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NCT01386658·Phase 3·INTERVENTIONAL

A Pharmacokinetic, Tolerability and Safety Study of Icatibant in Children and Adolescents With Hereditary Angioedema

IcatibantOtherCompleted

Status

Completed

Phase

Phase 3

Enrollment

32

Locations

26

Results

Posted

Publications

1

Study summary

What the protocol is testing.

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.

Full detailed description

Study HGT-FIR-086 will enroll 30 subjects from 2 to less than 18 years of age, divided into 2 groups: prepubertal and pubertal/postpubertal. At least 10 prepubertal children and at least 20 adolescents (including 10 treated during a HAE attack) must be enrolled in the study. After a qualifying screening period, the PK, safety/tolerability, and efficacy of treatment with SC icatibant will be evaluated in at least 20 subjects (10 prepubertal and 10 pubertal/postpubertal subjects) who present with cutaneous, abdominal, or laryngeal symptoms of an acute attack of HAE. The PK and safety/tolerability of SC icatibant will be evaluated in at least 10 additional pubertal/postpubertal subjects who meet screening criteria and receive treatment with SC icatibant in the absence of a current acute HAE attack. The planned duration of active participation for subjects who present with an initial attack of acute HAE will consist of treatment with a single subcutaneous injection of icatibant on Day 1 through follow up at day 90. After having received initial treatment with icatibant, either during or in the absence of an attack, at least 10 pubertal/postpubertal subjects who subsequently experience an acute HAE attack may continue to receive treatment with icatibant as a single SC administration per attack for a total of 3 eligible icatibant-treated attacks. The period of active participation in the study for prepubertal subjects will be approximately 90 days, while that for pubertal/postpubertal subjects could be a maximum of approximately 270 or 360 days (3 separate active periods of approximately 90 days for those treated with icatibant during an attack; 4 separate active periods for those treated without an attack), with each active period separated by periods of inactive participation of variable duration.

Interventions

Treatment arms and agents.

DRUG

icatibant

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

Timeline

From registration to results.

  1. First posted

    Jul 1, 2011

  2. Study start

    Jan 27, 2012

  3. Primary completion

    Mar 12, 2018

  4. Study completion

    Mar 12, 2018

  5. Results posted

    Mar 25, 2019

  6. Registry updated

    Jun 8, 2021

Outcomes

What the study measures.

Primary outcomes

Time to Peak Concentration (Tmax) of a Single Subcutaneous (SC) Dose of Icatibant

Time frame · Pre-dose; 0.25, 0.5, 0.75, 1, 2, 4 and 6 hours post-dose on Day 1

Time to peak concentration (Tmax) of a single SC dose of icatibant was reported.

Maximum Plasma Concentration (Cmax) of a Single Subcutaneous (SC) Dose of Icatibant

Time frame · Pre-dose; 0.25, 0.5, 0.75, 1, 2, 4 and 6 hours post-dose on Day 1

Maximum plasma concentration (Cmax) of a single SC dose of icatibant was reported.

Total Plasma Clearance (CL/F) of a Single Subcutaneous (SC) Dose of Icatibant

Time frame · Pre-dose; 0.25, 0.5, 0.75, 1, 2, 4 and 6 hours post-dose on Day 1

Total plasma clearance (CL/F) of a single SC dose of icatibant was reported.

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

Time frame · Pre-dose; 0.25, 0.5, 0.75, 1, 2, and 4 hours post-dose on Day 1

Area under the plasma concentration-time curve from time zero to 4 hours post-dose (AUC0-4) of a single SC dose of icatibant was reported.

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

Time frame · Pre-dose; 0.25, 0.5, 0.75, 1, 2, 4 and 6 hours post-dose on Day 1

Area under the plasma concentration-time curve from time zero to 6 hours post-dose (AUC0-t) of a single SC dose of icatibant was reported.

