Clinical trial 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
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)
Study type: EXPANDED_ACCESS
Overall status: NO_LONGER_AVAILABLE
Conditions: Hereditary Angioedema (HAE)
Angioedema
Lead sponsor: Takeda
Sponsor class: INDUSTRY
Collaborators: name: Takeda Development Center Americas, Inc.
First posted: 2020-10-12
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT

Brief 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. 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.


Eligibility criteria: 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.

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: Scottsdale
country: United States
facility: Medical Research of Arizona
state: Arizona
city: Santa Monica
country: United States
facility: AIRE Medical of Los Angeles
state: California
city: Walnut Creek
country: United States
facility: Allergy & Asthma Clinical Research
state: California
city: Overland Park
country: United States
facility: Henry J. Kanarek- Allergy, Asthma & Immunology
state: Kansas
city: Chevy Chase
country: United States
facility: Institute Asthma and Allergy
state: Maryland
city: Chevy Chase
country: United States
facility: Institute For Asthma and Allergy
state: Maryland
city: Ann Arbor
country: United States
facility: University of Michigan Allergy and Immunology Specialty Clinic
state: Michigan
city: Belleville
country: United States
facility: Hudson-Essex Allergy
state: New Jersey
city: New York
country: United States
facility: Icahn School of Medicine at Mount Sinai
state: New York
city: Charlotte
country: United States
facility: Clinical Research Center of Charlotte
state: North Carolina
city: Cincinnati
country: United States
facility: Bernstein Clinical Research Center
state: Ohio
city: Columbus
country: United States
facility: Optimed Research, LTD
state: Ohio
city: Toledo
country: United States
facility: Toledo Institute of Clinical Research Asthma & Allergy Center
state: Ohio
city: Dallas
country: United States
facility: AARA Research Center
state: Texas
city: Ottawa
country: Canada
facility: Yang Medicine
state: Ontario
city: Berlin
country: Germany
facility: Charité - Universitätsmedizin Berlin.
city: Frankfurt
country: Germany
facility: Klinikum der Johann-Wolfgang Goethe-Universitat.
city: Moerfelden-Walldorf
country: Germany
facility: Hämophilie Zentrum Rhein Main GmbH
city: Budapest
country: Hungary
facility: Semmelweis Egyetem.
city: Madrid
country: Spain
facility: Hospital Universitario La Paz. Paseo de la Castellana
Study officials:
References: