Phase 3
Completed N=12
Efficacy and Safety of Lanadelumab (SHP643) in Japanese Participants With Hereditary Angioedema (HAE)
Hereditary Angioedema (HAE)
Source: ClinicalTrials.gov NCT04180163 ↗
Enrolled (actual)
12
Serious AEs
4.2%
Results posted
Sep 2022
Primary outcomePrimary: Number of Participants Achieving Attack-Free Status for the Efficacy Evaluation Period of Day 0 Through Day 182 — 5 Participants
◆ Published Evidence
No publication linked
No peer-reviewed publication reporting this trial's results has been linked yet. This can indicate results are unpublished — a known publication-bias signal. We re-check periodically.
Summary
The purpose of this phase 3, open-label, multi-center study is to evaluate the safety and efficacy of lanadelumab in Japanese participants with HAE Type I or II.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants Achieving Attack-Free Status for the Efficacy Evaluation Period of Day 0 Through Day 182 |
5 | — |
| SECONDARY Number of Investigator-Confirmed Hereditary Angioedema (HAE) Attacks During Each of the Efficacy Evaluation Periods |
92; 189; 55; 152 | — |
| SECONDARY Number of Investigator-Confirmed Hereditary Angioedema (HAE) Attacks Requiring Acute Treatment During Each of the Efficacy Evaluation Periods |
79; 168; 46; 135 | — |
| SECONDARY Number of Moderate or Severe Investigator-Confirmed Hereditary Angioedema (HAE) Attacks During Each of the Efficacy Evaluation Periods |
72; 153; 41; 122 | — |
| SECONDARY Number of Participants With Maximum Hereditary Angioedema (HAE) Attack Severity During Each of the Efficacy Evaluation Periods |
5; 2; 4; 1; 2; 1 | — |
| SECONDARY Number of High-Morbidity Investigator-Confirmed Hereditary Angioedema (HAE) Attacks During Each of the Efficacy Evaluation Periods |
5; 11; 4; 10 | — |
| SECONDARY Time to First Hereditary Angioedema (HAE) Attack After Day 0 for the Efficacy Evaluation Period |
91.0 | — |
| SECONDARY Time to First Hereditary Angioedema (HAE) Attack After Day 0 for the Efficacy Evaluation Period |
91.0 | — |
| SECONDARY 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 Each of Efficacy Evaluation Periods |
10; 8; 6; 10; 8; 6 | — |
| SECONDARY Number of Participants Achieving Normalized Number of Attacks (NNA) <1.0 Per 4 Weeks, <0.75 Per 4 Weeks, <0.50 Per 4 Weeks and <0.25 Per 4 Weeks for Each of the Efficacy Evaluation Periods |
9; 9; 6; 6; 9; 8 | — |
| SECONDARY Number of Participants Achieving Attack-Free Status for the Efficacy Evaluation Period Day 0 Through Day 364, Day 70 Through Day 182, and Day 70 Through Day 364 |
2; 5; 2 | — |
| SECONDARY Number of Participants Achieving Attack-Free Status for Monthly Increments |
7; 8; 9; 7; 9; 7 | — |
| SECONDARY Number of Participants Achieving Investigator-Confirmed Hereditary Angioedema (HAE) Attack-Free Intervals |
5 | — |
| SECONDARY Percentage of Attack Free Days During Each of the Efficacy Evaluation Periods |
88.53; 89.04; 90.34; 89.85 | — |
| SECONDARY Number of Participants With Treatment Emergent Adverse Events (TEAEs) Including Adverse Events of Special Interest (AESI) and Serious Adverse Events (SAEs) |
10; 8; 9; 8; 0; 3 | — |
| SECONDARY Plasma Concentrations of Lanadelumab |
25.584; 23630.973; 24142.776; 24613.885; 23679.526; 19269.249 | — |
| SECONDARY Change From Baseline in Angioedema Quality of Life (AE-QoL) Questionnaire Total Score |
46.57; 26.35; 28.43; 26.96; 32.60; 21.69 | — |
| SECONDARY Plasma Kallikrein (pKal) Activity |
63.04; 30.88; 32.40; 35.67; 30.31; 21.69 | — |
| SECONDARY Number of Participants With Positive Anti-drug Antibody (ADA) in Plasma |
0; 0; 0; 0; 0; 0 | — |
| SECONDARY Number of Participants With TEAEs Related to Clinical Laboratory Tests |
0; 0; 0; 0; 0; 0 | — |
| SECONDARY Number of Participants With TEAEs Related to Vital Signs |
0; 0; 0; 0; 0; 0 | — |
| SECONDARY Number of Participants With TEAEs Related to Electrocardiogram (ECG) |
0; 0; 0; 0; 0; 0 | — |
Eligibility Criteria
Inclusion Criteria
- Be of Japanese descent, defined as born in Japan and having Japanese parents and Japanese maternal and paternal grandparents.
- The participant is male or female and >= 12 years of age at the time of informed consent.
- Documented diagnosis of HAE (Type I or II) based upon all of the following:
- Documented clinical history consistent with HAE (subcutaneous or mucosal, nonpruritic swelling episodes without accompanying urticaria).
- Diagnostic testing results obtained during screening that confirm HAE Type I or II: C1 inhibitor (C1-INH) functional level 3x upper limit of normal, or aspartate aminotransferase (AST) >3x upper limit of normal or bilirubin >2x upper limit of normal (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 (eg, history of substance abuse, or dependence, significant preexisting illnesses or major comorbidity the investigator considers may confound the interpretation of the study results).
- Participant has a known hypersensitivity to the IP or its components.
Data sourced from ClinicalTrials.gov (NCT04180163). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.