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

Shire

Status and phase: Withdrawn; Phase 3; Phase 2

Conditions: Hereditary Angioedema (HAE)

Treatments: Biological: ecallantide

Study type: Interventional

Funder types: Industry

Identifiers

NCT01253382

2010-022716-39 (EudraCT Number)

DX-88/26

Details and patient eligibility

About

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

Sex: All

Ages: 2 to 17 years old

Volunteers: No 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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Single Group Assignment

Masking: Quadruple Blind

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0 participants in 2 patient groups, including a placebo group

ecallantide

Active Comparator group

Treatment:

Biological: ecallantide

placebo

Placebo Comparator group

Description:

phosphate buffered saline

Treatment:

Biological: ecallantide