Study to Evaluate the Real-world Effectiveness of Lanadelumab in Participants With Hereditary Angioedema (HAE)

IDENTIFIERS
Trial Protocol: SHP643-403
Clinicaltrials.gov: NCT03845400
PURPOSE

This observational, prospective study aims to evaluate the real-world effectiveness of lanadelumab in participants with hereditary angioedema (HAE). Participant-reported attack diaries and patient-reported outcomes (PROs) as well as physician assessments at standard of care (SoC) visits will be used to describe HAE attack rates, treatment patterns, healthcare utilization and participant quality of life.

Recruiting
US
CA
PR

Trial at a glance

What medical condition is being studied?   Hereditary Angioedema (HAE)
Are placebos part of the trial?   No
How many participants are being enrolled?   270
When is the trial being conducted?    - 
How long is participation in the trial?   Participants will be enrolled in this observational study in sites in the United States and Canada as follows: 1. New lanadelumab users: a) participants who have not started lanadelumab at the time of enrollment already or b) already started lanadelumab prior to enrollment and have received <4 lanadelumab doses prior to enrollment date. 2. Prevalent lanadelumab users: participants who already started lanadelumab prior to enrollment and have received at least 4 lanadelumab doses prior to enrollment date. 3. New prophylaxis users: participants who are newly initiating HAE prophylaxis for the first time at enrollment (<4 prophylaxis HAE doses used prior to enrollment date).4. Prevalent prophylaxis users: participants who received at least 4 prophylaxis HAE doses prior to the enrollment date. Participants will be followed for 36 months after the enrollment date. Data from routine participant visits will be collected. Standardized patient-reported outcome (PRO) questionnaires will be collected from the participant via mobile phone application every 3 months. Additionally, participants will need to complete a participant HAE attack diary using the mobile phone application to self-report attacks at each time point an attack occurs.

Key requirements

Sex   
All
Age   
All
Accepts Healthy Volunteers?   
No

Trial Details / Participation Requirements

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Trial Phase:   N/A
Trial Type:   Observational
Trial Arms/Groups or Cohorts:   
Type I or Type II HAE Participants: Participants will be followed for 36 months after the enrollment date up to the time of withdrawal, lost to follow-up, death or end of follow-up (36 months) whichever comes first.
Primary Outcome Measure(s):
Number of Hereditary Angioedema (HAE) Attacks [Time Frame: From enrollment up to 36 months]
Secondary Outcome Measure(s):   
Dose of Lanadelumab Injections [Time Frame: From enrollment up to 36 months]
Frequency of Lanadelumab Injections [Time Frame: From enrollment up to 36 months]
Proportion of Injections Based on the Type of Administration of Lanadelumab [Time Frame: From enrollment up to 36 months]
Time From Diagnosis to Lanadelumab Initiation [Time Frame: From enrollment up to 36 months]
Proportion of Participants who Discontinue Lanadelumab [Time Frame: From enrollment up to 36 months]
Number of Lanadelumab Injections Before Discontinuation per Reason for Injection [Time Frame: From enrollment up to 36 months]
Number of Hospitalizations due to Hereditary Angioedema (HAE) Attack(s) [Time Frame: From enrollment up to 36 months]
Number of Emergency Room (ER) Visits due to Hereditary Angioedema (HAE) Attack(s) [Time Frame: From enrollment up to 36 months]
Number of Physician Visits due to Hereditary Angioedema (HAE) Attack(s) [Time Frame: From enrollment up to 36 months]
Rescue Medications Taken at Time of Hereditary Angioedema (HAE) Attack(s) [Time Frame: From enrollment up to 36 months]
Hereditary Angioedema (HAE) Attack Control Score Before and After Lanadelumab Initiation, as Measured by the Angioedema Control Test (AECT) [Time Frame: From enrollment up to 36 months (every 3 months)]
Angioedema Quality of Life (AE-QoL) Score [Time Frame: From enrollment up to 36 months (every 3 months)]
Work Productivity and Activity Impairment: General Health (WPAI:GH) Score [Time Frame: From enrollment up to 36 months (every 3 months)]
Treatment Satisfaction (TSQM-9) Score [Time Frame: From enrollment up to 36 months (every 3 months)]
Entry Criteria:
Inclusion Criteria: - Voluntarily provide written, signed, and dated (personally or via a legally-authorized representative) informed consent/and assent as applicable to participate in the study. Expression of understanding and agreement by fully informed parent(s) or legal guardian is required to permit the investigator to enroll a child in this study. The choice of the terms parental consent or parental permission in different regions may reflect local legal/regulatory and ethical considerations. - Diagnosis of HAE Type I or Type II. - Ability to use a mobile device for data collection in the study. Exclusion Criteria: - Participation in any interventional clinical trial at the time of enrollment. - Unable to provide written, signed, and dated informed consent/assent. - Investigator believes that the participant is not a suitable candidate for the study.