Area Under the Plasma Concentration-time Curve From Time Zero to Infinity (AUC0-inf) of a Single Subcutaneous (SC) Dose of Icatibant

Time frame · Pre-dose; 0.25, 0.5, 0.75, 1, 2, 4 and 6 hours post-dose on Day 1

Area under the plasma concentration-time curve from time zero to infinity (AUC0-inf) of a single SC dose of icatibant was reported.

Volume of Distribution (Vz/F) of a Single Subcutaneous (SC) Dose of Icatibant

Time frame · Pre-dose; 0.25, 0.5, 0.75, 1, 2, 4 and 6 hours post-dose on Day 1

Volume of distribution (Vz/F) of a single SC dose of icatibant was reported.

Elimination Half-life (t1/2) of a Single Subcutaneous (SC) Dose of Icatibant

Time frame · Pre-dose; 0.25, 0.5, 0.75, 1, 2, 4 and 6 hours post-dose on Day 1

Elimination half-life (t1/2) of a single SC dose of icatibant was reported.

Secondary outcomes

Time to Onset of Symptom Relief (TOSR) for Composite Investigator-Assessed Symptom Scores for Icatibant Exposure Number 1

Time frame · From start of study drug administration up to 8.5 hours post-dose

The TOSR was defined as the duration of time in hours from study drug administration to the earliest time post-treatment at which there was at least a 20 percent (%) improvement in the average post-treatment symptom score with no worsening of any single component score for the initial icatibant exposure. The investigator used a symptom score to assess the severities of symptoms of acute cutaneous, abdominal, and laryngeal attacks of hereditary angioedema (HAE) using the following 5-point scale: 0=none (absence of symptoms), 1=mild (no to mild interference with daily activities), 2=moderate (moderate interference with daily activities), 3=severe (severe interference with daily activities) and 4=very severe (very severe interference with daily activities). TOSR for participants who received initial icatibant administration was reported.

Time to Onset of Symptom Relief (TOSR) for Composite Investigator-Assessed Symptom Scores for Icatibant Exposure Number 2 and 3

Time frame · From start of study drug administration up to 12 hours post-dose

The TOSR was defined as the duration of time in hours from study drug administration to the earliest time post-treatment at which there was at least a 20% improvement in the composite (or average) post-treatment symptom score with no worsening of any single component score. The investigator used a symptom score to assess the severities of symptoms of acute cutaneous, abdominal, and laryngeal attacks of HAE using the following 5-point scale: 0=none (absence of symptoms), 1=mild (no to mild interference with daily activities), 2=moderate (moderate interference with daily activities), 3=severe (severe interference with daily activities) and 4=very severe (very severe interference with daily activities). TOSR for participants who received subsequent icatibant administration by HCP administration or by caregiver/ self-administration was reported.

Time to Onset of Symptom Relief (TOSR) for Faces Pain Scale-Revised (FPS-R) Scores for Icatibant Exposure Number 1

Time frame · From start of study drug administration up to 52 hours post-dose

The TOSR was defined as the earliest time at which the post-treatment score improved by at least one level. Participants of 4 years age and older self-assessed their HAE-related pain using the FPS-R instrument. FPS-R is a self-reported measure used to assess the intensity of children's pain and it is scored using a 0 to 10 scale (0=no pain to 10=very much pain). TOSR for participants who received initial icatibant administration was reported.

Time to Onset of Symptom Relief (TOSR) for Faces Pain Scale-Revised (FPS-R) Scores for Icatibant Exposure Number 2 and 3

Time frame · From start of study drug administration up to 28 hours post-dose

The TOSR was defined as the earliest time at which the post-treatment score improved by at least one level. Participants of 4 years age and older self-assessed their HAE-related pain using the FPS-R instrument. FPS-R is a self-reported measure used to assess the intensity of children's pain and it is scored using a 0 to 10 scale (0=no pain to 10=very much pain). TOSR for participants who received subsequent icatibant administration by HCP administration or by caregiver/ self-administration was reported.

