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A Study of Lanadelumab (SHP643) in Chinese Participants With Hereditary Angioedema (HAE)

Primary Purpose

Hereditary Angioedema (HAE)

Status
Recruiting
Phase
Phase 3
Locations
China
Study Type
Interventional
Intervention
Lanadelumab
Sponsored by
Takeda
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hereditary Angioedema (HAE)

Eligibility Criteria

12 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. Be of Chinese descent, defined as born in China and having Chinese parents and Chinese maternal and paternal grandparents.
  2. The participant is male or female and greater than or equal to (>=) 12 years of age at the time of informed consent.
  3. Documented diagnosis of HAE Type I or Type II based upon all of the following:

    • Documented clinical history consistent with HAE (subcutaneous [SC] or mucosal, nonpruritic swelling episodes without accompanying urticaria).
    • Diagnostic testing results obtained during screening by a laboratory (approved by the sponsor) that confirm HAE Type I or Type II: C1 esterase inhibitor (C1-INH) functional level <40% of the normal level. Participants with functional C1-INH level 40% to 50% of the normal level may be enrolled if they also have a C4 level below the normal range. Participants may begin participating in the run-in period before these diagnostic results are available. Participants may be re-tested if results are incongruent with clinical history or believed by the investigator to be confounded by recent long-term prophylaxis (LTP) use.
    • At least one of the following: Age at reported onset of first angioedema symptoms less than or equal to (<=) 30 years, a family history consistent with HAE Type I or Type II, or C1q within normal range.
  4. Attack rate:

    • At the time of enrollment, participants must experience at least 1 investigator-confirmed HAE attack per 4 weeks during the run-in period.

  5. The participant (or the participant's parent/legal guardian, if applicable) has provided written informed consent approved by the institutional review board (IRB)/ institutional ethical committee (IEC).

    • If the participant is an adult, be informed of the nature of the study and provide written informed consent before any study-specific procedures are performed.

    OR

    • If the participant is a minor (that is <18 years of age), have a parent/legal guardian who is informed of the nature of the study provide written informed consent (that is, permission) for the minor to participate in the study before any study-specific procedures are performed. Assent will be obtained from minor participants.

  6. Males, or non-pregnant, non-lactating females who are fertile and sexually active and who agree to be abstinent or agree to comply with the applicable contraceptive requirements of this protocol for the duration of the study, or females of non-childbearing potential, defined as surgically sterile (status post hysterectomy, bilateral oophorectomy, or bilateral tubal ligation) or postmenopausal for at least 12 months.
  7. Agree to adhere to the protocol-defined schedule of assessments and procedures.

Exclusion Criteria:

  1. Concomitant diagnosis of another form of chronic, recurrent angioedema, such as acquired angioedema, HAE with normal C1 esterase inhibitor (C1-INH) (also known as HAE Type III), idiopathic angioedema, or recurrent angioedema associated with urticaria.
  2. Participation in a prior lanadelumab study.
  3. Dosing with investigational drug or exposure to an investigational device within 4 weeks prior to entering to screening.
  4. Exposure to angiotensin-converting enzyme inhibitors or any estrogen-containing medications with systematic absorption (such as oral contraceptives or hormonal replacement therapy) within 4 weeks prior to screening.
  5. Exposure to androgens (that is, danazol, methyltestosterone, testosterone) within 2 weeks prior to entering the run-in period.
  6. Use of LTP therapy (defined as continued use) for HAE (C1-INH, attenuated androgens, or anti-fibrinolytics) for adult participants within 2 weeks prior to entering the run-in period. Adolescent participants (>=12 to <18 years of age) who are on LTP therapy for HAE are allowed to enter the study.
  7. Use of short-term prophylaxis for HAE 7 days prior to entering the run-in period. Short-term prophylaxis is defined as fresh frozen plasma (FFP), C1-INH, attenuated androgens, or antifibrinolytics used to avoid angioedema complications from medically indicated procedures. Note: Currently, C1-INH therapies are not available in China.
  8. Any of the following liver function abnormalities: alanine aminotransferase (ALT) greater than (>)3* upper limit of normal (ULN), or aspartate aminotransferase (AST) >3* ULN or bilirubin >2* ULN (unless the bilirubin is a result of Gilbert's syndrome).
  9. Pregnancy or breast feeding.
  10. Participant has any condition that in the opinion of the investigator or sponsor, may compromise their safety or compliance, preclude successful conduct of the study, or interfere with interpretation of the results (example, history of substance abuse or dependence, significant pre-existing illnesses or major comorbidity the investigator considers may confound the interpretation of the study results).

