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Trial details imported from ClinicalTrials.gov

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




Registration number
NCT01386658
Ethics application status
Date submitted
28/06/2011
Date registered
1/07/2011

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
Time to Peak Concentration (Tmax) of a Single Subcutaneous (SC) Dose of Icatibant
Timepoint [1] 0 0
Pre-dose; 0.25, 0.5, 0.75, 1, 2, 4 and 6 hours post-dose on Day 1
Primary outcome [2] 0 0
Maximum Plasma Concentration (Cmax) of a Single Subcutaneous (SC) Dose of Icatibant
Timepoint [2] 0 0
Pre-dose; 0.25, 0.5, 0.75, 1, 2, 4 and 6 hours post-dose on Day 1
Primary outcome [3] 0 0
Total Plasma Clearance (CL/F) of a Single Subcutaneous (SC) Dose of Icatibant
Timepoint [3] 0 0
Pre-dose; 0.25, 0.5, 0.75, 1, 2, 4 and 6 hours post-dose on Day 1
Primary outcome [4] 0 0
Area Under the Plasma Concentration-time Curve From Time Zero to 4 Hours Post-dose (AUC0-4) of a Single Subcutaneous (SC) Dose of Icatibant
Timepoint [4] 0 0
Pre-dose; 0.25, 0.5, 0.75, 1, 2, and 4 hours post-dose on Day 1
Primary outcome [5] 0 0
Area Under the Plasma Concentration-time Curve From Time Zero to 6 Hours Post-dose (AUC0-t) of a Single Subcutaneous (SC) Dose of Icatibant
Timepoint [5] 0 0
Pre-dose; 0.25, 0.5, 0.75, 1, 2, 4 and 6 hours post-dose on Day 1
Primary outcome [6] 0 0
Area Under the Plasma Concentration-time Curve From Time Zero to Infinity (AUC0-inf) of a Single Subcutaneous (SC) Dose of Icatibant
Timepoint [6] 0 0
Pre-dose; 0.25, 0.5, 0.75, 1, 2, 4 and 6 hours post-dose on Day 1
Primary outcome [7] 0 0
Volume of Distribution (Vz/F) of a Single Subcutaneous (SC) Dose of Icatibant
Timepoint [7] 0 0
Pre-dose; 0.25, 0.5, 0.75, 1, 2, 4 and 6 hours post-dose on Day 1
Primary outcome [8] 0 0
Elimination Half-life (t1/2) of a Single Subcutaneous (SC) Dose of Icatibant
Timepoint [8] 0 0
Pre-dose; 0.25, 0.5, 0.75, 1, 2, 4 and 6 hours post-dose on Day 1
Primary outcome [9] 0 0
Number of Participants With Clinically Significant Changes in Vital Signs
Timepoint [9] 0 0
Pre-dose up to 97 days post-dose
Primary outcome [10] 0 0
Number of Participants With Clinically Significant Changes in Electrocardiograms (ECGs)
Timepoint [10] 0 0
6 - 8 hours post-dose on Day 1
Primary outcome [11] 0 0
Number of Participants With Clinically Significant Changes in Clinical Laboratory Evaluations
Timepoint [11] 0 0
Pre-dose up to 97 days post-dose
Primary outcome [12] 0 0
Number of Participants Who Reported Presence of Anti-icatibant Antibodies
Timepoint [12] 0 0
Pre-dose up to 97 days post-dose
Primary outcome [13] 0 0
Number of Participants With Adverse Events (AEs)
Timepoint [13] 0 0
From the start of study drug administration up to 97 days post-dose
Primary outcome [14] 0 0
Number of Participants Who Reported Injection Site Reactions (ISR) for Icatibant Exposure Number 1
Timepoint [14] 0 0
1 h post-dose on Day 1 up to 9 days post-dose
Primary outcome [15] 0 0
Number of Participants Who Reported Injection Site Reactions (ISR) for Icatibant Exposure Number 2 and 3
Timepoint [15] 0 0
1 h post-dose up to 9 days post-dose
Primary outcome [16] 0 0
Number of Participants With Clinically Significant Changes in Reproductive Hormones
Timepoint [16] 0 0
Pre-dose up to 97 days post-dose
Secondary outcome [1] 0 0
Time to Onset of Symptom Relief (TOSR) for Composite Investigator-Assessed Symptom Scores for Icatibant Exposure Number 1
Timepoint [1] 0 0
From start of study drug administration up to 8.5 hours post-dose
Secondary outcome [2] 0 0
