A Study of Long-Term Safety and Efficacy of Lanadelumab for Prevention of Acute Attacks of Non-histaminergic Angioedema With Normal C1-Inhibitor
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT04444895|
Recruitment Status : Not yet recruiting
First Posted : June 24, 2020
Last Update Posted : October 12, 2020
|Condition or disease||Intervention/treatment||Phase|
|Angioedema||Drug: Lanadelumab||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||75 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||An Open-Label Study to Evaluate the Long-Term Safety and Efficacy of Lanadelumab for Prevention Against Acute Attacks of Non-histaminergic Angioedema With Normal C1-Inhibitor (C1-INH)|
|Estimated Study Start Date :||December 1, 2020|
|Estimated Primary Completion Date :||June 30, 2023|
|Estimated Study Completion Date :||June 30, 2023|
Rollover participants from SHP643-303 (NCT04206605) will receive 300 milligram (mg) of lanadelumab solution in prefilled syringe subcutaneously (SC) for 26 weeks once every 2 weeks (Q2W) or once every 4 weeks (Q4W) if well controlled during SHP643-303 (NCT04206605) with up to 13 doses.
Rollover participants will receive 300 mg of lanadelumab solution in a PFS SC injection once Q2W or Q4W for 26 consecutive weeks.
- Number of Participants with Treatment Emergent Adverse Events (TEAEs) [ Time Frame: From start of the study up to follow-up (Day 196) ]A TEAE is defined as any event emerging or manifesting at or after the initiation of treatment with an investigational product or medicinal product or any existing event that worsens in either intensity or frequency following exposure to the investigational product or medicinal product. A SAE is any untoward clinical manifestation of signs, symptoms or outcomes (whether considered related to investigational product or not and at any dose: results in death, is life-threatening, requires inpatient hospitalization or prolongation of hospitalization, results in persistent or significant disability/incapacity, congenital abnormality/birth defect, an important medical event. AESI will include hypersensitivity reactions, events of disordered coagulation such as bleeding AESI, hypercoagulable AESI. Number of participants with TEAEs including AESI and SAE will be assessed.
- Number of Investigator-Confirmed Angioedema Attacks During the Treatment Period of Day 0 Through Day 182 [ Time Frame: Day 0 through Day 182 ]An angioedema attack is defined as the symptoms or signs consistent with an attack in at least 1 of the following locations: peripheral angioedema (cutaneous swelling involving an extremity, the face, neck, torso, and/or genitourinary region), abdominal angioedema (abdominal pain, with or without abdominal distention, nausea, vomiting, or diarrhea), laryngeal angioedema (stridor, dyspnea, difficulty speaking, difficulty swallowing, throat tightening, or swelling of the tongue, palate, uvula, or larynx). Number of investigator-confirmed angioedema attacks during the treatment period of Day 0 through Day 182 will be assessed.
- Number of Moderate or Severe Angioedema Attacks During the Treatment Period of Day 0 Through Day 182 [ Time Frame: Day 0 through Day 182 ]The overall severity of angioedema attack will be determined by the site using following definitions: mild (transient or mild discomfort), moderate (mild to moderate limitation in activity), severe (marked limitation in activity). Number of moderate or severe angioedema attacks during the treatment period of Day 0 through Day 182 will be assessed.
- Number of High-Morbidity Angioedema Attacks During the Treatment Period of Day 0 Through Day 182 [ Time Frame: Day 0 through Day 182 ]A high morbidity angioedema attack is defined as any attack that has at least 1 of the following characteristics: severe, results in hospitalization (except hospitalization for observation less than (<) 24 hours), hemodynamically significant (systolic blood pressure less than < 90, requires intravenous (IV) hydration, or associated with syncope or near syncope) or laryngeal. Number of high-morbidity angioedema attacks during the treatment period of Day 0 through Day 182 will be assessed.
- Pharmacokinetic (PK) Plasma Concentrations of Lanadelumab [ Time Frame: Day 0, 84, 140 and 182 ]Pharmacokinetic plasma concentrations of lanadelumab will be assessed.
- Plasma Kallikrein (pKal) Activity [ Time Frame: Day 0, 84, 140 and 182 ]Plasma Kallikrein activity will be measured by biomarker cleaved high molecular weight kininogen (cHMWK ) with factor XIIa activation level to assess pharmacodynamics of lanadelumab.
- Number of Participants With Positive Antidrug Antibodies (ADA) in Plasma [ Time Frame: Day 0, 84, 140 and 182 ]Number of participants with positive ADA including evaluation of neutralizing antibodies in plasma will be assessed.
- Angioedema Quality of life (AE-QoL) Questionnaire [ Time Frame: Day 0, 28, 84, 140 and 182 ]The AE-QoL questionnaire is a self-administered validated instrument to assess health related (HR)QoL among participants with recurrent angioedema (including HAE). The AE-QoL consists of 17 disease-specific quality-of-life items, to produce a total AE-QoL score and 4 domain scores (functioning, fatigue/mood, fear/shame, and nutrition) and each of the 17 items has a five-point response scale ranging from 1 (Never) to 5 (Very Often).
- Lanadelumab Injection Report During the Treatment Period of Day 0 Through Day 168 [ Time Frame: Day 0 through Day 168 ]An injection report will be completed by the participant (or parent/caregiver) following each dose administration of lanadelumab injection used during the treatment period of Day 0 through Day 168 will be assessed.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04444895
|Contact: Takeda Development Center Americas Contact||+1 866 842 5335||ClinicalTransparency@takeda.com|
|United States, Alabama|
|Clinical Research Center of Alabama|
|Birmingham, Alabama, United States, 35209|
|Contact: Site Contact 205-209-4134 Janderson@alabamaallergy.com|
|Principal Investigator: John Anderson|
|United States, Arizona|
|Medical Research of Arizona a division of Allergy, Asthma & Immunology Associates, LTD|
|Scottsdale, Arizona, United States, 85251|
|Contact: Site Contact 480-675-8982 firstname.lastname@example.org|
|Principal Investigator: Michael E Manning|
|United States, California|
|UCSD Angioedema Center|
|San Diego, California, United States, 92122|
|Contact: Site Contact 858-657-5350 email@example.com|
|Principal Investigator: Alexander Kim|
|United States, Colorado|
|Asthma and Allergy Associates, PC|
|Colorado Springs, Colorado, United States, 80907|
|Contact: Site Contact 719-473-8330 firstname.lastname@example.org|
|Principal Investigator: Daniel F Soteres|
|United States, Maryland|
|Institute for Asthma & Allergy, P.C.|
|Chevy Chase, Maryland, United States, 20815|
|Contact: Site Contact 301-986-0670 email@example.com|
|Principal Investigator: Huamin Henry Li|
|United States, Michigan|
|University of Michigan Specialty Allergy Clinic and Food Allergy Clinic|
|Ann Arbor, Michigan, United States, 48106|
|Contact: Site Contact 734-936-5634 firstname.lastname@example.org|
|Principal Investigator: Alan Baptist|
|United States, Missouri|
|Washington University School of Medicine|
|Saint Louis, Missouri, United States, 63414|
|Contact: Site Contact 314-996-8339 email@example.com|
|Principal Investigator: H. James Wedner|
|United States, Ohio|
|Optimed Research, LTD|
|Columbus, Ohio, United States, 43235|
|Contact: Site Contact 614-430-8022 firstname.lastname@example.org|
|Principal Investigator: Donald L McNeil|
|Study Director:||Study Director||Takeda Development Center Americas|