A 3-Part Study to Evaluate the Pharmacokinetics, Safety, and Efficacy of Subcutaneous Ecallantide in Prepubertal Paediatric Patients Experiencing Acute Attacks of Hereditary Angioedema

Phase 1

• Conditions: Hereditary Angioedema; MedDRA version: 12.1Level: LLTClassification code 10019860Term: Hereditary angioedema

• Registration Number: EUCTR2010-022716-39-DE
• Lead Sponsor: Dyax Corp.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2010-12-06
• Last Update Posted: 6 November 2023

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 22

Inclusion Criteria

Each patient must meet the following criteria:

1.Male or female patient from 2 years of age and prepubertal (up through Tanner Stage 1; at the screening visit)
2.Physician diagnosis and history of HAE (Type I or II) including laboratory results showing C1-INH activity below the lower limit of normal or up to 15% above the lower limit of the normal range, as defined by the reference laboratory (at the screening visit)
3.Present at the site with moderate (interferes with normal daily activities) to severe (inability to perform normal daily activities) signs and symptoms of an acute attack of HAE within 8 hours of recognition of the onset of the attack (at pre-dose of Parts 1, 2, and 3). Spontaneous resolution of the attack must not have begun before the administration of study drug
4.Must have a signed informed consent by parent or caregiver

Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Patients who meet any of the following criteria will be excluded from the study.
1.Are <2 years of age or have reached puberty (as assessed by Tanner Stage 2; at the screening visit)
2.Received treatment with ecallantide within previous 72 hours (at pre-dose of Parts 1, 2, and 3)
3.Received an investigational (non-licensed) drug or device, other than ecallantide, within 30 days prior to the screening visit
4.For Part 2 only: pharyngeal/laryngeal symptoms (at pre-dose of Part 2)
5.Mild attacks including mild edema of the extremities and mild abdominal attacks (at pre-dose of Parts 1, 2, and 3)
6.Are unable or unwilling to give informed consent (patient 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 (at the screening visit and at pre-dose of Parts 1, 2, and 3)

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: •To assess the safety and tolerability of ecallantide in paediatric patients treated for acute attacks of HAE;Secondary Objective: •To evaluate the PK profile of ecallantide in paediatric patients treated for acute attacks of HAE<br>•To assess the efficacy of ecallantide in paediatric patients treated for moderate (interferes with normal daily activities) to severe (inability to perform normal daily activities) acute attacks of HAE<br>;Primary end point(s): Safety and tolerability of ecallantide. This will include vital signs, electrocardiogram (ECG), physical examination, clinical laboratory parameters (serum chemistry including liver function tests, haematology, coagulation, anti ecallantide antibodies), and adverse events (AEs).