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

Phase 2

Withdrawn

• Conditions: Hereditary Angioedema (HAE)
• Interventions: Biological: ecallantide

• Registration Number: NCT01253382
• Lead Sponsor: Shire

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

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2010-12-02
• Study First Submitted QC: 2010-12-02
• Study First Posted: 2010-12-03
• Study Start: 2012-06-01
• Primary Completion: 2015-04-01
• Status Verified: 2021-05
• Last Update Submitted: 2021-05-14
• Last Update Posted: 2021-05-18

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: Not specified

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
ecallantide	ecallantide	-
placebo	ecallantide	phosphate buffered saline

Primary Outcome Measures

Name	Time	Method
safety and tolerability of ecallantide	4 years

Secondary Outcome Measures

Name	Time	Method
assess the efficacy of ecallantide	4 years
evaluate the PK profile of ecallantide	4 years