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

NCT ID: NCT01386658

Last Updated: 2021-06-08

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 32 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2012-01-27
• Study Completion Date: 2018-03-12

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.

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.

Conditions

Hereditary Angioedema (HAE)

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Icatibant

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

Group Type: EXPERIMENTAL

icatibant

Intervention Type: DRUG

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

Interventions

icatibant

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

Intervention Type: DRUG

Other Intervention Names

Firazyr

Eligibility Criteria

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.

• Minimum Eligible Age: 2 Years
• Maximum Eligible Age: 17 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Shire

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Study Director

Role: STUDY_DIRECTOR

Takeda

Locations

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

Unidad de Alergia, Edif. Consultas Externas, Planta Baja HOSPITAL UNIVERSITARIO LA PAZ

Madrid, , Spain

University Hospital, Pediatric Pulmonology and Allergy Unit

Valencia, , Spain

Countries

United States; Australia; Austria; Canada; Colombia; Germany; Hungary; Israel; Italy; Spain

References

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.

Reference Type: DERIVED

PMID: 28601641 (View on PubMed)

Provided Documents

Document Type: Study Protocol: Protocol Original

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Document Type: Study Protocol: Protocol Amendment 1

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Document Type: Study Protocol: Protocol Amendment 2

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Document Type: Study Protocol: Protocol Amendment 3

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Document Type: Study Protocol: Protocol Amendment 4

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Document Type: Statistical Analysis Plan

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Other Identifiers

2011-003825-81

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

HGT-FIR-086

Identifier Type: -

Identifier Source: org_study_id