Back to resultsStudy to Assess the Tolerability and Safety of Ecallantide in Children and Adolescents With Hereditary Angioedema
Primary Purpose
Hereditary Angioedema Types I and II
Status
Withdrawn
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Ecallantide subcutaneous dosing
Sponsored by
About this trial
This is an interventional treatment trial for Hereditary Angioedema Types I and II focused on measuring Hereditary Angioedema, Children, Adolescents, Ecallantide
Eligibility Criteria
Inclusion Criteria:
- 2 through 15 years of age, inclusive (ie, from the second birthday through the day prior to the sixteenth birthday) at the time of the subject's first attack.
- Documented diagnosis of HAE type I or II. Diagnosis must be confirmed by a documented immunogenic (below the lower limit of normal) and/or functional (< 50% of normal levels) C1- Inhibitor deficiency. Diagnosis may be on the basis of historic data or by diagnostic testing conducted at the time of screening.
- Informed consent (and subject assent as appropriate) signed by the subject's parent(s) or legal guardian(s).
Exclusion Criteria:
- History of an adverse reaction (AE) to Ecallantide in the past
- Diagnosis of angioedema other than HAE
- Participation in another clinical study during the 30 days prior to treatment
- Any known factor/disease that might interfere with the treatment compliance, study conduct, or result interpretation
- Congenital or acquired cardiac anomalies that interfere significantly with cardiac function.
- Treatment with angiotensin converting enzyme (ACE) inhibitors within 7 days prior to treatment.
- Use of hormonal contraception within the 90 days prior to treatment for females of childbearing potential
- The subject is pregnant or breastfeeding
Sites / Locations
- Winthrop-University Hosptial Clinical Trials Center
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Ecallantide
Arm Description
Study Medication, Dose, and Mode of Administration: Single dose of ecallantide subcutaneous dosing: Age less than 10: Weight <25 Kg: 10mg subcutaneously at one site; 25-50kg: 20mg subcutaneously, 10mg per site for 2 separate sites; >50 kg 30mg subcutaneously, 10mg per site for 3 separate sites. Dosing will not exceed 30mg. Age greater than 10: 10mg per site for 3 separate sites. Dosing will not exceed 30mg.
Outcomes
Primary Outcome Measures
Measurement of time to symptomatic improvement from acute attacks of hereditary angioedema
Time to symptom relief. Time to minimal symptoms. An improvement assessment will also be performed at each of the following timepoints: pre -treatment, every 30 minutes for the first 2 hours and then hourly through discharge and at the 28 day clinic visit.
Incidence of need for rescue medication. Incidence of worsening despite use of ecallantide.
Secondary Outcome Measures
Number of Partcipants with Adverse Events
Study Partcipants will be monitored for adverse events, changes in laboratory values, physical exam, vital sign changes and ECG changes. Vital signs, including body temperature, heart rate and sitting blood pressure, will be assessed at screening, Pre-treatment, every 30 minutes for the first 2 hours and then hourly through discharge and at the 28 day clinic visit.
Full Information
NCT ID
NCT01832896
First Posted
April 4, 2013
Last Updated
March 28, 2023
Sponsor
NYU Langone Health
Collaborators
Dyax Corp.
1. Study Identification
Unique Protocol Identification Number
NCT01832896
Brief Title
Study to Assess the Tolerability and Safety of Ecallantide in Children and Adolescents With Hereditary Angioedema
Official Title
A Multicenter, Open-Label Study to Assess the Tolerability and Safety of a Single, Subcutaneous Administration of Ecallantide in Children and Adolescents With Hereditary Angioedema
Study Type
Interventional
2. Study Status
Record Verification Date
January 2017
Overall Recruitment Status
Withdrawn
Why Stopped
PI indicated departure from institution, inability to complete study.
Study Start Date
August 2013 (undefined)
Primary Completion Date
July 2017 (Actual)
Study Completion Date
December 2017 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
NYU Langone Health
Collaborators
Dyax Corp.
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
The objective of this study is to evaluate the safety and tolerability of a single Subcutaneous (SC) dose of Ecallantide in children and adolescents with Hereditary Angioedema (HAE).