Sites / Locations

  • The Second Affiliated Hospital of Guangzhou Medical UniversityRecruiting
  • Tongji Hospital, Tongji Medical College, Huazhong University of Science & TechnologyRecruiting
  • Yantai Yuhuangding HospitalRecruiting
  • Peking Union Medical College HospitalRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Lanadelumab 300 mg

Arm Description

Participants will receive lanadelumab 300 milligram (mg), subcutaneously, once every 2 weeks (Q2W) from Day 0 to Day 182 (26 weeks).

Outcomes

Primary Outcome Measures

Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
TEAE is defined as adverse event (AE) with onset at the time of or following initial dosing with study drug (lanadelumab), or medical conditions present prior to the start of study drug but increasing in severity or relationship at the time of or following the start of treatment, up to the last follow-up visit. 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. Number of participants with TEAEs and SAEs will be reported.
Number of Participants With Adverse Events of Special Interest (AESIs)
Hypersensitivity reactions and events of disordered coagulation will be considered as AESIs. Number of participants with AESIs will be reported.
Number of Participants With Clinically Significant Changes in Clinical Laboratory Parameter
Clinical laboratory parameter includes hematology, clinical chemistry, coagulation, and urinalysis. Number of participants with clinically significant changes in clinical laboratory parameter will be reported.
Number of Participants With Clinically Significant Changes in Vital Sign Parameter
Vital signs includes blood pressure (BP), heart rate (HR), body temperature, and respiratory rate. Number of participants with clinically significant changes in vital sign parameter will be reported.
Number of Participants With Clinically Significant 12-Lead Electrocardiogram (ECG) Values
Number of participants with clinically significant changes in 12-lead ECG values will be reported.
Number of Participants With Clinically Significant Changes in Physical Examination
Number of participants with clinically significant changes in physical examination will be reported.

