Trial · NCT04444895
A Study of Long-Term Safety and Efficacy of Lanadelumab for Prevention of Acute Attacks of Non-histaminergic Angioedema With Normal C1-Inhibitor
NCT04444895, Phase 3, Completed, Shire; results first posted 2024-06-17
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study of Long-Term Safety and Efficacy of Lanadelumab for Prevention of Acute Attacks of Non-histaminergic Angioedema With Normal C1-Inhibitor. Trials, record NCT04444895. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04444895
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study of Long-Term Safety and Efficacy of Lanadelumab for Prevention of Acute Attacks of Non-histaminergic Angioedema With Normal C1-Inhibitor
- NCT number
- NCT04444895
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 73 participants, actual
- Lead sponsor
- Shire
- Conditions
- Angioedema
- Interventions
- Lanadelumab
- First posted
- 2020-06-24
- Last updated
- 2024-06-17
- Other registries' IDs
- eudract:2019-004823-20
- Results first posted
- 2024-06-17
Source ClinicalTrials.gov
Registered outcomes14
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Treatment Emergent Adverse Events (TEAEs) Including Adverse Events of Special Interest (AESI) and Serious Adverse Events (SAEs) During Treatment Period | From Day 0 up to Day 182 | TEAE: Any event emerging or manifesting at or after initiation of treatment with investigational product (IP) or medicinal product or any existing event that worsens in either intensity or frequency following exposure to IP or medicinal product including clinically meaningful findings in laboratory safety tests, vital signs, weight, and electrocardiogram (ECG) findings. SAE: Any untoward clinical manifestation of signs, symptoms or outcomes (whether considered related to IP 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 included hypersensitivity reactions, events of disordered coagulation such as bleeding AESI, hypercoagulable AESI. TEAEs were classified and reported as angioedema attack and non-angioedema attack adverse events in this outcome measure. |
| Primary | Number of Participants With Treatment Emergent Adverse Events (TEAEs) Including Adverse Events of Special Interest (AESI) and Serious Adverse Events (SAEs) During Follow-up | From Day 183 up to Day 196 | TEAE: Any event emerging or manifesting at or after initiation of treatment with investigational product (IP) or medicinal product or any existing event that worsens in either intensity or frequency following exposure to IP or medicinal product including clinically meaningful findings in laboratory safety tests, vital signs, weight, and electrocardiogram (ECG) findings. SAE: Any untoward clinical manifestation of signs, symptoms or outcomes (whether considered related to IP 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 included hypersensitivity reactions, events of disordered coagulation such as bleeding AESI, hypercoagulable AESI. TEAEs were classified and reported as angioedema attack and non-angioedema attack adverse events in this outcome measure. |
| Secondary | Number of Investigator-Confirmed Angioedema Attacks During the Treatment Period of Day 0 Through Day 182 | From Day 0 up to Day 182 | An angioedema attack was 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 was assessed. |
| Secondary | Number of Moderate or Severe Angioedema Attacks During the Treatment Period of Day 0 Through Day 182 | From Day 0 up to Day 182 | The overall severity of angioedema attack was 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 was assessed. |
| Secondary | Number of High-Morbidity Angioedema Attacks During the Treatment Period of Day 0 Through Day 182 | From Day 0 up to Day 182 | A high-morbidity angioedema attack was defined as any attack that has at least one of the following characteristics: severe, results in hospitalization (except hospitalization for observation \<24 hours), hemodynamically significant (systolic blood pressure (BP) \<90 millimetres of mercury (mmHg), requires intravenous hydration, or associated with syncope or near-syncope) or laryngeal. |
| Secondary | Pharmacokinetic (PK) Plasma Concentrations of Lanadelumab | Predose on Days 0, 84, and 140 and postdose on Day 182 | The data was partitioned by dosing regimen and reported accordingly to appropriately attribute to each dosing regimen. |
| Secondary | Plasma Kallikrein (pKal) Activity | Predose on Days 0, 84, and 140 and postdose on Day 182 | Plasma kallikrein activity was measured by biomarker cleaved high molecular weight kininogen (cHMWK) with factor XIIa activation level to assess the pharmacodynamics of lanadelumab. The data was partitioned by dosing regimen and reported accordingly to appropriately attribute to each dosing regimen. |
| Secondary | Number of Participants With Neutralizing Antidrug Antibodies (ADA) in Plasma | Predose on Days 0, 84, and 140 and postdose on Day 182 | Number of participants with positive ADA including evaluation of neutralizing antibodies in plasma was assessed. As pre-specified in the statistical analysis plan (SAP), data for this outcome measure was collected and analyzed as a single group irrespective of dosing regimen. |
