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

July 20, 2023 updated by: Shire

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
No longer available

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Expanded Access

Expanded Access Type

Expanded Access Type

Describes the category of expanded access under U.S. Food and Drug Administration (FDA) regulations. There are three types of expanded access:
  • Individual Patients: Allows a single patient, with a serious disease or condition who cannot participate in a clinical trial, access to a drug or biological product that has not been approved by the FDA. This category also includes access in an emergency situation.
  • Intermediate-size Population: Allows more than one patient (but generally fewer patients than through a Treatment IND/Protocol) access to a drug or biological product that has not been approved by the FDA. This type of expanded access is used when multiple patients with the same disease or condition seek access to a specific drug or biological product that has not been approved by the FDA.
  • Treatment IND/Protocol: Allows a large, widespread population access to a drug or biological product that has not been approved by the FDA. This type of expanded access can only be provided if the product is already being developed for marketing for the same use as the expanded access use.
  • Individual Patients

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Canada
    • Ontario
      • Ottawa, Ontario, Canada, K1G6C6
        • Yang Medicine
  • Germany
      • Berlin, Germany, 10117
        • Charité - Universitätsmedizin Berlin.
      • Frankfurt, Germany, 60590
        • Klinikum der Johann-Wolfgang Goethe-Universitat.
      • Moerfelden-Walldorf, Germany, 64546
        • Hamophilie Zentrum Rhein Main GmbH
  • Hungary
      • Budapest, Hungary, 1125
        • Semmelweis Egyetem.
  • Spain
      • Madrid, Spain, 28046
        • Hospital Universitario La Paz. Paseo de la Castellana
  • United States
    • Arizona
      • Scottsdale, Arizona, United States, 85251
        • Medical Research of Arizona
    • California
      • Santa Monica, California, United States, 90404
        • AIRE Medical of Los Angeles
      • Walnut Creek, California, United States, 94598
        • Allergy & Asthma Clinical Research
    • Kansas
      • Overland Park, Kansas, United States, 66211
        • Henry J. Kanarek- Allergy, Asthma & Immunology
    • Maryland
      • Chevy Chase, Maryland, United States, 20815
        • Institute for Asthma and Allergy
      • Chevy Chase, Maryland, United States, 20815
        • Institute Asthma and Allergy
    • Michigan
      • Ann Arbor, Michigan, United States, 48109-5008
        • University of Michigan Allergy and Immunology Specialty Clinic
    • New Jersey
      • Belleville, New Jersey, United States, 07109
        • Hudson-Essex Allergy
    • New York
      • New York, New York, United States, 10029
        • ICAHN School of Medicine at Mount Sinai
    • North Carolina
      • Charlotte, North Carolina, United States, 28277
        • Clinical Research Center of Charlotte
    • Ohio
      • Cincinnati, Ohio, United States, 45231
        • Bernstein Clinical Research Center
      • Columbus, Ohio, United States, 43235
        • Optimed Research, LTD
      • Toledo, Ohio, United States, 43617
        • Toledo Institute of Clinical Research Asthma & Allergy Center
    • Texas
      • Dallas, Texas, United States, 75231
        • AARA Research Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

1 year and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

N/A

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Shire

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
Takeda Development Center Americas, Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
October 1, 2020

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
October 1, 2020

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
October 12, 2020

Study Record Updates

Last Update Posted (Estimated)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
July 24, 2023

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
July 20, 2023

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
July 1, 2023

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • TAK-743-6001
  • TAK-743-5012 (Other Identifier: Takeda)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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