Secondary Outcome Measures

Plasma Concentrations of Lanadelumab
Plasma concentrations of lanadelumab will be assessed.
Plasma Kallikrein (pKal) Activity
pKal activity will be measured by biomarker cleaved high molecular weight kininogen (cHMWK) level.
Number of Investigator-Confirmed HAE Attacks During the Efficacy Evaluation Periods
An HAE attack will be confirmed by following symptoms or signs consistent with an attack in at least one of the following locations: 1) Peripheral angioedema: cutaneous swelling involving an extremity, the face, neck, torso, and/or genitourinary region; 2) Abdominal angioedema: abdominal pain, with or without abdominal distention, nausea, vomiting, or diarrhea; 3) Laryngeal angioedema: stridor, dyspnea, difficulty speaking, difficulty swallowing, throat tightening, or swelling of the tongue, palate, uvula, or larynx. Efficacy evaluation period will consist of 2 periods: Day 0 (after study drug administration) through Day 182 and presumed steady-state period from Day 70 through Day 182. Number of investigator-confirmed HAE attacks during the efficacy evaluation periods will be reported.
Number of Investigator-Confirmed HAE Attacks Requiring Acute Treatment During the Efficacy Evaluation Periods
An HAE attack will be confirmed by following symptoms or signs consistent with an attack in at least one of the following locations: 1) Peripheral angioedema: cutaneous swelling involving an extremity, the face, neck, torso, and/or genitourinary region; 2) Abdominal angioedema: abdominal pain, with or without abdominal distention, nausea, vomiting, or diarrhea; 3) Laryngeal angioedema: stridor, dyspnea, difficulty speaking, difficulty swallowing, throat tightening, or swelling of the tongue, palate, uvula, or larynx. Efficacy evaluation period will consist of 2 periods: Day 0 (after study drug administration) through Day 182 and presumed steady-state period from Day 70 through Day 182. Number of investigator-confirmed HAE attacks requiring acute treatment during the efficacy evaluation periods will be reported.
Number of Moderate or Severe Investigator-Confirmed HAE Attacks During the Efficacy Evaluation Periods
The severity of the Investigator-Confirmed HAE attacks is defined per the HAE attack assessment and reporting procedures (HAARP) definitions: severe (marked limitation in activity, assistance required), moderate (mild to moderate limitation in activity, some assistance needed). Efficacy evaluation period will consist of 2 periods: Day 0 (after study drug administration) through Day 182 and presumed steady-state period from Day 70 through Day 182. Number of moderate or severe investigator-confirmed HAE attacks during the efficacy evaluation periods will be reported.
Number of Participants with Maximum Attack Severity During the Efficacy Evaluation Periods
Maximum HAE attack severity is the most severe attack reported by the participant. Efficacy evaluation period will consist of 2 periods: Day 0 (after study drug administration) through Day 182 and presumed steady-state period from Day 70 through Day 182. Number of participants with maximum attack severity during the efficacy evaluation periods will be reported.
Time to First HAE Attack for the Efficacy Evaluation Period of Day 0 Through Day 182
The time to the first HAE attack (days) after Day 0 for the efficacy evaluation period of Day 0 through Day 182 will be calculated from the date and time of the first dose of lanadelumab for the efficacy evaluation period (Day 0 through Day 182) to the date and time of the first in HAE attack after the first open-label dose for the efficacy evaluation period of Day 0 through Day 182. The time to the first HAE attack will be summarized using Kaplan-Meier (KM) estimates.
Time to First HAE Attack for the Efficacy Evaluation Period of Day 70 Through Day 182
