CINRYZE for the Treatment of Hereditary Angioedema Attacks in Children Under the Age of 12

NCT ID: NCT01095510

Last Updated: 2021-06-03

Study Results

Results available

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

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

9 participants

Study Classification

INTERVENTIONAL

Study Start Date

2010-06-02

Study Completion Date

2012-04-17

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

The objectives of this study were to evaluate: (1) the dose response and (2) the pharmacokinetics (PK) and pharmacodynamics (PD) of intravenous (IV) administration of CINRYZE for the treatment of acute angioedema attacks in children above and below 25 kg and less than 12 years of age with hereditary angioedema (HAE); and (3) to determine the safety and tolerability following IV administration of CINRYZE in this study population.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Each subject received CINRYZE for treatment of a single acute angioedema attack.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Hereditary Angioedema (HAE)

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

500 U CINRYZE (10-25 kg body weight)

Single IV dose of 500 U CINRYZE

Group Type EXPERIMENTAL

CINRYZE

Intervention Type BIOLOGICAL

1000 U CINRYZE (10-25 kg body weight)

Single IV dose of 1000 U CINRYZE

Group Type EXPERIMENTAL

CINRYZE

Intervention Type BIOLOGICAL

1000 U CINRYZE (>25 kg body weight)

Single IV dose of 1000 U CINRYZE

Group Type EXPERIMENTAL

CINRYZE

Intervention Type BIOLOGICAL

1500 U CINRYZE (>25 kg body weight)

Single IV dose of 1500 U CINRYZE

Group Type EXPERIMENTAL

CINRYZE

Intervention Type BIOLOGICAL

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

CINRYZE

Intervention Type BIOLOGICAL

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

C1 inhibitor [human]

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

To be eligible for this protocol, subjects must:

1. Be at least 10 kg of body weight.
2. Have a confirmed diagnosis of HAE.
3. Have an acute HAE attack and be able to initiate treatment within 8 hours after onset of symptoms.

Exclusion Criteria

To be eligible for this protocol, subjects must not:

1. Have any active infectious illness.
2. Have had a prior HAE attack and/or received any C1 INH product within 7 days prior to dosing with study drug.
3. Have received therapy with antifibrinolytics (e.g., tranexamic acid), androgens (e.g., danazol, oxandrolone, stanozolol, or testosterone), ecallantide (Kalbitor®), or icatibant (Firazyr®) within 7 days prior to dosing with study drug.
4. Have a history of allergic reaction to C1 INH products, including CINRYZE (or any of the components of CINRYZE), or other blood products.
5. Have participated in any other investigational drug evaluation within 30 days prior to dosing with study drug, or have previously received treatment with CINRYZE in this study at any time.
Minimum Eligible Age

2 Years

Maximum Eligible Age

11 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Shire

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Study Director

Role: STUDY_DIRECTOR

Takeda

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Asthma & Allergy Associates, P.C.

Colorado Springs, Colorado, United States

Site Status

University of South Florida Asthma, Allergy and Immunology Clinical Research Unit

Tampa, Florida, United States

Site Status

Institute for Asthma and Allergy, PC

Chevy Chase, Maryland, United States

Site Status

Allergy & Asthma Research Group

Eugene, Oregon, United States

Site Status

Baker Allergy, Asthma and Dermatology Research Center, LLC

Lake Oswego, Oregon, United States

Site Status

AARA Research Center

Dallas, Texas, United States

Site Status

Allergy and Asthma Research Center, P.A.

San Antonio, Texas, United States

Site Status

Marycliff Allergy Specialists

Spokane, Washington, United States

Site Status

Charité Universitätsmedizin Berlin, Dept. of Dermatology and Allergy

Berlin, , Germany

Site Status

Klinikum rechts der Isar, Technical University Munich, ENT Clinic

Munich, , Germany

Site Status

Semmelweis University, Allergy and Angioedema Outpatients Clinic, Kútvölgyi Clinical Center

Budapest, , Hungary

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States Germany Hungary

References

Explore related publications, articles, or registry entries linked to this study.

Lumry W, Soteres D, Gower R, Jacobson KW, Li HH, Chen H, Schranz J. Safety and efficacy of C1 esterase inhibitor for acute attacks in children with hereditary angioedema. Pediatr Allergy Immunol. 2015 Nov;26(7):674-80. doi: 10.1111/pai.12444. Epub 2015 Aug 11.

Reference Type RESULT
PMID: 26171584 (View on PubMed)

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

2011-000369-11

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

0624-203

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.

PRecIsion Medicine in CardiomyopathY
NCT04036799 ACTIVE_NOT_RECRUITING