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
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)
2 other identifiers
expanded_access
N/A
5 countries
20
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.
Trial Health
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20 active sites
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Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
October 1, 2020
CompletedFirst Posted
Study publicly available on registry
October 12, 2020
CompletedJuly 24, 2023
July 1, 2023
October 1, 2020
July 20, 2023
Conditions
Interventions
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.
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.
Eligibility Criteria
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).
- +9 more criteria
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Shirelead
- Takeda Development Center Americas, Inc.collaborator
Study Sites (20)
Medical Research of Arizona
Scottsdale, Arizona, 85251, United States
AIRE Medical of Los Angeles
Santa Monica, California, 90404, United States
Allergy & Asthma Clinical Research
Walnut Creek, California, 94598, United States
Henry J. Kanarek- Allergy, Asthma & Immunology
Overland Park, Kansas, 66211, United States
Institute Asthma and Allergy
Chevy Chase, Maryland, 20815, United States
Institute For Asthma and Allergy
Chevy Chase, Maryland, 20815, United States
University of Michigan Allergy and Immunology Specialty Clinic
Ann Arbor, Michigan, 48109-5008, United States
Hudson-Essex Allergy
Belleville, New Jersey, 07109, United States
Icahn School of Medicine at Mount Sinai
New York, New York, 10029, United States
Clinical Research Center of Charlotte
Charlotte, North Carolina, 28277, United States
Bernstein Clinical Research Center
Cincinnati, Ohio, 45231, United States
Optimed Research, LTD
Columbus, Ohio, 43235, United States
Toledo Institute of Clinical Research Asthma & Allergy Center
Toledo, Ohio, 43617, United States
AARA Research Center
Dallas, Texas, 75231, United States
Yang Medicine
Ottawa, Ontario, K1G6C6, Canada
Charité - Universitätsmedizin Berlin.
Berlin, 10117, Germany
Klinikum der Johann-Wolfgang Goethe-Universitat.
Frankfurt, 60590, Germany
Hämophilie Zentrum Rhein Main GmbH
Mörfelden-Walldorf, 64546, Germany
Semmelweis Egyetem.
Budapest, 1125, Hungary
Hospital Universitario La Paz. Paseo de la Castellana
Madrid, 28046, Spain