The time to the first HAE attack (days) after Day 0 for the efficacy evaluation period of Day 70 through Day 182 will be calculated from the date and time of the first dose of lanadelumab for the efficacy evaluation period (Day 70 through Day 182) to the date and time of the first in HAE attack after the first open-label dose for the efficacy evaluation period of Day 70 through Day 182. The time to the first HAE attack will be summarized using KM estimates.
Number of Participants Achieving Attack-Free Status for the Efficacy Evaluation Periods
An HAE attack will be confirmed by following symptoms or signs consistent with an attack in at least one of the following locations: 1) Peripheral angioedema: cutaneous swelling involving an extremity, the face, neck, torso, and/or genitourinary region; 2) Abdominal angioedema: abdominal pain, with or without abdominal distention, nausea, vomiting, or diarrhea; 3) Laryngeal angioedema: stridor, dyspnea, difficulty speaking, difficulty swallowing, throat tightening, or swelling of the tongue, palate, uvula, or larynx. Efficacy evaluation period will consist of 2 periods: Day 0 (after study drug administration) through Day 182 and presumed steady-state period from Day 70 through Day 182. Number of participants achieving attack-free status for the efficacy evaluation periods will be assessed.
Number of Participants Achieving at Least 50 %, 70% and 90% Reduction in the Investigator-Confirmed Normalized Number of Attacks (NNA) per 4 Weeks Relative to the Run-in Period NNA for the Efficacy Evaluation Periods
Run in period will be 4 weeks and may be extended up to 8 weeks to determine their baseline attack rate. The normalized number of investigator-confirmed HAE attacks during efficacy evaluation period will be expressed as a monthly (28 days) HAE attack rate. Efficacy evaluation period will consist of 2 periods: Day 0 (after study drug administration) through Day 182 and presumed steady-state period from Day 70 through Day 182. Number of participants achieving at least 50 percent (%), 70% and 90% reduction in the investigator-confirmed NNA per 4 weeks relative to the run-in period normalized NNA for the efficacy evaluation periods will be assessed.
Number of Participants Achieving NNA Less than (<) 1.0 per 4 Weeks for the Efficacy Evaluation Periods
The normalized number of investigator-confirmed HAE attacks during each efficacy evaluation period will be expressed as a monthly (28 days) HAE attack rate. Efficacy evaluation period will consist of 2 periods: Day 0 (after study drug administration) through Day 182 and presumed steady-state period from Day 70 through Day 182. Number of participants achieving NNA <1.0 per 4 weeks for the efficacy evaluation periods will be assessed.
Number of Participants With Neutralizing or Non-neutralizing Antidrug Antibodies (ADA) in Plasma
Number of participants with neutralizing or non-neutralizing ADA in plasma will be assessed.
Effect of Presence or Absence of Neutralizing or Non-neutralizing Antidrug Antibodies (ADA) in Plasma on Lanadelumab Plasma Concentrations
The effect of presence or absence of neutralizing or non-neutralizing ADA in plasma on the lanadelumab plasma concentrations will be assessed.
Effect of Presence or Absence of Neutralizing or Non-neutralizing Antidrug Antibodies (ADA) in Plasma on cHMWK Levels
The effect of presence or absence of neutralizing or non-neutralizing ADA in plasma on the cHMWK levels will be assessed.
Effect of Presence or Absence of Neutralizing or Non-neutralizing Antidrug Antibodies (ADA) in Plasma on Number of Investigator-confirmed HAE Attacks During the Efficacy Evaluation Periods
Effect of presence or absence of neutralizing or non-neutralizing ADA in plasma on the number of investigator-confirmed HAE attacks during the efficacy evaluation periods will be assessed.

