Hereditary Angioedema (HAE)NCT04583007
Expanded Access for the Prevention of Acute Attacks of 1) Hereditary Angioedema (HAE) in Children and 2) Non-histaminergic Angioedema With Normal C1-Inhibitor (C1-INH) in Teenagers and Adults
A study testing Lanadelumab 150 mg and Lanadelumab 300 mg in people with Hereditary Angioedema (HAE) and Angioedema, run by Shire at 20 sites in 5 countries.
Trial 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 angioedema attacks. Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Hereditary Angioedema (HAE), Angioedema
- What is being tested?
- Lanadelumab 150 mg and Lanadelumab 300 mg
- Type of treatment
- Injection or infusion
- How many people
- Not given
- Where
- 20 sitesin 5 countries
- Study dates
- Start not givento not given
- Phase
- Expanded access
- 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 2 years and older
- All sexes
- Child, Adult, Older adult
Who may be able to join
- TAK-743-6001 Study (Acute Attacks of HAE):
- Pediatric participants, 2 to <12 years of age, with HAE who completed the SHP643-301 (SPRING) study and are deriving continued clinical benefit from lanadelumab treatment will be invited to take part in this program
- Participants have no suitable alternative treatment options and cannot enter a lanadelumab clinical trial
- Participant has a parent(s)/legal guardian who is informed of the nature of the expanded access program and can provide written informed consent for the child to participate (with assent from child when appropriate) prior to treatment
- Participants does not have any of the following liver function test abnormalities : alanine aminotransferase (ALT) greater than (>)3x upper limit of normal (ULN), or aspartate aminotransferase (AST) >3x ULN, or total bilirubin >2x ULN (unless the bilirubin elevation is a result of Gilbert's syndrome)
- Participant does not have any condition that in the opinion of the Investigator may compromise their safety
Show all 24Show fewer
- Participant does not have a known hypersensitivity to lanadelumab or its components
- Participant (greater than or equal to >or=6 years) and/or caregiver is willing to self-administer lanadelumab at home and has received documented training on self-administration during the SHP643-301 (SPRING) Study
- For Female participants of childbearing potential only:
- Participant has agreed to be abstinent or agreed to comply with the applicable contraceptive requirements of this protocol for the duration of the expanded access program and for 70 days after completion
- Participant is not pregnant or breast-feeding Note: Participants who do not satisfy the above criteria will be excluded.
- TAK-743-5012 Study (Acute Attacks of Non-histaminergic Angioedema With Normal C1-Inhibitor (C1-INH)):
- Participant has completed the treatment period of the TAK-743-3001 study (all Visit 14 assessments have been completed).
- Participant is demonstrating continued benefit from lanadelumab treatment.
- Participant and/or a parent(s)/legal guardian is informed of the nature of the expanded access program and can provide written informed consent for themselves or the child to participate (with assent from child when appropriate) prior to treatment).
- Participant does not have any condition including lab test that in the opinion of the Investigator may compromise their safety.
- Participant does not have a known hypersensitivity to lanadelumab or its components.
- Participant and/or caregiver is willing to self-administer lanadelumab at home and has received documented training on self-administration during the Study TAK-743-3001 OR the participant and/or caregiver is willing to self-administer lanadelumab at home and will receive training at the first two dose administrations in accordance with protocol.
- Female Participants of childbearing potential only:
- Participant has agreed to be abstinent or agreed to comply with the applicable contraceptive requirements of this protocol for the duration of the expanded access program and for 70 days after completion of the last dose.
- Participant is not pregnant or breast-feeding.
- Male Participants only:
- Participant has agreed to comply with the applicable contraceptive requirements of this protocol for the duration of the expanded access program and for 70 days following the last dose of lanadelumab
- Participant has agreed to not donate sperm during the course of the program and for 70 days following the last dose of lanadelumab.
Who cannot join
None listed.
Trial locations and contacts
20 sites in 5 countries.
20 sites in 5 countries
United States14 sites
University of Michigan Allergy and Immunology Specialty Clinic
Ann Arbor, Michigan, 48109-5008
Hudson-Essex Allergy
Belleville, New Jersey, 07109
Clinical Research Center of Charlotte
Charlotte, North Carolina, 28277
Institute Asthma and Allergy
Chevy Chase, Maryland, 20815
Institute For Asthma and Allergy
Chevy Chase, Maryland, 20815
Bernstein Clinical Research Center
Cincinnati, Ohio, 45231
Optimed Research, LTD
Columbus, Ohio, 43235
AARA Research Center
Dallas, Texas, 75231
Icahn School of Medicine at Mount Sinai
New York, New York, 10029
Henry J. Kanarek- Allergy, Asthma & Immunology
Overland Park, Kansas, 66211
AIRE Medical of Los Angeles
Santa Monica, California, 90404
Medical Research of Arizona
Scottsdale, Arizona, 85251
Toledo Institute of Clinical Research Asthma & Allergy Center
Toledo, Ohio, 43617
Allergy & Asthma Clinical Research
Walnut Creek, California, 94598
Germany3 sites
Charité - Universitätsmedizin Berlin.
Berlin, 10117
Klinikum der Johann-Wolfgang Goethe-Universitat.
