Phase 3
N=12
Expanded Access Program With Lanadelumab for Japanese People With Hereditary Angioedema
Hereditary Angioedema
Bottom Line
View on ClinicalTrials.gov: NCT04687137 ↗Enrolled (actual)
12
Serious AEs
8.3%
Results posted
Jun 2023
Primary outcome: Primary: Number of Participants With Treatment Emergent Adverse Events (TEAEs) Including Serious TEAEs and Adverse Events of Special Interest (AESI) — 11; 1; 8; 1 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- TAK-743 300 mg (Drug)
- Age
- Pediatric, Adult, Older Adult · 12+ yrs
- Sex
- All
- Sponsor
- Takeda
- Primary completion
- Jun 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Treatment Emergent Adverse Events (TEAEs) Including Serious TEAEs and Adverse Events of Special Interest (AESI) |
11; 1; 8; 1; 1; 0 | — |
| PRIMARY Number of Participants With Clinically Significant Abnormalities in Clinical Laboratory Tests |
0; 0 | — |
| PRIMARY Number of Participants With Clinically Significant Abnormalities in Vital Signs |
0; 0 | — |
| SECONDARY Time to First HAE Attack After Day 0 for the Efficacy Evaluation Period for Non-Rollover Participants |
0.0 | — |
Summary
The expanded access program allows people to gain access to an unlicensed treatment on compassionate grounds. Lanadelumab, also known as TAK-743, is a medicine to help prevent hereditary angioedema attacks. Lanadelumab is not yet licensed for use in Japan.
The main aim of this study is to allow Japanese teenagers and adults with type I or type II hereditary angioedema to be treated with lanadelumab, through the expanded access program in Japan.
Participants can either have taken part in the previous study SHP643-302 or can be new participants. Participants just completing study SHP643-302 who reach the criteria can automatically take part in this study. However, for new participants, the study doctor will check who can take part at the first study visit.
For those who can take part, new participants will receive injections of lanadelumab just under the skin. Eventually, after training, some of these will be able to inject themselves with lanadelumab in the same way. Participants who injected themselves with lanadelumab in study SHP643-302 can continue to do so during this study.
The study doctors will decide if each participant will be treated with lanadelumab every 2 weeks or every 4 weeks. Treatment with lanadelumab will continue until lanadelumab is commercially available in Japan or the sponsor (Takeda) stops the study.
Participants can visit the clinic during treatment if needed. If treatment continues after 6 months, participants will visit the clinic every 12 weeks for a check-up. This will include noting any hereditary angioedema attacks and side effects from the treatment. After 7 months of treatment, the study staff will check-up with each participant every 2 weeks by telephone.
After treatment has finished, participants will visit the clinic for a final-check-up 4 weeks later.
Eligibility Criteria
Inclusion Criteria
- In the opinion of the Investigator, the participant is capable of understanding and complying with protocol requirements.
- Be of Japanese descent, defined as born in Japan and having Japanese parents and Japanese maternal and paternal grandparents.
- Male and female HAE participants who are 12 years of age or older at the time of screening.
- Documented diagnosis of disease HAE (Type I or II) based on all of the following:
- Documented clinical history consistent with HAE (subcutaneous [SC] or mucosal, nonpruritic swelling episodes without accompanying urticaria).
- Diagnostic testing results obtained during screening (or a prior lanadelumab study) that confirm HAE Type I or II: C1-inhibitor (C1-INH) functional level 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).
- Pregnancy or breast feeding.
- Have any uncontrolled underlying medical condition which would require treatment adjustment during the study treatment period, that, in the opinion of the Investigator or sponsor, may confound the results of the safety assessments or may place the participant at risk. Participants with stable treatment for at least 3 months prior to screening and NOT expecting any change to their treatment regimen for 6 months during the study treatment period, will not be excluded.
- Participant has a known hypersensitivity to the study drug or its components.
Data sourced from ClinicalTrials.gov (NCT04687137). 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.