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Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema

NCT ID: NCT00262301

Last Updated: 2012-10-02

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

75 participants

Study Classification

INTERVENTIONAL

Study Start Date

2004-06-30

Study Completion Date

2009-10-31

Brief Summary

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Hereditary angioedema ("HAE") is a genetic disorder characterized by sudden recurrent attacks of local swelling (angioedema). These attacks are often painful and disabling, and, in some cases, life-threatening. "HAE" is caused by mutations in the "C1INH" gene that leads to a decrease in the blood level of functional "C1INH". This multi-center study was designed to assess the safety and tolerability, efficacy and pharmacodynamics/ pharmacokinetics of recombinant human C1 inhibitor ("rhC1INH") in the treatment of acute hereditary angioedema attacks.

Detailed Description

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A prospectively planned interim analysis will be performed on the double-blind data.

Conditions

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Hereditary Angioedema Angioneurotic Edema Genetic Disorders

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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100 IU/kg "rhC1INH"

100 IU/kg recombinant human C1 inhibitor

Group Type EXPERIMENTAL

recombinant human C1 inhibitor

Intervention Type DRUG

IV

Saline

Saline solution

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

IV

Interventions

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recombinant human C1 inhibitor

IV

Intervention Type DRUG

Placebo

IV

Intervention Type DRUG

Other Intervention Names

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"rhC1INH" Ruconest conestat alfa saline physiological salt solution

Eligibility Criteria

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Inclusion Criteria

* Clear clinical and laboratory diagnosis of HAE
* Baseline plasma level of functional C1INH of less than 50% of normal
* Evidence for exacerbation or development of a severe abdominal, oro-facial/ pharyngeal/ laryngeal, genito-urinary and/or peripheral HAE attack

Exclusion Criteria

* Acquired angioedema
* Pregnancy or breastfeeding
* Participation in another clinical study within prior 3 months
Minimum Eligible Age

16 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Pharming Technologies B.V.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Jan Nuijens, MD, PhD

Role: STUDY_CHAIR

Pharming Group N.V.

Locations

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For information on sites, please contact Pharming Medical Affairs Deparment

Leiden, , Netherlands

Site Status

Emergency County Hospital, Internal Medicin Clinica, Allergology-Immunology Department

Târgu Mureş, , Romania

Site Status

Countries

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Netherlands Romania

References

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Zuraw B, Cicardi M, Levy RJ, Nuijens JH, Relan A, Visscher S, Haase G, Kaufman L, Hack CE. Recombinant human C1-inhibitor for the treatment of acute angioedema attacks in patients with hereditary angioedema. J Allergy Clin Immunol. 2010 Oct;126(4):821-827.e14. doi: 10.1016/j.jaci.2010.07.021.

Reference Type RESULT
PMID: 20920772 (View on PubMed)

Moldovan D, Reshef A, Fabiani J, Kivity S, Toubi E, Shlesinger M, Triggiani M, Montinaro V, Cillari E, Realdi G, Cancian M, Visscher S, Zanichelli A, Relan A, Cicardi M. Efficacy and safety of recombinant human C1-inhibitor for the treatment of attacks of hereditary angioedema: European open-label extension study. Clin Exp Allergy. 2012 Jun;42(6):929-35. doi: 10.1111/j.1365-2222.2012.03984.x.

Reference Type RESULT
PMID: 22909164 (View on PubMed)

Bernstein JA, Relan A, Harper JR, Riedl M. Sustained response of recombinant human C1 esterase inhibitor for acute treatment of hereditary angioedema attacks. Ann Allergy Asthma Immunol. 2017 Apr;118(4):452-455. doi: 10.1016/j.anai.2017.01.029. Epub 2017 Mar 9.

Reference Type DERIVED
PMID: 28284978 (View on PubMed)

Other Identifiers

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C1 1304-01

Identifier Type: -

Identifier Source: org_study_id