Back to results

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

Primary Purpose

Hereditary Angioedema (HAE)

Status

Withdrawn

Phase

Phase 2

Locations

Study Type

Interventional

Intervention

ecallantide

About this trial

This is an interventional treatment trial for Hereditary Angioedema (HAE)

Eligibility Criteria

2 Years - 17 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Male or female from 2 years of age and prepubertal
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
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.
Must have signed informed consent by parent or caregiver.

Exclusion Criteria:

<2 years of age or have reached puberty
Received treatment with ecallantide within previous 72 hours
Received an investigational drug or device, other than ecallantide, within 30 days prior to the screening visit
Pharyngeal/laryngeal symptoms
Mild attacks including mild edema of the extremities and mild abdominal attacks
Are unable or unwilling to give informed consent (parent or caregiver)
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

Sites / Locations

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

Placebo Comparator

Arm Label

ecallantide

placebo

Arm Description

phosphate buffered saline

Outcomes

Primary Outcome Measures

safety and tolerability of ecallantide

Secondary Outcome Measures

evaluate the PK profile of ecallantide

assess the efficacy of ecallantide

Full Information

NCT ID

NCT01253382

First Posted

December 2, 2010

Last Updated

May 14, 2021

Sponsor

Shire

1. Study Identification

Unique Protocol Identification Number

NCT01253382

Brief Title

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

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 Type

Interventional

2. Study Status

Record Verification Date

May 2021

Overall Recruitment Status

Withdrawn

Study Start Date

June 1, 2012 (Actual)

Primary Completion Date

April 1, 2015 (Anticipated)

Study Completion Date

undefined (undefined)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Shire

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Hereditary Angioedema (HAE)

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2, Phase 3

Interventional Study Model

Single Group Assignment

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

0 (Actual)

8. Arms, Groups, and Interventions

Arm Title

ecallantide

Arm Type

Active Comparator

Arm Title

placebo

Arm Type

Placebo Comparator

Arm Description

phosphate buffered saline

Intervention Type

Biological

Intervention Name(s)

ecallantide

Intervention Description

10 - 30mg subcutaneous injection.

Primary Outcome Measure Information:

Title

safety and tolerability of ecallantide

Time Frame

4 years

Secondary Outcome Measure Information:

Title

evaluate the PK profile of ecallantide

Time Frame

4 years

Title

assess the efficacy of ecallantide

Time Frame

4 years

10. Eligibility

Sex

All

Minimum Age & Unit of Time

2 Years

Maximum Age & Unit of Time

17 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Male or female from 2 years of age and prepubertal 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 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. Must have signed informed consent by parent or caregiver. Exclusion Criteria: <2 years of age or have reached puberty Received treatment with ecallantide within previous 72 hours Received an investigational drug or device, other than ecallantide, within 30 days prior to the screening visit Pharyngeal/laryngeal symptoms Mild attacks including mild edema of the extremities and mild abdominal attacks Are unable or unwilling to give informed consent (parent or caregiver) 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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Study Director

Organizational Affiliation

Takeda

Official's Role

Study Director

12. IPD Sharing Statement

Learn more about this trial

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

We'll reach out to this number within 24 hrs