A clinical study to assess the safety of Lanadelumab to prevent episodes of severe swelling in children

SPRING STUDY: An Open-Label, Multicenter, Phase 3 Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Lanadelumab for Prevention Against Acute Attacks of Hereditary Angioedema (HAE) in Pediatric Subjects 2 to <12 Years of Age - Safety, Pharmacokinetics, and Pharmacodynamics Study of Lanadelumab to Prevent Hereditary Angioedema

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2018-002093-42-DE

Enrollment

Registered

2019-07-10

Start date

2020-02-04

Completion date

Unknown

Last updated

2022-05-02

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Hereditary Angioedema (HAE) MedDRA version: 23.1 Level: PT Classification code 10019860 Term: Hereditary angioedema System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 23.1 Level: LLT Classification code 10075280 Term: Hereditary angioedema attack System Organ Class: 10010331 - Congenital, familial and genetic disorders

Interventions

Trade Name: Takhzyro Product Name: Lanadelumab Product Code: TAK743

SHP643 Pharmaceutical Form: Solution for injection INN or Proposed INN: LANADELUMAB CAS Number: 1426055-14-2 Current Sponsor code: TAK743

SHP643 Other descriptive name: DX-2930 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 150-

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Be a child (male or female) 2 to =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 (AAE), HAE with normal C1-INH, idiopathic angioedema, or recurrent angioedema associated with urticaria. 2. Dosing with an investigational drug or exposure to an investigational device within 4 weeks prior to screening. 3. Be pregnant or breastfeeding. 4. Have initiated androgen treatment (eg, stanozolol, danazol, oxandrolone, methyltestosterone, and testosterone) within 2 weeks prior to entering the observation period. 5. Exposure to angiotensin-converting enzyme (ACE) inhibitors or any estrogen-containing medications with systemic absorption (such as oral contraceptives or hormonal replacement therapy) within 4 weeks prior to screening. 6. Have any active infectious illness or fever defined as an oral temperature >38°C (100.4°F), tympanic >38.5°C (101.3°F), axillary >38°C (100.4°F), or rectal/core >38.5°C (101.3°F) within 24 hours prior to the first dose of study drug in Treatment Period A. 7. Have any HAE attack that is not resolved prior to the first dose of study drug in Treatment Period A. 8. Have any of the following liver function test abnormalities: alanine aminotransferase (ALT) >3x upper limit of normal (ULN), or aspartate aminotransferase (AST) >3x ULNl, or total bilirubin >2x ULN (unless the bilirubin elevation is a result of Gilbert’s syndrome). 9. Have any condition (any surgical or medical condition) that, in the opinion of the investigator or sponsor, may compromise their safety or compliance, preclude the successful conduct of the study, or interfere with interpretation of the results (eg, significant pre-existing illness or other major comorbidity that the investigator considers may confound the interpretation of study results). 10. Subject has a known hypersensitivity to the investigational product or its components.

Design outcomes

Primary

Measure	Time frame
Timepoint(s) of evaluation of this end point: • Overall treatment period (Day 0 [after study drug administration] through Day 364 [Week 52]) • Treatment Period A (Day 0 [after study drug administration] through Day 182 [Week 26]) • Treatment Period B (Day 183 through Day 364 [Week 52]) • Overall presumed steady state period (Day 70 [Week 10] through Day 364 [Week 52]) • Presumed steady state period for Treatment Period A (Day 70 [Week 10] through Day 182 [Week 26]).;Main Objective: The primary objective of the study is to evaluate the safety and pharmacokinetics (PK) of lanadelumab in children (2 to <12 years of age) with HAE.;Secondary Objective: The secondary objectives of the study are: • To evaluate the clinical outcomes of lanadelumab in preventing HAE attacks in children (2 to <12 years of age) with HAE. • To characterize the pharmacodynamics (PD) of lanadelumab in children (2 to <12 years of age) with HAE. • To assess the immunogenicity of chronically administered lanadelumab and its effect on PK, PD, clinical outcomes, and safety. The exploratory objectives are: • To evaluate the effect of lanadelumab on health-related quality of life (HRQoL). • To evaluate the effect of lanadelumab on exploratory biomarker(s) of angioedema disease-state bioactivity.;Primary end point(s): Measures of safety include: • Adverse events including SAEs and AESI. • Clinical laboratory testing (hematology, clinical chemistry, coagulation) • Vital signs including blood pressure, heart rate, body temperature, and respiratory rate. Measures of PK include: • Plasma concentrations of lanadelumab • PK parameters in plasma, by age group, will be determined by a modelling and simulation approach and reported separately: o Cmax,ss: Maximum observed concentration at steady state o Cavg,ss: Average concentration over dosing interval at steady state o Ctrough,ss: Predose concentration at steady state o tmax: Time to reach Cmax in plasma o AUCtau,ss: Area under the concentration-time curve ov	—