Full Information

First Posted
July 13, 2022
Last Updated
March 20, 2023
Sponsor
Takeda
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1. Study Identification

Unique Protocol Identification Number
NCT05460325
Brief Title
A Study of Lanadelumab (SHP643) in Chinese Participants With Hereditary Angioedema (HAE)
Official Title
A Multi-center, Open-label Study to Evaluate the Safety, Pharmacokinetics, and Efficacy of Lanadelumab (SHP643) in Chinese Subjects With Hereditary Angioedema
Study Type
Interventional

2. Study Status

Record Verification Date
March 2023
Overall Recruitment Status
Recruiting
Study Start Date
June 22, 2022 (Actual)
Primary Completion Date
June 6, 2024 (Anticipated)
Study Completion Date
June 6, 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Takeda

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

Brief Summary
The main aim of this study is to evaluate the safety of lanadelumab in Chinese participants with HAE. Participants will be treated with lanadelumab for 26 weeks.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hereditary Angioedema (HAE)

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
20 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Lanadelumab 300 mg
Arm Type
Experimental
Arm Description
Participants will receive lanadelumab 300 milligram (mg), subcutaneously, once every 2 weeks (Q2W) from Day 0 to Day 182 (26 weeks).
Intervention Type
Drug
Intervention Name(s)
Lanadelumab
Other Intervention Name(s)
SHP643, TAKHZYRO, TAK-743, DX-2930, Lanadelumab Injection
Intervention Description
Lanadelumab subcutaneous injection.
Primary Outcome Measure Information:
Title
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Description
TEAE is defined as adverse event (AE) with onset at the time of or following initial dosing with study drug (lanadelumab), or medical conditions present prior to the start of study drug but increasing in severity or relationship at the time of or following the start of treatment, up to the last follow-up visit. 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. Number of participants with TEAEs and SAEs will be reported.
Time Frame
Up to Day 210
Title
Number of Participants With Adverse Events of Special Interest (AESIs)
Description
Hypersensitivity reactions and events of disordered coagulation will be considered as AESIs. Number of participants with AESIs will be reported.
Time Frame
Up to Day 210
Title
Number of Participants With Clinically Significant Changes in Clinical Laboratory Parameter
Description
Clinical laboratory parameter includes hematology, clinical chemistry, coagulation, and urinalysis. Number of participants with clinically significant changes in clinical laboratory parameter will be reported.
Time Frame
Up to Day 210
Title
Number of Participants With Clinically Significant Changes in Vital Sign Parameter
Description
Vital signs includes blood pressure (BP), heart rate (HR), body temperature, and respiratory rate. Number of participants with clinically significant changes in vital sign parameter will be reported.
Time Frame
Up to Day 210
Title
Number of Participants With Clinically Significant 12-Lead Electrocardiogram (ECG) Values
Description
Number of participants with clinically significant changes in 12-lead ECG values will be reported.
Time Frame
Up to Day 210
Title
Number of Participants With Clinically Significant Changes in Physical Examination
Description
Number of participants with clinically significant changes in physical examination will be reported.
Time Frame
Up to Day 210
Secondary Outcome Measure Information:
Title
Plasma Concentrations of Lanadelumab
Description
Plasma concentrations of lanadelumab will be assessed.
Time Frame
At Days 0 and 210; and pre-dose at Days 14, 56, 98, 140, 182
Title
Plasma Kallikrein (pKal) Activity
Description
pKal activity will be measured by biomarker cleaved high molecular weight kininogen (cHMWK) level.
Time Frame
At Days 0 and 210; and pre-dose at Days 14, 56, 98, 140, 182
Title
Number of Investigator-Confirmed HAE Attacks During the Efficacy Evaluation Periods
Description
An HAE attack will be confirmed by following symptoms or signs consistent with an attack in at least one of the following locations: 1) Peripheral angioedema: cutaneous swelling involving an extremity, the face, neck, torso, and/or genitourinary region; 2) Abdominal angioedema: abdominal pain, with or without abdominal distention, nausea, vomiting, or diarrhea; 3) Laryngeal angioedema: stridor, dyspnea, difficulty speaking, difficulty swallowing, throat tightening, or swelling of the tongue, palate, uvula, or larynx. Efficacy evaluation period will consist of 2 periods: Day 0 (after study drug administration) through Day 182 and presumed steady-state period from Day 70 through Day 182. Number of investigator-confirmed HAE attacks during the efficacy evaluation periods will be reported.
Time Frame
Day 0 through Day 182; Day 70 through Day 182
Title
Number of Investigator-Confirmed HAE Attacks Requiring Acute Treatment During the Efficacy Evaluation Periods
Description
An HAE attack will be confirmed by following symptoms or signs consistent with an attack in at least one of the following locations: 1) Peripheral angioedema: cutaneous swelling involving an extremity, the face, neck, torso, and/or genitourinary region; 2) Abdominal angioedema: abdominal pain, with or without abdominal distention, nausea, vomiting, or diarrhea; 3) Laryngeal angioedema: stridor, dyspnea, difficulty speaking, difficulty swallowing, throat tightening, or swelling of the tongue, palate, uvula, or larynx. Efficacy evaluation period will consist of 2 periods: Day 0 (after study drug administration) through Day 182 and presumed steady-state period from Day 70 through Day 182. Number of investigator-confirmed HAE attacks requiring acute treatment during the efficacy evaluation periods will be reported.
Time Frame
Day 0 through Day 182; Day 70 through Day 182
Title
Number of Moderate or Severe Investigator-Confirmed HAE Attacks During the Efficacy Evaluation Periods
Description