Time to Onset of Symptom Relief (TOSR) for Faces, Legs, Activity, Cry, and Consolability (FLACC) Scores

Time frame · From start of study drug administration up to 8.5 hours post-dose

The TOSR was defined as the earliest time at which a 20% improvement was seen in the total post-treatment score. Participants of 4 years age and younger underwent investigator assessment of HAE-related pain (cutaneous, abdominal, and laryngeal) using the FLACC comportmental pain scale. Each of the 5 categories was scored from 0 to 2. Face(F): 0 (no particular expression/smile) - 2 (frequent to constant frown clenched jaw quivering chin); Legs(L): 0 (normal position/relaxed) - 2 (kicking/legs drawn up); Activity(A): 0 (lying quietly, normal position, moves easily) - 2 (arched rigid/jerking); Cry(C): 0 (No cry \[awake/asleep\]) - 2 (crying steadily/screams/sobs or frequent complaints); Consolability(C): 0 (content/relaxed) - 2 (difficult to console/comfort), resulting in a total score between 0 and 10.

Time to Minimum Symptoms for Composite Investigator-Assessed Symptom Scores for Icatibant Exposure Number 1

Time frame · From start of study drug administration up to 8.5 hours post-dose

Time to minimum symptom was defined as the duration of time in hours from study drug administration to the earliest time post-treatment at which all symptoms were either mild or absent for the investigator-reported symptom score. The investigator used a symptom score to assess the severities of symptoms of acute cutaneous, abdominal, and laryngeal attacks of HAE using the following 5-point scale: 0=none (absence of symptoms), 1=mild (no to mild interference with daily activities), 2=moderate (moderate interference with daily activities), 3=severe (severe interference with daily activities) and 4=very severe (very severe interference with daily activities). Time to minimum symptom for participants who received initial icatibant administration was reported.

Time to Minimum Symptoms for Composite Investigator-Assessed Symptom Scores for Icatibant Exposure Number 2 and 3

Time frame · From start of study drug administration up to 12 hours post-dose

Time to minimum symptom was defined as the duration of time in hours from study drug administration to the earliest time post-treatment at which all symptoms were either mild or absent for the investigator-reported symptom score. The investigator used a symptom score to assess the severities of symptoms of acute cutaneous, abdominal, and laryngeal attacks of HAE using the following 5-point scale: 0=none (absence of symptoms), 1=mild (no to mild interference with daily activities), 2=moderate (moderate interference with daily activities), 3=severe (severe interference with daily activities) and 4=very severe (very severe interference with daily activities). Time to minimum symptom for participants who received subsequent icatibant administration by HCP administration or by caregiver/ self-administration was reported.

Time to Minimum Symptom for Faces Pain Scale-Revised (FPS-R) Scores for Icatibant Exposure Number 1

Time frame · From start of study drug administration up to 52 hours post-dose

Time to minimum symptoms was defined as the duration of time in hours from study drug administration to the earliest time at which post-treatment score improved to zero (or no pain). Participants of 4 years of age and older self-assessed their HAE-related pain using the FPS-R instrument. FPS-R is a self-reported measure used to assess the intensity of children's pain and it is scored using a 0 to 10 scale (0=no pain to 10=very much pain). Time to minimum symptom for participants who received initial icatibant administration was reported.

Time to Minimum Symptom for Faces Pain Scale-Revised (FPS-R) Scores for Icatibant Exposure Number 2 and 3

Time frame · From start of study drug administration up to 28 hours post-dose

Time to minimum symptoms was defined as the duration of time in hours from study drug administration to the earliest time at which post-treatment score improved to zero (or no pain). Participants of 4 years of age and older self-assessed their HAE-related pain using the FPS-R instrument. FPS-R is a self-reported measure used to assess the intensity of children's pain and it is scored using a 0 to 10 scale (0=no pain to 10=very much pain). Time to minimum symptom for participants who received subsequent icatibant administration by HCP administration or by caregiver/ self-administration was reported.

