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

NCT01253382 | Withdrawn Phase 2 DMC

• 2 other identifiers: DX-88/262010-022716-39
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

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

Conditions

Hereditary Angioedema (HAE)

Outcome Measures

Primary Outcomes (1)

• safety and tolerability of ecallantide

  4 years

Secondary Outcomes (2)

• evaluate the PK profile of ecallantide

  4 years

• assess the efficacy of ecallantide

  4 years

Study Arms (2)

ecallantide

ACTIVE COMPARATOR

Biological: ecallantide

placebo

PLACEBO COMPARATOR

phosphate buffered saline

Biological: ecallantide

Interventions

ecallantide BIOLOGICAL

10 - 30mg subcutaneous injection.

ecallantide; placebo

Eligibility Criteria

• Age: 2 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• \<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

Sponsors & Collaborators

• Shire: lead

MeSH Terms

Conditions

Angioedemas, Hereditary

Interventions

ecallantide

Condition Hierarchy (Ancestors)

Angioedema; Vascular Diseases; Cardiovascular Diseases; Hereditary Complement Deficiency Diseases; Primary Immunodeficiency Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Urticaria; Skin Diseases, Vascular; Skin Diseases; Skin and Connective Tissue Diseases; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases; Immunologic Deficiency Syndromes

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 2, 2010
• First Posted: December 3, 2010
• Study Start: June 1, 2012
• Primary Completion: April 1, 2015
• Last Updated: May 18, 2021

Record last verified: 2021-05