| Secondary | Change From Baseline in Total Angioedema Quality of Life (AE-QoL) Questionnaire Total Score at End of Treatment Period | Baseline (Day 0) up to end of treatment period (Day 182) | The AE-QoL questionnaire is self-administered validated instrument to assess health related (HR)QoL among participants with recurrent angioedema(including hereditary angioedema\[HAE\]). It consists of 17 disease-specific QOL items, to produce total AE-QoL score \& 4 domain scores(functioning,fatigue/mood,fear/shame,nutrition) each of 17 items had 5-point response scale ranging from 1(Never) to 5(Very Often). It was scored according to developers' guidelines to produce 4 domain scores yielding total score. The raw total score(mean of all item scores) was rescaled using linear transformations into final percentage scores ranging 0-100, based on maximum possible score, where higher score, greater QoL impairment. Negative change from Baseline indicates better QoL. Baseline: Last non-missing value prior to first exposure to study drug(based on date or date/time). As pre-specified in SAP, data for this outcome measure was collected and analyzed as single group irrespective of dosing regimen. |
| Secondary | Number of Participants With Any Pause During Injection | Days 0, 14, 28, 42, 56, 70, 84, 98, 112, 126, 140, 154, and 168 | An injection report was completed by the participant (or parent/caregiver) following each dose administration of lanadelumab injection used during the treatment period and any kind of pause during injection was captured. Categories with at least one participant with event are reported. |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 registered outcomesOutcomes14
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Treatment Emergent Adverse Events (TEAEs) Including Adverse Events of Special Interest (AESI) and Serious Adverse Events (SAEs) During Treatment Period | From Day 0 up to Day 182 | The SFAS included all participants who received any study drug after entering this study (i.e., any exposure to open-label lanadelumab). The data was partitioned by dosing regimen and reported accordingly to appropriately attribute to each dosing regimen. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Treatment Emergent Adverse Events (TEAEs) Including Adverse Events of Special Interest (AESI) and Serious Adverse Events (SAEs) During Follow-up | From Day 183 up to Day 196 | The SFAS included all participants who received any study drug after entering this study (i.e., any exposure to open-label lanadelumab). The data was partitioned by dosing regimen and reported accordingly to appropriately attribute to each dosing regimen. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Investigator-Confirmed Angioedema Attacks During the Treatment Period of Day 0 Through Day 182 | From Day 0 up to Day 182 | The SFAS included all participants who received any study drug after entering this study (i.e., any exposure to open-label lanadelumab). The data was partitioned by dosing regimen and reported accordingly to appropriately attribute to each dosing regimen. | angioedema attacks | NUMBER |
| SECONDARY | Number of Moderate or Severe Angioedema Attacks During the Treatment Period of Day 0 Through Day 182 | From Day 0 up to Day 182 | The SFAS included all participants who received any study drug after entering this study (i.e., any exposure to open-label lanadelumab). The data was partitioned by dosing regimen and reported accordingly to appropriately attribute to each dosing regimen. | angioedema attacks | NUMBER |
| SECONDARY | Number of High-Morbidity Angioedema Attacks During the Treatment Period of Day 0 Through Day 182 | From Day 0 up to Day 182 | The SFAS included all participants who received any study drug after entering this study (i.e., any exposure to open-label lanadelumab). The data was partitioned by dosing regimen and reported accordingly to appropriately attribute to each dosing regimen. | angioedema attacks | NUMBER |
| SECONDARY | Pharmacokinetic (PK) Plasma Concentrations of Lanadelumab | Predose on Days 0, 84, and 140 and postdose on Day 182 | The Pharmacokinetic (PK) Set included all participants in the SFAS who had at least 1 evaluable postdose PK concentration value. Overall number analyzed is the number of participants with data available for analyses. Number analyzed is the number of participants with data available for analysis at the specified time point. | nanograms per milliliter (ng/mL) | MEAN |
| SECONDARY | Plasma Kallikrein (pKal) Activity | Predose on Days 0, 84, and 140 and postdose on Day 182 | The Pharmacodynamic (PD) Set included all participants in the SFAS who had at least 1 evaluable postdose PD concentration value. Overall number analyzed is the number of participants with data available for analyses. Number analyzed is the number of participants with data available for analysis at the specified time point. | percentage of cHMWK | MEAN |
| SECONDARY | Number of Participants With Neutralizing Antidrug Antibodies (ADA) in Plasma | Predose on Days 0, 84, and 140 and postdose on Day 182 | The SFAS included all participants who received any study drug after entering this study (i.e., any exposure to open-label lanadelumab). Overall number analyzed is the number of participants with data available for analyses. Number analyzed is the number of participants with data available for analysis at the specified time point. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline in Total Angioedema Quality of Life (AE-QoL) Questionnaire Total Score at End of Treatment Period | Baseline (Day 0) up to end of treatment period (Day 182) | The SFAS included all participants who received any study drug after entering this study (i.e., any exposure to open-label lanadelumab). Overall number analyzed is the number of participants with data available for analyses. | score on a scale | MEAN |