The severity of the Investigator-Confirmed HAE attacks is defined per the HAE attack assessment and reporting procedures (HAARP) definitions: severe (marked limitation in activity, assistance required), moderate (mild to moderate limitation in activity, some assistance needed). Efficacy evaluation period will consist of 2 periods: Day 0 (after study drug administration) through Day 182 and presumed steady-state period from Day 70 through Day 182. Number of moderate or severe investigator-confirmed HAE attacks during the efficacy evaluation periods will be reported.
Time Frame
Day 0 through Day 182; Day 70 through Day 182
Title
Number of Participants with Maximum Attack Severity During the Efficacy Evaluation Periods
Description
Maximum HAE attack severity is the most severe attack reported by the participant. Efficacy evaluation period will consist of 2 periods: Day 0 (after study drug administration) through Day 182 and presumed steady-state period from Day 70 through Day 182. Number of participants with maximum attack severity during the efficacy evaluation periods will be reported.
Time Frame
Day 0 through Day 182; Day 70 through Day 182
Title
Time to First HAE Attack for the Efficacy Evaluation Period of Day 0 Through Day 182
Description
The time to the first HAE attack (days) after Day 0 for the efficacy evaluation period of Day 0 through Day 182 will be calculated from the date and time of the first dose of lanadelumab for the efficacy evaluation period (Day 0 through Day 182) to the date and time of the first in HAE attack after the first open-label dose for the efficacy evaluation period of Day 0 through Day 182. The time to the first HAE attack will be summarized using Kaplan-Meier (KM) estimates.
Time Frame
Day 0 through Day 182
Title
Time to First HAE Attack for the Efficacy Evaluation Period of Day 70 Through Day 182
Description
The time to the first HAE attack (days) after Day 0 for the efficacy evaluation period of Day 70 through Day 182 will be calculated from the date and time of the first dose of lanadelumab for the efficacy evaluation period (Day 70 through Day 182) to the date and time of the first in HAE attack after the first open-label dose for the efficacy evaluation period of Day 70 through Day 182. The time to the first HAE attack will be summarized using KM estimates.
Time Frame
Day 70 through Day 182
Title
Number of Participants Achieving Attack-Free Status for the Efficacy Evaluation Periods
Description
An HAE attack will be confirmed by following symptoms or signs consistent with an attack in at least one of the following locations: 1) Peripheral angioedema: cutaneous swelling involving an extremity, the face, neck, torso, and/or genitourinary region; 2) Abdominal angioedema: abdominal pain, with or without abdominal distention, nausea, vomiting, or diarrhea; 3) Laryngeal angioedema: stridor, dyspnea, difficulty speaking, difficulty swallowing, throat tightening, or swelling of the tongue, palate, uvula, or larynx. Efficacy evaluation period will consist of 2 periods: Day 0 (after study drug administration) through Day 182 and presumed steady-state period from Day 70 through Day 182. Number of participants achieving attack-free status for the efficacy evaluation periods will be assessed.
Time Frame
Day 0 through Day 182; Day 70 through Day 182
Title
Number of Participants Achieving at Least 50 %, 70% and 90% Reduction in the Investigator-Confirmed Normalized Number of Attacks (NNA) per 4 Weeks Relative to the Run-in Period NNA for the Efficacy Evaluation Periods
Description
Run in period will be 4 weeks and may be extended up to 8 weeks to determine their baseline attack rate. The normalized number of investigator-confirmed HAE attacks during efficacy evaluation period will be expressed as a monthly (28 days) HAE attack rate. Efficacy evaluation period will consist of 2 periods: Day 0 (after study drug administration) through Day 182 and presumed steady-state period from Day 70 through Day 182. Number of participants achieving at least 50 percent (%), 70% and 90% reduction in the investigator-confirmed NNA per 4 weeks relative to the run-in period normalized NNA for the efficacy evaluation periods will be assessed.
Time Frame
Day 0 through Day 182; Day 70 through Day 182
Title
Number of Participants Achieving NNA Less than (<) 1.0 per 4 Weeks for the Efficacy Evaluation Periods
Description
The normalized number of investigator-confirmed HAE attacks during each efficacy evaluation period will be expressed as a monthly (28 days) HAE attack rate. Efficacy evaluation period will consist of 2 periods: Day 0 (after study drug administration) through Day 182 and presumed steady-state period from Day 70 through Day 182. Number of participants achieving NNA <1.0 per 4 weeks for the efficacy evaluation periods will be assessed.
Time Frame
Day 0 through Day 182; Day 70 through Day 182
Title
Number of Participants With Neutralizing or Non-neutralizing Antidrug Antibodies (ADA) in Plasma
Description
Number of participants with neutralizing or non-neutralizing ADA in plasma will be assessed.
Time Frame
At Days 0 and 210; and pre-dose at Days 56, 98, 140, 182
Title
Effect of Presence or Absence of Neutralizing or Non-neutralizing Antidrug Antibodies (ADA) in Plasma on Lanadelumab Plasma Concentrations
Description
The effect of presence or absence of neutralizing or non-neutralizing ADA in plasma on the lanadelumab plasma concentrations will be assessed.
Time Frame
At Days 0, 56, 98, 140, 182 and 210
Title
Effect of Presence or Absence of Neutralizing or Non-neutralizing Antidrug Antibodies (ADA) in Plasma on cHMWK Levels
Description
The effect of presence or absence of neutralizing or non-neutralizing ADA in plasma on the cHMWK levels will be assessed.
Time Frame
At Days 0, 56, 98, 140, 182 and 210
Title
Effect of Presence or Absence of Neutralizing or Non-neutralizing Antidrug Antibodies (ADA) in Plasma on Number of Investigator-confirmed HAE Attacks During the Efficacy Evaluation Periods
Description
Effect of presence or absence of neutralizing or non-neutralizing ADA in plasma on the number of investigator-confirmed HAE attacks during the efficacy evaluation periods will be assessed.
Time Frame
Day 0 through Day 182; Day 70 through Day 182