Trial Locations

Any
Recruiting
Active, not recruiting
Completed
Not yet recruiting
Any
RESULTS
Clinical Research Center of Alabama

504 Brookwood Blvd #250, Birmingham, AL 35209, USA

Recruiting
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Medical Research of Arizona

7514 E Monterey Way, Scottsdale, AZ 85251, USA

Recruiting
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AIRE Medical of Los Angeles

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

Recruiting
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Asthma and Allergy Associates, PC

2709 N Tejon St, Colorado Springs, CO 80907, USA

Recruiting
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University of Kansas Medical Center Research Institute, Inc.

3901 Rainbow Blvd, Kansas City, KS 66160, USA

Recruiting
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Institute for Asthma & Allergy - Chevy Chase

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

Recruiting
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Massachusetts General Hospital

55 Fruit St, Boston, MA 02114, USA

Recruiting
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University of Michigan

500 S State St, Ann Arbor, MI 48109, USA

Recruiting
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Washington University

4559 Scott Ave, St. Louis, MO 63110, USA

Recruiting
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Jay M Kashkin, MD Allergy, Asthma and Immunology

23-00 NJ-208, Fair Lawn, NJ 07410, USA

Recruiting
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The Mount Sinai Hospital

1468 Madison Ave, New York, NY 10029, USA

Recruiting
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Clinical Research of Charlotte

8045 Providence Rd #300, Charlotte, NC 28277, USA

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Portland Clinical Research/AAIM Care

8740 SE Sunnybrook Blvd, Clackamas, OR 97015, USA

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AARA Research Center

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

Recruiting
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Clinique Specialisée en Allergie de la Capitale

2600 Boulevard Laurier Bureau 880, Québec, QC G1V 4W1, Canada

Recruiting
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Rafael H Zaragoza-Urdaz

317 Av. Ing. Manuel Domenech, San Juan, 00918, Puerto Rico

Recruiting
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St. Michael's Hospital

30 Bond St, Toronto, ON M5B 1W8, Canada

Will be Recruiting
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CHUM Hôtel-Dieu

3840 Rue Saint-Urbain, Montréal, QC H2W 1T8, Canada

Will be Recruiting
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University of California San Diego

9500 Gilman Dr, La Jolla, CA 92093, USA

Recruitment Complete
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Yale University School of Medicine

15 York St, New Haven, CT 06510, USA

Recruitment Complete
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University of South Florida Asthma, Allergy & Immunology

4503 N Armenia Ave, Tampa, FL 33603, USA

Recruitment Complete
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Riverside Medical Group, Belleville

5 S, Franklin Ave Suite 302, Belleville, NJ 07109, USA

Recruitment Complete
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Duke Asthma, Allergy & Airway Center

1821 Hillandale Rd Suite 25-A, Durham, NC 27705, USA

Recruitment Complete
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Bernstein Clinical Research Center, LLC

8444 Winton Rd, Cincinnati, OH 45231, USA

Recruitment Complete
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Penn State Univ. Milton S. Hershey Medical Center

700 HMC Cres Rd, Hershey, PA 17033, USA

Recruitment Complete
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University of Calgary - Heritage Medical Research Clinic

3310 Hospital Dr NW, Calgary, AB T2N 4N1, Canada

Recruitment Complete
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McMaster University Health Sciences Center

1200 Main St W, Hamilton, ON L8N 3Z5, Canada

Recruitment Complete
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Mid West Immunology Clinic

15700 37th Ave N STE 110, Plymouth, MN 55446, USA

Completed
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Trial Protocol ID: SHP643-403
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