Safety and Efficacy Study of CINRYZE for Prevention of Angioedema Attacks in Children Ages 6-11 With Hereditary Angioedema

NCT ID: NCT02052141

Last Updated: 2021-06-03

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

12 participants

Study Classification

INTERVENTIONAL

Study Start Date

2014-03-20

Study Completion Date

2017-05-04

Brief Summary

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Primary Objective - To assess the relative efficacy of two dose levels of CINRYZE (500 Units and 1000 Units) administered by intravenous (IV) injection every 3 or 4 days to prevent angioedema attacks in children 6 to 11 years of age with hereditary angioedema (HAE).

Secondary Objectives - To assess the safety and tolerability, characterize the pharmacokinetics (PK) and pharmacodynamics (PD), and assess the immunogenicity of two dose levels of CINRYZE administered by IV injection in children 6 to 11 years of age with HAE.

Detailed Description

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Conditions

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Hereditary Angioedema (HAE)

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

Primary Study Purpose

PREVENTION

Blinding Strategy

SINGLE

Participants

Study Groups

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500/1000

500 Units of CINRYZE administered by IV injection twice per week for 12 weeks followed by 1000 Units of CINRYZE administered by IV injection twice per week for 12 weeks

Group Type EXPERIMENTAL

CINRYZE 500

Intervention Type BIOLOGICAL

500 Units of CINRYZE administered by IV injection

CINRYZE 1000

Intervention Type BIOLOGICAL

1000 Units of CINRYZE administered by IV injection

1000/500

1000 Units of CINRYZE administered by IV injection twice per week for 12 weeks followed by 500 Units of CINRYZE administered by IV injection twice per week for 12 weeks

Group Type EXPERIMENTAL

CINRYZE 500

Intervention Type BIOLOGICAL

500 Units of CINRYZE administered by IV injection

CINRYZE 1000

Intervention Type BIOLOGICAL

1000 Units of CINRYZE administered by IV injection

Interventions

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CINRYZE 500

500 Units of CINRYZE administered by IV injection

Intervention Type BIOLOGICAL

CINRYZE 1000

1000 Units of CINRYZE administered by IV injection

Intervention Type BIOLOGICAL

Eligibility Criteria

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Inclusion Criteria

* Diagnosis of Type I or Type II HAE.
* History of angioedema attacks.

Exclusion Criteria

* History of bleeding or clotting abnormality.
* Diagnosis of acquired angioedema or known to have C1 INH antibodies.
* History of allergic reaction to C1 esterase inhibitor or other blood products.
* Receipt of any experimental agents other than those required for prevention or treatment of angioedema attacks within 30 days prior to screening.
Minimum Eligible Age

6 Years

Maximum Eligible Age

11 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Shire

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Study Director

Role: STUDY_DIRECTOR

Takeda

Locations

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Asthma and Allergy Associates, P.C

Colorado Springs, Colorado, United States

Site Status

Nevada Access to Research and Education Society

Las Vegas, Nevada, United States

Site Status

Oregon Allergy Associates

Eugene, Oregon, United States

Site Status

Klinikum der J.W. Goethe Universitat

Frankfurt, , Germany

Site Status

HZRM Hamophilie Zentrum Rhein Main GmbH

Mörfelden-Walldorf, , Germany

Site Status

Tel Aviv Sourasky Medical Center

Tel Aviv, , Israel

Site Status

Instituto Nacional de Pediatria

Mexico City, , Mexico

Site Status

Clinical County Hospital Mures

Târgu Mureş, , Romania

Site Status

Countries

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United States Germany Israel Mexico Romania

References

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Aygoren-Pursun E, Soteres D, Moldovan D, Christensen J, Van Leerberghe A, Hao J, Schranz J, Jacobson KW, Martinez-Saguer I. Preventing Hereditary Angioedema Attacks in Children Using Cinryze(R): Interim Efficacy and Safety Phase 3 Findings. Int Arch Allergy Immunol. 2017;173(2):114-119. doi: 10.1159/000477541. Epub 2017 Jun 30.

Reference Type RESULT
PMID: 28662509 (View on PubMed)

Beard N, Frese M, Smertina E, Mere P, Katelaris C, Mills K. Interventions for the long-term prevention of hereditary angioedema attacks. Cochrane Database Syst Rev. 2022 Nov 3;11(11):CD013403. doi: 10.1002/14651858.CD013403.pub2.

Reference Type DERIVED
PMID: 36326435 (View on PubMed)

Aygoren-Pursun E, Soteres DF, Nieto-Martinez SA, Christensen J, Jacobson KW, Moldovan D, Van Leerberghe A, Tang Y, Lu P, Vardi M, Schranz J, Martinez-Saguer I. A randomized trial of human C1 inhibitor prophylaxis in children with hereditary angioedema. Pediatr Allergy Immunol. 2019 Aug;30(5):553-561. doi: 10.1111/pai.13060. Epub 2019 May 29.

Reference Type DERIVED
PMID: 30968444 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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2013-002453-29

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

SHP616-301

Identifier Type: OTHER

Identifier Source: secondary_id

0624-301

Identifier Type: -

Identifier Source: org_study_id

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