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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT01386658




Registration number
NCT01386658
Ethics application status
Date submitted
28/06/2011
Date registered
30/06/2011
Date last updated
21/03/2018

Titles & IDs
Public title
A Pharmacokinetic, Tolerability and Safety Study of Icatibant in Children and Adolescents With Hereditary Angioedema
Scientific title
A Multicenter, Open-Label, Non-Randomized Study to Assess the Pharmacokinetics, Tolerability, and Safety of a Single Subcutaneous Administration of Icatibant in Children and Adolescents With Hereditary Angioedema
Secondary ID [1] 0 0
2011-003825-81
Secondary ID [2] 0 0
HGT-FIR-086
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hereditary Angioedema (HAE) 0 0
Condition category
Condition code
Cardiovascular 0 0 0 0
Diseases of the vasculature and circulation including the lymphatic system
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Blood 0 0 0 0
Other blood disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - icatibant

Experimental: Icatibant - Single dose of icatibant 0.4 mg/kg subcutaneous(SC) up to a maximal dose of 30 mg


Treatment: Drugs: icatibant
Single dose of icatibant 0.4 mg/kg subcutaneous(SC) up to a maximal dose of 30 mg

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Pharmacokinetic (PK) Profile after a single SC injection (in prepubertal children with an acute attack of HAE and pubertal/postpubertal children with or without an acute attack of HAE) - PK parameter estimates will include, where appropriate: actual icatibant and metabolite concentrations at each sampling time, time to peak concentration (Tmax), actual peak (Cmax) and minimum (Cmin) concentrations, clearance (CL/F), actual area under the plasma concentration-time-curve (AUC0-last and AUC0-inf), mean residence time (MRT), volume of distribution at steady state (Vss/F) and elimination half-life (t1/2).
Timepoint [1] 0 0
Administration through 6 hours
Primary outcome [2] 0 0
Safety of a single SC dose of icatibant - Safety and tolerability will be assessed by standard criteria including injection site reactions, adverse events, vital signs, electrocardiogram (ECG) recordings, physical examination, clinical laboratory parameters (serum chemistry [including liver function tests], hematology, urinalysis), reproductive hormone levels, and immunogenicity (presence of anti-icatibant antibodies).
Timepoint [2] 0 0
Treatment through day 90
Secondary outcome [1] 0 0
Time to onset of relief of symptoms and time to minimal symptoms, as measured byinvestigator- and subject-reported outcomes (only for subjects treated with icatibant during a HAE attack) - • For subjects 2 to less than 18 years of age: investigator assessment and scoring of cutaneous, abdominal and laryngeal symptoms of acute HAE attacks by an investigator-rated symptom score.• For subjects 4 years of age and older only: subject self-assessment of HAE related pain using the Faces Pain Scale-Revised (FPS-R).• For subjects less than 4 years of age only: investigator assessment of HAE-related pain (cutaneous, abdominal and laryngeal) using a validated pain scale (Faces, Legs, Activity, Cry, and Consolability [FLACC]).
Timepoint [1] 0 0
Treatment through 8 hours
Secondary outcome [2] 0 0
Proportion of subjects with worsened intensity of clinical HAE symptoms between 2 and 4 hours after treatment with icatibant for subjects who have experienced HAE attack only
Timepoint [2] 0 0
Treatment through 4 hours
Secondary outcome [3] 0 0
Incidence of rescue medication use for subjects who have experienced HAE attack only - Rescue medications are only monitored through the current attack and not through 90 days
Timepoint [3] 0 0
Treatment through day 90

Eligibility
Key inclusion criteria
1. Two through <18 years of age at the time of first HAE attack.

- Prepubertal and pubertal/postpubertal subjects experiencing and acute cutaneous,
abdominal, or laryngeal HAE attack treated with icatibant as part of this study.

- Pubertal/postpubertal subjects with HAE who are treated with icatibant, but not
during an attack.

2. Documented diagnosis of HAE Type I or II.

3. Informed consent (and subject assent as appropriate) signed by the subject's
parent(s)or legal guardian(s).
Minimum age
2 Years
Maximum age
17 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Diagnosis of angioedema other than HAE.

2. Participation in another clinical trial that involves the use of any investigational
product (drug or device)within 30 days prior to study enrollment or at any time during
the study.

3. Any known factor/disease that might interfere with the treatment compliance, study
conduct,or result interpretation.

4. Congenital or acquired cardiac anomalies that interfere significantly with cardiac
function.

5. Treatment with ACE inhibitors within 7 days prior to treatment.

6. Use of hormonal contraception within the 90 days prior to treatment.

7. Androgen use (eg, stanozolol, danazol, oxandrolone, methyltestosterone, testosterone)
within the 90 days prior to treatment.

8. Pregnancy or breastfeeding.

9. A physical condition that interferes with pubertal status determination.

Study design
Purpose of the study
Treatment
Allocation to intervention
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Campbelltown Hospiital - New South Wales
Recruitment postcode(s) [1] 0 0
2560 - New South Wales
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Florida
Country [3] 0 0
United States of America
State/province [3] 0 0
Louisiana
Country [4] 0 0
United States of America
State/province [4] 0 0
Maryland
Country [5] 0 0
United States of America
State/province [5] 0 0
Massachusetts
Country [6] 0 0
United States of America
State/province [6] 0 0
Missouri
Country [7] 0 0
United States of America
State/province [7] 0 0
Ohio
Country [8] 0 0
United States of America
State/province [8] 0 0
Oklahoma
Country [9] 0 0
United States of America
State/province [9] 0 0
Oregon
Country [10] 0 0
United States of America
State/province [10] 0 0
Pennsylvania
Country [11] 0 0
United States of America
State/province [11] 0 0
Texas
Country [12] 0 0
Austria
State/province [12] 0 0
Graz
Country [13] 0 0
Canada
State/province [13] 0 0
Ontario
Country [14] 0 0
Colombia
State/province [14] 0 0
Cundinamarca
Country [15] 0 0
Germany
State/province [15] 0 0
Frankfurt
Country [16] 0 0
Germany
State/province [16] 0 0
Mainz
Country [17] 0 0
Germany
State/province [17] 0 0
Walldorf
Country [18] 0 0
Hungary
State/province [18] 0 0
Budapest
Country [19] 0 0
Israel
State/province [19] 0 0
Haifa
Country [20] 0 0
Israel
State/province [20] 0 0
Tel Aviv
Country [21] 0 0
Israel
State/province [21] 0 0
Tel Hashomer
Country [22] 0 0
Italy
State/province [22] 0 0
Naples
Country [23] 0 0
Spain
State/province [23] 0 0
Madrid
Country [24] 0 0
Spain
State/province [24] 0 0
Valencia

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Shire
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
HGT-FIR-086 is a multicenter, open-label, non-randomized, single-arm study to evaluate the
Pharmacokinetics, tolerability,safety, and efficacy on reproductive hormones, of a single
subcutaneous (SC) administration of icatibant in approximately 30 pediatric subjects with
Hereditary Angioedema (HAE) during an initial acute attack.
Trial website
https://clinicaltrials.gov/show/NCT01386658
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Study Physician
Address 0 0
Shire Orphan Therapies, LLC
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

No data has been provided for results reporting
Summary results
Other publications