Angioedemas, HereditaryNCT04955964
A Study of Lanadelumab in Teenagers and Adults With Hereditary Angioedema (HAE) in Argentina
An observational study of Angioedemas, Hereditary, run by Takeda at 1 site in Argentina.
Trial summary
The main aim of this study is to learn about the safety profile of lanadelumab in teenagers and adults with hereditary angioedema (HAE) in Argentina as part of routine routine practice. This study is about collecting data only. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Angioedemas, Hereditary
- How many people
- 48 participants
- Where
- 1 siteArgentina
- Study dates
- 28 Jul 2021to 7 Aug 2023
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Study will include all participants treated with lanadelumab according to approved indications in routine clinical practice settings in Argentina.
Who may be able to join
- Participants (>=12 years) with investigator confirmed diagnosis of HAE.
- Have received at least one dose of lanadelumab according to approved indications.
- Signed the mandatory consent that has been agreed with national regulatory authorities (ANMAT) as applicable.
Who cannot join
- Hypersensitivity to the active substance or to any of the excipients.
Trial locations and contacts
1 site in 1 country.
Argentina1 site
Ic Projects Srl
Ciudad Autónoma de Buenos Aires, Buenos Aires, C1055ADD
About this study
Official title: Post Authorization Study to Monitor Efficacy, Effectiveness and Safety of Lanadelumab (Takhzyro®) in Patients Aged 12 Years and Older With Hereditary Angioedema (HAE) in Argentina
The main aim of this study is to learn about the safety profile of lanadelumab in teenagers and adults with hereditary angioedema (HAE) in Argentina as part of routine routine practice.
This study is about collecting data only. The study sponsor will not be involved in how participants are treated but will provide instructions on how the clinics will record what happens during the study. The study will collect data from the participant's medical records.
Participants do not need to visit their doctor in addition to their normal visits.
- Conditions
- Angioedemas, Hereditary
- Keywords
- Lanadelumab, Post-authorization study
- Medical subject headings
- Angioedemas, Hereditary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| All Participants | Participants with diagnosis of HAE who have received at least one dose of lanadelumab according to currently approved indications in routine clinical practice settings in Argentina will be observed in this study. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants With Adverse Events of Special Interest (AESIs)
An adverse event (AE) is any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. Percentage of participants with AESIs such as hypersensitivity, immunogenicity, liver toxicity, and embryo-fetal toxicity will be reported.
Measured: Baseline up to end of study (up to 24 months)
Percentage of Participants With Serious and Non-serious Adverse Events (AEs)
An AE is any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. Serious AE is any untoward medical occurrence (whether considered to be related to study assigned treatment or not) that at any dose resulted in death, is life-threatening event, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, results in a congenital abnormality/birth defect, or was an important medical event. AEs including both serious and non-serious adverse events will be reported.
Measured: Baseline up to end of study (up to 24 months)
Secondary outcomes (4)
Monthly Mean Rate of Investigator Confirmed HAE Attacks
HAE attack is defined as a discrete episode during which the participant progressed from no angioedema to symptoms of angioedema based on investigator judgement. In general, the event must have symptoms or signs consistent with an attack in greater than or equal to (>=) 1 of the typical locations (peripheral angioedema; abdominal angioedema; laryngeal/tongue angioedema). Monthly mean rate of investigator confirmed HAE attacks with assessments at Weeks 12 and 24 will be reported.
Measured: At Weeks 12 and 24
Monthly Mean Rate of Investigator Confirmed Moderate or Severe HAE Attacks
The overall severity of attack will be determined by the investigator using following definitions: mild (transient or mild discomfort), moderate (mild to moderate limitation in activity), severe (marked limitation in activity). Monthly mean rate of investigator confirmed moderate or severe HAE attacks with assessments at Weeks 12 and 24 will be reported.
Measured: At Weeks 12 and 24
Monthly Mean Rate of Investigator Confirmed HAE Attacks Requiring Acute Treatment
HAE attack is defined as a discrete episode during which the participant progressed from no angioedema to symptoms of angioedema based on investigator judgement. In general, the event must have symptoms or signs consistent with an attack in >=1 of the typical locations (peripheral angioedema; abdominal angioedema; laryngeal/tongue angioedema). Monthly mean rate of investigator confirmed HAE attacks requiring acute treatment with assessments at Weeks 12 and 24 will be reported.
