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Withdrawn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
About this trial
This is an interventional treatment trial for Hereditary Angioedema (HAE)
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
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
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Withdrawn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
We'll reach out to this number within 24 hrs