Phase 3
N=73
A Study of Long-Term Safety and Efficacy of Lanadelumab for Prevention of Acute Attacks of Non-histaminergic Angioedema With Normal C1-Inhibitor
Angioedema
Bottom Line
View on ClinicalTrials.gov: NCT04444895 ↗Enrolled (actual)
73
Serious AEs
10.7%
Results posted
Jun 2024
Primary outcome: 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 — 55; 1; 61; 1 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Lanadelumab (Drug)
- Age
- Pediatric, Adult, Older Adult · 12+ yrs
- Sex
- All
- Sponsor
- Shire
- Primary completion
- May 2023
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| 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 |
55; 1; 61; 1; 1; 0 | — |
| 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 |
4; 0; 19; 0; 0; 0 | — |
| SECONDARY Number of Investigator-Confirmed Angioedema Attacks During the Treatment Period of Day 0 Through Day 182 |
595; 2 | — |
| SECONDARY Number of Moderate or Severe Angioedema Attacks During the Treatment Period of Day 0 Through Day 182 |
391; 2 | — |
| SECONDARY Number of High-Morbidity Angioedema Attacks During the Treatment Period of Day 0 Through Day 182 |
232; 1 | — |
| SECONDARY Pharmacokinetic (PK) Plasma Concentrations of Lanadelumab |
14419.068; NA; 17021.002; 7047.860; 21138.057; 4944.010 | — |
| SECONDARY Plasma Kallikrein (pKal) Activity |
15.306; NA; 17.586; 21.600; 17.238; 44.700 | — |
| SECONDARY Number of Participants With Neutralizing Antidrug Antibodies (ADA) in Plasma |
2; 2; 3; 4 | — |
| SECONDARY Change From Baseline in Total Angioedema Quality of Life (AE-QoL) Questionnaire Total Score at End of Treatment Period |
-12.73 | — |
| SECONDARY Number of Participants With Any Pause During Injection |
3; 0; 5; 0; 5; 0 | — |
| SECONDARY Number of Participants With Treatment Emergent Adverse Events (TEAEs) Including Adverse Events of Special Interest (AESI) and Serious Adverse Events (SAEs) in Participants Who Switched Dosing Regimen |
2; 1; 0; 1; 0; 0 | — |
| SECONDARY Number of Investigator-Confirmed Angioedema Attacks During the Treatment Period of Day 0 Through Day 182 in Participants Who Switched Dosing Regimen |
0; 2 | — |
| SECONDARY Number of Moderate or Severe Angioedema Attacks During the Treatment Period of Day 0 Through Day 182 in Participants Who Switched Dosing Regimen |
0; 2 | — |
| SECONDARY Number of High-Morbidity Angioedema Attacks During the Treatment Period of Day 0 Through Day 182 in Participants Who Switched Dosing Regimen |
0; 1 | — |
Summary
The purpose of this study is to evaluate the long-term safety and efficacy of repeated subcutaneous (SC) administration of lanadelumab in adolescents and adults with non-histaminergic angioedema with normal C1-inhibitor who completed study SHP643-303 (NCT04206605).
Eligibility Criteria
Inclusion Criteria
- Males and females, 12 years of age and older diagnosed with non-histaminergic normal C1-INH angioedema at the time of enrollment into the antecedent Study SHP643-303 (NCT04206605).
- Participants must have completed the treatment period (through Visit 26/Day 182) of Study SHP643-303 (NCT04206605) without reporting a clinically significant TEAE that would preclude subsequent exposure to lanadelumab.
- Agree to adhere to the protocol-defined schedule of treatments, assessments, and procedures.
- Males, or non-pregnant, non-lactating females who are of child-bearing potential 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 post-menopausal for at least 12 months.
- The participant (or the participant's parent/legal guardian, if applicable) has provided written informed consent approved by the institutional review board/research ethics board/ethics committee (IRB/REB/EC) at any time prior to study start. If the participant is a minor (i.e. lesser then (<) 18 years of age), have a parent/legal guardian who is informed of the nature of the study provide written informed consent (i.e. permission) for the minor to participate in the study before any study-specific procedures are performed. Assent will be obtained from minor participants.
Exclusion Criteria
- Discontinued from Study SHP643-303 (NCT04206605) after enrollment but before Visit 26 for any reason.
- Presence of important safety concerns identified in Study SHP643-303 (NCT04206605) that would preclude participation in this study.
- Dosing with an investigational product (IP, not including IP defined in antecedent Study SHP643-303 [NCT04206605]) or exposure to an investigational device within 4 weeks prior to Day 0.
- Participants has a known hypersensitivity to the investigational product or its components.
- Have any condition (surgical or medical) 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 (e.g. significant pre-existing illness or other major comorbidities that the investigator considers may confound the interpretation of study results).
Data sourced from ClinicalTrials.gov (NCT04444895). 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.