Measure

Time frame

Timepoint(s) of evaluation of this end point: • Overall treatment period (Day 0 [after study drug administration] through Day 364 [Week 52]) • Treatment Period A (Day 0 [after study drug administration] through Day 182 [Week 26]) • Treatment Period B (Day 183 through Day 364 [Week 52]) • Overall presumed steady state period (Day 70 [Week 10] through Day 364 [Week 52]) • Presumed steady state period for Treatment Period A (Day 70 [Week 10] through Day 182 [Week 26]).;Main Objective: The primary objective of the study is to evaluate the safety and pharmacokinetics (PK) of lanadelumab in children (2 to <12 years of age) with HAE.;Secondary Objective: The secondary objectives of the study are: • To evaluate the clinical outcomes of lanadelumab in preventing HAE attacks in children (2 to <12 years of age) with HAE. • To characterize the pharmacodynamics (PD) of lanadelumab in children (2 to <12 years of age) with HAE. • To assess the immunogenicity of chronically administered lanadelumab and its effect on PK, PD, clinical outcomes, and safety. The exploratory objectives are: • To evaluate the effect of lanadelumab on health-related quality of life (HRQoL). • To evaluate the effect of lanadelumab on exploratory biomarker(s) of angioedema disease-state bioactivity.;Primary end point(s): Measures of safety include: • Adverse events including SAEs and AESI. • Clinical laboratory testing (hematology, clinical chemistry, coagulation) • Vital signs including blood pressure, heart rate, body temperature, and respiratory rate. Measures of PK include: • Plasma concentrations of lanadelumab • PK parameters in plasma, by age group, will be determined by a modelling and simulation approach and reported separately: o Cmax,ss: Maximum observed concentration at steady state o Cavg,ss: Average concentration over dosing interval at steady state o Ctrough,ss: Predose concentration at steady state o tmax: Time to reach Cmax in plasma o AUCtau,ss: Area under the concentration-time curve ov

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Secondary

Measure	Time frame
Secondary end point(s): • Normalized number of investigator-confirmed HAE attacks. • Time to the first attack, ie, duration that a subject is attack-free until their first attack. • Normalized number of investigator-confirmed HAE attacks requiring acute therapy use. • Normalized number of moderate or severe investigator-confirmed HAE attacks. • Normalized number of high morbidity investigator-confirmed HAE attacks. • Characteristics of investigator-confirmed HAE attacks, including duration, severity, attack location, and rescue medication use. • Achievement of attack-free status. • Plasma kallikrein activity (as measured by cHMWK level).;Timepoint(s) of evaluation of this end point: • Overall treatment period (Day 0 [after study drug administration] through Day 364 [Week 52]) • Treatment Period A (Day 0 [after study drug administration] through Day 182 [Week 26]) • Treatment Period B (Day 183 through Day 364 [Week 52]) • Overall presumed steady state period (Day 70 [Week 10] through Day 364 [Week 52]) • Presumed steady state period for Treatment Period A (Day 70 [Week 10] through Day 182 [Week 26]).	—

Measure

Time frame

Secondary end point(s): • Normalized number of investigator-confirmed HAE attacks. • Time to the first attack, ie, duration that a subject is attack-free until their first attack. • Normalized number of investigator-confirmed HAE attacks requiring acute therapy use. • Normalized number of moderate or severe investigator-confirmed HAE attacks. • Normalized number of high morbidity investigator-confirmed HAE attacks. • Characteristics of investigator-confirmed HAE attacks, including duration, severity, attack location, and rescue medication use. • Achievement of attack-free status. • Plasma kallikrein activity (as measured by cHMWK level).;Timepoint(s) of evaluation of this end point: • Overall treatment period (Day 0 [after study drug administration] through Day 364 [Week 52]) • Treatment Period A (Day 0 [after study drug administration] through Day 182 [Week 26]) • Treatment Period B (Day 183 through Day 364 [Week 52]) • Overall presumed steady state period (Day 70 [Week 10] through Day 364 [Week 52]) • Presumed steady state period for Treatment Period A (Day 70 [Week 10] through Day 182 [Week 26]).

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Countries

Canada, Germany, Hungary, Spain, United States

Contacts

Public ContactRobert Bradley

Takeda Pharmaceutical Company Limited

robert.bradley@takeda.com+17814821638

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026