Time to Minimum Symptom for Faces, Legs, Activity, Cry, and Consolability (FLACC) Scores

Time frame · From start of study drug administration up to 8.5 hours post-dose

Time to minimum symptoms was defined as the duration of time in hours from study drug administration to the earliest time at which the total post-treatment score improved to zero. Participants of 4 years age and younger underwent investigator assessment of HAE-related pain (cutaneous, abdominal, and laryngeal) using the FLACC comportmental pain scale. Each of the 5 categories was scored from 0 to 2. (F) Face: 0 (no particular expression/smile) - 2 (frequent to constant frown clenched jaw quivering chin); (L) Legs: 0 (normal position/relaxed) - 2 (kicking/legs drawn up); (A) Activity: 0 (lying quietly, normal position, moves easily) - 2 (arched rigid/jerking); (C) Cry: 0 (No cry \[awake/asleep\]) - 2 (crying steadily/screams/sobs or frequent complaints); (C) Consolability: 0 (content/relaxed) - 2 (difficult to console/comfort), resulting in a total score between 0 and 10.

Eligibility

Who can take part.

Minimum age
2 Years
Maximum age
17 Years
Sex
ALL
Healthy volunteers
No

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). 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 locations

26 registered sites.

Australia · Austria · Canada · Colombia · Germany · Hungary · Israel · Italy · Spain · United States. Showing up to 24 locations stored in the fast local snapshot.

Children's Hospital Los Angeles

Los Angeles, California, United States

University of South Florida

Tampa, Florida, United States

Breathe America

Shreveport, Louisiana, United States

Institute for Asthma and Allergy, PC

Chevy Chase, Maryland, United States

Boston Children's Hospital

Boston, Massachusetts, United States

Washington University School of Medicine

St Louis, Missouri, United States

Bernstein Clinical Research Center, LLC

Cincinnati, Ohio, United States

Toledo Institute of Clinical Research

Toledo, Ohio, United States

Oklahoma Institute of Allergy and Asthma Clinical Research, LLC

Oklahoma City, Oklahoma, United States

Allergy Asthma Dermatology Research Center

Lake Oswego, Oregon, United States

Penn State University

Hershey, Pennsylvania, United States

AARA Research Center

Dallas, Texas, United States

Campbelltown Hospital

Campbelltown, New South Wales, Australia

Medizinische Universität Graz Hautklinik

Graz, Austria

McMaster University

Hamilton, Ontario, Canada

Hospital Infantil Universitario de San Jose

Bogota, Cundinamarca, Colombia

Klinikum der Johann Wolfgang Goethe University

Frankfurt, Germany

Johannes-Gutenberg University Clinical Research Center

Mainz, Germany

HZRM Hämophilie Zentrum Rhein Main GmbH

Walldorf, Germany

Heim Pal Childrens Hospital

Budapest, Hungary

Bnai Zion Medical Center, Allergy and Immunology Institute

Haifa, Israel

Tel Aviv Sourasky Medical Center, Pulmonology and Allergy Unit

Tel Aviv, Israel

Sheba Medical Center Allergy and Immunology Angioedema Center

Tel Litwinsky, Israel

University of Naples Federico II, Dipartimento di Medicina Interna

Naples, Italy

Publications

Results and literature.

PMID 28601641 ↗Farkas H, Reshef A, Aberer W, Caballero T, McCarthy L, Hao J, Nothaft W, Schranz J, Bernstein JA, Li HH. Treatment Effect and Safety of Icatibant in Pediatric Patients with Hereditary Angioedema. J Allergy Clin Immunol Pract. 2017 Nov-Dec;5(6):1671-1678.e2. doi: 10.1016/j.jaip.2017.04.010. Epub 2017 Jun 7.

Primary links

Continue at the source.

ClinicalTrials.gov ↗Search PubMed ↗Search FDA ↗Search EMA ↗

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