Measured: At Weeks 12 and 24
Percentage of Participants who are Attack Free
A participant will be considered as attack free during an efficacy evaluation period if the participant has no investigator-confirmed HAE attacks during treatment period. HAE attack is defined as a discrete episode during which the participant progressed from no angioedema to symptoms of angioedema based on investigator judgement. In general, the event must have symptoms or signs consistent with an attack in >=1 of the typical locations (peripheral angioedema; abdominal angioedema; laryngeal/tongue angioedema). Percentage of participants who are attack free with assessments at 12 and 24 weeks will be reported.
Measured: At Weeks 12 and 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jul 2021
- Study start28 Jul 2021
- Primary completion7 Aug 2023
- Study completion7 Aug 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Angioedemas, HereditaryStudies of the same condition, open and under way.
- More trials run by TakedaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 48 participants (actual)
Sponsor and investigators
- Lead sponsor
- Takeda
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04955964
- Sponsor's protocol ID
- TAK-743-4008
- Status last verified
- Oct 2023
- First submitted
- 30 Jun 2021
- Last updated
- 4 Oct 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
Study at a glance
From the registry record, last updated by the sponsor on 4 Oct 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Post Authorization Study to Monitor Efficacy, Effectiveness and Safety of Lanadelumab (Takhzyro®) in Patients Aged 12 Years and Older With Hereditary Angioedema (HAE) in Argentina
- ClinicalTrials.gov ID
- NCT04955964
- Acronym / sponsor ID
- TAK-743-4008
- Conditions
- Angioedemas, Hereditary
- Keywords
- Lanadelumab, Post-authorization study
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 48 participants (actual)
- Lead sponsor
- TakedaIndustry (pharma, biotech, devices)
- Study start
- 28 Jul 2021 (actual)
- Primary completion
- 7 Aug 2023 (actual)
- Study completion
- 7 Aug 2023 (actual)
- First posted
- 9 Jul 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The main aim of this study is to learn about the safety profile of lanadelumab in teenagers and adults with hereditary angioedema (HAE) in Argentina as part of routine routine practice.
This study is about collecting data only. The study sponsor will not be involved in how participants are treated but will provide instructions on how the clinics will record what happens during the study. The study will collect data from the participant's medical records.
Participants do not need to visit their doctor in addition to their normal visits.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| All Participants | – | –Participants with diagnosis of HAE who have received at least one dose of lanadelumab according to currently approved indications in routine clinical practice settings in Argentina will be observed in this study. |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Study will include all participants treated with lanadelumab according to approved indications in routine clinical practice settings in Argentina.
Key inclusion criteria (3 listed)
- Participants (>=12 years) with investigator confirmed diagnosis of HAE.
- Have received at least one dose of lanadelumab according to approved indications.
- Signed the mandatory consent that has been agreed with national regulatory authorities (ANMAT) as applicable.
Key exclusion criteria (1 listed)
- Hypersensitivity to the active substance or to any of the excipients.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Percentage of Participants With Adverse Events of Special Interest (AESIs)
Time frame: Baseline up to end of study (up to 24 months)
An adverse event (AE) is any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. Percentage of participants with AESIs such as hypersensitivity, immunogenicity, liver toxicity, and embryo-fetal toxicity will be reported.
Percentage of Participants With Serious and Non-serious Adverse Events (AEs)
Time frame: Baseline up to end of study (up to 24 months)
An AE is any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. Serious AE is any untoward medical occurrence (whether considered to be related to study assigned treatment or not) that at any dose resulted in death, is life-threatening event, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, results in a congenital abnormality/birth defect, or was an important medical event. AEs including both serious and non-serious adverse events will be reported.
4 secondary outcomes
Monthly Mean Rate of Investigator Confirmed HAE Attacks
Time frame: At Weeks 12 and 24
Monthly Mean Rate of Investigator Confirmed Moderate or Severe HAE Attacks
Time frame: At Weeks 12 and 24
Monthly Mean Rate of Investigator Confirmed HAE Attacks Requiring Acute Treatment
Time frame: At Weeks 12 and 24
Percentage of Participants who are Attack Free
Time frame: At Weeks 12 and 24
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Oct 2023.
- To obtain more information on the study, click here/on this linkLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Study Director study director
Takeda
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Angioedemas, Hereditary, run by Takeda at 1 site in Argentina.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 4 Oct 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Argentina. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of Participants With Adverse Events of Special Interest (AESIs) (time frame: Baseline up to end of study (up to 24 months)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Oct 2023.