Frankfurt, 60590
Hämophilie Zentrum Rhein Main GmbH
Mörfelden-Walldorf, 64546
About this study
Official title: Expanded Access Program: Lanadelumab for the Prevention of Acute Attacks of Hereditary Angioedema (HAE) in Pediatric Patients 2 to <12 Years of Age and Lanadelumab for the Prevention Against Acute Attacks of Non-histaminergic Angioedema With Normal C1-Inhibitor (C1-INH)
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 angioedema attacks. This expanded access program enables these participants with a high unmet medical need to continue receiving lanadelumab during the interim period between completion of either the SHP643-301 (NCT04070326; SPRING study) or the TAK-743-3001 (NCT04444895) study and potential licensure of lanadelumab for the respective age group and/or treatment.
- Conditions
- Hereditary Angioedema (HAE), Angioedema
- Being tested
- Lanadelumab 150 mg, Lanadelumab 300 mg
- Medical subject headings
- Angioedemas, Hereditary, Angioedema
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Lanadelumab 150 mgDrug
Also called TAK-743, SHP643, DX-2930
TAK-743-6001: Participants will receive subcutaneous (SC) injection of lanadelumab at a dose of 150 milligrams (mg) at the same frequency (for every 2 weeks [Q2W] or for every 4 weeks [Q4W]) given at the completion of SPRING study until benefit-risk no longer favors the individual participant or an appropriate alternative therapy becomes available or lanadelumab 150 mg/ 1 milliliter (mL) becomes commercially available or the participants turns 12 years of age or participant chooses to discontinue treatment. For participants aged 6-12 years, a dose frequency may be modified based on a benefit-risk assessment and medical decision from the treating physician.
Lanadelumab 300 mgDrug
Also called TAK-743, SHP643, DX-2930
TAK-743-5012: Participants will receive SC injection of lanadelumab in prefilled syringe at a dose of 300 mg, Q2W or Q4W if well controlled for 26 weeks across Study TAK-743-3001 and this (TAK-743-5012) program.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Oct 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hereditary Angioedema (HAE)Studies of the same condition, open and under way.
- 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
- Expanded access
Sponsor and investigators
- Lead sponsor
- Shire
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Takeda Development Center Americas, Inc.
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04583007
- Sponsor's protocol ID
- TAK-743-6001
- Other IDs
- TAK-743-5012
- Status last verified
- Jul 2023
- First submitted
- 1 Oct 2020
- Last updated
- 24 Jul 2023
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Jul 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Expanded Access Program: Lanadelumab for the Prevention of Acute Attacks of Hereditary Angioedema (HAE) in Pediatric Patients 2 to <12 Years of Age and Lanadelumab for the Prevention Against Acute Attacks of Non-histaminergic Angioedema With Normal C1-Inhibitor (C1-INH)
- ClinicalTrials.gov ID
- NCT04583007
- Acronym / sponsor ID
- TAK-743-6001
- Conditions
- Hereditary Angioedema (HAE), Angioedema
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- ShireIndustry (pharma, biotech, devices)
- Collaborators
- Takeda Development Center Americas, Inc.
- First posted
- 12 Oct 2020 (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 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 angioedema attacks. This expanded access program enables these participants with a high unmet medical need to continue receiving lanadelumab during the interim period between completion of either the SHP643-301 (NCT04070326; SPRING study) or the TAK-743-3001 (NCT04444895) study and potential licensure of lanadelumab for the respective age group and/or treatment.
Study design
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (24 listed)
- Pediatric participants, 2 to <12 years of age, with HAE who completed the SHP643-301 (SPRING) study and are deriving continued clinical benefit from lanadelumab treatment will be invited to take part in this program
- Participants have no suitable alternative treatment options and cannot enter a lanadelumab clinical trial
- Participant has a parent(s)/legal guardian who is informed of the nature of the expanded access program and can provide written informed consent for the child to participate (with assent from child when appropriate) prior to treatment
- Participants does not have any of the following liver function test abnormalities : alanine aminotransferase (ALT) greater than (>)3x upper limit of normal (ULN), or aspartate aminotransferase (AST) >3x ULN, or total bilirubin >2x ULN (unless the bilirubin elevation is a result of Gilbert's syndrome)
- Participant does not have any condition that in the opinion of the Investigator may compromise their safety
Key exclusion criteria (0 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Lanadelumab 150 mgAlso: TAK-743, SHP643, DX-2930TAK-743-6001: Participants will receive subcutaneous (SC) injection of lanadelumab at a dose of 150 milligrams (mg) at the same frequency (for every 2 weeks [Q2W] or for every 4 weeks [Q4W]) given at the completion of SPRING study until benefit-risk no longer favors the individual participant or an appropriate alternative therapy becomes available or lanadelumab 150 mg/ 1 milliliter (mL) becomes commercially available or the participants turns 12 years of age or participant chooses to discontinue treatment. For participants aged 6-12 years, a dose frequency may be modified based on a benefit-risk assessment and medical decision from the treating physician. | Drug | – |
| Lanadelumab 300 mgAlso: TAK-743, SHP643, DX-2930TAK-743-5012: Participants will receive SC injection of lanadelumab in prefilled syringe at a dose of 300 mg, Q2W or Q4W if well controlled for 26 weeks across Study TAK-743-3001 and this (TAK-743-5012) program. | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jul 2023.
- To obtain more information on the study, click here/on this linkLink provided by the sponsor.
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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.
The record lists no contact. Use the official record to reach the sponsor.
20 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Lanadelumab 150 mg and Lanadelumab 300 mg in people with Hereditary Angioedema (HAE) and Angioedema, run by Shire at 20 sites in 5 countries.
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 24 inclusion and 0 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: status not confirmed, as of the sponsor's last update on 24 Jul 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 20 sites in United States, Germany, Canada, Hungary, Spain. 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?
Results are not available in the source record.
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.
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 24 Jul 2023.