| SECONDARY | Number of Participants With Any Pause During Injection | Days 0, 14, 28, 42, 56, 70, 84, 98, 112, 126, 140, 154, and 168 | The SFAS included all participants who received any study drug after entering this study (i.e., any exposure to open-label lanadelumab). Number analyzed is the number of participants with data available for analysis at the specified time point. The data was partitioned by dosing regimen and reported accordingly to appropriately attribute to each dosing regimen. | Participants | COUNT_OF_PARTICIPANTS |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 outcomesResults by group95
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) Including Adverse Events of Special Interest (AESI) and Serious Adverse Events (SAEs) During Treatment Period | Lanadelumab 300 mg Q2W | Any TEAEs: Non-Angioedema Attack | 55 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) Including Adverse Events of Special Interest (AESI) and Serious Adverse Events (SAEs) During Treatment Period | Lanadelumab 300 mg Q4W | Any TEAEs: Non-Angioedema Attack | 1 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) Including Adverse Events of Special Interest (AESI) and Serious Adverse Events (SAEs) During Treatment Period | Lanadelumab 300 mg Q2W | Any TEAEs: Angioedema Attack | 61 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) Including Adverse Events of Special Interest (AESI) and Serious Adverse Events (SAEs) During Treatment Period | Lanadelumab 300 mg Q4W | Any TEAEs: Angioedema Attack | 1 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) Including Adverse Events of Special Interest (AESI) and Serious Adverse Events (SAEs) During Treatment Period | Lanadelumab 300 mg Q2W | AESI: Non-Angioedema Attack | 1 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) Including Adverse Events of Special Interest (AESI) and Serious Adverse Events (SAEs) During Treatment Period | Lanadelumab 300 mg Q4W | AESI: Non-Angioedema Attack | 0 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) Including Adverse Events of Special Interest (AESI) and Serious Adverse Events (SAEs) During Treatment Period | Lanadelumab 300 mg Q2W | AESI: Angioedema Attack | 0 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) Including Adverse Events of Special Interest (AESI) and Serious Adverse Events (SAEs) During Treatment Period | Lanadelumab 300 mg Q4W | AESI: Angioedema Attack | 0 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) Including Adverse Events of Special Interest (AESI) and Serious Adverse Events (SAEs) During Treatment Period | Lanadelumab 300 mg Q2W | Any SAEs: Non-Angioedema Attack | 5 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) Including Adverse Events of Special Interest (AESI) and Serious Adverse Events (SAEs) During Treatment Period | Lanadelumab 300 mg Q4W | Any SAEs: Non-Angioedema Attack | 1 | Participants |
1 to 10 of 95Source ClinicalTrials.gov
See all 95 results by groupParticipant flow4
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Lanadelumab 300 mg Q2W | 73 | |
| Overall Study | Reduced-dose Safety AnalysisSet(RD-SFAS) | Lanadelumab 300 mg Q2W | 2 | |
| Overall Study | COMPLETED | Lanadelumab 300 mg Q2W | 64 | |
| Overall Study | NOT COMPLETED | Lanadelumab 300 mg Q2W | 9 |
Source ClinicalTrials.gov
Adverse events42
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Angioedema | Skin and subcutaneous tissue disorders | Lanadelumab 300 mg Every 2 Weeks | 61 | 73 | |
| other | Angioedema | Skin and subcutaneous tissue disorders | Lanadelumab 300 mg Every 4 Weeks | 1 | 2 | |
| other | Arthralgia | Musculoskeletal and connective tissue disorders | Lanadelumab 300 mg Every 2 Weeks | 7 | 73 | |
| other | Arthralgia | Musculoskeletal and connective tissue disorders | Lanadelumab 300 mg Every 4 Weeks | 0 | 2 | |
| other | COVID-19 | Infections and infestations | Lanadelumab 300 mg Every 2 Weeks | 17 | 73 | |
| other | COVID-19 | Infections and infestations | Lanadelumab 300 mg Every 4 Weeks | 0 | 2 | |
| other | Eye swelling | Eye disorders | Lanadelumab 300 mg Every 2 Weeks | 0 | 73 | |
| other | Eye swelling | Eye disorders | Lanadelumab 300 mg Every 4 Weeks | 1 | 2 | |
| other | Fatigue | General disorders | Lanadelumab 300 mg Every 2 Weeks | 1 | 73 | |
| other | Fatigue | General disorders | Lanadelumab 300 mg Every 4 Weeks | 1 | 2 |
1 to 10 of 42Source ClinicalTrials.gov
See all 42 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Lanadelumab 300 mg Every 2 Weeks | 0 | 73 |
| Total, all-cause mortality | Lanadelumab 300 mg Every 4 Weeks | 0 | 2 |
| Total, other adverse events | Lanadelumab 300 mg Every 2 Weeks | 65 | 73 |
| Total, other adverse events | Lanadelumab 300 mg Every 4 Weeks | 1 | 2 |
| Total, serious adverse events | Lanadelumab 300 mg Every 2 Weeks | 7 | 73 |
| Total, serious adverse events | Lanadelumab 300 mg Every 4 Weeks | 1 | 2 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 40469312 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Lanadelumab 300 mg Every 2 Weeks vs Lanadelumab 300 mg Every 4 Weeks: deaths from any cause | Trial comparisons |
| Lanadelumab 300 mg Every 2 Weeks vs Lanadelumab 300 mg Every 4 Weeks: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
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GET https://conductscience.org/api/trials/v1/NCT04444895/results?page=2