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

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)

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

Study Overview

• Status: Withdrawn
• Conditions: Hereditary Angioedema (HAE)
• Intervention / Treatment: Biological: ecallantide

• Study Type: Interventional
• Phase: Phase 2; Phase 3

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years to 17 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Single Group Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
safety and tolerability of ecallantide	4 years

Secondary Outcome Measures

Outcome Measure	Time Frame
evaluate the PK profile of ecallantide	4 years
assess the efficacy of ecallantide	4 years

Collaborators and Investigators

• Sponsor: Shire

Study record dates

Study Major Dates

• Study Start (Actual): June 1, 2012
• Primary Completion (Anticipated): April 1, 2015

Study Registration Dates

• First Submitted: December 2, 2010
• First Submitted That Met QC Criteria: December 2, 2010
• First Posted (Estimate): December 3, 2010

Study Record Updates

• Last Update Posted (Actual): May 18, 2021
• Last Update Submitted That Met QC Criteria: May 14, 2021
• Last Verified: May 1, 2021

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Cardiovascular Diseases; Vascular Diseases; Skin Diseases; Immunologic Deficiency Syndromes; Immune System Diseases; Hypersensitivity, Immediate; Genetic Diseases, Inborn; Skin Diseases, Vascular; Hypersensitivity; Urticaria; Hereditary Complement Deficiency Diseases; Primary Immunodeficiency Diseases; Angioedema; Angioedemas, Hereditary; Physiological Effects of Drugs; Peripheral Nervous System Agents; Analgesics; Sensory System Agents; Anti-Inflammatory Agents, Non-Steroidal; Analgesics, Non-Narcotic; Anti-Inflammatory Agents; Antirheumatic Agents; Ecallantide
• Other Study ID Numbers: DX-88/26; 2010-022716-39 (EudraCT Number)