment Cannot have had certain liver function test abnormalities as defined by the study protocol

Trial Locations

Any
Recruiting
Not yet recruiting
Any
RESULTS
AIRE Medical of Los Angeles

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

Recruiting
2,184 miles (3,514 km)  awayfrom
Toronto, Ontario
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Allergy & Asthma Clinical Research

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

Recruiting
2,240 miles (3,605 km)  awayfrom
Toronto, Ontario
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IMMUNOe Research Centers

6801 S Yosemite St, Centennial, CO 80112, USA

Recruiting
1,341 miles (2,158 km)  awayfrom
Toronto, Ontario
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Institute Asthma and Allergy

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

Recruiting
345 miles (556 km)  awayfrom
Toronto, Ontario
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Hudson-Essex Allergy

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

Recruiting
332 miles (534 km)  awayfrom
Toronto, Ontario
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Clinical Research Center of Charlotte

600 Towne Centre Blvd, Pineville, NC 28134, USA

Recruiting
598 miles (962 km)  awayfrom
Toronto, Ontario
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Bernstein Clinical Research Center

8444 Winton Rd, Cincinnati, OH 45231, USA

Recruiting
405 miles (652 km)  awayfrom
Toronto, Ontario
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Toledo Institute of Clinical Research Asthma & Allergy Center

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

Recruiting
259 miles (417 km)  awayfrom
Toronto, Ontario
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AARA Research Center

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

Recruiting
1,197 miles (1,926 km)  awayfrom
Toronto, Ontario
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Icahn School of Medicine at Mount Sinai

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

Will be Recruiting
341 miles (549 km)  awayfrom
Toronto, Ontario
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University of Alberta Hospital

8440 112 St NW, Edmonton, AB T6G 2B7, Canada

Will be Recruiting
1,682 miles (2,707 km)  awayfrom
Toronto, Ontario
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Yang Medicine

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

Will be Recruiting
220 miles (354 km)  awayfrom
Toronto, Ontario
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Charité - Universitätsmedizin Berlin.

Charitépl. 1, 10117 Berlin, Germany

Will be Recruiting
4,023 miles (6,474 km)  awayfrom
Toronto, Ontario
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Klinikum der Johann-Wolfgang Goethe-Universitat.

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

Will be Recruiting
3,935 miles (6,332 km)  awayfrom
Toronto, Ontario
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Hämophilie Zentrum Rhein Main GmbH

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

Will be Recruiting
3,936 miles (6,334 km)  awayfrom
Toronto, Ontario
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Semmelweis Egyetem.

Budapest, Üllői út 26, 1085 Hungary

Will be Recruiting
4,428 miles (7,126 km)  awayfrom
Toronto, Ontario
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Hospital Universitario La Paz. Paseo de la Castellana

Paseo de la Castellana, 261, 28046 Madrid, Spain

Will be Recruiting
3,749 miles (6,033 km)  awayfrom
Toronto, Ontario
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Washington University School of Medicine

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

663 miles (1,067 km)  awayfrom
Toronto, Ontario
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View all (18)
Trial Protocol ID: SHP643-301
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