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Study to Evaluate Ecallantide in Paediatric Patients With Acute Attacks of Hereditary Angioedema

Primary Purpose

Hereditary Angioedema (HAE)

Status
Withdrawn
Phase
Phase 2
Locations
Study Type
Interventional
Intervention
ecallantide
Sponsored by
Shire
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hereditary Angioedema (HAE)

Eligibility Criteria

2 Years - 17 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. Male or female from 2 years of age and prepubertal
  2. Physician diagnosis and history of HAE (Type I or II) including laboratory results showing C1-1NH activity below the lower limit of normal or up to 15% above the lower limit of the normal range
  3. Present at the site with moderate to severe signs and symptoms of an acute attack of HAE within 8 hours of recognition of the onset of the attack. Spontaneous resolution of the attack must not have begun before the administration of study drug.
  4. Must have signed informed consent by parent or caregiver.

Exclusion Criteria:

  1. <2 years of age or have reached puberty
  2. Received treatment with ecallantide within previous 72 hours
  3. Received an investigational drug or device, other than ecallantide, within 30 days prior to the screening visit
  4. Pharyngeal/laryngeal symptoms
  5. Mild attacks including mild edema of the extremities and mild abdominal attacks
  6. Are unable or unwilling to give informed consent (parent or caregiver)
  7. Any other condition that, in the opinion of the investigator, may compromise the safety or compliance of the patient or would preclude the patient from successful completion of the study

Sites / Locations

    Arms of the Study

    Arm 1

    Arm 2

    Arm Type

    Active Comparator

    Placebo Comparator

    Arm Label

    ecallantide

    placebo

    Arm Description

    phosphate buffered saline

    Outcomes

    Primary Outcome Measures

    safety and tolerability of ecallantide

    Secondary Outcome Measures

    evaluate the PK profile of ecallantide
    assess the efficacy of ecallantide

    Full Information

    First Posted
    December 2, 2010
    Last Updated
    May 14, 2021
    Sponsor
    Shire
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    1. Study Identification

    Unique Protocol Identification Number
    NCT01253382
    Brief Title
    Study to Evaluate Ecallantide in Paediatric Patients With Acute Attacks of Hereditary Angioedema
    Official Title
    A 3-Part Study to Evaluate the Pharmacokinetics, Safety, and Efficacy of Subcutaneous Ecallantide in Prepubertal Paediatric Patients Experiencing Acute Attacks of Hereditary Angioedema (HAE)
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    May 2021
    Overall Recruitment Status
    Withdrawn
    Study Start Date
    June 1, 2012 (Actual)
    Primary Completion Date
    April 1, 2015 (Anticipated)
    Study Completion Date
    undefined (undefined)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    Shire

    4. Oversight

    Data Monitoring Committee
    Yes

    5. Study Description

    Brief Summary
    The primary objective of this study is: assess the safety and tolerability of ecallantide in paediatric patients for acute attacks of HAE The secondary objectives are: evaluate the pharmacokinetic profile of ecallantide in paediatric patients treated for acute attacks of HAE assess the efficacy of ecallantide in paediatric patients treated for moderate to severe acute attacks of HAE

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Hereditary Angioedema (HAE)

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 2, Phase 3
    Interventional Study Model
    Single Group Assignment
    Masking
    ParticipantCare ProviderInvestigatorOutcomes Assessor
    Allocation
    Randomized
    Enrollment
    0 (Actual)

    8. Arms, Groups, and Interventions

    Arm Title
    ecallantide
    Arm Type
    Active Comparator
    Arm Title
    placebo
    Arm Type
    Placebo Comparator
    Arm Description
    phosphate buffered saline
    Intervention Type
    Biological
    Intervention Name(s)
    ecallantide
    Intervention Description
    10 - 30mg subcutaneous injection.
    Primary Outcome Measure Information:
    Title
    safety and tolerability of ecallantide
    Time Frame
    4 years
    Secondary Outcome Measure Information:
    Title
    evaluate the PK profile of ecallantide
    Time Frame
    4 years
    Title
    assess the efficacy of ecallantide
    Time Frame
    4 years

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    2 Years
    Maximum Age & Unit of Time
    17 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    Inclusion Criteria: Male or female from 2 years of age and prepubertal Physician diagnosis and history of HAE (Type I or II) including laboratory results showing C1-1NH activity below the lower limit of normal or up to 15% above the lower limit of the normal range Present at the site with moderate to severe signs and symptoms of an acute attack of HAE within 8 hours of recognition of the onset of the attack. Spontaneous resolution of the attack must not have begun before the administration of study drug. Must have signed informed consent by parent or caregiver. Exclusion Criteria: <2 years of age or have reached puberty Received treatment with ecallantide within previous 72 hours Received an investigational drug or device, other than ecallantide, within 30 days prior to the screening visit Pharyngeal/laryngeal symptoms Mild attacks including mild edema of the extremities and mild abdominal attacks Are unable or unwilling to give informed consent (parent or caregiver) Any other condition that, in the opinion of the investigator, may compromise the safety or compliance of the patient or would preclude the patient from successful completion of the study
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Study Director
    Organizational Affiliation
    Takeda
    Official's Role
    Study Director

    12. IPD Sharing Statement

    Learn more about this trial

    Study to Evaluate Ecallantide in Paediatric Patients With Acute Attacks of Hereditary Angioedema

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