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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01253382
Recruitment Status : Withdrawn
First Posted : December 3, 2010
Last Update Posted : January 24, 2018
Information provided by (Responsible Party):

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

Condition or disease Intervention/treatment Phase
Hereditary Angioedema (HAE) Biological: ecallantide Phase 2 Phase 3

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
Allocation: Randomized
Intervention Model: Single Group Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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 Start Date : June 1, 2012
Estimated Primary Completion Date : April 1, 2015

Resource links provided by the National Library of Medicine

Drug Information available for: Ecallantide
U.S. FDA Resources

Arm Intervention/treatment
Active Comparator: ecallantide Biological: ecallantide
10 - 30mg subcutaneous injection.
Placebo Comparator: placebo
phosphate buffered saline
Biological: ecallantide
10 - 30mg subcutaneous injection.

Primary Outcome Measures :
  1. safety and tolerability of ecallantide [ Time Frame: 4 years ]

Secondary Outcome Measures :
  1. evaluate the PK profile of ecallantide [ Time Frame: 4 years ]
  2. assess the efficacy of ecallantide [ Time Frame: 4 years ]

Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Responsible Party: Shire Identifier: NCT01253382     History of Changes
Other Study ID Numbers: DX-88/26
2010-022716-39 ( EudraCT Number )
First Posted: December 3, 2010    Key Record Dates
Last Update Posted: January 24, 2018
Last Verified: January 2018

Additional relevant MeSH terms:
Angioedemas, Hereditary
Vascular Diseases
Cardiovascular Diseases
Skin Diseases, Vascular
Skin Diseases
Hypersensitivity, Immediate
Immune System Diseases
Genetic Diseases, Inborn