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

NCT ID: NCT04583007

Last Updated: 2023-07-24

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

NO_LONGER_AVAILABLE

Study Classification

EXPANDED_ACCESS

Brief Summary

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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.

Detailed Description

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Conditions

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Hereditary Angioedema (HAE) Angioedema

Interventions

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Lanadelumab 150 mg

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.

Intervention Type DRUG

Lanadelumab 300 mg

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.

Intervention Type DRUG

Other Intervention Names

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TAK-743 SHP643 DX-2930 TAK-743 SHP643 DX-2930

Eligibility Criteria

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Inclusion Criteria

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
* 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.
Minimum Eligible Age

2 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Takeda Development Center Americas, Inc.

INDUSTRY

Sponsor Role collaborator

Shire

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Medical Research of Arizona

Scottsdale, Arizona, United States

Site Status

AIRE Medical of Los Angeles

Santa Monica, California, United States

Site Status

Allergy & Asthma Clinical Research

Walnut Creek, California, United States

Site Status

Henry J. Kanarek- Allergy, Asthma & Immunology

Overland Park, Kansas, United States

Site Status

Institute Asthma and Allergy

Chevy Chase, Maryland, United States

Site Status

Institute For Asthma and Allergy

Chevy Chase, Maryland, United States

Site Status

University of Michigan Allergy and Immunology Specialty Clinic

Ann Arbor, Michigan, United States

Site Status

Hudson-Essex Allergy

Belleville, New Jersey, United States

Site Status

Icahn School of Medicine at Mount Sinai

New York, New York, United States

Site Status

Clinical Research Center of Charlotte

Charlotte, North Carolina, United States

Site Status

Bernstein Clinical Research Center

Cincinnati, Ohio, United States

Site Status

Optimed Research, LTD

Columbus, Ohio, United States

Site Status

Toledo Institute of Clinical Research Asthma & Allergy Center

Toledo, Ohio, United States

Site Status

AARA Research Center

Dallas, Texas, United States

Site Status

Yang Medicine

Ottawa, Ontario, Canada

Site Status

Charité - Universitätsmedizin Berlin.

Berlin, , Germany

Site Status

Klinikum der Johann-Wolfgang Goethe-Universitat.

Frankfurt, , Germany

Site Status

Hämophilie Zentrum Rhein Main GmbH

Mörfelden-Walldorf, , Germany

Site Status

Semmelweis Egyetem.

Budapest, , Hungary

Site Status

Hospital Universitario La Paz. Paseo de la Castellana

Madrid, , Spain

Site Status

Countries

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United States Canada Germany Hungary Spain

Related Links

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Other Identifiers

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TAK-743-5012

Identifier Type: OTHER

Identifier Source: secondary_id

TAK-743-6001

Identifier Type: -

Identifier Source: org_study_id

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