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

Shire

Status and phase: Completed; Phase 2

Conditions: Hereditary Angioedema (HAE)

Treatments: Biological: CINRYZE

Study type: Interventional

Funder types: Industry

Identifiers

NCT01095510

2011-000369-11 (EudraCT Number)

0624-203

Details and patient eligibility

About

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.

Full description

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

Enrollment: 9 patients

Sex: All

Ages: 2 to 11 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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9 participants in 4 patient groups

500 U CINRYZE (10-25 kg body weight)

Experimental group

Description:

Single IV dose of 500 U CINRYZE

Treatment:

Biological: CINRYZE

1000 U CINRYZE (10-25 kg body weight)

Experimental group

Description:

Single IV dose of 1000 U CINRYZE

Treatment:

Biological: CINRYZE

1000 U CINRYZE (>25 kg body weight)

Experimental group

Description:

Single IV dose of 1000 U CINRYZE

Treatment:

Biological: CINRYZE

1500 U CINRYZE (>25 kg body weight)

Experimental group

Description:

Single IV dose of 1500 U CINRYZE

Treatment:

Biological: CINRYZE