Phase 3
N=21
A Study of Lanadelumab to Prevent Hereditary Angioedema (HAE) Attacks in Children
Hereditary Angioedema
Bottom Line
View on ClinicalTrials.gov: NCT04070326 ↗Enrolled (actual)
21
Serious AEs
0.0%
Results posted
May 2022
Primary outcome: Primary: Number of Participants With Adverse Events Including Serious Adverse Events (SAEs) and Adverse Events of Special Interest (AESIs) — 0; 0; 0; 0 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Lanadelumab (Drug)
- Age
- Pediatric · 2+ yrs
- Sex
- All
- Sponsor
- Shire
- Primary completion
- Oct 2021
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Adverse Events Including Serious Adverse Events (SAEs) and Adverse Events of Special Interest (AESIs) |
0; 0; 0; 0 | — |
| PRIMARY Number of Participants With Clinically Significant Laboratory Assessment Abnormalities |
0; 0 | — |
| PRIMARY Number of Participants With Clinically Significant Vital Signs Measurements |
0; 0 | — |
| PRIMARY Plasma Concentrations of Lanadelumab Over The Treatment Period |
0.000; 11.735; 32407.215; 20349.609; 20443.818; 14589.883 | — |
| PRIMARY Maximum Observed Concentration at Steady State (Cmax,ss) of Lanadelumab in Plasma |
37.7; 39.0 | — |
| PRIMARY Average Concentration Over Dosing Interval at Steady State (Cavg,ss) of Lanadelumab in Plasma |
24.6; 33.2 | — |
| PRIMARY Minimum Concentration at Steady State (Cmin,ss) of Lanadelumab in Plasma |
11.1; 24.8 | — |
| PRIMARY Time to Reach Maximum Observed Concentration (Cmax) [Tmax] of Lanadelumab in Plasma |
122; 86.0 | — |
| PRIMARY Area Under the Concentration-Time Curve Over the Dosing Interval at Steady State (AUCtau,ss) of Lanadelumab in Plasma |
690; 464 | — |
| PRIMARY Terminal Half-life (t1/2) of Lanadelumab in Plasma |
11.7; 12.6 | — |
| PRIMARY Apparent Clearance (CL/F) of Lanadelumab |
0.00906; 0.0135 | — |
| PRIMARY Apparent Volume of Distribution (V/F) of Lanadelumab |
3.63; 5.59 | — |
| SECONDARY Normalized Number of Investigator-Confirmed Hereditary Angioedema (HAE) Attacks During Overall Treatment Period |
0.07; 0.08 | — |
| SECONDARY Normalized Number of Investigator-Confirmed HAE Attacks For Each Efficacy Evaluation Period Other Than the Overall Treatment Period |
0.09; 0.07; 0.15; 0.08; 0.25; 0.06 | — |
| SECONDARY Time to the First Investigator-Confirmed HAE Attack for Each Evaluation Period |
NA; NA; NA; NA; NA; NA | — |
| SECONDARY Normalized Number of HAE Attacks Requiring Acute Treatment for Each Efficacy Evaluation Period |
0.07; 0.07; 0.09; 0.06; 0.15; 0.07 | — |
| SECONDARY Normalized Number of Moderate or Severe Investigator-Confirmed HAE Attacks for Each Efficacy Evaluation Period |
0.07; 0.07; 0.09; 0.06; 0.15; 0.07 | — |
| SECONDARY Normalized Number of High Morbidity Investigator-Confirmed HAE Attacks for Each Efficacy Evaluation Period |
0.01; 0.01; 0.02; 0.01; 0.04; 0.00 | — |
| SECONDARY Number of Participants With Characteristics of Investigator-Confirmed HAE Attacks for Each Efficacy Evaluation Period |
3; 14; 1; 2; 0; 1 | — |
| SECONDARY Number of Participants With HAE Attack-Free Status for Each Evaluation Period |
3; 14; 2; 15; 9; 13 | — |
| SECONDARY Plasma Kallikrein (pKal) Activity |
30.2500; 45.6438; 16.4000; 32.2857; 9.5750; 25.7176 | — |
| SECONDARY Number of Participants With Immunogenicity Status as Positive or Negative |
4; 17; 0; 0; 0; 0 | — |
Summary
The main aims of this study are to learn how lanadelumab moves through a child's body and if the children have any medical problems from lanadelumab. Other aims are to learn if prophylactic treatment with lanadelumab reduces the number and severity of HAE attacks in children, how lanadelumab affects the child's body, and if the children develop antibodies to lanadelumab.
The study doctors will treat acute HAE attacks according to their standard practice.
Participants will receive lanadelumab for up to 52 weeks. When they start treatment, participants will visit their clinic every week for the first 4 weeks. Then, they will visit their clinic every 4 weeks during treatment.
Eligibility Criteria
Inclusion Criteria
- Be a child (male or female) 2 to lesser than ( =)1.0 angioedema attacks per three months during the 12-week baseline observation period and who remain eligible per the inclusion criteria will enter the lanadelumab treatment period.
- Agree to adhere to the protocol-defined schedule of treatments, assessments, and procedures.
- Have a parent(s)/legal guardian who is informed of the nature of the study and can provide written informed consent for the child to participate in the study before any study-specific procedures are performed (with assent from the child when appropriate).
- Females of childbearing potential must agree to be abstinent or agree to comply with the applicable contraceptive requirements of this protocol through the duration of the study from screening through 70 days after the final study visit.
Exclusion Criteria
- 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.
- Dosing with an investigational drug or exposure to an investigational device within 4 weeks prior to screening.
- Be pregnant or breastfeeding.
- Have initiated androgen treatment (eg, stanozolol, danazol, oxandrolone, methyltestosterone, and testosterone) within 2 weeks prior to entering the observation period.
- 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.
- Have any active infectious illness or fever defined as an oral temperature greater than (>) 38 degree celsius (°C) (100.4 fahrenheit [°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.
- Have any HAE attack that is not resolved prior to the first dose of study drug in treatment period A.
- Have any of the following liver function test abnormalities: alanine aminotransferase (ALT) > 3*upper limit of normal (ULN), or aspartate aminotransferase (AST) > 3*ULN, or total bilirubin > 2*ULN (unless the bilirubin elevation is a result of Gilbert's syndrome).
- 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).
- Participant has a known hypersensitivity to the investigational product or its components.
Data sourced from ClinicalTrials.gov (NCT04070326). 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.