Study to Assess the Tolerability and Safety of Ecallantide in Children and Adolescents With Hereditary Angioedema

NCT01832896 | Withdrawn Phase 2

• 1 other identifier: DX-88/IST-5
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

The objective of this study is to evaluate the safety and tolerability of a single Subcutaneous (SC) dose of Ecallantide in children and adolescents with Hereditary Angioedema (HAE).

Conditions

Hereditary Angioedema Types I and II

Keywords

Hereditary Angioedema; Children; Adolescents; Ecallantide

Outcome Measures

Primary Outcomes (1)

• Measurement of time to symptomatic improvement from acute attacks of hereditary angioedema

  Time to symptom relief. Time to minimal symptoms. An improvement assessment will also be performed at each of the following timepoints: pre -treatment, every 30 minutes for the first 2 hours and then hourly through discharge and at the 28 day clinic visit. Incidence of need for rescue medication. Incidence of worsening despite use of ecallantide.

  28 days

Secondary Outcomes (1)

• Number of Partcipants with Adverse Events

  28 Days

Study Arms (1)

Ecallantide

EXPERIMENTAL

Study Medication, Dose, and Mode of Administration: Single dose of ecallantide subcutaneous dosing: * Age less than 10: Weight \<25 Kg: 10mg subcutaneously at one site; 25-50kg: 20mg subcutaneously, 10mg per site for 2 separate sites; \>50 kg 30mg subcutaneously, 10mg per site for 3 separate sites. Dosing will not exceed 30mg. * Age greater than 10: 10mg per site for 3 separate sites. Dosing will not exceed 30mg.

Drug: Ecallantide subcutaneous dosing

Interventions

Ecallantide subcutaneous dosing DRUG

For acute attacks of Hereditary Angioedema in children and adolescents, Ecallantide will be administered.

Ecallantide

Eligibility Criteria

• Age: 2 Years - 16 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• through 15 years of age, inclusive (ie, from the second birthday through the day prior to the sixteenth birthday) at the time of the subject's first attack.
• Documented diagnosis of HAE type I or II. Diagnosis must be confirmed by a documented immunogenic (below the lower limit of normal) and/or functional (\< 50% of normal levels) C1- Inhibitor deficiency. Diagnosis may be on the basis of historic data or by diagnostic testing conducted at the time of screening.
• Informed consent (and subject assent as appropriate) signed by the subject's parent(s) or legal guardian(s).

You may not qualify if:

• History of an adverse reaction (AE) to Ecallantide in the past
• Diagnosis of angioedema other than HAE
• Participation in another clinical study during the 30 days prior to treatment
• Any known factor/disease that might interfere with the treatment compliance, study conduct, or result interpretation
• Congenital or acquired cardiac anomalies that interfere significantly with cardiac function.
• Treatment with angiotensin converting enzyme (ACE) inhibitors within 7 days prior to treatment.
• Use of hormonal contraception within the 90 days prior to treatment for females of childbearing potential
• The subject is pregnant or breastfeeding

Sponsors & Collaborators

• NYU Langone Health: lead
• Dyax Corp.: collaborator

Study Sites (1)

Winthrop-University Hosptial Clinical Trials Center

Mineola, New York, 11501, United States

Location

MeSH Terms

Conditions

Hereditary Angioedema Types I and II; Angioedemas, Hereditary

Condition Hierarchy (Ancestors)

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

Study Officials

• Mark A Davis-Lorton, MD

  Winthrop University Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 4, 2013
• First Posted: April 16, 2013
• Study Start: August 1, 2013
• Primary Completion: July 1, 2017
• Study Completion: December 1, 2017
• Last Updated: March 31, 2023

Record last verified: 2017-01

Locations

US (1)