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

NO_LONGER_AVAILABLE

Conditions studied: Hereditary Angioedema (HAE), Angioedema

In brief

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.

Key facts

Study ID: NCT04583007
Run by: Shire
Last updated by the study team: 2023-07-24

Who can join

Age: 2 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

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.

Where it is running

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

Full record on ClinicalTrials.gov

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