Detailed Description
This pilot study is an open-label, non-randomized, single-arm study to evaluate the tolerability and safety of a single SC administration of ecallantide in up to approximately 10 pediatric subjects with HAE during an initial acute attack. The study is planned to enroll subjects 2 through 15 years of age who present with an acute cutaneous, abdominal, or laryngeal HAE attack. No more than 3 study sites will be included until a goal of 10 patients is achieved.
After treatment for an initial attack, one additional open label treatment with ecallantide will be offered to subjects contingent upon having been treated previously and presenting with a subsequent acute cutaneous, abdominal, or laryngeal attack of HAE at least 7 days after initial treatment. Open-label treatment for a second HAE attack will continue until 10 patients have been treated for an initial attack. Safety evaluations will be performed at each subsequent ecallantide-treated attack as for the initial treated attack
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hereditary Angioedema Types I and II
Keywords
Hereditary Angioedema, Children, Adolescents, Ecallantide
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
0 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Ecallantide
Arm Type
Experimental
Arm Description
Study Medication, Dose, and Mode of Administration:
Single dose of ecallantide subcutaneous dosing:
Age less than 10: Weight <25 Kg: 10mg subcutaneously at one site; 25-50kg: 20mg subcutaneously, 10mg per site for 2 separate sites; >50 kg 30mg subcutaneously, 10mg per site for 3 separate sites. Dosing will not exceed 30mg.
Age greater than 10: 10mg per site for 3 separate sites. Dosing will not exceed 30mg.
Intervention Type
Drug
Intervention Name(s)
Ecallantide subcutaneous dosing
Intervention Description
For acute attacks of Hereditary Angioedema in children and adolescents, Ecallantide will be administered.
Primary Outcome Measure Information:
Title
Measurement of time to symptomatic improvement from acute attacks of hereditary angioedema
Description
Time to symptom relief. Time to minimal symptoms. An improvement assessment will also be performed at each of the following timepoints: pre -treatment, every 30 minutes for the first 2 hours and then hourly through discharge and at the 28 day clinic visit.
Incidence of need for rescue medication. Incidence of worsening despite use of ecallantide.
Time Frame
28 days
Secondary Outcome Measure Information:
Title
Number of Partcipants with Adverse Events
Description
Study Partcipants will be monitored for adverse events, changes in laboratory values, physical exam, vital sign changes and ECG changes. Vital signs, including body temperature, heart rate and sitting blood pressure, will be assessed at screening, Pre-treatment, every 30 minutes for the first 2 hours and then hourly through discharge and at the 28 day clinic visit.
Time Frame
28 Days
10. Eligibility
Sex
All
Minimum Age & Unit of Time
2 Years
Maximum Age & Unit of Time
16 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
2 through 15 years of age, inclusive (ie, from the second birthday through the day prior to the sixteenth birthday) at the time of the subject's first attack.
Documented diagnosis of HAE type I or II. Diagnosis must be confirmed by a documented immunogenic (below the lower limit of normal) and/or functional (< 50% of normal levels) C1- Inhibitor deficiency. Diagnosis may be on the basis of historic data or by diagnostic testing conducted at the time of screening.
Informed consent (and subject assent as appropriate) signed by the subject's parent(s) or legal guardian(s).
Exclusion Criteria:
History of an adverse reaction (AE) to Ecallantide in the past
Diagnosis of angioedema other than HAE
Participation in another clinical study during the 30 days prior to treatment
Any known factor/disease that might interfere with the treatment compliance, study conduct, or result interpretation
Congenital or acquired cardiac anomalies that interfere significantly with cardiac function.
Treatment with angiotensin converting enzyme (ACE) inhibitors within 7 days prior to treatment.
Use of hormonal contraception within the 90 days prior to treatment for females of childbearing potential
The subject is pregnant or breastfeeding
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Mark A Davis-Lorton, MD
Organizational Affiliation
Winthrop University Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
Winthrop-University Hosptial Clinical Trials Center
City
Mineola
State/Province
New York
ZIP/Postal Code
11501
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Study to Assess the Tolerability and Safety of Ecallantide in Children and Adolescents With Hereditary Angioedema
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