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? 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
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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University of Alberta Hospital
8440 112 St NW, Edmonton, AB T6G 2B7, Canada
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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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View all (18)
Trial Protocol ID: SHP643-301
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