Time to Onset of Symptom Relief (TOSR) for Composite Investigator-Assessed Symptom Scores for Icatibant Exposure Number 2 and 3
Timepoint [2] 0 0
From start of study drug administration up to 12 hours post-dose
Secondary outcome [3] 0 0
Time to Onset of Symptom Relief (TOSR) for Faces Pain Scale-Revised (FPS-R) Scores for Icatibant Exposure Number 1
Timepoint [3] 0 0
From start of study drug administration up to 52 hours post-dose
Secondary outcome [4] 0 0
Time to Onset of Symptom Relief (TOSR) for Faces Pain Scale-Revised (FPS-R) Scores for Icatibant Exposure Number 2 and 3
Timepoint [4] 0 0
From start of study drug administration up to 28 hours post-dose
Secondary outcome [5] 0 0
Time to Onset of Symptom Relief (TOSR) for Faces, Legs, Activity, Cry, and Consolability (FLACC) Scores
Timepoint [5] 0 0
From start of study drug administration up to 8.5 hours post-dose
Secondary outcome [6] 0 0
Time to Minimum Symptoms for Composite Investigator-Assessed Symptom Scores for Icatibant Exposure Number 1
Timepoint [6] 0 0
From start of study drug administration up to 8.5 hours post-dose
Secondary outcome [7] 0 0
Time to Minimum Symptoms for Composite Investigator-Assessed Symptom Scores for Icatibant Exposure Number 2 and 3
Timepoint [7] 0 0
From start of study drug administration up to 12 hours post-dose
Secondary outcome [8] 0 0
Time to Minimum Symptom for Faces Pain Scale-Revised (FPS-R) Scores for Icatibant Exposure Number 1
Timepoint [8] 0 0
From start of study drug administration up to 52 hours post-dose
Secondary outcome [9] 0 0
Time to Minimum Symptom for Faces Pain Scale-Revised (FPS-R) Scores for Icatibant Exposure Number 2 and 3
Timepoint [9] 0 0
From start of study drug administration up to 28 hours post-dose
Secondary outcome [10] 0 0
Time to Minimum Symptom for Faces, Legs, Activity, Cry, and Consolability (FLACC) Scores
Timepoint [10] 0 0
From start of study drug administration up to 8.5 hours post-dose
Secondary outcome [11] 0 0
Time to Use of Rescue Medication for the Treatment of Symptoms of the Hereditary Angioedema (HAE) Attack Following Study Drug Administration
Timepoint [11] 0 0
From the start of study drug administration up to 52 hours post-dose
Secondary outcome [12] 0 0
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 1
Timepoint [12] 0 0
From 2 hours post-dose to 4 hours post-dose
Secondary outcome [13] 0 0
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
Timepoint [13] 0 0
From 2 hours post-dose to 4 hours post-dose

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
Sex
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
NA
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
27/01/2012
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
12/03/2018
Sample size
Target
Accrual to date
Final
32
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 0 0
Campbelltown Hospital - Campbelltown
Recruitment postcode(s) [1] 0 0
2560 - Campbelltown
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
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Study Director
Address 0 0
Takeda
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

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.

Supporting document/s available: Study protocol, Statistical analysis plan (SAP), Informed consent form (ICF), Clinical study report (CSR)
When will data be available (start and end dates)?
Available to whom?
IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: https://vivli.org/ourmember/takeda/


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT01386658