CClinicalTrials.gg
No longer availableNCT04583007Updated Jul 24, 2023

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

An expanded access record providing Lanadelumab 150 mg and Lanadelumab 300 mg in Hereditary Angioedema (HAE) and Angioedema, sponsored by Shire. No longer available at 20 sites in 5 countries. Open to participants aged 2 Years and older. Per ClinicalTrials.gov, last updated 2023-07-24.

Sponsored by Shire · Expanded access

Study type
Expanded access
Access type
Individual patients
Ages
2 Years and older
Sex
All
01

Study 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.

02

Conditions studied

  • Hereditary Angioedema (HAE)
  • Angioedema
03

In context

Angioedema

164 studies on the registry are indexed under Angioedema; 19 are open to participants now.

Browse Angioedema studies →

Lead sponsor

Shire is the lead sponsor of 346 studies on the registry; 2 are open to participants now.

Of its 47 completed or terminated interventional studies of FDA-regulated products, 47 (100%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
2 Years and older
Sexes eligible
All

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.
05

Access details

Study type
Expanded access
Access type
Individual patients

Available treatment

  • DrugLanadelumab 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.

    Also known as: TAK-743, SHP643, DX-2930

  • DrugLanadelumab 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.

    Also known as: TAK-743, SHP643, DX-2930

06

Where to request access

20 sites
  • 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
    Moerfelden-Walldorf, 64546, Germany
  • Semmelweis Egyetem.
    Budapest, 1125, Hungary
  • Hospital Universitario La Paz. Paseo de la Castellana
    Madrid, 28046, Spain
07

References and documents

08

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Jul 24, 2023, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
09

Registry details

Key details

Study ID
NCT04583007
Lead sponsor
Shire
Collaborators
Takeda Development Center Americas, Inc.
Responsible party
Sponsor
First posted
Oct 12, 2020
Last update
Jul 24, 2023
View the source record on ClinicalTrials.gov ↗

Requesting access

Expanded access is arranged between your doctor and the company. Ask your care team to contact the provider listed on this record.

No contact was published for this record. The registry link below has the sponsor’s details.

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