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Study to Assess the Tolerability and Safety of Ecallantide in Children and Adolescents With Hereditary Angioedema

This study is not yet open for participant recruitment. (see Contacts and Locations)
Verified April 2013 by Winthrop University Hospital
Sponsor:
Information provided by (Responsible Party):
Winthrop University Hospital
ClinicalTrials.gov Identifier:
NCT01832896
First received: April 4, 2013
Last updated: April 15, 2013
Last verified: April 2013
  Purpose

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).


Condition Intervention Phase
Hereditary Angioedema Types I and II
Drug: Ecallantide subcutaneous dosing
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Winthrop University Hospital:

Primary Outcome Measures:
  • Measurement of time to symptomatic improvement from acute attacks of hereditary angioedema [ Time Frame: 28 days ] [ Designated as safety issue: No ]

    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 worstening despite use of ecallantide.



Secondary Outcome Measures:
  • Number of Partcipants with Adverse Events [ Time Frame: 28 Days ] [ Designated as safety issue: Yes ]
    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.


Estimated Enrollment: 10
Study Start Date: May 2013
Estimated Study Completion Date: May 2015
Estimated Primary Completion Date: December 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Ecallantide

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.
Drug: Ecallantide subcutaneous dosing
For acute attacks of Hereditary Angioedema in children and adolescents, Ecallantide will be administered.

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

  Eligibility

Ages Eligible for Study:   2 Years to 16 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. 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.
  2. 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-INH (Inhibitor) deficiency. Diagnosis may be on the basis of historic data or by diagnostic testing conducted at the time of screening.
  3. Informed consent (and subject assent as appropriate) signed by the subject's parent(s) or legal guardian(s).

Exclusion Criteria:

  1. History of an adverse reaction (AE) to Ecallantide in the past
  2. Diagnosis of angioedema other than HAE
  3. Participation in another clinical study during the 30 days prior to treatment
  4. Any known factor/disease that might interfere with the treatment compliance, study conduct, or result interpretation
  5. Congenital or acquired cardiac anomalies that interfere significantly with cardiac function.
  6. Treatment with angiotensin converting enzyme (ACE) inhibitors within 7 days prior to treatment.
  7. Use of hormonal contraception within the 90 days prior to treatment for females of childbearing potential
  8. The subject is pregnant or breastfeeding
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01832896

Contacts
Contact: Mark A Davis-Lorton, MD 516-663-2097 mdavislorton@winthrop.org

Locations
United States, New York
Winthrop-University Hosptial Clinical Trials Center Not yet recruiting
Mineola, New York, United States, 11501
Contact: Mark A Davis-Lorton, MD    516-663-2097    mdavislorton@winthrop.org   
Principal Investigator: Mark A Davis-Lorton, MD         
Sponsors and Collaborators
Winthrop University Hospital
Investigators
Principal Investigator: Mark A Davis-Lorton, MD Winthrop University Hospital
  More Information

No publications provided

Responsible Party: Winthrop University Hospital
ClinicalTrials.gov Identifier: NCT01832896     History of Changes
Other Study ID Numbers: DX-88/IST-5
Study First Received: April 4, 2013
Last Updated: April 15, 2013
Health Authority: United States: Institutional Review Board

Keywords provided by Winthrop University Hospital:
Hereditary Angioedema
Children
Adolescents
Ecallantide

Additional relevant MeSH terms:
Angioedemas, Hereditary
Hereditary Angioedema Types I and II
Angioedema
Vascular Diseases
Cardiovascular Diseases
Urticaria
Skin Diseases, Vascular
Skin Diseases
Hypersensitivity, Immediate
Hypersensitivity
Immune System Diseases
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on July 20, 2014