Hereditary Angioedema (HAE)
Conditions
Interventions
Trade Name: Takhzyro
Product Name: TAKHZYRO
Product Code: TAK-743, SHP643, DX-2930
Pharmaceutical Form: Solution for injection in vial
INN or Proposed INN: Lanadelumab
CAS Number: 1426055-14-2
Current
Sponsors
Takeda
Eligibility
Sex/Gender
All
Inclusion criteria
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 =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
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 or use any lanadelumab prior to the 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 ) 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).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the safety of repeated subcutaneous (SC) administrations of lanadelumab in Chinese subjects with hereditary angioedema (HAE).;Secondary Objective: - To evaluate the pharmacokinetics (PK) of repeated SC administrations of lanadelumab in Chinese subjects with HAE - To evaluate the pharmacodynamics (PD) of repeated SC administrations of lanadelumab in Chinese subjects with HAE - To evaluate the efficacy of repeated SC administrations of lanadelumab in Chinese subjects with HAE - To evaluate the immunogenicity of repeated SC administration of lanadelumab and the effect on lanadelumab PK, PD, efficacy, and safety;Primary end point(s): - Treatment-emergent adverse events (TEAEs), including serious adverse events (SAEs) and adverse events of special interest (AESIs) - Clinical laboratory testing (hematology, clinical chemistry, coagulation, and urinalysis) - Vital signs including blood pressure (BP), heart rate (HR), body temperature, and respiratory rate (RR) - 12-lead electrocardiogram (ECG) - Physical examination;Timepoint(s) of evaluation of this end point: Up to Day 210 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - Plasma concentrations of lanadelumab - Plasma kallikrein (pKal) activity as measured by cleaved high molecular weight kininogen (cHMWK) level (ie, plasma concentrations of cHMWK) - Number of investigator-confirmed HAE attacks during the efficacy evaluation periods - Number of investigator-confirmed HAE attacks requiring acute treatment during the efficacy evaluation periods - Number of investigator-confirmed moderate or severe HAE attacks during the efficacy evaluation periods - Maximum attack severity during the efficacy evaluations periods - Time to first HAE attack during the efficacy evaluation periods - Achievement of attack-free status during the efficacy evaluation periods - Number and percentage of subjects meeting the criterion of at least a 50%, 70%, 90% and 100% reduction in the investigator-confirmed normalized number of attacks (NNA) per 4 weeks relative to the run-in period NNA, and the number and percentage of subjects achieving NNA <1.0 per 4 weeks - Presence or absence of antidrug antibody (ADA) in plasma (neutralizing or non-neutralizing antibodies in plasma) - - Effect on: Lanadelumab plasma concentrations, cHMWK level, Number of investigator-confirmed HAE attacks during the efficacy evaluation periods;Timepoint(s) of evaluation of this end point: Up to Day 210 | — |
Countries
China
Contacts
Public ContactStudy Director
Takeda
Outcome results
None listed