10. Eligibility

Sex
All
Minimum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Be of Chinese descent, defined as born in China and having Chinese parents and Chinese maternal and paternal grandparents. The participant is male or female and greater than or equal to (>=) 12 years of age at the time of informed consent. Documented diagnosis of HAE Type I or Type II based upon all of the following: Documented clinical history consistent with HAE (subcutaneous [SC] or mucosal, nonpruritic swelling episodes without accompanying urticaria). Diagnostic testing results obtained during screening by a laboratory (approved by the sponsor) that confirm HAE Type I or Type II: C1 esterase inhibitor (C1-INH) functional level <40% of the normal level. Participants with functional C1-INH level 40% to 50% of the normal level may be enrolled if they also have a C4 level below the normal range. Participants may begin participating in the run-in period before these diagnostic results are available. Participants may be re-tested if results are incongruent with clinical history or believed by the investigator to be confounded by recent long-term prophylaxis (LTP) use. At least one of the following: Age at reported onset of first angioedema symptoms less than or equal to (<=) 30 years, a family history consistent with HAE Type I or Type II, or C1q within normal range. Attack rate: • At the time of enrollment, participants must experience at least 1 investigator-confirmed HAE attack per 4 weeks during the run-in period. The participant (or the participant's parent/legal guardian, if applicable) has provided written informed consent approved by the institutional review board (IRB)/ institutional ethical committee (IEC). • If the participant is an adult, be informed of the nature of the study and provide written informed consent before any study-specific procedures are performed. OR • If the participant is a minor (that is <18 years of age), have a parent/legal guardian who is informed of the nature of the study provide written informed consent (that is, permission) for the minor to participate in the study before any study-specific procedures are performed. Assent will be obtained from minor participants. Males, or non-pregnant, non-lactating females who are fertile and sexually active and who agree to be abstinent or agree to comply with the applicable contraceptive requirements of this protocol for the duration of the study, or females of non-childbearing potential, defined as surgically sterile (status post hysterectomy, bilateral oophorectomy, or bilateral tubal ligation) or postmenopausal for at least 12 months. Agree to adhere to the protocol-defined schedule of assessments and procedures. Exclusion Criteria: Concomitant diagnosis of another form of chronic, recurrent angioedema, such as acquired angioedema, HAE with normal C1 esterase inhibitor (C1-INH) (also known as HAE Type III), idiopathic angioedema, or recurrent angioedema associated with urticaria. Participation in a prior lanadelumab study or use any lanadelumab prior to the study. Dosing with investigational drug or exposure to an investigational device within 4 weeks prior to entering to screening. Exposure to angiotensin-converting enzyme inhibitors or any estrogen-containing medications with systematic absorption (such as oral contraceptives or hormonal replacement therapy) within 4 weeks prior to screening. Exposure to androgens (that is, danazol, methyltestosterone, testosterone) within 2 weeks prior to entering the run-in period. Use of LTP therapy (defined as continued use) for HAE (C1-INH, attenuated androgens, or anti-fibrinolytics) for adult participants within 2 weeks prior to entering the run-in period. Adolescent participants (>=12 to <18 years of age) who are on LTP therapy for HAE are allowed to enter the study. Use of short-term prophylaxis for HAE 7 days prior to entering the run-in period. Short-term prophylaxis is defined as fresh frozen plasma (FFP), C1-INH, attenuated androgens, or antifibrinolytics used to avoid angioedema complications from medically indicated procedures. Note: Currently, C1-INH therapies are not available in China. Any of the following liver function abnormalities: alanine aminotransferase (ALT) greater than (>) 3* upper limit of normal (ULN), or aspartate aminotransferase (AST) > 3* ULN or bilirubin > 2* ULN (unless the bilirubin is a result of Gilbert's syndrome). Pregnancy or breast feeding. Participant has any condition that in the opinion of the investigator or sponsor, may compromise their safety or compliance, preclude successful conduct of the study, or interfere with interpretation of the results (example, history of substance abuse or dependence, significant pre-existing illnesses or major comorbidity the investigator considers may confound the interpretation of the study results).
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Takeda Contact
Phone
+1-877-825-3327
Email
medinfoUS@takeda.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
S Director
Organizational Affiliation
Takeda
Official's Role
Study Director
Facility Information:
Facility Name
The Second Affiliated Hospital of Guangzhou Medical University
City
Guangzhou
State/Province
Guangdong
ZIP/Postal Code
510260
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
86-18926299848
Email
1102796746@qq.com
First Name & Middle Initial & Last Name & Degree
He Lai
Facility Name
Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology
City
Wuhan
State/Province
Hubei
ZIP/Postal Code
430030
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
86-18986292602
Email
zrf13092@163.com
First Name & Middle Initial & Last Name & Degree
Rongfei Zhu
Facility Name
Yantai Yuhuangding Hospital
City
Yantai
State/Province
Shandong
ZIP/Postal Code
264000
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
86-139 0535 6473
Email
ytchenxm@163.com
First Name & Middle Initial & Last Name & Degree
Xiumei Chen
Facility Name
Peking Union Medical College Hospital
City
Beijing
ZIP/Postal Code
100730
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
86-13426303007
Email
yuxiang_zhi@126.com
First Name & Middle Initial & Last Name & Degree
Yuxiang Zhi

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing Plan Description
Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
IPD Sharing Access Criteria
IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/ For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
IPD Sharing URL
https://vivli.org/ourmember/takeda/

Learn more about this trial

A Study of Lanadelumab (SHP643) in Chinese Participants With Hereditary Angioedema (HAE)

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